2017 >475,000 ANNUAL PATIENTS REPORT Sharing Knowledge. Sharing Hope.
>2,800 RESEARCHERS
>420 CENTERS
>1,200 PUBLICATIONS
>55 COUNTRIES
2017 Annual Report January – December
Milwaukee Campus Minneapolis Campus Medical College of Wisconsin National Marrow Donor Program/ 9200 W Wisconsin Ave, Be The Match Suite C5500 500 N 5th St Milwaukee, WI 53226 Minneapolis, MN 55401 (414) 805-0700 (763) 406-5800
cibmtr.org
CIBMTR 2017 Annual Report TABLE OF CONTENTS TABLE OF CONTENTS 1.0 WHO WE ARE ...... 1 1.1 Mission ...... 1 1.2 Value to the Community ...... 1 1.3 Organizational Structure ...... 2 1.3.1 Scientific Working Committees ...... 2 2.0 WHAT WE DO ...... 7 2.1 Clinical Outcomes Research Program ...... 12 2.1.1 Scientific Working Committees ...... 12 2.1.2 Cellular Therapy and Other Research Initiatives ...... 16 2.2 Immunobiology Research Program ...... 19 2.3 Clinical Trials Support Program ...... 21 2.3.1 Blood and Marrow Transplant Clinical Trials Network ...... 21 2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation ...... 23 2.4 Health Services Research Program ...... 24 2.5 Bioinformatics Research Program ...... 26 2.6 Statistical Methodology Research Program ...... 27 2.7 Stem Cell Therapeutic Outcomes Database (SCTOD) ...... 28 2.8 Corporate Program ...... 30 3.0 HOW WE SHARE KNOWLEDGE ...... 31 3.1 Information Request Service ...... 36 3.2 Internet Presence ...... 37 3.2.1 CIBMTR Public Website ...... 37 3.2.2 CIBMTR Collaborative Site ...... 38 3.2.3 CIBMTR Portal Site ...... 39 3.2.4 Be The Match Public Website...... 40 3.2.5 Be The Match Clinical Website ...... 40 3.2.6 HRSA Blood Cell Transplant Website ...... 40 3.2.7 Other Applications and Data Exchange Standards ...... 41 3.3 BMT Tandem Meetings ...... 42 3.4 Data Management Training ...... 43 4.0 HOW WE COLLECT AND MANAGE DATA ...... 44 4.1 Research Data Life Cycle ...... 44 4.2 Collecting and Storing Data ...... 45 4.2.1 FormsNet ...... 45 4.2.2 Research Database ...... 45 4.2.3 Integrated Data Warehouse ...... 45 4.3 Ensuring Data Quality ...... 46 iii CIBMTR 2017 Annual Report TABLE OF CONTENTS 4.3.1 Continuous Process Improvement ...... 46 4.3.2 Verification and Validation ...... 46 4.3.3 On‐Site Data Audit Program ...... 47 4.4 Protecting Patients and Data ...... 49 4.4.1 Human Subjects / HIPAA Compliance ...... 49 4.4.2 Information Security and Data Privacy ...... 49 5.0 WHAT WE WILL DO NEXT...... 50 2017 KEY ACCOMPLISHMENTS ...... 53 APPENDIX A: CENTERS ...... 57 Appendix A1: US Centers...... 58 Appendix A2: International Centers ...... 68 APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP ...... 83 Appendix B1: Organizational Structure – Milwaukee Campus ...... 84 Appendix B2: Organizational Structure – Minneapolis Campus ...... 85 Appendix B3: Coordinating Center Leadership ...... 88 APPENDIX C: COMMITTEE MEMBERSHIP ...... 98 Appendix C1: Advisory Committee Membership ...... 98 Appendix C2: Executive Committee Membership ...... 101 Appendix C3: Consumer Advocacy Committee Membership ...... 103 Appendix C4: Nominating Committee Membership ...... 104 Appendix C5: Scientific Working Committee Leadership ...... 105 Appendix C6: Immunobiology Steering Committee Membership ...... 109 Appendix C7: Clinical Trials Advisory Committee Membership ...... 110 APPENDIX D: PUBLICATIONS ...... 111 Appendix D1: Scientific Working Committee Publications ...... 111 Appendix D2: BMT CTN Publications ...... 126 Appendix D3: Health Services Research Program Publications ...... 128 Appendix D4: Bioinformatics Research Program Publications ...... 129 Appendix D5: Statistical Methodology Research Program Publications ...... 131 Appendix D6: 2017 Publications Not Previously Reported ...... 132 APPENDIX E: PRESENTATIONS ...... 133 APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS ...... 143 APPENDIX G: CLINICAL STUDIES AND TRIALS ...... 146 Appendix G1: BMT CTN Clinical Trials Open for Enrollment ...... 146 Appendix G2: RCI BMT Clinical studies ...... 149 APPENDIX H: FORMS SUBMISSION PROCESS ...... 153 APPENDIX I: WEBSITES ...... 154 APPENDIX J: GLOSSARY ...... 155
iv CIBMTR 2017 Annual Report TABLE OF CONTENTS FIGURES AND TABLES Figure 1.1 CIBMTR Research Programs...... 1 Figure 1.2 Scientific Organizational Structure ...... 3 Figure 1.3 Functional Organizational Structure with Scientific Oversight ...... 4 Table 1.4 Committee Structure ...... 5 Figure 2.1 Continued Growth in the Number of Patients Registered with the CIBMTR ...... 8 Figure and Table 2.2 Distribution of Patients in the CIBMTR Research Database by Graft Source ...... 8 Table 2.3 Distribution of Patients in the CIBMTR Research Database by Therapy and Indication ...... 9 Table 2.4 2017 CIBMTR Publications by Journal ...... 10 Figure 2.5 CIBMTR Publications by Year ...... 11 Table 2.6 2017 CIBMTR Presentations by Meeting ...... 11 Table 2.7 2017 Working Committee Studies ...... 13 Figure 2.8 2017 CIBMTR Publications by Program ...... 14 Figure 2.9 Working Committee Study Proposal Review Process...... 15 Table 2.10 CMS CED Studies ...... 17 Figure 3.1 How to Access CIBMTR Knowledge ...... 31 Table 3.2 How to Access Information Online at cibmtr.org ...... 32 Table 3.3 How to Access Data Online at cibmtr.org ...... 32 Table 3.4 How to Access Tools Online at cibmtr.org ...... 33 Table 3.5 How to Access Biospecimens Online at cibmtr.org ...... 33 Table 3.6 Standard Reports Published by the CIBMTR ...... 34 Table 3.7 Data Requests Addressed by the CIBMTR in 2017 ...... 36 Figure 4.1 Research Data Life Cycle ...... 44 Figure 4.2 Audit Process ...... 48 Table 5.1 Plans to Enhance Data ...... 50 Table 5.2 Plans to Expand Knowledge Sharing ...... 51 Table 5.3 Plans to Increase Impact ...... 52 Figure A.1 Location of Centers that Submit Data to the CIBMTR ...... 57
WEB LINKS Throughout this report, electronic links to webpages and documents are provided; they are underlined and italicized for identification. If you are unable to access items using the links provided, enter the underlined and italicized words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).
v CIBMTR 2017 Annual Report 1.0 WHO WE ARE 1.0 WHO WE ARE
The CIBMTR® (Center for International Blood outcomes database, and a unique biospecimen and Marrow Transplant Research®) is a repository. research collaboration between the National Marrow Donor Program® (NMDP)/Be The 1.2 VALUE TO THE COMMUNITY Match® and the Medical College of Wisconsin The CIBMTR has been collecting health (MCW). outcomes data worldwide for >45 years, 1.1 MISSION resulting in a Research Database with information on >475,000 patients. These data The CIBMTR collaborates with the global are available to investigators with interest in scientific community to advance hematopoietic treatments for cancer, marrow failure cell transplantation (HCT) and cellular therapy syndromes, and other life‐threatening diseases. worldwide to increase survival and enrich CIBMTR research involves 6 major programs quality of life for patients. The CIBMTR (Figure 1.1). The CIBMTR has become a facilitates critical observational and respected leader in outcomes research by interventional research through scientific and providing a unique resource of information and statistical expertise, a large network of expertise to the medical and scientific transplant centers, an extensive clinical communities.
Figure 1.1 CIBMTR Research Programs
Clinical 15 Scientific Working Committees utilize the CIBMTR’s Research Database to answer clinically important questions. Each committee focuses on a specific Outcomes disease, use of HCT, or complication of HCT therapy.
Immuno‐ The CIBMTR maintains a repository of paired tissue samples (from donors and recipients, related and unrelated) used in studying the genetic, cellular, and biology immunologic factors that influence the outcomes of HCT and cellular therapy.
The BMT CTN conducts multicenter Phase II and III trials focusing on HCT issues Clinical Trials while the RCI BMT supports Phase I and II trials and other prospective studies that bridge the gap between single‐center studies and larger trials.
The CIBMTR conducts research in health disparities, health policy, and system Health Services capacity issues. Current studies focus on costs and cost‐effectiveness, insurance coverage, individualized care plans, and informed consent.
The CIBMTR provides expertise in, and conducts research on, translational and Bioinformatics operational bioinformatics. Recent endeavors include improving the HLA matching algorithm and analyzing next generation sequencing typing data.
Statistical The CIBMTR Coordinating Center provides advice and statistical consultation to researchers developing protocols for HCT studies and investigates new Methodology statistical approaches and techniques for analyzing HCT data.
Page | 1 CIBMTR 2017 Annual Report 1.0 WHO WE ARE
1.3 ORGANIZATIONAL STRUCTURE The CIBMTR (Figures 1.2 and 1.3) represents a Scientific Working Committees network of >420 participating centers in >55 countries (Appendix A) that submit outcomes‐ Acute Leukemia related data for patients. The CIBMTR Autoimmune Diseases and Cellular Coordinating Center, staffed by approximately Therapies 210 employees (Appendix B), provides data Chronic Leukemia acquisition, management, and statistical support for analyses of these data. Donor Health and Safety The Chief Scientific Director is responsible for Graft Sources and Manipulation all administrative and scientific operations. The Graft‐versus‐Host Disease CIBMTR utilizes a dyad organizational structure, Health Services and International Studies which partners administrative management with Senior Scientific Directors who provide Immunobiology scientific input to each functional area of the Infection and Immune Reconstitution Coordinating Center (Figure 1.3). CIBMTR administrative committees (Table 1.4) provide Late Effects and Quality of Life input and advice to the leadership team, Lymphoma ensuring the continued support of the needs Pediatric Cancer and priorities of the scientific and medical communities. Plasma Cell Disorders and Adult Solid Tumors 1.3.1 Scientific Working Committees Primary Immune Deficiencies, Inborn Errors To ensure broad input into the research of Metabolism, and Other Non‐Malignant process and efficient use of resources, the Marrow Disorders CIBMTR looks to 15 Scientific Working Regimen‐Related Toxicity and Supportive Committees focused on specific research areas. Care Total Working Committee membership exceeds 2,800 researchers. Membership is open to any The Working Committee structure encourages researcher willing to take an active role in a collaborative but rigorous methodological developing and conducting studies that use approach to all CIBMTR activities. CIBMTR data and / or resources. While most of these individuals are clinical researchers, statisticians and basic scientists also Working Committee Leadership participate. PhD‐level statistical faculty and Chairs (usually 3‐4) Master’s‐level statisticians from the CIBMTR Coordinating Center provide unique expertise MD Scientific Director in data analysis. Basic scientists investigating PhD Statistical Director human leukocyte antigen (HLA), MS‐level Statistician immunogenetics, pharmacogenetics, stem cell biology, and other areas related to HCT provide essential expertise in their respective areas. Working Committee leadership is listed in Appendix C5.
Page | 2 CIBMTR 2017 Annual Report 1.0 WHO WE ARE Figure 1.2 Scientific Organizational Structure
National Marrow Donor Joint Affiliation Committee Medical College of Transplant Centers Program / Be The Match Executive Director Wisconsin CR Mills, PhD Associate Scientific Director for CIBMTR Minneapolis CIBMTR Senior Research Advisor Executive Committee Advisory role D Confer, MD Assembly D Weisdorf, MD Senior Scientific Director for Research Operations Advisory M Eapen, MBBS, MS Committee Chief Statistical Chief Scientific Director Director Senior Scientific Director M Horowitz, MD, MS MJ Zhang, PhD for Patient-Reported Outcomes K Flynn, PhD
Senior Scientific Director for SCTOD JD Rizzo, MD, MS
Scientific Working Committees Consumer Advocacy Advisory role Steering Committees Committee Senior Scientific Director for Data Operations B Shaw, MD, PhD
Statistical Clinical Trials Health Services Immunobiology Bioinfomatics Methodology Clinical Outcomes Support Program Research Program Research Program Research Program Research Program M Eapen, MBBS, MS M Pasquini, MD, MS L Burns, MD S Lee, MD, MPH M Maiers, MS MJ Zhang, PhD B Shaw, MD, PhD
Blood and Marrow Transplant Clinical Resource for Clinical Investigations in Trials Network Blood and Marrow Transplantation (BMT CTN) (RCI BMT) M Pasquini, MD, MS B Shaw, MD, PhD
Page | 3 CIBMTR 2017 Annual Report 1.0 WHO WE ARE Figure 1.3 Functional Organizational Structure with Scientific Oversight
Research & Administration M Horowitz & D Confer
Faculty CIBMTR Administration Finance, Grants & Contracts
M Horowitz & D Confer P Steinert & R King MCW & NMDP
MDs PhDs
Statistics & Human Research Clinical Studies Quality Information Business Office Advancement Auditing & Bioinformatics Data Operations BMT CTN Observational Protection Support Assurance Technology Monitoring Research Research Program E Leckrone TBD E Bergman & S Lorenz & S Fisher D Christianson C Kennedy J Brunner & A Foley W Perez & R King MPLS MKE M Prestegaard P Vespalec MKE MPLS MPLS M Matlack MPLS MKE & MPLS S Spellman MPLS MKE MKE & MPLS MKE & MPLS (M Eapen, MBBS, (D Confer, MD) MS) (B Shaw, MD, (M Horowitz, MD, (D Rizzo, MD, MS) (M Horowitz, MD, (B Shaw, MD, (M Maiers, MS) (B Shaw, MD, (M Pasquini, (M Eapen, MBBS, MS) (**B Lindberg, JD) PhD) MS) (B Shaw, MD, PhD) MS) PhD) PhD) MD, MS)
Immunobiology Statistics & RCI BMT Survey Project Quality Data Corporate Meetings Data HCT HCT Donor Cellular Research Clinical Outcomes Research Management Assurance Solutions Management Recipient Data Therapy Group & Analysis Special Data Management Data Projects Management Management W Perez & T Moerke V Yarra E Bergman S Fisher T Houseman S Spellman S Spellman E Leckrone D Mattila & K Gee MPLS J Pollack MKE MPLS J Brunner & S Meiers & A Hauck T Hunt MPLS MKE & MPLS MPLS MPLS MKE & MPLS MKE & MPLS M Matlack K Gardner MPLS MKE (MJ Zhang, PhD) MKE & MPLS MKE & (M Eapen, MPLS (D Confer, MD) MBBS, MS) (S Lee, MD, (M Eapen, MBBS, (B Shaw, MD, (B Shaw, MD, (M Eapen, (M Horowitz, MD, (B Shaw, MD, (B Shaw, (D Confer, (M Pasquini, MPH) MS) PhD) (D Confer, MD) PhD) MBBS, MS) MS) PhD) MD, PhD) MD) MD)
Health Services Applications Technical Communications Data Entry & Data Training Research* Services Imaging Support
E Denzen E Bergman T Moerke S Fisher B Levesque M Matlack M Matlack MPLS & E Chan MKE MKE MPLS MPLS MPLS MKE & MPLS
(M Eapen, MBBS, MS) (D Rizzo, MD, MS) (D Rizzo, MD, (M Horowitz, MD, (B Shaw, (B Shaw, (B Shaw, (L Burns, MD) (B Shaw, MD, PhD) MS) MS) MD, PhD) MD, PhD) MD, PhD) *Staffed through NMDP Patient Services ( ) Indicates Scientific oversight **Institutional official oversight
Page | 4 CIBMTR 2017 Annual Report 1.0 WHO WE ARE Table 1.4 Committee Structure
Committee Function Meetings Roster Joint Affiliation Reviews and approves the CIBMTR Annually Board budget and research plan Amends the terms of the affiliation agreement, as necessary Reviews and approves data access and confidentiality policies Assembly Includes representatives from each Annually during the center that submits CRF‐level data BMT Tandem Elects members of the Advisory, Meetings Nominating, and Clinical Trials Advisory Committees Advisory Oversees CIBMTR policies and In person annually at Appendix C1 Committee scientific agenda the BMT Tandem Partners with the Working Meetings Committees to prioritize scientific By teleconference studies quarterly and as Oversees operations of the SCTOD needed Executive Provides scientific and policy advice Four times annually Appendix C2 Committee to the Chief Scientific Director and by teleconference (subcommittee of Coordinating Center Advisory Reviews audit results and makes Committee) recommendations for
improvement Consumer Provides patient and donor In person annually at Appendix C3 Advocacy perspectives during the the BMT Tandem Committee development of the CIBMTR Meetings (subcommittee of research agenda By teleconference Advisory Communicates CIBMTR research periodically Committee) results and data to the non‐medical
community Nominating Prepares a slate of candidates for At least once Appendix C4 Committee open positions on the Advisory, annually each Fall by
Nominating, and Clinical Trials teleconference Advisory Committees Makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments
Page | 5 CIBMTR 2017 Annual Report 1.0 WHO WE ARE
Committee Function Meetings Roster Scientific Working Design and conduct relevant In person annually at Leadership ‐ Committees studies using CIBMTR data, the BMT Tandem Appendix C5 (Section 1.3.1) statistical resources, networks, and Meetings
/ or centers Leadership ‐ by Set priorities for clinical outcomes teleconference studies every 4‐8 weeks Assess and revise CIBMTR data collection forms, as needed
Immunobiology Reviews and approves the use of In person twice Appendix C6 Steering donor‐recipient specimens from annually, in summer Committee / the Research Repository in CIBMTR and at the BMT NMDP/Be The studies Tandem Meetings Match Histocompatibility Advisory Group
Clinical Trials Assists in the review, approval, and In person annually at Appendix C7 Steering oversight of proposals and the BMT Tandem Committee protocols for clinical trials Meetings submitted to the RCI BMT By teleconference as needed
Page | 6 CIBMTR 2017 Annual Report 2.0 WHAT WE DO
2.0 WHAT WE DO
The CIBMTR collects data for approximately At any given time, the CIBMTR has >200 23,000 new transplantations annually as well retrospective, correlative, or methodologic as a continually increasing volume of follow‐up studies and 15 prospective trials ongoing in 6 data on previously reported recipients and major areas of research activity. donors. Centers submit transplant data at two levels: A Transplant Essential Data (TED) level, Areas of Research Activity which captures basic data, and a Comprehensive Report Form (CRF) level, which Clinical Outcomes Research (Section 2.1) captures more detail. Centers submit cellular Immunobiology Research (Section 2.2) therapy data with a suite of Cellular Therapy Essential Data (CTED) forms. Clinical Trials Support (Section 2.3) Submission of outcomes data is mandatory for Blood and Marrow Transplant Clinical allogeneic transplants in the US and those Trials Network (BMT CTN) outside the US that use a US donor; all other (Section 2.3.1) submissions are voluntary. The CIBMTR Resource for Clinical Investigations in estimates that almost 100% of US allogeneic Blood and Marrow Transplantation transplants and about 90% of US autologous (RCI BMT) (Section 2.3.2) transplants are reported. Cellular therapy Health Services Research (Section 2.4) outcomes data are also reported to the CIBMTR; since July 2016, 300 patients who Bioinformatics Research (Section 2.5) received cellular therapy were included in the Statistical Methodology Research CIBMTR Research Database. Data for (Section 2.6) approximately 7,600 non‐US patients are collected annually. Publications The CIBMTR Research Database contains In 2017, the CIBMTR published 82 information on >475,000 patients. Figure 2.1 shows the continued growth in the number of manuscripts, 81 in peer‐reviewed journals patients registered with the CIBMTR. The listed in Table 2.4. As of December 31, an distribution of patients in the database by additional 17 manuscripts were submitted and graft type is displayed in Figure and Table 2.2 are under review. A complete list of 2017 and by disease in Table 2.3. publications is provided in Appendix D. Figure 2.5 displays the number of CIBMTR Research publications annually since 2004. The CIBMTR is dedicated to improving survival, Presentations treatment, and quality of life for transplant and cellular therapy patients. We provide In 2017, CIBMTR study investigators presented many opportunities to conduct research in HCT 71 abstracts (38 oral and 33 poster) at and cellular therapy, and we encourage both national and international conferences (Table senior and junior investigators to participate. 2.6). A complete list of presentations is provided in Appendix E.
Page | 7 CIBMTR 2017 Annual Report 2.0 WHAT WE DO
Figure 2.1 Continued Growth in the Number of Patients Registered with the CIBMTR
Years (Data are incomplete for 2017)
Figure and Table 2.2 Distribution of Transplant Patients in the CIBMTR Research Database by Graft Source
Allogeneic Autologous TOTAL TED CRF TED CRF Bone Marrow 52,021 59,883 10,180 5,913 127,997 Peripheral Blood 78,727 36,025 181,942 38,545 335,239 Cord Blood 5,614 9,369 28 6 15,017 TOTAL 136,362 105,277 192,150 44,464 478,253
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Table 2.3 Distribution of Patients in the CIBMTR Research Database by Therapy and Indication
TRANSPLANT DATA Allogeneic Autologous TOTAL Indication TED CRF TED CRF Lymphoma 13,048 6,746 75,095 14,649 109,538 Plasma cell disorders 2,693 1,446 76,294 13,929 94,362 Acute myelogenous leukemia 45,607 29,768 6,154 2,434 83,963 Other malignant diseases1 6,344 3,925 31,487 12,420 54,176 Acute lymphoblastic leukemia 23,534 17,541 1,181 477 42,733 Chronic myelogenous leukemia 14,363 15,263 420 287 30,333 Myelodysplastic / myeloproliferative 14,610 12,265 222 90 27,187 syndromes Aplastic anemia / PNH2 6,783 7,652 ‐ ‐ 14,435 Immune disorders3 3,593 4,052 ‐ ‐ 7,645 Hemoglobinopathy4 2,790 3,098 ‐ ‐ 5,888 Inherited bone marrow failure5 1,356 1,689 ‐ ‐ 3,045 Inborn errors of metabolism 1,146 1,572 ‐ ‐ 2,718 Other nonmalignant disorders6 182 162 872 161 1,377 Other diseases 313 98 425 17 853 TOTAL 136,362 105,277 192,150 44,464 478,253 CELLULAR THERAPY DATA Indication Non‐Hodgkin lymphoma 55 Acute lymphocytic leukemia 50 Acute myeloid leukemia 27 Multiple myeloma 11 Hodgkin disease 7 Other malignancies 30 Neurologic disorders 60 Infection 26 Other 34 TOTAL 300 Genetically Modified Cells 140
Page | 9 CIBMTR 2017 Annual Report 2.0 WHAT WE DO
Footnotes for Table 2.3: 1. Includes other leukemia (allogeneic, n=8,863 autologous, n=976), solid tumors (allogeneic, n=1,406; autologous, n=42,931) 2. Includes SAA (allogeneic, n=13,804; autologous, n=0), PNH (allogeneic, n=631; autologous, n=0) 3. Includes immune deficiencies (allogeneic, n=6,030; autologous, n=0), histiocytic disorders (allogeneic, n=1,615; autologous, n=0) 4. Includes sickle cell anemia (allogeneic, n=1,662; autologous, n=0), sickle cell thalassemia (allogeneic, n=141; autologous, n=0), thalassemia major (allogeneic, n=4085; autologous, n=0) 5. Includes Schwachman‐Diamond (allogeneic, n=70; autologous, n=0), Fanconi anemia (allogeneic, n=2,212; autologous, n=0), Diamond‐Blackfan anemia (allogeneic, n=415; autologous, n=0), other inherited abnormalities of erythrocyte (allogeneic, n=348; autologous, n=0) 6. Includes platelet disorders (allogeneic, n=207; autologous, n=7), autoimmune deficiencies (allogeneic, n=137; autologous, n=1026) 7. Neuroblastoma, sarcoma, and glioma 8. CAR T cells for BCMA, CD19, CD22, CD30, CD16v, CD123, CD171, NY‐ESO‐1
Table 2.4 2017 CIBMTR Publications by Journal Journal Number of Publications Biology of Blood and Marrow Transplantation 28 Cancer 8 Bone Marrow Transplantation 7 Blood 4 Haematologica 4 Journal of Clinical Oncology 3 Leukemia 3 Lancet Haematology 2 Other Journals* 23 TOTAL* 82
*One publication each in the 2017 IEEE International Conference on Healthcare Informatics, American Journal of Epidemiology, Blood Advances, British Journal of Haematology, Clinical Transplantation, Genes and Immunity, Human Immunology, Immunogenetics, JAMA Clinical Pediatrics Philadelphia, JAMA Neurology, Journal of Allergy and Clinical Immunology, Journal of Cancer, Journal of Hematology and Oncology, Journal of Oncology Practice, Journal of the American Medical Informatics Association, Lifetime Data Analysis, New England Journal of Medicine, Pharmacogenomics Journal, PLOS Computational Biology, Proceedings of the National Academy of Sciences of the United States of America, Statistical Methods in Medical Research, Statistics in Medicine, and Transplantation.
Page | 10 CIBMTR 2017 Annual Report 2.0 WHAT WE DO
Figure 2.5 CIBMTR Publications by Year
100 90 80 70 60 Publications
50 of 40 30
Number 20 10 0 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 Year
Table 2.6 2017 CIBMTR Presentations by Meeting Conference Oral Poster Total American Society of Hematology (ASH) Annual Meeting 15 17 32 BMT Tandem Meetings 13 6 19 American Society of Clinical Oncology (ASCO) Annual Meeting 2 1 3 International Myeloma Workshop 3 0 3 European Federation for Immunogenetics (EFI) 0 2 2 Other Meetings and Conferences* 5 7 12 TOTAL 38 33 71 *One oral presentation each at the AI Genomic Hackathon, American Society of Pediatric Hematology / Oncology (ASPHO), American Statistical Association Joint Statistical Meeting, Biostatistics in the Modern Computing Era conference, and International Chinese Statistical Association Meeting. One poster presentation each at the European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting, Individualizing Medicine Conference Advancing Care Through Genomics, International Cancer Education Conference, International Conference on Long‐Term Complications of Treatment of Children and Adolescents for Cancer, International Society for Cellular Therapy, Patient‐ Centered Outcomes Research Institute (PCORI) Annual Meeting, and Society for Clinical Trials.
Page | 11 CIBMTR 2017 Annual Report 2.0 WHAT WE DO
2.1 CLINICAL OUTCOMES RESEARCH PROGRAM Clinical Outcomes Research using the CIBMTR Research Database is a core activity of the Key Working Committee organization. These studies address a wide Publications this Year range of issues, focusing on questions that are difficult or impossible to address in single‐ Bashey A et al. Mobilized PBSC vs center studies or randomized trials because unstimulated bone marrow as a graft diseases studied are uncommon, single centers source for T‐cell‐replete haploidentical treat few patients with a given disorder, and donor HCT using post‐transplant not all important questions are amenable to a cyclophosphamide. Journal of Clinical randomized research design. Oncology. 2017 Sep 10; 35(26):3002‐3009. Epub 2017 Jun 23. PMC5590802. 2.1.1 Scientific Working Committees Kumar SK et al. Early relapse after Program Activities autologous HCT remains a poor The 15 Scientific Working Committees oversee prognostic factor in multiple myeloma but most of the CIBMTR’s clinical outcomes outcomes have improved over time. research. Currently, there are approximately Leukemia. Epub 2017 Nov 16. 175 studies in progress. Numbers for studies in progress, publications, and presentations by Ustun C et al. Outcomes of UCB each Working Committee in 2017 are transplantation are comparable in FLT3+ displayed in Table 2.7. In progress and recently AML: Results of CIBMTR, EUROCORD and published studies are detailed in the 2017 EBMT collaborative analysis. Leukemia. Working Committee Research Portfolio on the 2017 Jun 1; 31(6):1408‐1414. Epub 2017 CIBMTR website. Jan 25. PMC5462854. The Working Committees reviewed 207 new Muffly L et al. Increasing use of allogeneic study proposals before the 2017 BMT Tandem HCT in patients aged 70 years and older in Meetings. 80 proposals were presented at the the US. Blood. 2017 Aug 31; 130(9):1156‐ 1164. Epub 2017 Jul 3. PMC5580273. annual meeting, and 33 were approved. The prioritization and selection process ensures Arnold SD et al. Clinical risks and that the most important issues can be healthcare utilization of HCT for sickle cell addressed in a timely manner. disease in the US using merged databases. Publications Haematologica. 2017 Nov 1; 102(11):1823‐ 1832. Epub 2017 Aug 17. PMC5664386. In 2017, Working Committee study investigators published 59 manuscripts, all of which were peer‐reviewed journal articles. Presentations This is 74% of the total number of CIBMTR In 2017, Working Committee study publications this year (Figure 2.8). As of investigators presented 30 abstracts (17 oral December 31, an additional 14 manuscripts and 13 poster). A complete list of CIBMTR were submitted for publication and are under presentations is provided in Appendix E. review. A complete list of Working Committee publications is provided in Appendix D1.
Page | 12 CIBMTR 2017 Annual Report 2.0 WHAT WE DO
Table 2.7 2017 Working Committee Studies
Studies in Working Committee Publications Presentations Progress
Acute Leukemia 13 6 2 (1 oral / 1 poster)
Autoimmune Diseases and Cellular Therapies 5 1 0 (0 oral / 0 poster)
Chronic Leukemia 12 3 3 (2 oral / 1 poster)
Donor Health and Safety 13 2 1 (0 oral / 1 poster)
Graft Sources and Manipulation 7 3 1 (1 oral / 0 poster)
Graft‐versus‐Host Disease 13 3 2 (1 oral / 1 poster)
Health Services and International Studies 10 2 0 (0 oral / 0 poster)
Immunobiology 38 10 5 (1 oral / 4 poster)
Infection and Immune Reconstitution 7 0 1 (0 oral / 1 poster)
Late Effects and Quality of Life 13 6 4 (4 oral / 0 poster)
Lymphoma 10 6 3 (3 oral / 0 poster)
Pediatric Cancer 1 3 0 (0 oral / 0 poster)
Plasma Cell Disorders and Adult Solid Tumors 8 7 3 (1 oral / 2 poster)
Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non‐Malignant 13 4 1 (0 oral / 1 poster) Marrow Disorders
Regimen‐Related Toxicity and Supportive Care 12 3 4 (3 oral / 1 poster)
TOTAL 175 59 30
Page | 13 CIBMTR 2017 Annual Report 2.0 WHAT WE DO Funding Successful Working Committee Support for the Working Committees is primarily provided by the National Institutes of Study Proposals are Health (NIH) grant # U24CA076518 from the National Cancer Institute (NCI); National Heart, Feasible. Utilize data available in the Lung, and Blood Institute (NHLBI); and National CIBMTR Research Database. Institute for Allergy and Infectious Disease (NIAID). Unique. Fill a gap not addressed by current studies or publications. How to Get Involved Working Committees are collaborative in Important. Impact the field by nature, and all interested individuals are improving transplant procedures or encouraged to participate. Please feel free to results. attend annual in‐person meetings of the Working Committees at the BMT Tandem See the CIBMTR How to Propose a Meetings in February. Additionally, anyone Study webpage and, specifically, the willing to follow the study development and Study Proposal Outline on that management process (Appendix F) is eligible webpage for additional guidelines and to propose a study to the Working Committees advice. (Figure 2.9). Figure 2.8 2017 CIBMTR Publications by Program
Bioinformatics (10)
Clinical Outcomes – BMT CTN Working Committees (7) (59)
Statistical Methodology (4) Health Services (2)
Page | 14 CIBMTR 2017 Annual Report 2.0 WHAT WE DO
Figure 2.9 Working Committee Study Proposal Review Process
•By mid‐November, study investigator submits proposal to the CIBMTR Coordinating Center for consideration at the next BMT Tandem Meetings. Submission
•Working Committee Leadership reviews for feasibility with CIBMTR data, potential conflict with active studies, scientific merit, and ability to complete the study in a Initial timely fashion. Researchers with similar concepts may be advised to combine Review their proposals.
•If Working Committee Leadership clears the proposal to move forward, the MS‐ level Statistician contacts the study investigator and prepares a table of Preliminary characteristics of patient data based on the population defined in the proposal. Assessment
• Study investigator presents the proposal at the Working Committee meeting at the February BMT Tandem Meetings. Presentation
•Working Committee members vote for each proposal, assigning a scientific impact score to each. Voting
•Working Committee Leadership utilizes member feedback in determining which Final proposals to pursue. Advisory Committee approves the CIBMTR research agenda. Approval
•Working Committee Leadership contacts study investigator to notify of study approval / rejection by the end of April. Notification
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2.1.2 Cellular Therapy and Other Research Initiatives In addition to collecting data on HCT patients, The pilot, which began in July 2016, included the CIBMTR continues to increase its data beta testing the forms and data collection collection of cellular and other therapies that processes in selected centers, including do not involve replacement of blood stem intensive staff training. Focus groups to get cells. The CIBMTR also works collaboratively feedback were conducted in October 2016 at a with the Primary Immune Deficiency Disease second Cellular Therapy Forum, at the 2017 Consortium to add transplant outcomes data BMT Tandem Meetings, and via to data on non‐transplant therapy collected by teleconferences with diverse stakeholders. the Consortium. In parallel, the CIBMTR initiated a process to Cellular Therapy Outcomes Registry harmonize cellular therapy data collection with The CIBMTR presented a suite of CTED forms EBMT and the Japanese Society for HCT; this for public commentary at a first Cellular occurred through a series of in‐person Therapy Registry Forum in October 2015, meetings and teleconferences. These activities which included representatives from centers, informed the next version of CTED forms, NIH, the Food and Drug Administration (FDA), released in July 2017. industry, and other stakeholders. Regenerative Medicine Outcomes Registry Implementation and piloting the new data Initial efforts in cellular therapy focused on collection initiative was funded, in part, by an uses in cancer, infection, and genetic NCI administrative supplement to disorders. However, in August 2017, the U24CA76518. CIBMTR expanded its focus and hosted a Regenerative Medicine Outcomes Registry Cellular Therapy strategy meeting. Meeting goals were to Data Collection Forms determine how to establish and demonstrate (Revised in July 2017) feasibility and early utility of a Regenerative Medicine Outcomes Registry, identify potential Pre‐Cellular Therapy Essential Data barriers to success and methods for mitigating (pre‐CTED, 4000) those issues, and determine initial areas of Functions like the pre‐TED in focus. Participants included key stakeholders: collecting pre‐infusion data for physicians and scientists as well as commercial cellular therapies and government representatives involved in using a wide range of cells to restore tissue Cellular Therapy Infusion (4006) and organ function. Replaces the currently required F2006 and collects all cellular Regenerative Medicine therapy infusion data Outcomes Registry
Post‐Cellular Therapy Essential Data Initial Disease Focus (post‐CTED, 4100) Cardiovascular Collects post‐infusion data similar Neurologic to the F2100 post‐TED Musculoskeletal
Page | 16 CIBMTR 2017 Annual Report 2.0 WHAT WE DO CMS Coverage with Evidence CMS CED studies allow CMS to provide Development (CED) Studies coverage to patients enrolled on clinical studies that inform policy decisions. The Many patients with specific diseases and / or at certain ages are denied access to HCT CIBMTR is currently engaged in 5 CMS CED therapy in the US due to lack of insurance studies (Table 2.10). For additional coverage by the Centers for Medicare and information, visit the CIBMTR Medicare Clinical Medicaid Services (CMS). Trials webpage.
Table 2.10 CMS CED Studies
Disease Patient Population Enrollment Dates
Myelodysplastic Elderly patients with 134 centers Launched in Syndrome (MDS) MDS 3,425 patients 2010 (10‐CMS‐MDS) 2,151 patients ≥65 years old NCT# 01166009 1,046 patients 55‐64 years old 228 patients <54 years old
Myelofibrosis Patients aged ≥55 104 centers Launched in December 2016 (16‐CMS‐MF) years with primary 56 patients myelofibrosis or post‐ 14 related (matched 6/6) NCT# 02934477 essential thrombocythemia / 33 unrelated (matched 8/8) polycythemia vera 6 haploidentical 5 unknown
Multiple Elderly patients with 71 centers Launched in Myeloma Stage II or III multiple 3 patients July 2017 (17‐CMS‐MM) myeloma or primary plasma cell leukemia NCT# 03127761 who are eligible to receive allogeneic HCT
Sickle Cell Adolescents and young 31 centers Launched in Disease adults with severe 39 patients October 2016 (BMT CTN 1503) sickle cell disease NCT# 02766465
Sickle Cell Patients aged 15‐50 47 centers Launched in Disease years with severe sickle 200 patients planned November 2017 (17‐CMS‐SCD) cell disease NCT# 01166009
Page | 17 CIBMTR 2017 Annual Report 2.0 WHAT WE DO International Initiatives World Marrow Donor Association (WMDA). This year the CIBMTR continued to strengthen WMDA has a committed partnership with the of processing its international collaborations with clinical CIBMTR in a global aspect standardization and data collection and centers and international registries. sharing – all with the vision of improved European Society for Blood and Marrow outcomes of HCT. Transplantation. The CIBMTR and EBMT Since WDMA’s inception, the CIBMTR has collaborate to collect and exchange HCT data worked closely with the organization to from transplant teams, share data for research improve global accessibility to donors for studies, and define and revise common data elements and collection instruments. The unrelated recipients in need of HCT, combined resources of the registries have standardize global identification systems to for data produced high quality studies with significant match donors and recipients reporting, and conduct important research impact that are unlikely to have been possible about donor outcomes. in single center or single registry studies. Canadian BMT Group and Japan Society for In 2017, the CIBMTR and EBMT enhanced data HCT. The CIBMTR partners with the Canadian alignment and exchange and formalized a BMT Group and Japan Society for HCT to commitment to study collaboration. collect data from centers in Canada and Japan Leadership from both organizations meet in‐ person twice annually and have increased and return those data to the regional groups to their own national outcomes registries. relationships to maintain harmonization and create Using this approach, the regional groups develop standard data collection for the leverage the CIBMTR’s data collection emerging field of cellular therapy. mechanisms at little or no additional expense Worldwide Network for Blood and Marrow to them and provide the CIBMTR with valuable Transplantation (WBMT). The WBMT international data. collaborates with CIBMTR leaders in a wide Data Management. In February 2017, the range of areas but with a particular focus on harmonizing data collection forms and specific CIBMTR Data Operations team hosted the data elements to share transplant data Clinical Research Professionals' Data Conference during the BMT worldwide. These data are used in reports of Management international transplant utilization and to Tandem Meetings in Orlando, Florida. >240 support meaningful health services research. individuals attended this three‐day training The CIBMTR and WBMT are utilizing these and educational opportunity; 22 were same processes to define standards for data international participants. collection for emerging indications of cellular The CIBMTR Data Operations team conducted therapy. on‐site international training at 2 international In January 2017, 6 CIBMTR Scientific Directors sites in 2017. In August 2017, in collaboration spoke at the WBMT Workshop and Scientific with Sociedade Brasileira de Transplante de Symposium in Riyadh, Saudi Arabia. In Medula Óssea (SBTMO), the CIBMTR hosted a February 2017, the CIBMTR hosted the Latin training at the Meeting of Data Managers on American BMT Society’s meeting at the BMT Bone Marrow Transplantation in São Paulo, Tandem Meetings. Brazil. In October 2017, the CIBMTR hosted a CIBMTR Data Management Workshop in
Sydney, Australia.
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2.2 IMMUNOBIOLOGY RESEARCH PROGRAM The CIBMTR maintains a Research Repository of paired tissue samples from donors and Research Repository recipients, both unrelated and related. The Immunobiology Research group manages the 2,591,469 aliquots Research Repository inventory and immunogenetic testing programs that add 17,995 cell lines critical HLA and killer‐cell immunoglobulin‐like receptors (KIR) data for use in CIBMTR clinical 67,097 samples from unrelated donors outcomes studies. and 7,858 from related donors The CIBMTR leverages the NMDP/Be The Match’s investment in the Unrelated Donor 60,171 samples from unrelated Research Repository with the NIH’s investment recipients and 8,171 from related in the CIBMTR Research Database. Linking recipients outcomes data to immunologic data available in the Research Repository supports studies 11,707 samples from unrelated cord that include genetic and immunobiologic data blood units and clinical phenotype data. Samples from complete pairs: The Related Donor Research Repository, supported by the Health Resources and 39,234 from complete unrelated adult Services Administration (HRSA), is a unique donor‐recipient pairs opportunity to enhance immunobiologic 6,943 from complete related donor‐ research. Related donor and recipient samples recipient pairs are better matched than unrelated recipients for HLA, a measure of immunological 5,368 from unrelated cord‐recipient compatibility, thus reducing the confounding pairs effects of HLA disparity in clinical research. The combination of the Unrelated and Related recipient pairs that have been retrospectively Donor Research Repositories facilitates an high resolution typed for HLA‐A, ‐B, ‐C, ‐DRB1 organized approach to studying transplant and ‐DQB1; >70% include ‐DPB1, and biology across the spectrum of allogeneic HCT. >14,000 include KIR. Program Activities The Immunobiology Research group In 2017, 182 centers (130 transplant centers, distributed 8,289 research samples in support 35 donor centers, and 17 cord blood banks) of Working Committee studies this year. provided samples to the Research Repository. Publications The Immunobiology Research group enhanced the Research Repository inventory and The Immunobiology Research Program Immunogenetic Database this year by supports investigators’ publications by completing high resolution HLA and KIR typing providing research samples. 16 manuscripts on 613 related and 3,268 unrelated HCT published this year by Working Committee and donor / cord and recipient pairs, bringing the BMT CTN investigators utilized samples from total to >30,000 unrelated donor / cord and the Research Repository.
Page | 19 CIBMTR 2017 Annual Report 2.0 WHAT WE DO Funding Key Publications Support for the Immunobiology Research supported by the Immunobiology Program is primarily provided by the Office of Research Program this Year Naval Research grant # N00014‐17‐1‐2388, NIH grant # U24CA076518, and HRSA contract # Lindsley RC et al. Prognostic mutations in HHSH250201700006C. myelodysplastic syndrome after stem‐ cell transplantation. New England Journal The Immunobiology Research Program offers of Medicine. 2017 Feb 9; 376(6):536‐547. limited research funds supporting Epub 2017 Feb 9. PMC5438571. immunobiology research studies. The grants are intended to subsidize lab tests, sample Boudreau JE et al. KIR3DL1/HLA‐B collection, or costs associated with the use of subtypes govern acute myelogenous research samples. These grants are available to leukemia relapse after hematopoietic approved CIBMTR studies that support cell transplantation. Journal of Clinical organizational research priorities. For Oncology. 2017 Jul 10; 35(20):2268‐2278. additional information, visit the CIBMTR Epub 2017 May 18. PMC5501362. Grants for Immunobiology Research webpage. How to Get Involved Eapen M et al. Allele‐level HLA matching for umbilical cord blood transplantation All interested parties may attend the annual for non‐malignant diseases in children: a in‐person meeting of the Immunobiology retrospective analysis. The Lancet Working Committee at the BMT Tandem Haematology. 2017 Jul 1; 4(7):e325‐e333. Meetings in February. Additionally, the Epub 2017 Jun 13. PMC5699478. Immunobiology Working Committee encourages highly translational, hypothesis‐ driven proposals through the Working Committee Study Proposal Review Process (Figure 2.9).
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2.3 CLINICAL TRIALS SUPPORT PROGRAM The CIBMTR manages a wide array of studies, 2.3.1 Blood and Marrow Transplant Clinical including multi‐center trials, surveys, and Trials Network correlative studies. Access to the CIBMTR The BMT CTN, sponsored by NHLBI and NCI, is Research Database and use of data from the US network charged with developing and observational studies are important resources conducting multicenter Phase II and III clinical to support decisions regarding design of trials focused on HCT. The CIBMTR is the lead prospective clinical trials. institution for the BMT CTN Data and Coordinating Center, which it runs in CIBMTR Coordinating Center collaboration with NMDP/Be The Match and Support of Clinical Trials the Emmes Corporation, a contract research organization based in Rockville, MD. Trial Planning. Oversee study Program Activities development, including patient population identification, site selection, The BMT CTN has launched 46 trials (4 this training, and financial administration. year) and completed accrual for 32 of these trials (1 this year). The Network has accrued Data Collection. Collect new data and collaborate to share existing data across >9,900 patients to its trials from >100 systems and centers. centers, including >650 this year. Among trials currently open for enrollment, the BMT CTN Study Conduct. Oversee data accuracy achieved an overall accrual rate in 2017 that is and protocol compliance through approximately 98% of projections. The monitoring and auditing. Network has established a Research Sample Repository that currently includes >398,000 Statistical Consultation. Provide biospecimens. Additionally, the BMT CTN has expert statistical review of protocols. conducted 36 ancillary and correlative studies, with another 36 in progress. Patient Interviews. Engage patients to collect information. More detail regarding Network activities and protocols is provided in the Annual Progress Real‐Time Accrual Assessment. Report on the BMT CTN website. A list of Review characteristics of enrolled and Network trials open for enrollment is provided non‐enrolled patients to address in Appendix G1. potential accrual barriers. Publications Trial Interpretation. Evaluate results In 2017, BMT CTN study investigators of clinical trials, including through the published 7 manuscripts, all of which were provision of matched controls. peer‐reviewed journal articles. These bring the total number of Network publications to 81, Long‐Term Follow‐Up Data. Capture including 21 primary results papers. A follow‐up data for long‐term or complete list of 2017 BMT CTN publications is secondary analyses, resulting in provided in Appendix D2. considerable cost‐savings.
Page | 21 CIBMTR 2017 Annual Report 2.0 WHAT WE DO Presentations Key BMT CTN Publications this Year BMT CTN study investigators presented 17 abstracts (12 oral and 5 poster) at national Scott BL et al. Myeloablative versus and international conferences in 2017.These reduced‐intensity HCT for AML and MDS. bring the total number of Network Journal of Clinical Oncology. 2017 Apr 10; presentations to 91. A complete list of 2017 35(11):1154‐1161. Epub 2017 Feb 13. CIBMTR presentations is provided in PMC5455603. Appendix E. Holstein SA et al. Updated analysis of Funding CALGB (Alliance) 100104 assessing Support for the BMT CTN Data and lenalidomide versus placebo Coordinating Center is provided by the NIH maintenance after single autologous HCT grant # U10HL069294 and U24HL138660 from for multiple myeloma: A randomised, the NHLBI and NCI. double‐blind, Phase 3 trial. The Lancet Haematology. 2017 Sep 1; 4(9):e431‐ How to Get Involved e442. Epub 2017 Aug 17. PMC5718627. The Network is committed to widespread participation in its trials. If you would like to Holtan SG et al. Low EGF in serve as an Affiliate Center, visit the BMT CTN myeloablative allotransplantation: website for more information. Additionally, Association with severe acute GVHD in you may act as a Center Principal Investigator BMT CTN 0402. Bone Marrow or champion a trial to increase patient accrual Transplantation. 2017 Sep 1; 52(9):1300‐ at your Center, serve on a Protocol Team or an 1303. Epub 2017 Jun 5. PMC5699445. Endpoint Review Committee, or act as a Medical Monitor. You may also propose an ancillary study, which uses data, biospecimens, and / or analyses outside the specific objectives of a primary BMT CTN study.
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2.3.2 Resource for Clinical Investigations in telephone interviews as well as self‐ Blood and Marrow Transplantation administered questionnaires. In 2017, the The RCI BMT provides researchers in the fields Survey Research Group supported 5 active of HCT and cellular therapy with infrastructure studies and participated in the development of and expertise in clinical trial conduct and 2 upcoming studies. analysis. The program’s goal is to help In 2017, the RCI BMT began development of a investigators generate data allowing novel and new electronic patient‐reported outcomes innovative ideas to move into the larger Phase (ePRO) system to be used by the Survey II or Phase III setting with groups such as the Research Group to support clinical trials, long BMT CTN or other national trials groups. It also term follow‐up and other research studies facilitates large survey and cohort studies. with patients and donors, utilizing PROMIS Program Activities (Patient Reported Outcome Measurement Information System) and other measures. The RCI BMT has launched 17 studies, Presentations including 1 this year. In 2017, the RCI BMT accrued >3,000 patients, bringing the total In 2017, RCI BMT study investigators number of accrued patients to approximately presented 2 oral abstracts and 3 posters at 33,500, of which >21,000 were enrolled in a national and international conferences. A cohort study examining long‐term outcomes of complete list of CIBMTR presentations is unrelated donors. provided in Appendix E. The RCI BMT is currently managing 2 FDA Funding investigational new drug (IND) protocols for Support for the RCI BMT is primarily provided NMDP/Be The Match. Peripheral Blood Stem by NMDP/Be The Match and corporate and Cell (PBSC) Procurement accrued >2,300 private sponsors of specific studies. subjects this year, and Cord Blood Access The RCI BMT team can work with study accrued >450. These protocols allow US investigators to seek funding from a variety of centers to access peripheral blood and sources, including government agencies, unlicensed cord blood for transplantation. foundations, pharmaceutical companies, and private corporations. The RCI BMT also supported 4 studies involving unrelated donor data or sample How to Get Involved collection for investigators. A complete list of Study investigators may solicit clinical trials RCI BMT studies is provided in Appendix G2. services from the RCI BMT, including Survey Research Group assistance with funding proposals; protocol development and approvals; management of The Survey Research Group is a team within study conduct; data auditing, management, the RCI BMT created to assist researchers in and analysis; and financial administration. developing and conducting research involving Study investigators may also contract for questionnaires, direct subject interviews, and specific services as needed, such as support patient reported outcomes. The group is with surveys, site selection and management, responsible for collecting high quality, sample management, and more. For additional scientifically valid data from donors, patients, information, visit the RCI BMT webpage. and their families. The Survey Research Group utilizes standardized and semi‐structured
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2.4 HEALTH SERVICES RESEARCH PROGRAM Health services research is the multi‐ Treatment Decision‐Making Support. The disciplinary field of scientific investigation that Health Services Research Program studies how social factors, financial systems, administered a survey to US transplant organizational structures and processes, physicians to identify factors that affect technology, and behavior affect treatment physicians’ preferred graft source (PBSC versus outcomes, quality, and cost. marrow) for HCT and learn more about how HCT research findings translate into clinical Health Services practice change. In partnership with the American Society for Blood and Marrow Research Focus Areas Transplantation (ASBMT) Palliative Care Healthcare disparities in access to and Special Interest Group, US physicians were outcomes of HCT surveyed to better understand their perceptions of palliative care for HCT Economic aspects of HCT, such as costs recipients. The program also provided and cost‐effectiveness technical writing support and developed 4 easy‐to‐read consent forms, 3 easy‐to‐read Treatment decision‐making support assent forms, and 2 patient information sheets Survivorship and quality of life for BMT CTN protocols.
Program Activities Select Health Services Research Studies in Progress The Health Services Research Program currently has 9 studies in progress. In 2017, Individualized care plans for HCT the program completed analysis for 4 research survivors studies. Developing a patient‐centered HCT Health Economics. In collaboration with outcomes research agenda NMDP/Be The Match’s Public and Payer Policy Department, the Health Services Research Reimbursement analysis of HCT in older Program conducted 4 studies to evaluate cost patients and reimbursement of HCT‐related health services. Using Optum Clinformatics data, Physician perceptions of palliative care investigators compared costs for HCT versus chemotherapy alone. Also, Medicare data was Impact of results from BMT CTN linked to the CIBMTR Research Database to protocol 0201 on selection and analyze reimbursement and utilization for utilization of stem cell source for older patients diagnosed with de novo acute unrelated donor HCT myeloid leukemia (AML). The Health Services Payer‐partnered approach to Research Program partnered with investigators community‐based referral for HCT from Kansas University to explore cost of readmissions following HCT.
Page | 24 CIBMTR 2017 Annual Report 2.0 WHAT WE DO Survivorship. In 2017, the program continued efforts to develop a patient‐centered Health Services Research outcomes research agenda in HCT, including Publications this Year sponsoring a symposium at the 2017 BMT Tandem Meetings. Working groups comprised Preussler JM et al. Healthcare costs and of patients, family members, and health care utilization for patients age 50 to 64 years providers prioritized comparative effectiveness with AML treated with chemotherapy or research questions in 6 topic areas. Central to with chemotherapy and allogeneic HCT. this project is the engagement of patients and Biology of Blood and Marrow caregivers in all aspects of planning, Transplantation. 2017 Jun 1; 23(6):1021‐ implementation, and dissemination. 1028. Epub 2017 Mar 2. PMC5497312.
Patient‐Centered Outcomes Neumann JL et al. Burnout, moral Research Topic Areas distress, work‐life balance and career satisfaction among HCT professionals. Physical health and fatigue Biology of Blood and Marrow Transplantation. Epub 2017 Dec 2. Emotional, cognitive, and social health Funding Financial burden Health Services Research Program studies are Models of care delivery ‐ survivorship funded via a variety of mechanisms. and late effects Individualized care plans for HCT survivors is supported by the PCORI award # CD‐12‐11‐ Patient, caregiver, and family education 4062; Engaging patients in developing a and support patient‐centered HCT research agenda is supported by the PCORI engagement award # Sexual health and relationships EAIN‐2956; and A payer‐partnered approach to community‐based referral for HCT is supported by the grant # 11762021 from the National More detail regarding program activities is Comprehensive Care Network / Pfizer. provided in the Health Services Research Additional support for the Health Services Annual Report on the CIBMTR website. Research Program is provided by NMDP/Be Publications The Match.
In 2017, Health Services Research investigators How to Get Involved published 2 manuscripts in peer‐reviewed For more information about the Health journals. A list of program publications is Services Research Program, contact Ellen provided in Appendix D3. Denzen, MS, Senior Manager, at [email protected] or 612.884.8562. Presentations
In 2017, program investigators presented 6 abstracts (1 oral and 5 poster) at scientific meetings. A complete list of CIBMTR presentations is provided in Appendix E.
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2.5 BIOINFORMATICS RESEARCH PROGRAM The Bioinformatics Research Program provides expertise in, and conducts research on, Key Bioinformatics translational and operational bioinformatics. Publications this Year Program Activities Xie C et al. Fast and accurate HLA typing from short‐read next‐generation Current Bioinformatics sequence data with xHLA. Proceedings of Research Goals the US National Academy of Sciences. 2017 Jul 3; 114(30):8059‐8064. Epub Develop pipelines to analyze Next 2017 Jul 3. PMC5544337. Generation Sequencing typing data, including full‐gene HLA, KIR, and genome‐ Alter I et al. HLA class I haplotype wide sequencing, to refine our diversity is consistent with selection for understanding of genetic matching frequent existing haplotypes. PLOS Computational Biology. Epub 2017 Aug Investigate the role of genetic ancestry in 28. PMC5590998. transplantation, including the best way to match multiracial individuals Jones RB et al. Stem cell transplantation and informatics ‐ current considerations. Develop data standards and tools for Biology of Blood and Marrow making immunogenetic data portable for Transplantation. Epub 2017 Dec 27. research and clinical use Funding Investigate HLA data from other countries to better understand global frequencies Support for the Bioinformatics Research and improve matching Program is primarily provided by the grant # N00014‐17‐1‐2850 from the Office of Naval Develop methods for HLA association Research as well as several grants from the studies NIH. Mapping the intersection: Self‐ identification and genetic ancestry is Publications supported by the R21 grant # PA‐11‐251, and Solutions for immunogenetic data In 2017, Bioinformatics study investigators management and analysis is supported by the published 10 manuscripts in peer‐reviewed R01 grant # AI128775. journals. A complete list of program How to Get Involved publications is provided in Appendix D4. For more information about the Bioinformatics Presentations Research Program, contact Martin Maiers, Bioinformatics study investigators presented Director, at [email protected] or 8 abstracts (2 oral and 6 poster) at national 612.627.5892. and international conferences in 2017. A complete list of CIBMTR presentations is provided in Appendix E.
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2.6 STATISTICAL METHODOLOGY RESEARCH PROGRAM The CIBMTR has enjoyed a positive, collaborative association with the Division of Key Statistical Methodology Biostatistics in the MCW Institute for Health Publications this Year and Society since 1980, an association that is a distinctive asset and crucial to the success of Logan BR et al. Decision making and CIBMTR research. Biostatisticians ensure the uncertainty quantification for statistical integrity of CIBMTR scientific individualized treatments using Bayesian activities, contribute to results in articles on Additive Regression Trees. Statistical HCT‐related statistical issues for clinical Methods in Medical Research. Epub 2017 audiences, and support Working Committee Dec 18 study investigators in developing scientific study protocols using CIBMTR data. CIBMTR Zheng C et al. Instrumental variable with biostatisticians have pioneered novel competing risk model. Statistics in methodologic approaches to analyzing HCT Medicine. 2017 Apr 15; 36(8):1240‐1255. data. Epub 2017 Jan 8. PMC5479873. Program Activities Funding HCT is a complex process with multiple competing risks and dramatic changes in the Support for the Statistical Methodology risks of specific events over time. The CIBMTR Research Program is primarily provided by the has developed and evaluated the statistical NIH grant # U24CA076518 from the NCI, models used in HCT research and helped guide NHLBI, and NIAID and the HRSA contract the research community in appropriate # HHSH25020170006C. application and interpretation of these How to Get Involved sophisticated models. During the BMT Tandem Meetings in February, PhD‐level biostatisticians plan and present Statistical Methodology educational sessions related to statistical Research Goals design and analysis, and they provide 1:1 statistical consultation to researchers writing Develop new statistical models proposals or developing protocols for CIBMTR studies. Any interested individual may Compare new statistical models with participate in these sessions. Additionally, the existing solutions using the CIBMTR MCW Division of Biostatistics presents a Research Database lecture series and a seminar series throughout the year in Milwaukee. Publications In 2017, PhD‐level biostatisticians in the Statistical Methodology Research Program published 4 manuscripts, 3 in peer‐reviewed journals. A list of program publications is provided in Appendix D5.
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2.7 STEM CELL THERAPEUTIC OUTCOMES DATABASE (SCTOD) The CIBMTR administers the SCTOD contract for the HRSA‐sponsored C.W. Bill Young Cell SCTOD Contract Requirements Transplantation Program, established by the Stem Cell Therapeutic and Research Act of Collect HCT outcomes data for: 2005. Continued support is provided through the Stem Cell Therapeutic and Research All allogeneic HCTs performed in the Reauthorization Acts of 2010 and 2015. US using related or unrelated donors
C.W. Bill Young Cell Transplantation All allogeneic HCTs worldwide that Program Goals Fulfilled by the SCTOD use grafts procured through the C.W. Bill Young Cell Transplantation Collect, analyze, and report outcomes Program data for all allogeneic transplants and other therapeutic uses of blood stem Clinical applications of cells hematopoietic stem cells other than hematopoietic cell recovery Publicize information about HCT to patients, families, health care Use the data collected for the SCTOD to professionals, and the public evaluate the performance of centers
Define better processes for identifying Provide specific SCTOD data to the unrelated matched marrow donors, public PBSC donors, and cord blood units through one electronic system Collect a basic set of data for analyses of program use, center‐specific Increase availability of unrelated adult outcomes, donor registry, cord blood volunteer donors and cord blood units inventory size, and patient access to HCT Expand research to improve patient outcomes Establish a Related Donor‐Recipient Research Sample Repository Program Activities (Section 2.2) Annually, the CIBMTR publishes HCT volumes and performance data by transplant center Center‐Specific Volumes and Survival and provides public access to this information Analysis. As part of the contract to operate the via the HRSA Blood Cell Transplant website. SCTOD, the CIBMTR provides the annual Study Summaries for Patients. In conjunction volume of transplants performed at each with NMDP/Be the Match and the Consumer center and performs a center‐specific survival Advocacy Committee, the CIBMTR publishes analysis evaluating the one‐year survival rates lay summaries of CIBMTR publications for among US centers. The report assesses patients and their loved ones. In 2017, the transplants from both related and unrelated donors. CIBMTR published 8 patient‐friendly research summaries.
Page | 28 CIBMTR 2017 Annual Report 2.0 WHAT WE DO The most recent analysis was completed in neurologic, musculoskeletal, and September 2017 and contains information on endocrinologic diseases with the intent to all first allogeneic transplants performed in US treat relapsed / progressive disease, treat or centers from January 1, 2013, through prevent infections, and / or improve organ December 31, 2015. function. As of November 30, the cellular therapy data repository includes data for Center Outcomes Forums. The CIBMTR has patients. conducted 5 Center Outcomes Forums to >2,000 engage relevant stakeholders in the center‐ The CIBMTR Cellular Therapy Outcomes specific outcomes reporting process. The most Registry initiative (Section 2.1.2) expands and recent, October 2016, meeting was held in replaces the cellular therapy data repository to conjunction with a meeting of Implementing support more detailed data collection of more Quality and Value in HCT, sponsored by Be The therapies (e.g. CAR T‐cell therapy). In October Match’s Advisory Group on Financial Barriers 2017, the CIBMTR held a third Cellular Therapy in Transplantation. Recommendations were Forum to discuss how to further develop this generated related to the center‐specific registry. collection of quality of life data, and analysis, Funding development of tools to enhance centers’ quality improvement efforts. Support for the SCTOD is provided by the HRSA contract # HHSH250201700006C. Quality of Life Assessments. In 2011, the CIBMTR launched a pilot program at 5 adult How to Get Involved and 3 pediatric centers. The principal goal was All US centers performing allogeneic HCTs to test feasibility of center‐based recruitment provide data to the CIBMTR for the SCTOD. of patients to a quality‐of‐life data collection These data are used to generate reports, program, followed by communication between which are distributed to transplant center CIBMTR Coordinating Center staff and patients medical directors and posted on the HRSA to collect longitudinal quality‐of‐life Blood Cell Transplant website. information. This pilot program is unique in that the CIBMTR collects data directly from Publicly Available Reports developed patients rather than through transplant by the CIBMTR for the SCTOD centers. Accrual closed in 2013 with 390 patients enrolled. The manuscript was Transplant Outcomes and Data published in August 2017. Cellular Therapies. The SCTOD contract US Patient Survival Report mandates data collection on uses of cells found in bone marrow, peripheral blood, and US Transplant Data by Center Report umbilical cord blood for alternative therapeutic applications. US Transplant Data by Disease Report The CIBMTR captures uses of cells for the Transplant Activity Report treatment of diseases without the intention of replacing the recipient’s hematopoietic These reports are available on the function. These therapies include, but are not HRSA Blood Cell Transplant website. limited to, treatment of malignancies as well as infectious, cardiovascular, rheumatologic,
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2.8 CORPORATE PROGRAM The CIBMTR Corporate Program provides Corporate Studies and Projects. Corporate opportunities for industry collaborators to partners may contract with the CIBMTR to access CIBMTR data and statistical support to conduct a study, support a project involving address questions specific to their business more complex analyses, or license a specified needs through Corporate Membership as well data set. Organizations interested in funding a as Corporate Studies and Projects. study, such as one comparing HCT with one or more non‐HCT therapies, or using historical Corporate Membership. The CIBMTR controls, can contract with the CIBMTR for Corporate Membership program provides a data and / or expert statistical analyses. variety of resource materials to corporations needing access to the most current and Program Activities comprehensive data on HCT. These materials The CIBMTR engaged in 14 studies with are useful for Marketing Managers, Medical Directors, Research Directors, Product corporate partners in 2017. Currently 16 Managers, Case Managers, and Transplant organizations participate in the CIBMTR Coordinators. The CIBMTR offers 4 levels of Corporate Membership program, including 15 Corporate Membership, each described on the that joined or renewed this year. CIBMTR Corporate Membership Program How to Get Involved webpage. If you would like to learn more about the CIBMTR Corporate Program, visit the CIBMTR Corporate Membership Benefits Corporate Membership Program webpage or contact Sherry Fisher, Director of CIBMTR Report on Survival Statistics for Advancement, at [email protected] or BMT 414.805.0687. If you are a Corporate Member requesting analyses, please complete the Center Volumes Dataset Corporate Member Information Request Form Worldwide CIBMTR Directory of BMT on the CIBMTR website. Physicians
Reduced registration rates at CIBMTR meetings and educational forums, including the BMT Tandem Meetings
Access to CIBMTR data and resources
Page | 30 CIBMTR 2017 Annual Report 3.0 HOW WE SHARE KNOWLEDGE
3.0 HOW WE SHARE KNOWLEDGE
The CIBMTR is committed to sharing the data The CIBMTR shares its knowledge in different we collect as well as the information and ways. To determine the best way to access knowledge produced from our data and our specific types of CIBMTR knowledge, review extensive collaborations with investigators in Figure 3.1 and Tables 3.2‐3.5. the HCT field. Figure 3.1 How to Access CIBMTR Knowledge
Details provided in Tables 3.2‐3.5.
Page | 31 CIBMTR 2017 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Table 3.2 How to Access Information Table 3.3 How to Access Data Online at Online at cibmtr.org* cibmtr.org*
Information Data
GENERAL TYPES In addition to reviewing this report, Review the baseline and follow‐up data access the Facts and Figures report on available for recipients and donors on the Administrative and Progress Reports the Types of Data Available for webpage. Read editions of the quarterly Research or Request webpage. newsletter on the Newsletters webpage, and email cibmtr‐[email protected] to be STANDARD REPORTS added to the electronic distribution list. Access the Summary Slides, BMT Survival Statistics Report, Center ACTIVITIES Transplant Activity Report, Patient Review Section 2 or visit the What We Transplant Outcomes Reports, and Do webpage. Visit the Studies webpage, Center‐Specific Survival Reports via SCTOD webpage, or Corporate Support Table 3.6 or on the Slides and Reports webpage to learn more about CIBMTR webpage. research programs, the SCTOD, or the Corporate Program, respectively. ELECTRONIC RETURN OF CENTER DATA MEETINGS Utilize the eDBtC application on the Visit the BMT Tandem Meetings Portal site to download TED‐ and webpage to view agendas, register, selected CRF‐level variables that have reserve housing, and submit abstracts. been validated and processed in the TRAINING CIBMTR Research Database. Review Section 3.4 or visit the Training RESEARCH STUDY and Reference webpage to access the Propose a study as explained on the Center Reference Guide, Forms How to Propose a Study webpage, or Instruction Manual, FormsNet and participate in one of the existing AGNIS trainings, and online courses. studies listed on the Working PUBLICATIONS Committee Study Lists webpage. Review the CIBMTR’s >1,200 CUSTOM ANALYSIS publications on the Publication List Complete the Custom Information webpage. For summaries of selected Request Form or, for Corporate CIBMTR publications written specifically Members, the Corporate Member for patients and the lay public, visit the Information Request Form. Study Summaries for Patients webpage. OTHER OTHER Email [email protected] Email [email protected]
Page | 32 CIBMTR 2017 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Table 3.4 How to Access Tools Online Table 3.5 How to Access Biospecimens at cibmtr.org* Online at cibmtr.org*
Tools Biospecimens
CENTER PERFORMANCE SAMPLES TYPES AND INVENTORY ANALYTICS Review the >2 million samples On the Portal site, authorized users available in the Research Repository may compare their center’s data to via the Sample Types and Inventory aggregated center data filtered by Summary webpage. various factors, view their own one‐ year survival rate, or create and REQUESTING SAMPLES implement ad hoc queries. For studies that include recipient clinical outcome data, propose a study DISEASE RISK INDEX ASSIGNMENT as explained on the How to Propose a TOOL Study webpage. Access this tool on the DRI Assignment For studies that do not include clinical Tool webpage to categorize patients outcome data, review the How to undergoing allogeneic HCT for Request Samples from the Research hematologic malignancy by disease Sample Repository webpage. risk. OTHER PATIENT ONE‐YEAR SURVIVAL Email research‐[email protected] CALCULATOR FOR ALLOGENEIC TRANSPLANTS Transplant Center Medical Directors may access this tool on the Portal site to predict one‐year survival for individual allogeneic HCT recipients.
*If you are unable to access items using the electronic links provided, enter the underlined and italicized words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).
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Table 3.6 Standard Reports Published by the CIBMTR
Report Title Description Accessibility* CIBMTR Annual Information on the CIBMTR's goals Published on the CIBMTR Report and achievements as well as Administrative and Progress operational details on how the Reports webpage CIBMTR is funded, supported, Released: February promoted, and maintained Format: PDF CIBMTR Summary Charts and figures summarizing Published on the CIBMTR Slides current uses and outcomes of Summary Slides webpage allogeneic and autologous HCT; Released: February developed in conjunction with the Format: PPT BMT Tandem Meetings CIBMTR Facts and High level summary of CIBMTR fiscal Published on the CIBMTR Figures year accomplishments and high Administrative and Progress impact publications Reports webpage Released: September Format: PDF US Centers Annual Dataset containing center‐specific pre‐ Published on the HRSA Blood Cell Transplant Activity transplant patient‐, disease‐, and Transplant website Report transplant‐related characteristics data Released: September for nearly all allogeneic and a majority Format: PDF of autologous HCTs performed in the US annually since 2008 CIBMTR Report of Highly detailed report on survival Via Corporate Membership Survival Statistics for statistics that describes use and Program (Section 2.8) or by BMT outcome of autologous and allogeneic request from physicians for HCT in the >500 centers that have making treatment decisions or participated in the CIBMTR clinical investigators planning clinical studies to [email protected] Released: November Format: Word US Patient Center‐ Comparison of observed to expected Published on the Be The Match Specific Survival one‐year survival rates among centers Transplant Center Directory Report in the C.W. Bill Young Cell webpage; available as a Word Transplantation Program network; document upon request to evaluates outcomes for transplants [email protected] using both related and unrelated Released: December donors Format: Web
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Report Title Description Accessibility* US Patient Transplant Disseminated in 3 different reports: Published on the HRSA Blood Cell Outcomes US Patient Survival Report: 100‐ Transplant website day, 1‐year, and 3‐year survival rate Released: December estimates for US HCT recipients by Format: Web disease and donor type US Transplant Data by Center Report: Number of bone marrow and cord blood transplants performed at a specific center US Transplant Data by Disease Report: Number of bone marrow and cord blood transplants for a specific disease US Allogeneic Report containing patient, disease, Via Corporate Membership Transplant Activity donor HLA match, donor age, and Program (Section 2.8) Report gender match information for Released: January, April, July and allogeneic transplant activity in the US October since 2010 Format: PDF CIBMTR Newsletter Articles regarding Working Published on the CIBMTR Committees, the SCTOD, data Newsletters webpage and management and collection, and distributed via email; contact noteworthy events in the HCT cibmtr‐[email protected] to be community added to the distribution list Released: February, May, August and November Format: Web Study Summaries for Summaries of CIBMTR research Published on the CIBMTR Study Patients publications written for patients and Summaries for Patients webpage others in the lay public and the Be The Match Recent Research webpage Released: Ongoing Format: PDF
*If you are unable to access items using the electronic links provided, enter the underlined and italicized words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).
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3.1 INFORMATION REQUEST SERVICE The CIBMTR Information Request Service Table 3.7 Data Requests Addressed by the provides timely access to data on CIBMTR in 2017 transplantation to patients, physicians, Number of hospitals, pharmaceutical companies, Requestor insurance companies, and others involved in Requests healthcare. Requests range from simple Physician / Researcher 341 queries of patient, disease, and transplant Pharmaceutical / Biotech frequencies to those with greater complexity 32 Company involving specific data combinations and / or statistical analysis of outcomes. Patient or Relative 17 Student 16 Potential Reasons for Market Research Firm 11 Information Requests Federal Government Agency 5 Self‐education and decision making Cord Blood Bank 4 Patient counseling or clinical News Media 3 decision making Insurance Company 1 Presentation support TOTAL 430
Center assessments How to Access For more information about requesting data Clinical trial planning from the Research Database, visit the CIBMTR Market assessments How to Request Data webpage. If you would like a one‐time, custom analysis, complete the Custom Information Request Form on that Coordinating Center staff members fulfill webpage. If you have questions about requests related to clinical decision making requesting CIBMTR data, please contact within three days and most other requests [email protected]. within three weeks. If a request will take more than an estimated four weeks to fulfill, a Coordinating Center staff member will contact the requestor to discuss an appropriate timeline. Accomplishments In 2017, the CIBMTR fulfilled 430 requests for information and data (Table 3.7).
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3.2 INTERNET PRESENCE The CIBMTR Internet presence provides the Studies scientific community and the public with Working Committee Studies Lists access to HCT information. Current websites (4,324 unique page views in 2017) include general information about HCT and A summary of the planned, in‐progress, and CIBMTR activities; training and support; a recently published clinical outcomes studies shared communications and collaborative for each Working Committee. environment for member centers; and secure web access to CIBMTR data for Working Meetings Committees, centers, scientific investigators, Annual BMT Tandem Meetings Materials and CIBMTR staff members. (19,150 unique page views in 2017) 3.2.1 CIBMTR Public Website Provides access to agendas, handouts, and educational materials from specific meetings The CIBMTR public website (cibmtr.org) is at the BMT Tandem Meetings: Working unrestricted and provides information about Committee Meetings and Clinical Research the CIBMTR and its research. It supports the Professionals / Data Management Working Committees and BMT CTN with Conferences. information regarding proposal submission, access to a listing and summaries of all studies Reference Center in process, and access to a summary of all Summary Slides CIBMTR publications. The website facilitates (10,884 unique page views in 2017) data and information requests, and it provides Includes charts and figures summarizing access to all current and past data collections current uses and outcomes of allogeneic and forms, training manuals, and videos as well as autologous HCT. other materials for both investigators and data professionals. The website information is, in Web‐based US Transplant Reports part, supported by DISCO (Data and (24,945 unique page views in 2017) Information for Statistical Center Operations), Directs users to the Be The Match US Center an application which maintains data on >850 Listing Report and customizable reports of studies, >1,200 publications, and >2,700 patient survival and transplant available through the HRSA Blood Cell Transplant authors and their institutions at time of website. publication. In 2017, the CIBMTR public website had 374,025 unique page views. Publication List About CIBMTR (5,272 unique page views in 2017) Administrative and Progress Reports Searchable descriptive list of >1,200 (1,217 unique page views in 2017) publications resulting from the use of CIBMTR data and statistical resources. Provides access to the CIBMTR’s annual Progress Report, annual Summary of Accomplishments, and Manual of Operations.
Page | 37 CIBMTR 2017 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Newsletter Data Collection Forms (4,389 unique page views in 2017) (40,673 unique page views in 2017) Published 4 times per year. Articles feature Provides access to current and retired versions updates on Working Committees, the SCTOD, of the forms used by the CIBMTR to collect data management and collection, and standard data elements for all transplant noteworthy events in the HCT community. recipients. Patient Resources Training and Reference (5,563 unique page views in 2017) (74,314 unique page views in 2017) Includes lay summaries of CIBMTR research Provides access to a wide variety of CIBMTR articles as well as post‐transplant care data management training and reference recommendations for adult and pediatric materials. autologous and allogeneic HCT recipients to help patients and clinicians understand and 3.2.2 CIBMTR Collaborative Site plan for the specialized care of transplant The CIBMTR Collaborative site recipients. 8 lay summaries were published in (collaborate.cibmtr.org) uses the SharePoint 2017. Enterprise Collaboration platform to promote cooperative work among CIBMTR staff Statistical Resources members and provides a communication (5,417 unique page views in 2017) platform for specific studies and initiatives. Provides access to resources offered through This site is secured by username and the unique partnership between the CIBMTR password, and user‐specific security and MCW Division of Biostatistics, including credentials are assigned centrally. biostatistical publications, a series of statistical lectures targeted at basic and clinical The CIBMTR uses the site for storing and investigators, and research tools, such as the sharing protocol and consent documents, Disease Risk Index Assignment Tool. donor / recipient tracking tools, confidential committee information, data, manuscript Data Management drafts, and other relevant information. While Data Management Manual only two Working Committees currently use (273,065 unique page views in 2017) the Collaborate site on a regular basis, it is A comprehensive reference document for available to all Working Committees for completing CIBMTR data collection forms. The sharing information. manual also details reporting requirements, describes protocols and the consent process, and includes downloadable report forms. Data Back to Centers Applications (2,095 unique page views in 2017) Links to the Data Back to Centers (DBtC) enhanced DBtC (eDBtC) applications that provide CIBMTR member centers the ability to retrieve all TED‐ and selected CRF‐level data their center has reported to the CIBMTR through FormsNet or AGNIS (A Growable Network Information System).
Page | 38 CIBMTR 2017 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.2.3 CIBMTR Portal Site Data for RFI The CIBMTR Portal site (portal.cibmtr.org) In 2017, the CIBMTR launched the Data for RFI delivers applications and data to CIBMTR (Request for Information) application in centers and other partners. In 2017, external response to requests from the BMT visitors viewed 18,977 Portal pages. community and as part of CIBMTR’s growing 6 applications are currently hosted on this site: portfolio of tools to help centers access and use the outcome data they share with the Data Back to Centers CIBMTR. Data for RFI provides centers with the DBtC ‐ Data Back to Centers ability to access, view, reconcile and format The DBtC application provides authorized data submitted to CIBMTR for use in fulfilling users the ability to download CIBMTR TED‐ submissions to third party payers and other level data variables for their centers. The data organizations. Data for RFI leverages the same have been validated and processed in the data available in eDBtC but translates these to CIBMTR Research Database and are reviewed the standard RFI format developed by ASBMT. and refreshed quarterly. Legacy International This application also incorporates the standard Bone Marrow Transplant Registry (IBMTR) data ASBMT RFI rules for determining survival and from as far back as 1964 and some legacy for differentiating between adult and pediatric NMDP/Be The Match data from as far back as populations. Centers can export data into the 1987 are available. In 2017, 2,095 unique, standard format developed by ASBMT and then supplement with additional data not non‐CIBMTR visitors viewed 2,992 DBtC pages collected by CIBMTR. In 2017, 328 Data for RFI and downloaded data 443 times. sessions were accessed by 43 unique users. eDBtC ‐ Enhanced Data Back to Centers In 2016, CIBMTR deployed eDBtC in Qlikview, a Center Performance Analytics business intelligence and data analytics In 2016, the CIBMTR also deployed Center platform. eDBtC provides centers with self‐ Performance Analytics in the Qlikview service access to a limited set of their CRF‐ and platform. Center Performance Analytics TED‐level data, including descriptive statistics supports center performance and quality and outcomes. eDBtC enables centers to view initiatives by allowing authorized users to and filter outcomes, including a five‐year compare their center’s data to aggregated overall survival curve as well as acute graft‐ center data. Predefined filters for this versus‐host disease (GVHD), chronic GVHD, comparison include geographic region, and other outcomes, for the center's historical performance, volume of transplants population. An ad‐hoc query tab allows users as a proxy for size, and patient population to create and implement their own custom served. Centers can also view their center's query of these data. eDBtC data are extracted own one‐year survival rate, based on a rolling from the CIBMTR Research Database and are three‐year period of data included in the validated, reviewed, and refreshed monthly. Transplant Center Specific Survival dataset. Users are also able to export filtered data in Like eDBtC, users can create and implement Excel file formats. In 2017, 3,766 eDBtC their own customized, ad hoc query and sessions have been accessed by 242 unique export a download of the source dataset for users, and 463 eDBtC data downloads were their center. In 2017, 466 Center Performance accessed by 109 unique users. Analytics sessions were accessed by 96 unique users.
Page | 39 CIBMTR 2017 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Patient One‐Year Survival Calculator US Center Listing Report for Allogeneic Transplants Transplant Center Directory Accessible by medical directors, the Patient Provides transplant center specific information One‐Year Survival Calculator for Allogeneic about facilities, personnel, diseases treated, Transplants provides centers with a tool to cost, and transplant experience, including the predict one‐year survival for individual number of transplants performed and survival allogeneic HCT recipients. The calculator data rates by age, disease type, and disease stage. are updated annually to reflect new information contained in the center outcomes 3.2.5 Be The Match Clinical Website analysis. In 2017, 3,352 unique, non‐CIBMTR The Be The Match Clinical website visitors accessed the survival calculator 5,702 (bethematchclinical.org) is designed for times. clinicians, network participants, payors, and bioinformatics professionals. For clinicians, the Center Volumes Portal website provides access to evidence‐based The Center Volumes Portal allows centers to tools, clinical guidelines, outcomes data, and preview; correct, if necessary; and approve education courses on HCT. The website also center volume data published annually on the provides information specific to types of HRSA Blood Cell Transplant website. Centers network participants: Transplant centers, may display and download the previous five donor centers, apheresis and collection years of volume data (2012‐2016). This year, centers, and cord blood banks. For payors, the 1,657 external visitors accessed the Center website offers information to help individuals Volumes Portal 1,232 times. understand BMT, determine coverage, and answer employer and patient questions. 3.2.4 Be The Match Public Website Related to bioinformatics, the website provides resources for immunogenetic‐focused The Be The Match public website research and operational bioinformatics as (bethematch.org) is designed for patients and well as frequently used HLA tools. families, donors, and supporters. It incorporates detailed information about 3.2.6 HRSA Blood Cell Transplant Website transplantation and donation written for the public. The website provides scientific The HRSA Blood Cell Transplant website information in lay terms for donors related to (bloodcell.transplant.hrsa.gov) provides the donation process and for patients related information for the public, physicians, and to specific diseases, various treatment options, other constituents. It incorporates transplant the process of transplantation, and life after resources, donor information, and cord blood transplant. It also addresses concerns related information as well as research, data, and to specific populations, including children and outcomes. CIBMTR data and research findings caregivers. The CIBMTR collaborates with are incorporated in numerous ways, including NMDP/Be The Match to provide content for through provision of datasets and CIBMTR‐ several areas of this website, including data for created reports: the US Center Listing Report.
Page | 40 CIBMTR 2017 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Transplant Outcomes and Data AGNIS US Patient Survival Report AGNIS allows participating centers to Provides disease‐specific post‐HCT survival electronically collect and share data with the estimates by the length of time after CIBMTR as well as others who link to AGNIS. transplant: 100 days, 1 year, and 3 years. Data are entered once and then distributed Survival estimates are also available by patient and synchronized among databases. In 2017, a age, patient gender, patient race, or cell total of 26,090 forms for 15,091 patients source. were submitted through AGNIS by 25 US Transplant Data by US Center Report centers and by EBMT for 68 of their affiliated Displays the number of adult donor and cord centers. blood transplants performed at a specific BRIDG center. The BRIDG (Biomedical Research Integrated Transplant Data by Disease Report Domain Group) Model is an information Displays the number of adult donor and cord model, representing a shared view of the blood transplants reported for a specific concepts of basic, pre‐clinical, clinical, and disease. Totals are also available by patient translational research. Common data elements age, patient gender, patient race, cell source, (CDEs) for certain standard CIBMTR forms have and the year the transplant was performed. been extracted and associated in the BRIDG Transplant Activity Report model to one of three contexts: Recipient, Displays the number of transplants performed donor, or stem cell product. This year the at US centers, including autologous as well as CIBMTR modeled the CDEs contained in the related and unrelated allogeneic. Numbers are new Cellular Therapies forms to BRIDG 5.0 and also available by patient age, patient gender, is in the process of publishing these to the patient race, cell source, disease, center Cancer Data Standards Registry and location by state, and year in which the Repository. The CIBMTR is also upgrading all transplant was performed. existing CDEs with BRIDG mappings to the new BRIDG 5.0 standard. 3.2.7 Other Applications and Data Exchange Standards Disease Risk Index Assignment Tool The CIBMTR has multiple methods for sharing In 2015, the CIBMTR launched a Disease Risk data. Those hosted on the CIBMTR Portal site Index Assignment Tool developed by (DBtC, eDBtC, Data for RFI, Center investigators at the Dana Farber Cancer Performance Analytics, Patient One‐Year Institute and validated in a large CIBMTR Survival Calculator, and Center Volumes study. It is intended for use by clinical Portal) were described in Section 3.2.3. Other researchers. The tool was developed for the methods for sharing data are: primary outcome of overall survival after HCT and, at present, only applies to adult patients with hematologic malignancies. It is not intended to give an accurate prognosis for individual patients. In 2017, 2,315 unique, non‐CIBMTR visitors viewed the Disease Risk Index Assignment Tool 8,235 times.
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3.3 BMT TANDEM MEETINGS The BMT Tandem Meetings, co‐sponsored BMT Administrative Director Conference with the ASBMT, are held annually in February. With approximately 225 attendees, this They include 5 days of plenary sessions, conference focused on many topics related to concurrent scientific sessions, and other leadership and quality, including methods for meetings. Reports on recent progress in basic improving accuracy and value, staffing models science, translational research, and clinical and resilience, and Medicare coverage. studies are targeted to worldwide physicians, BMT Pharmacists Conference scientists, and other health professionals With 150 attendees, this conference interested in HCT. presented the latest research and best pharmacy practices with a focus on specific diseases and therapies. Transplant Nursing Conference 2017 BMT Tandem Meetings With 400 attendees, this conference presented the latest research and best nursing With attendees from countries, the 3,491 44 practices with a focus on communication 2017 BMT Tandem Meetings included 5 techniques and quality of life. plenary sessions, 9 concurrent sessions, 96 BMT Clinical Education Conference oral abstracts, 2 poster sessions, 9 corporate‐ This conference is designed for Nurse supported symposia, and 7 product theaters. Practitioners, Physician Assistants, Fellows, Continuing Medical Education (CME) and and Junior Faculty. With almost 300 Continuing Education credits were issued attendees, the conference focused not only on through MCW to physicians and allied health the latest clinical research but also ethics and professionals. In addition to the extensive resiliency. scientific agenda, many educational opportunities focused on young investigators and other allied health professionals. Clinical Research Professionals / Data Management Conference 2018 BMT Tandem Meetings With almost 250 attendees, this conference The 2018 BMT Tandem Meetings are expected provided forms and subject matter training, to attract approximately 3,500 attendees. The which increases the accuracy with which CIBMTR forms are completed. Meetings will include 5 plenary sessions, 9 concurrent sessions, 96 oral abstracts, 1 late BMT CTN Coordinators and Investigators breaking abstract session, 2 poster sessions, Meetings corporate‐supported symposia, and With approximately 100 and >400 attendees, 8 respectively, these meetings focused on study 9 product theaters. management, such as promoting studies and reporting adverse events; procedures, such as enrollment; specific clinical trials; and general HCT subject matters.
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3.4 DATA MANAGEMENT TRAINING The CIBMTR has developed comprehensive, Newsletters and eBlasts secure, and efficient applications to allow Read archived issues of the Data Matters centers to electronically submit data to the Training Newsletter and eBlasts. CIBMTR. Visit the CIBMTR Data Management Tip Sheets Training and Reference webpage to access Access sheets providing tips and instructions resources. for various CIBMTR forms. Data Management Guide Online Training Learn about participation in CIBMTR research, Review educational modules developed for center membership, access to FormsNet, data new and seasoned data managers. Modules manager education, mentor program, forms are available on the Online Training webpage. submission process and many useful tips and links. Online Trainings Manuals New this Year Find the answers to your data submission questions by accessing the Forms Instructions APPLICATION SERIES Manual, which includes general instructions and instructions for each form type. FormsNet3 Recipient Application Training FormsNet Data for RFI (Request for Information) Learn how to submit data to the CIBMTR via the FormsNet application, a secure clinical eDBtC Data Sharing research management system, which is in Center Performance Analytics Tutorial compliance with SCTOD requirements. Query Functionality Conference Materials HLA SERIES Access meeting materials as well as audio and visual presentations on the form submission Genetics for HLA process presented at Clinical Research DISEASE SPECIFIC SERIES Professionals / Data Management Conferences. Multiple Myeloma 101 (2 parts) AGNIS Veno‐Occlusive Disease / Sinusoidal Learn how to retrieve and transmit form data, Obstructive Syndrome (VOD / SOS) extracted directly from your own institution’s Reporting Form 2553 database, directly to the FormsNet application Institutional Review Board (IRB) using AGNIS, a secure, standards‐based system. NMDP IRB New Member Orientation
Legacy Data GENERAL TOPICS Review retired data manuals, forms, and other archived documents for reference purposes Research Sample Submission and to assist in making changes to legacy data. Adverse Events Learn how to report adverse events and product issues through FormsNet.
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4.0 HOW WE COLLECT AND MANAGE DATA
4.1 RESEARCH DATA LIFE CYCLE The Research Data Life Cycle (Figure 4.1) Data Sharing completes the cycle, providing describes the path of data from the point of data for analysis that have been collected and capture to its ultimate use in analysis, curated to ensure research value. These data reporting, and publication. are extracted from the Research Database in The process begins with data collection. Most monthly and quarterly retrievals to serve a centers enter data in FormsNet, a web range of research and stakeholder needs. The application now in its third generation. Centers data retrievals provide the basis for research data files, reports, and externally‐ that have implemented local or third‐party study systems can also capture and submit data requested datasets. TED‐level data are also electronically using AGNIS. The goal of these directly available to centers through use of the applications is to capture high quality data as DBtC application (Section 3.2.3). efficiently as possible. Following collection, data undergo quality assessment and validation and are extracted and loaded into the CIBMTR Research Database on a monthly basis.
Figure 4.1 Research Data Life Cycle
Abbreviations: AGNIS = A Growable Network Information System, CRID = CIBMTR Recipient Identification Number, CRF = Comprehensive Report Form, FN = FormsNet, IDW = Integrated Data Warehouse, RDB = Research Database
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4.2 COLLECTING AND STORING DATA
4.2.1 FormsNet 4.2.3 Integrated Data Warehouse More than 98% of data collected by the Looking to the future, the CIBMTR continues to CIBMTR is submitted electronically via develop an Integrated Data Warehouse to FormsNet, a comprehensive electronic data robustly support future database submission system containing >120 forms requirements. The Integrated Data Warehouse related to capturing HCT outcomes for donors uses Oracle's enterprise database and business and recipients. The application was updated in intelligence infrastructure. The warehouse’s 2017 to provide key enhancements to support implementation makes use of industry operational efficiencies. The CIBMTR also standard data warehousing design patterns in released a number of new forms this year in a addition to some novel techniques for continuing effort to maintain alignment managing metadata and data quality. The between data collection and treatment Integrated Data Warehouse is intended to practices as the HCT field changes and serve as both the aggregation and improves. Additionally, all updated forms have dissemination point for data collected by the been dual purposed to be appropriate for CIBMTR to support its data and research either HCT or cellular therapies, with a core set programs as well as external data of cellular therapy forms also released. dissemination. The warehouse accommodates the integration of data coming from multiple 4.2.2 Research Database sources and spanning multiple domains, such as patient outcomes, product data, HLA, The CIBMTR Research Database now contains biospecimens, and study data. information on >475,000 patients. Submission of outcomes data is mandatory for This year the CIBMTR completed integration of allogeneic HCTs in the US and those outside new data sources to meet warehouse users’ the US that use a US donor; all other reporting needs. This integration includes submissions are voluntary. The CIBMTR sample data stored in LabVantage system, HLA and match grade data, prospective research estimates that almost 100% of US allogeneic data stored in Clinical Trials Rave (an electronic transplants and about 80% of US autologous data capture and clinical trials management HCTs are reported. Additionally, the CIBMTR system) software, and FormsNet Cell Therapy receives data for approximately 7,600 non‐US forms data. The CIBMTR also completed a patients annually. proof of concept extract for the Transplant Center Specific Analysis, in support of advancing the Integrated Data Warehouse. Furthermore, the CIBMTR implemented a revised suite of cord blood outcome reports to assist cord blood banks with quality assurance processes, and it provided a Business Intelligence environment for internal Audit staff, which provides end user defined reports and ad hoc analysis capabilities.
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4.3 ENSURING DATA QUALITY
4.3.1 Continuous Process Improvement 4.3.2 Verification and Validation Robust data collection is critical to the success FormsNet of the CIBMTR. The Continuous Process When data are entered into FormsNet, a series Improvement (CPI) program ensures timeliness of entry level validation checks takes place to and completeness of data forms submissions ensure data consistency. This process flags (Appendix H). certain errors at the time of entry and allows Recipient Forms the CIBMTR to contact the center data manager so errors can be corrected Throughout each trimester, US centers receive immediately while source documents are CPI reports listing the number of follow‐up readily available. If a data field does not pass forms that were due in the previous trimester and the number and percentage of each the FormsNet validation checks, an error submitted within the trimester. Letters are comment is generated, and the data manager is navigated to an error review page to review, sent 3 times per year (January, May, and resolve, or override the unresolved errors. September) to the Medical Director and Lastly, an error report is generated that lists Primary Data Manager notifying them if their any unresolved errors as well as errors that center met CIBMTR CPI criteria for the have been overridden. trimester. A form is not officially submitted until errors are resolved and all applicable The CIBMTR reacts to data quality issues information is submitted and approved. To be detected downstream by incorporating compliant, centers must submit ≥90% of additional data quality validations in the forms due for the trimester, for all transplants upstream data capture system (FormsNet). (autologous and allogeneic, related and Detection of issues earlier in the process unrelated), which occurred since December 3, provides real time feedback to the parties 2007. most capable of correcting the problem and improves the overall quality of the data. Donor Forms
The Donor Data Management Team oversees submission of donor work‐up and donation forms from NMDP/Be The Match donor, collection, and apheresis centers. Donor CPI reports are generated 4 times per year (January, April, July, and October) for US centers. To be compliant, centers must submit 100% of the forms required for that CPI period.
Page | 46 CIBMTR 2017 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA Research Database Consolidation of FACT‐CIBMTR Audits Previously, the CIBMTR and Foundation for the Data extracted from FormsNet and loaded into Accreditation of Cellular Therapy (FACT) the Research Database each month undergo individually audited data quality at centers. To comprehensive validation and verification. diminish duplications in the parallel programs These data are rigorously validated for and ease the reporting and compliance consistency, completeness, and uniqueness burdens for centers, the organizations using business rules implemented in custom consolidated data audit efforts this year. logic for the categories provided below. The Within this collaboration, the CIBMTR Data Quality Team reviews errors and works conducts data quality audits and evaluations with centers to correct data. on behalf of both organizations, and FACT Rules across various categories are updated determines whether the results of a data and tested as part of ongoing Forms Revision. quality audit are satisfactory for accreditation. The CIBMTR continues work to reduce the number of data entry errors. Currently, the All verification of the accuracy of data against CIBMTR audit teams on rate of form rejection due to inconsistently source data is done by site according to their current practices and reported data is <2% and has decreased schedules. FACT verifies at each annual report because of recent enhancements in center and at each application for renewal education as well as enhanced validations built accreditation the status of CIBMTR data in at the point of entry in FormsNet. accuracy by requiring submission of centers’ 4.3.3 On‐Site Data Audit Program most recent CIBMTR audit results for error rates (random errors, systematic errors, and On‐going data audits are performed at all critical field error rates). FACT verifies CIBMTR participating centers. The audit completeness of data by requiring each center compares data in source documents to annually submit the most recent CPI report maintained at the center with data contained from the CIBMTR that demonstrates “In Good in the CIBMTR Research Database. Clinical Standing” related to the on‐time submission of Research Associates perform the on‐site completed reports at the rate of at least 90% center audits, spending 3‐4 days at each center of expected. reviewing original source documents. The overall audit process is displayed in Figure 4.2. Transplant programs submitting an Annual Report or Renewal Application to FACT are In 2017, 57 centers were scheduled for audit reviewed during the monthly FACT / CIBMTR (46 domestic, 11 international). As of Data Audit Committee meeting. Successful December 1, 2017, 56 centers were notified of FACT accreditation will depend on satisfactory their final audit results, including requested completion of this process. corrective action follow‐up. Of the centers sent reports, 82% passed with ≤3% critical field errors. Of the 10 centers that did not pass the audit, 5 completed all required corrective action; the remaining 5 centers are in the process of completing requested corrective action.
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Figure 4.2 Audit Process
Page | 48 CIBMTR 2017 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA
4.4 PROTECTING PATIENTS AND DATA Since 2008, the CIBMTR has held an Authority 4.4.1 Human Subjects / HIPAA Compliance to Operate from the Chief Information Officer The CIBMTR is committed to the ethical of HRSA. The certification was renewed in June conduct of research. All Coordinating Center 2015, and security audits are performed personnel maintain Collaborative IRB Training annually, most recently in September 2017. Initiative (CITI) certification. The NMDP/Be The The NMDP/Be The Match also holds an Match IRB, which is fully accredited by the Authority to Operate from HRSA, ensuring Association for the Accreditation of Human similar standards of information security are Research Protection Programs, reviews all applied to all CIBMTR and NMDP/Be The human subject research conducted by the Match systems. These controls, maintained by CIBMTR. The CIBMTR maintains compliance the CIBMTR and NMDP/Be The Match, protect with the Health Insurance Portability and the data and information in these systems in Accountability Act (HIPAA) Privacy Rule, as ways beyond those required by HIPAA. applicable. CIBMTR rules requiring the registration of all consecutive HCT recipients ensure the inclusion of women, minorities, and children, so the Research Database population includes women and minorities in the same proportion as found in the general HCT population. Children are included in most CIBMTR studies; their inclusion is dependent on the study focus.
4.4.2 Information Security and Data Privacy The CIBMTR protects the data and information received from centers and patients. The SCTOD contract requires specific protections through minimum security controls, policies, and standards. The CIBMTR’s data systems are maintained in accordance with the Federal Information Systems Management Act of 2002, and with information security guidance provided by the National Institute of Standards and Technology. In accordance with National Institute of Standards and Technology Special Publication 800‐18, and in collaboration with HRSA’s Office of Information Technology, the CIBMTR maintains a System Security Plan that outlines management, operational, and technical controls.
Page | 49 CIBMTR 2017 Annual Report 5.0 WHAT WE WILL DO NEXT
5.0 WHAT WE WILL DO NEXT
In 2018, the CIBMTR will continue to conduct high quality research through a collaborative process. Plans are listed in Tables 5.1‐5.3.
Table 5.1 Plans to Enhance Data
Enhance the quality and scope of CIBMTR data
Further develop the Cellular Therapy Outcomes Registry, including revision of data collection forms
Harmonize cellular therapy data collection with global partners
Establish the capability for whole genome sequence analysis of donor‐recipient HCT pairs with the goal of identifying markers associated with improved outcomes
Continually implement key data validations in FormsNet, close to the point of data capture
Enhance functionality within clinical trial and survey research data collection systems related to risk‐based monitoring, lab sample management, and patient reported outcomes
Implement initial phases of CPI for forms submission for international centers reporting to the CIBMTR
Improve ability to make changes to forms more frequently
Increase flexibility in how and when CIBMTR releases software
Expand onsite and online data management training support for centers
Implement electronic collection of PROs as a data resource for clinical investigation
Design data collection for a Regenerative Medicine Outcomes Registry
Page | 50 CIBMTR 2017 Annual Report 5.0 WHAT WE WILL DO NEXT Table 5.2 Plans to Expand Knowledge Sharing
Expand data and knowledge sharing
In collaboration with the cellular therapy community, evaluate and improve the quality and integrity of data submitted on CTED forms
Extend features of eDBtC to deliver cellular therapy data back to centers as well as methods to visualize these data in meaningful ways and link these data to relevant HCT data
Provide access to the US Cord Blood Quality Reports in the Data Analytics / Data Sharing environment to cord blood banks
In collaboration with ASBMT, host the BMT Tandem Meetings to share the latest developments in HCT basic science, translational research, and clinical studies
Publish easy to read summaries of key HCT publications
Continue curation of data elements aligned with accepted standards, including the Cancer Data Standards Registry and Repository, Clinical Data Interchange Standards Consortium (CDISC), and BRIDG model standards, in order to support electronic interoperability
Strengthen collaborations with international partners to facilitate data sharing and joint research studies
Explore integration with the Cancer Data Ecosystem and use of HL7/FHIR to integrate with electronic medical records systems
Expand regenerative medicine data collection
Explore linking with administrative payer claims data to facilitate research on access to, utilization of, and value of HCT
Page | 51 CIBMTR 2017 Annual Report 5.0 WHAT WE WILL DO NEXT Table 5.3 Plans to Increase Impact
Increase research productivity and scientific impact
Focus effort on studies with highest scientific impact and important clinical and policy implications
Support Working Committee Chairs to be active leaders in their committees
Regularly track and share performance metrics as a Working Committee management resource
Continue to provide data to CMS for CED studies to allow patients to receive transplants while developing evidence for their efficacy
Expand use of outcomes data to assist in the design, implementation, and long‐term follow‐up of clinical trials
Explore opportunities for innovative and important collaborative studies within the RCI BMT and Health Services Research Program
Develop new statistical methodologies
Evaluate opportunities to expand the collection and utilization of research samples for HCT and cellular therapy correlative research
Utilize an ePRO system to support clinical trials, long term follow‐up, and other research studies with patients and donors
Enhance data sharing by supporting public access to publication datasets to facilitate collaboration, validation, and expansion of research findings
Continue to partner with professional societies to translate advances in disease risk stratification and transplantation into clinical practice
Page | 52 CIBMTR 2017 Annual Report 2017 KEY ACCOMPLISHMENTS
2017 KEY ACCOMPLISHMENTS
CIBMTR by the Numbers Clinical Outcomes Research Program RESEARCH DATABASE >420 participating centers WORKING COMMITTEES >475,000 patients 15 committees with >2,800 researchers ~23,000 new patients annually worldwide PUBLICATIONS 47 HCT global experts voluntarily chair committees >1,200 publications since inception 175 ongoing studies 82 publications in 2017 59 from Working Committees 207 new study proposals; >35% were submitted by new investigators 10 from Bioinformatics Research Program 30 abstracts (17 oral and 13 poster) 7 from BMT CTN presented at national and international conferences 4 from Statistical Methodology Research Program 59 manuscripts published in peer‐ 2 from Health Services Research reviewed journals by 750 authors at 300 Program institutions worldwide PRESENTATIONS CELLULAR THERAPY AND OTHER RESEARCH INITIATIVES 71 presentations in 2017 (38 oral and 33 poster) Third Cellular Therapies Forum 33 abstracts (16 oral and 17 poster) New versions of cellular therapy data presented at the 2017 ASH collection forms Annual Meeting Pilot study of cellular therapy data 19 abstracts (13 oral and 6 poster) collection completed presented at the 2017 BMT Tandem Meetings Regenerative Medicine Outcomes 19 abstracts (9 oral and 10 poster) Registry strategy meeting presented at other national and New CMS CED study for sickle cell international conferences disease
Page | 53 CIBMTR 2017 Annual Report 2017 KEY ACCOMPLISHMENTS
Immunobiology Clinical Trials Research Program Support Program
Research sample collection and BMT CTN correlative testing for BMT CTN and RCI BMT prospective studies 4 new trials opened to accrual (46 overall) New Research Repository inventory management system >650 patients accrued to trials (>9,900 overall) Samples received from 182 centers (130 transplant centers, 35 donor centers, 14 open protocols with overall accrual and 17 cord blood banks) at about 98% of projections High resolution HLA and KIR typing on 17,471 new protocol‐related 613 related and 3,268 unrelated HCT biospecimens (398,311 overall) donor / cord and recipient pairs 17 abstracts (12 oral and 5 poster) 8,289 samples distributed to presented at national and international investigators for various studies conferences RESEARCH REPOSITORY 7 manuscripts published in peer‐ reviewed journals (approximate numbers) 4,300 new unrelated recipient samples RCI BMT (60,000 overall) 1 new study opened to accrual 1,400 new related recipient samples (17 overall) (8,100 overall) >3,000 patients accrued (approximately 4,300 new adult unrelated donor 33,500 overall) samples (67,000 overall) 5 abstracts (2 oral and 3 poster) 1,300 new related donor samples presented at national and international (7,800 overall) conferences 700 new unrelated cord blood samples 5 active studies supported and 2 (11,700 overall) upcoming studies developed by the Survey Research Group
Page | 54 CIBMTR 2017 Annual Report 2017 KEY ACCOMPLISHMENTS
Health Services Bioinformatics Research Program Research Program
AML MATTERS: Assessed AML Guidelines for reporting HLA and KIR knowledge and practice gaps among genotyping via Next Generation US health care professionals in Sequencing developed partnership with ASH; held 4 national Role of genetic ancestry in summits and a special education transplantation investigated session at ASH Annual Meeting HLA data from other countries Transplant physicians’ perspective on investigated palliative care in HCT: Survey conducted in partnership with ASBMT 8 abstracts (2 oral and 6 poster) presented at national and INSPIRE study, which provides stepped international conferences care self‐management program for survivors: Developed in partnership 10 manuscripts published in peer‐ with Fred Hutchinson Cancer Research reviewed journals Center and funded by NIH
Building a Patient‐Centered Outcomes Research Collaborative Community Symposium and 2 continuing education Statistical Methodology webinars: Prioritized comparative Research Program effectiveness research questions disseminated New statistical models developed Easy‐to‐read informed consent (BMT CTN 1205): Study completed Statistical integrity of CIBMTR scientific activities ensured 6 abstracts (1 oral and 5 poster) presented at national and Articles on HCT‐related statistical international conferences issues for clinical audiences supported 2 manuscripts published in peer‐ Working Committee study reviewed journals investigators supported in developing scientific study protocols using CIBMTR data 4 manuscripts, 3 published in peer‐ reviewed journals
Page | 55 CIBMTR 2017 Annual Report 2017 KEY ACCOMPLISHMENTS
SCTOD
SCTOD contract with HRSA renewed Annual Center‐Specific Survival Report for 2013‐2015, Transplant Center Volumes Data for 2011‐2015, and a Transplant Activity Report for transplants performed 2010‐2014 published 3‐year update of the report which analyzed the size, composition, and growth rate of the National Cord Blood Inventory and Adult Donor Registry produced Tools enhanced to support centers’ quality improvement efforts Quality of life manuscript published in August 2017; the project was unique in that the CIBMTR collected data directly from patients rather than through transplant centers >16,200 samples in the Related Donor‐ Recipient Sample Repository 8 lay summaries of CIBMTR research publications developed in conjunction with NMDP/Be The Match and the Consumer Advocacy Committee
Page | 56 CIBMTR 2017 Annual Report APPENDIX A: CENTERS APPENDIX A: CENTERS
Figure A.1 Location of Centers that Submit Data to the CIBMTR
Page | 57 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
APPENDIX A1: US CENTERS
The following table lists the US‐based centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED and CRF.
Participating Center City State MUD RELATED AUTO University of Alabama at Birmingham Birmingham AL CRF CRF CRF
Huntsville Hospital Huntsville AL N/A N/A TED
University of Arkansas for Medical Sciences Little Rock AR CRF TED TED
Banner MD Anderson Cancer Center Gilbert AZ N/A CRF CRF
Mayo Clinic Arizona and Phoenix Children's Phoenix AZ CRF CRF CRF Hospital Cancer Transplant Institute at Virginia G. Piper Scottsdale AZ CRF CRF CRF Cancer Center University of Arizona Medical Center Tucson AZ CRF TED TED
Alta Bates Comprehensive Cancer Center Berkeley CA N/A N/A CRF
City of Hope Duarte CA CRF TED TED
Scripps Blood & Marrow Transplant Program La Jolla CA CRF CRF CRF
University of California, San Diego Medical La Jolla CA CRF CRF CRF Center Loma Linda University Cancer Center Loma Linda CA CRF CRF CRF
Cedars‐Sinai Medical Center Los Angeles CA CRF TED N/A
Children's Hospital of Los Angeles Los Angeles CA CRF TED TED
UCLA Health Los Angeles CA CRF CRF TED
USC BMT Program Los Angeles CA CRF CRF CRF
Children's Hospital & Research Center Oakland Oakland CA CRF CRF CRF
Children's Hospital of Orange County Orange CA CRF CRF CRF
Page | 58 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO Lucile Packard Children's Hospital / Stanford Palo Alto CA CRF CRF TED Children's Health Sutter Medical Center Sacramento CA TED TED TED
University of California‐Davis Cancer Center Sacramento CA CRF TED TED
Rady Children's Hospital, San Diego San Diego CA CRF CRF CRF
University of California San Francisco Medical San Francisco CA CRF CRF CRF Center University of California San Francisco Medical San Francisco CA CRF CRF CRF Center ‐ Peds Stanford Health Care Stanford CA CRF CRF TED
University of Colorado ‐ Children's Hospital Aurora CO CRF CRF CRF
University of Colorado Hospital Aurora CO CRF TED TED
Colorado Blood Cancer Institute Denver CO CRF TED TED
Yale New Haven Hospital New Haven CT TED TED TED
Children's National Medical Center Washington DC CRF TED TED
MedStar Georgetown University Hospital's Washington DC CRF N/A CRF Stem Cell Transplant and Cellular Immunotherapy Program Christiana Care Newark DE CRF CRF CRF
Medical Oncology Hematology Consultants, PA Newark DE CRF CRF CRF
Alfred I. duPont Hospital for Children Wilmington DE CRF CRF CRF
Shands HealthCare & University of Florida Gainesville FL CRF CRF CRF
Mayo Clinic Florida Jacksonville FL CRF CRF CRF
Nemours/Wolfson Children's Clinic & Hospital Jacksonville FL CRF CRF CRF
Rejuva Stem Cell Center LLC Jupiter FL CRF CRF N/A
Page | 59 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO Baptist Hospital of Miami Miami FL N/A N/A CRF
Miami Children's Hospital Miami FL CRF TED TED
University of Miami Miami FL CRF CRF CRF
University of Miami/Jackson Memorial Hospital Miami FL CRF TED TED
Blood & Marrow Transplant Center, Florida Orlando FL CRF CRF CRF Hospital Medical Group Florida Center for Pediatric Cellular Therapy Orlando FL TED TED TED
Memorial Cancer Institute Pembroke FL TED TED TED Pines Johns Hopkins All Children's Hospital St. Petersburg FL CRF CRF CRF
H. Lee Moffitt Cancer Center and Research Tampa FL CRF TED TED Institute Children's Healthcare of Atlanta at Egleston Atlanta GA CRF CRF CRF
Emory University Hospital Atlanta GA CRF CRF CRF
The Blood and Marrow Transplant Program at Atlanta GA CRF CRF CRF Northside Hospital Georgia Cancer Center at Augusta University Augusta GA CRF CRF CRF
Cancer Treatment Centers of America ‐ Newnan GA CRF CRF CRF Southeastern Regional Medical Center Kapi'olani Medical Center for Women and Honolulu HI CRF CRF CRF Children University of Iowa Hospitals & Clinics ‐ 2 Iowa City IA CRF CRF CRF
St. Luke's Mountain States Tumor Institute Boise ID N/A N/A CRF
Ann & Robert H. Lurie Children's Hospital of Chicago IL CRF CRF CRF Chicago Comer Children's Hospital/University of Chicago IL CRF CRF CRF Chicago Medicine Northwestern Medicine Chicago IL CRF CRF TED
Rush University Medical Center Chicago IL CRF CRF CRF
Page | 60 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO University of Chicago Medicine Chicago IL CRF CRF CRF
University of Illinois Medical Center at Chicago Chicago IL CRF CRF CRF
NorthShore University HealthSystem Evanston IL N/A N/A CRF
Loyola University Medical Center Maywood IL CRF CRF CRF
Advocate Lutheran General Hospital Park Ridge IL CRF CRF CRF
Cancer Treatment Centers of America Zion IL CRF CRF CRF
Indiana Blood & Marrow Transplantation Indianapolis IN CRF CRF CRF (IBMT) Indiana University Hospital/Riley Hospital for Indianapolis IN CRF CRF TED Children University of Kansas Kansas City KS CRF CRF CRF
Via Christi Hospitals Wichita, Inc. Wichita KS N/A CRF CRF
University of Kentucky Chandler Medical Lexington KY CRF TED TED Center University of Louisville Hospital/James Brown Louisville KY CRF TED TED Cancer Center Children's Hospital / LSUHSC New Orleans LA CRF CRF CRF
Ochsner Medical Center New Orleans LA CRF CRF TED
Tulane University Medical Center New Orleans LA CRF CRF CRF
Louisiana State University Health Sciences Shreveport LA TED TED TED Center‐ Shreveport Beth Israel Deaconess Medical Center Boston MA CRF TED TED
Boston Medical Center Boston MA N/A N/A TED
Dana Farber Cancer Institute ‐ Adults Boston MA CRF CRF TED
Dana Farber Cancer Institute ‐ Peds Boston MA CRF CRF TED
Massachusetts General Hospital Boston MA CRF TED TED
Page | 61 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO Tufts Medical Center Boston MA CRF TED TED
Lahey Clinic Medical Center Burlington MA N/A N/A TED
UMass Memorial Medical Center Worcester MA CRF CRF CRF
Greenebaum Cancer Center U M of MD Baltimore MD CRF CRF CRF
The Sidney Kimmel Comprehensive Cancer Baltimore MD TED TED TED Center at Johns Hopkins National Cancer Institute ‐ NIH Bethesda MD N/A TED N/A
National Heart Lung and Blood Institute ‐ NIH Bethesda MD N/A TED N/A
National Institutes of Health ‐ MUD Program Bethesda MD CRF N/A N/A
NIAID ‐ NIH Bethesda MD N/A TED N/A
NIH‐NCI Experimental Transplantation and Bethesda MD N/A CRF CRF Immunology Branch The University of Michigan Ann Arbor MI CRF TED TED
Children's Hospital of Michigan Detroit MI CRF CRF CRF
Henry Ford Hospital Bone Marrow Transplant Detroit MI CRF CRF CRF Program Karmanos Cancer Institute Detroit MI CRF CRF CRF
Helen DeVos Children's Hospital Grand Rapids MI CRF CRF CRF
Spectrum Health Grand Rapids MI CRF CRF CRF
University of Minnesota Blood and Marrow Minneapolis MN CRF CRF CRF Transplant Program ‐ Adults University of Minnesota Blood and Marrow Minneapolis MN CRF CRF CRF Transplant Program ‐ Pediatrics Mayo Clinic Rochester Rochester MN CRF CRF CRF
The Children's Mercy Hospitals and Clinics Kansas City MO CRF CRF CRF
Barnes Jewish Hospital St. Louis MO CRF CRF TED
Page | 62 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO Cardinal Glennon Children's Medical Center St. Louis MO CRF CRF CRF
SSM Health Saint Louis University Hospital St. Louis MO CRF TED TED
Washington University/St. Louis Children's St. Louis MO CRF CRF CRF Hospital University of Mississippi Medical Center Jackson MS CRF TED TED
Billings Clinic Cancer Center Billings MT N/A N/A TED
University of North Carolina Hospitals Chapel Hill NC CRF CRF CRF
Levine Cancer Institute Charlotte NC N/A CRF CRF
Levine Children's Hospital Charlotte NC CRF CRF CRF
Duke University Medical Center Durham NC CRF TED TED
Duke University Medical Center; Pediatric Durham NC CRF CRF CRF Blood and Marrow Transplant Pediatric Immunology Bone Marrow Transplant Durham NC N/A TED N/A Center Wake Forest Baptist Health Winston‐Salem NC CRF TED TED
Nebraska Medicine Omaha NE CRF CRF CRF
Nebraska Methodist Hospital Omaha NE N/A N/A TED
Dartmouth Hitchcock Medical Center Lebanon NH CRF CRF CRF
Hackensack University Medical Center Hackensack NJ CRF CRF CRF
Robert Wood Johnson University Hospital New Brunswick NJ CRF CRF CRF
UNM Comprehensive Cancer Center Albuquerque NM N/A N/A TED
Children's Hospital at Montefiore Bronx NY CRF TED TED
Montefiore Medical Center Bronx NY CRF CRF TED
Roswell Park Cancer Institute Buffalo NY CRF CRF CRF
Page | 63 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO Westchester Medical Center Hawthorne NY CRF CRF CRF
North Shore University Hospital Lake Success NY CRF TED TED
Steven and Alexandra Cohen Children's New Hyde Park NY CRF CRF CRF Medical Center of New York Memorial Sloan Kettering Cancer Center ‐ New York NY CRF TED TED Adults Memorial Sloan Kettering Cancer Center ‐ Peds New York NY CRF TED TED
Morgan Stanley Children's Hospital of New New York NY CRF CRF CRF York‐Presbyterian ‐ Columbia University Medical Center Mount Sinai Medical Center ‐ 2 New York NY CRF CRF TED
New York Presbyterian Hospital at Cornell New York NY CRF CRF CRF
New York University Langone Medical Center New York NY CRF CRF CRF
NYPH/ Columbia University Medical Center New York NY CRF CRF CRF
Strong Memorial Hospital ‐ Univ. of Rochester Rochester NY CRF CRF CRF Med Ctr Stony Brook University Medical Center, SUNY Stony Brook NY TED TED CRF
SUNY University Hospital Syracuse NY N/A CRF CRF
Children's Hospital Medical Center of Akron Akron OH CRF CRF CRF
Cincinnati Children's Hospital Medical Center Cincinnati OH CRF CRF CRF
Jewish Hospital Blood and Marrow Transplant Cincinnati OH CRF CRF CRF Center University of Cincinnati Medical Center Cincinnati OH CRF CRF CRF
Cleveland Clinic Foundation Cleveland OH CRF CRF CRF
Seidman Cancer Center‐University Hospitals Cleveland OH CRF CRF CRF Cleveland Medical Center Nationwide Children's Hospital Columbus OH CRF CRF CRF
Page | 64 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO Ohio State Medical Center, James Cancer Columbus OH CRF CRF CRF Center HCA Health Services of Oklahoma, Inc., Univ of Oklahoma City OK CRF CRF CRF OK Saint Francis Hospital Tulsa OK N/A TED TED
Legacy Good Samaritan Hospital and Medical Portland OR N/A N/A CRF Center Oregon Health and Science University Portland OR CRF CRF CRF
Pediatric Blood & Marrow Transplant Program, Portland OR CRF CRF CRF Doernbecher Children's Hospital, OHSU Providence Portland Medical Center Portland OR N/A N/A CRF
The Center for Bone Marrow Transplantation Danville PA CRF TED TED at Geisinger Penn State Hershey Medical Center Hershey PA CRF CRF CRF
Abramson Cancer Center University of Philadelphia PA CRF CRF CRF Pennsylvania Medical Center Children's Hospital of Philadelphia Philadelphia PA TED TED N/A
Eastern Regional Medical Center Philadelphia PA N/A CRF CRF
Fox Chase Temple University Hospital Bone Philadelphia PA CRF TED TED Marrow Transplant Program Hahnemann University Hospital Philadelphia PA CRF TED TED
Pennsylvania Hospital Philadelphia PA N/A N/A CRF
St. Christopher's Hospital for Children Philadelphia PA TED TED TED
Thomas Jefferson University Hospital, Inc. Philadelphia PA TED TED TED
Children's Hospital of Pittsburgh of UPMC Pittsburgh PA CRF CRF CRF
University of Pittsburgh Medical Center ‐ Pittsburgh PA CRF CRF N/A Cancer Center West Penn Hospital Pittsburgh PA CRF CRF CRF
Roger Williams Medical Center Providence RI CRF TED TED
Page | 65 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO Medical University of South Carolina Charleston SC CRF TED TED
Roper Hospital Charleston SC N/A CRF CRF
GHS Cancer Institute Greenville SC CRF CRF CRF
Saint Francis Hospital ‐ 2 Greenville SC N/A CRF CRF
Avera McKennan Transplant Institute Sioux Falls SD CRF CRF CRF
Baptist Blood and Marrow Transplant Memphis TN TED TED TED
Methodist Healthcare Blood and Marrow Memphis TN CRF CRF CRF Transplant Center St. Jude Children's Research Hospital Memphis TN CRF TED N/A
Sarah Cannon BMT Program Nashville TN CRF TED TED
VA Tennessee Valley HCS HSCT Program Nashville TN TED TED N/A Nashville Vanderbilt University Medical Center Nashville TN CRF TED TED
Baylor University Medical Center Dallas TX CRF CRF CRF
Children's Medical Center Dallas Dallas TX CRF CRF CRF
Medical City Dallas Hospital Dallas TX CRF CRF CRF
UT Southwestern Medical Center ‐ BMT Dallas TX CRF CRF CRF Program Zachary and Elizabeth M. Fisher Bone Marrow Fort Sam TX TED TED TED Transplant Program Houston Cook Children's Medical Center Fort Worth TX CRF CRF CRF
Baylor College of Medicine Center for Cell and Houston TX CRF CRF CRF Gene Therapy M.D. Anderson Cancer Center Houston TX CRF CRF CRF
Covenant Health System Hematopoietic Lubbock TX N/A TED TED Transplant Program South Texas Veterans Health Care System San Antonio TX N/A CRF CRF
Page | 66 CIBMTR 2017 Annual Report APPENDIX A1: US CENTERS
Participating Center City State MUD RELATED AUTO Texas Transplant Institute San Antonio TX CRF CRF CRF
The Children's Hospital of San Antonio San Antonio TX CRF CRF CRF
Scott and White Memorial Hospital Temple TX N/A N/A CRF
LDS Hospital/Intermountain Healthcare Salt Lake City UT CRF CRF CRF
Utah Blood and Marrow Transplant Program ‐ Salt Lake City UT CRF CRF CRF Adults Utah Blood and Marrow Transplant Program ‐ Salt Lake City UT CRF CRF CRF Peds University of Virginia Health System Charlottesville VA CRF CRF CRF
Virginia Oncology Associates, Mid Atlantic Norfolk VA N/A N/A TED Consultants Virginia Commonwealth University Massey Richmond VA CRF CRF CRF Cancer Center Bone Marrow Transplant Program University of Vermont Medical Center Burlington VT N/A N/A TED
Fred Hutchinson Cancer Research Center Seattle WA CRF CRF CRF
VA Puget Sound Health Care System Seattle WA TED TED N/A
University of Wisconsin Hospital and Clinics Madison WI CRF CRF CRF
Marshfield Clinic, St. Joseph's Hospital Marshfield WI N/A N/A CRF
Aurora St. Luke's Medical Center Milwaukee WI N/A N/A CRF
Children's Hospital of Wisconsin Milwaukee WI CRF CRF CRF
Froedtert Memorial Lutheran Hospital Milwaukee WI CRF CRF TED
West Virginia University Hospitals, Inc. Morgantown WV CRF CRF CRF
Page | 67 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
APPENDIX A2: INTERNATIONAL CENTERS
The following table lists the international centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED and CRF.
Participating Center City Country MUD RELATED AUTO Alexander Fleming Institute Buenos Aires Argentina CRF CRF CRF
FUNDALEU Buenos Aires Argentina CRF CRF CRF
Hospital de Clínicas Jose de San Martin, Buenos Aires Argentina TED TED TED UBA Hospital de Ninos La Plata Buenos Aires Argentina CRF CRF CRF
Hospital de Pediatria, S.A.M.I.C. Garrahan Buenos Aires Argentina TED TED N/A
Hospital Universitario Austral Buenos Aires Argentina CRF CRF CRF
Hospital Universitario Fundacion Favaloro Buenos Aires Argentina CRF CRF CRF
Instituto Argentino de Diagnostico y Buenos Aires Argentina CRF CRF CRF Tratamiento Sanatorio Anchorena Caba Argentina N/A CRF CRF
Hospital Privado de Cordoba Cordoba Argentina CRF CRF CRF
Sanatorio Allende Cordoba Argentina N/A CRF CRF
Royal Adelaide Hospital Adelaide Australia CRF CRF N/A
Box Hill Hospital, Oncology Day Centre Box Hill Australia N/A N/A TED
Royal Prince Alfred Hospital Institute of Camperdown Australia CRF CRF TED Haematology St. Vincent's Hospital Darlinghurst Australia TED TED N/A
Austin Health Heidelberg Australia CRF CRF N/A
Royal Brisbane & Women's Hospital Herston Australia CRF CRF N/A
Royal Children's Hospital, Melbourne Melbourne Australia CRF CRF N/A
Page | 68 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO The Alfred Hospital Melbourne Australia TED TED TED
Fiona Stanley Hospital Perth Australia CRF CRF N/A
Princess Margaret Hospital for Children Perth Australia CRF CRF TED
Sydney Children's Hospital Randwick Australia CRF CRF TED
Lady Cilento Children's Hospital South Australia N/A CRF CRF Brisbane Royal North Shore Hospital St. Leonards, Australia TED TED N/A Sydney Royal Melbourne Hospital City Campus Victoria Australia CRF CRF N/A
The Children's Hospital at Westmead Westmead Australia CRF CRF TED
Westmead Hospital Westmead Australia CRF CRF N/A
Medizinische Universitatsklinik Graz Graz Austria CRF CRF N/A
Medical University of Vienna Vienna Austria TED TED N/A
ZNA Ziekenhuis Netwerk Antwerpen Antwerpen Belgium TED TED TED
A.Z. Sint‐Jan Brugge Belgium TED TED N/A
Institut Jules Bordet Brussels Belgium TED TED TED
Cliniques Universitaires Saint Luc, Bruxelles Belgium CRF CRF CRF University Louvain Kinderen Universitaire Ziekenhuis Fabiola Bruxelles Belgium TED TED TED
Universiteit Ziekenhuis Antwerpen (UZA) Edegem Belgium TED TED N/A
UZ Gent Gent Belgium MED‐A MED‐A N/A
University Hospital Gasthuisberg Leuven Belgium TED TED MED‐A
Universitaire de Liege, Centre Hospitalier Liege Belgium MED‐A MED‐A MED‐A Universitaire ‐ Sart‐Tilman Hospital de Câncer de Barretos, Fundacao Barretos Brazil TED TED N/A Pio XII
Page | 69 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO UFMG Hospital das Clínicas Servico de Belo Brazil TED TED TED Transplante de Medula Óssea Horizonte Instituto de Cardiologia do Distrito Brasilia Brazil CRF CRF CRF Federal ‐ Unidade de TMO Pietro Albuquerque UNICAMP ‐ HEMOCENTRO Campinas Brazil CRF CRF CRF
Hospital de Clínicas ‐ UFPR Curitiba Brazil CRF CRF CRF
Federal University of Ceara Fortaleza Brazil N/A N/A CRF
Hospital Amaral Carvalho Jau Brazil CRF CRF CRF
Natal Hospital Center Natal Brazil TED TED TED
Associação Hospitalar Moinhos de Vento Porto Alegre Brazil N/A TED TED
Hospital de Clínicas de Porto Alegre Porto Alegre Brazil TED TED TED
Hospital de Clínicas de Porto Alegre Porto Alegre Brazil TED TED TED (peds) HEMOPE / CTMO ‐ Centro de Transplante Recife Brazil TED TED TED de Medula Óssea Real Hospital Portugues Recife Brazil CRF CRF CRF
Univ de São Paulo, School of Medicine of Ribeirao Preto Brazil TED TED TED Ribeirao Preto Hospital Universitario Clementino Fraga Rio de Janeiro Brazil CRF CRF CRF Filho, Univ. Fed. RJ Instituto Nacional de Câncer Rio de Janeiro Brazil CRF CRF CRF
CTMO‐HCFMUSP São Paolo Brazil CRF CRF CRF
De São Jose De Campos São Paolo Brazil N/A N/A TED
Hospital Sírio Libanês São Paolo Brazil CRF CRF CRF
Instituto da Criança ‐ Hospital das Clínicas São Paolo Brazil N/A TED TED da Faculdade de Medicina da Universidade de São Paulo Instituto de Oncologia Pediatrica São Paolo Brazil TED TED TED
Page | 70 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO Santa Casa de Misericordia de São Paulo São Paolo Brazil N/A TED TED
Real e Benemérita Sociedade de Sao Paulo Brazil TED TED TED Beneficiência Portuguesa de São Paulo Albert Einstein Hospital São Paulo Brazil CRF CRF CRF
Bio Sana's São Paulo Brazil CRF CRF CRF
Hospital Samaritano São Paulo Brazil CRF CRF CRF
Hospital São Camilo São Paulo Brazil TED TED TED
Alberta Children's Hospital Calgary Canada CRF CRF CRF
Tom Baker Cancer Centre Calgary Canada TED TED TED
QE II Health Sciences Centre, Dalhousie Halifax Canada TED TED TED University Hamilton Health Sciences Hamilton Canada CRF CRF CRF
Kingston General Hospital Queen's Kingston Canada N/A N/A TED University Centre Hospitalier Universitaire Sainte‐ Montreal Canada CRF CRF CRF Justine Maisonneuve‐Rosemont Hospital Montreal Canada TED TED N/A
McGill University Health Centre (MUHC)‐ Montreal Canada TED TED N/A Royal Victoria Hospital Montreal Children's Hospital, McGill Univ. Montreal Canada TED TED TED Health Centre The Ottawa Hospital Blood & Marrow Ottawa Canada N/A N/A CRF Transplant Program Hotel‐Dieu de Quebec Hospital Quebec Canada TED TED TED
CHA‐Enfant‐Jesus Hospital Quebec City Canada TED TED TED
Saint John Regional Hospital Saint John Canada N/A N/A CRF
Saskatchewan Cancer Centre Saskatoon Canada TED TED TED
St. John's Health Science Center St. John's Canada N/A N/A TED
Page | 71 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO Health Sciences North Sudbury Canada N/A N/A TED
Hospital for Sick Children Toronto Canada TED TED TED
Princess Margaret Cancer Center ‐ BMT Toronto Canada CRF CRF CRF Program British Columbia's Children's Hospital, Vancouver Canada CRF CRF CRF UBC Vancouver General Hospital Vancouver Canada TED TED TED
CancerCare Manitoba/University of Winnipeg Canada CRF CRF CRF Manitoba Clinica Santa Maria Providencia Chile TED TED TED
Catholic University of Chile ‐ TC Santiago Chile CRF TED N/A
Peking University People's Hospital Beijing China CRF CRF N/A
Guangzhou First Municipal People's Guangzhou China TED TED TED Hospital First Affiliated Hospital Medical College HangZhou China CRF CRF CRF Zhejiang University Anhui Provincial Hospital, Anhui Medical Hefei China CRF CRF CRF University Ludaopei Hematology & Oncology Center Langfang China TED TED TED
Instituto de Trasplante de Médula Ósea Barranquilla Colombia TED TED TED de la Costa Fundacion HOMI Hospital de la Bogota Colombia N/A TED TED Misericordia Clinica de Marly Bogotá Colombia CRF CRF CRF
Hospital Pablo Tobon Uribe Medellin Colombia CRF CRF CRF
Instituto de Cancerologia, Clinica Las Medellin Colombia TED TED TED Americas Hospital Mexico San Jose Costa Rica TED TED N/A
Charles University Hospital Pilsen Pilsen Czech CRF CRF CRF Republic Institut of Hematology & Blood Praha Czech TED TED N/A Transfusion Republic
Page | 72 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO University Hospital Motol, Charles Praha Czech TED TED N/A University Hospital Republic University Hospital, Rigshospitalet Copenhagen Denmark CRF CRF N/A
Ain Shams Bone Marrow Transplantation Cairo Egypt N/A TED TED Unit Children's Cancer Hospital ‐ Egypt Cairo Egypt N/A TED TED
National Cancer Institute Cairo University Cairo Egypt TED TED N/A
Helsinki University Hospital, Helsinki Finland CRF CRF N/A Comprehensive Cancer Center Turku University Hospital Turku Finland TED TED N/A
CHRU ‐ Universitaire D'Angers Angers France TED TED N/A
Hopital Jean‐Minjoz Besançon France CRF CRF N/A
Hospital A. Michallon, CHU de Grenoble Grenoble France MED‐A MED‐A N/A
CHRU Hopital Claude Huriez Lille France MED‐A MED‐A N/A
Institut Paoli‐Calmettes Marseille France CRF CRF N/A
Centre Hospitalier Universitaire ARCHET 1 Nice France TED TED MED‐A
Hopital Robert Debre Paris France TED TED N/A
Hopital Saint Louis Paris France CRF CRF N/A
Hotel Dieu Paris France TED TED N/A
Centre Hospitalier Lyon Sud Pierre Bénite France MED‐A MED‐A MED‐A Cedex Hopital Jean Bernard ‐ Hopital La Miletrie Poitiers France CRF CRF N/A
University Hospital Charite ‐ Campus Berlin Germany CRF CRF CRF Virchow University Hospital Charite‐Virchow Berlin Germany CRF CRF N/A Kinderklinik Universitaetsklinikum Dresden Dresden Germany CRF CRF CRF
Page | 73 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO Universitatklinikum Dusseldorf Klinik fur Dusseldorf Germany CRF TED N/A Kinder Universitatsklinikum Essen KMT Essen Germany CRF CRF CRF
Klinik Fur Kinderheilkunde III Frankfurt Germany CRF CRF CRF
Universitatsklinikum Freiburg Freiburg Germany MED‐A MED‐A N/A
Ernst‐Moritz‐Arndt‐University Greifswald, Greifswald Germany TED TED N/A Haematology, Oncology Martin‐Luther‐University Halle‐ Halle Germany TED TED N/A Wittenberg Universitatsklinikum Hamburg, Hamburg Germany CRF CRF N/A Eppendorf Hannover Medical School Hannover Germany MED‐A MED‐A N/A
Universitaetsklinikum Heidelberg Heidelberg Germany CRF CRF N/A
Universitatklinikum Schleswig‐Holstein, Kiel Germany CRF CRF CRF Campus Kiel University Leipzig, Bone Marrow Leipzig Germany CRF N/A N/A Transplant Center University of Munich, Klinikum Munich Germany CRF CRF N/A Grosshadern Universitatsklinikum Münster Münster Germany MED‐A MED‐A MED‐A
Klinikum Nuernberg, Einheit für Nuernberg Germany MED‐A MED‐A MED‐A Knochenmarktransplantation Klinikum der Universitaet Regensburg Regensburg Germany MED‐A MED‐A N/A
Universitatsklinikum Tubingen Tubingen Germany CRF TED N/A
University Hospital Tubingen Tubingen Germany CRF TED N/A
Universitatsklinikum Ulm Ulm Germany CRF CRF TED
Universitatsklinikum Ulm Klinik fur Kinder Ulm Germany CRF CRF N/A
Deutsche Klinik fur Diagnostik ‐ Wiesbaden Germany CRF CRF N/A Wiesbaden Patras University Medical School Rio Patras Greece TED TED TED
Page | 74 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO Chinese University of Hong Kong, Prince Shatin Hong Kong CRF CRF N/A of Wales Hospital Gujrat Cancer & Research Institute (GCRI) Ahmedabad India CRF CRF CRF
SANKALP‐CIMS Centre for Paediatric Ahmedabad India N/A CRF CRF Bone Marrow Transplantation Manipal Hospital Bangalore India N/A N/A N/A
People Tree Centre for Paediatric Bone Bangalore India N/A CRF N/A Marrow Transplantation Apollo Cancer / Specialty Hospital Chennai India TED TED TED
Rajiv Gandhi Cancer Institute and Delhi India CRF CRF CRF Research Center, New Delhi Sir Ganga Ram Hospital Delhi India CRF CRF CRF
Asian Institute of Medical Sciences, Faridabad India CRF CRF CRF Faridabad Apollo Hospital International Ltd. Gandhinagar India N/A TED TED
Fortis Memorial Research Institute Gurgaon India N/A CRF CRF
Medanta ‐ The Medicity Hospital Gurgaon India CRF CRF CRF
Sri Aurobindo Medical College & Post Indore India N/A CRF CRF Graduate Institute South East Asia Institute for Thalassemia Jaipur India N/A CRF N/A
Christian Medical College, Ludhiana Ludhiana India TED TED TED
KDA Stem Cell Transplantation Unit Mumbai India CRF CRF CRF
Tata Memorial Hospital, Parel Mumbai India CRF CRF N/A
All India Institute of Medical Sciences New Delhi India TED TED N/A
Dr. B.L. Kapur Memorial Hospital, New New Delhi India N/A CRF CRF Delhi, India Genebandhu New Delhi India CRF N/A N/A
Institute Rotary Cancer Hospital, AIIMS New Delhi India CRF CRF CRF
Page | 75 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO Jawaharlal Institute of Postgraduate Puducherry India N/A TED TED Medical Education and Research Deenanath Mangeshkar Hospital Pune India CRF CRF CRF
Sahyadri Speciality Hospital Pune India CRF CRF CRF
Christian Medical College Hospital Vellore India CRF CRF N/A
St. James's Hospital, HOPE Directorate Dublin Ireland CRF CRF N/A Office Rambam Medical Center Haifa Israel CRF CRF CRF
Hadassah Medical Center Jerusalem Israel CRF CRF N/A
Davidoff Cancer Center, Rabin Medical Petah Tikva Israel CRF CRF CRF Center, Beilinson Hospital Schneider Children's Medical Center of Petah Tikva Israel CRF CRF N/A Israel Tel‐Aviv Sourasky Medical Center Tel‐Aviv Israel CRF CRF CRF
Chaim Sheba Medical Center (Pediatric) Tel‐Hashomer Israel CRF CRF CRF
Sheba Medical Center Tel‐Hashomer Israel CRF CRF N/A
Universita di Bologna Bologna Italy TED TED TED
Universita di Bologna ‐ 2 Bologna Italy MED‐A MED‐A N/A
Spedali Civili di Brescia Brescia Italy N/A N/A MED‐A
Ospedale Ferrarotto Catania Italy CRF CRF TED
Universita di Firenze, Ospedale di Careggi Firenze Italy CRF MED‐A MED‐A
Ospedale San Salvatore Pesaro Italy CRF CRF CRF
Ospedale Civile BMT Center Pescara Pescara Italy MED‐A MED‐A N/A
Policlinic Tor Vergata University ‐ Rome Rome Italy CRF CRF N/A Transplant Network Universita Cattolica Sacro Cuore Rome Italy TED TED TED
Page | 76 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO University La Sapienza Rome Italy TED TED N/A
Ospedale Molinette Torino Italy MED‐A MED‐A N/A
Udine University Hospital Udine Italy CRF CRF N/A
Kyushu University Hospital Fukuoka Japan CRF CRF CRF
Tokai University Hospital Isehara Japan TED TED N/A
Hyogo College of Medicine Nishinomiya Japan CRF CRF CRF
Osaka City University Graduate School of Osaka Japan CRF CRF N/A Medicine Jichi Medical University Hospital Tochigi Japan TED TED TED
National Cancer Center Hospital Tokyo Japan CRF CRF N/A
Asan Medical Center, Ulsan University Seoul Korea (South) CRF CRF N/A Medical College Samsung Medical Center Seoul Korea (South) CRF CRF CRF
Seoul National University Hospital Seoul Korea (South) CRF CRF CRF
Seoul St. Mary's Hospital Catholic BMT Seoul Korea (South) CRF CRF CRF Center Hamid Al‐Essa MultiOrgan Transplant Safat Kuwait N/A N/A CRF Center, Kuwait Univ American University of Beirut Beirut Lebanon N/A N/A MED‐A
University of Malaya Medical Centre Kuala Lumpur Malaysia TED TED N/A PPUM Hospital Especialidades CMN La Raza Ciudad de Mexico CRF CRF CRF IMSS Mexico Instituto Nacional de Ciencias Medicas y Ciudad de Mexico CRF CRF CRF Nutricion Salvador Zubiran Mexico National Medical Center "20 de Ciudad de Mexico CRF CRF CRF Noviembre" Mexico The American British Cowdray Medical Ciudad de Mexico CRF CRF CRF Center Mexico Instituto Nacional de Pediatria Mexico Coyoacan Mexico CRF CRF CRF
Page | 77 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO Hospital Angeles de las Lomas Huixquilucan Mexico CRF TED TED
Hospital San Jose ‐ Tec de Monterrey Monterrey Mexico CRF CRF CRF
Hospital Universidad Autonoma de Monterrey Mexico CRF CRF CRF Nuevo Leon Clinica Ruiz de Puebla Puebla Mexico TED TED TED
Academic Medical Center Amsterdam Netherlands TED TED TED
VU Medical Center Amsterdam Netherlands TED TED TED
University Medical Centre Groningen Groningen Netherlands TED N/A N/A
Leiden University Medical Centre Leiden Netherlands CRF TED N/A
Academische Ziekenhuis Maastricht Maastricht Netherlands CRF TED N/A
Radboud University Medical Center Nijmegen Netherlands TED TED N/A
Dr. Daniel Den Hoed Cancer Center Rotterdam Netherlands TED TED N/A
University Medical Center Utrecht Utrecht Netherlands TED N/A N/A
University Medical Center Utrecht, Utrecht Netherlands CRF N/A N/A pediatrics Auckland City Hospital Auckland New Zealand CRF CRF N/A
Starship Children's Health, Univ. of Auckland New Zealand CRF CRF N/A Auckland Hospital Canterbury District Health Board (CDHB) Christchurch New Zealand CRF CRF TED
Wellington Blood and Cancer Centre Wellington New Zealand CRF CRF N/A
Rikshospitalet ‐ The National Hospital Oslo Norway CRF N/A N/A
Shifa International Hospitals Islamabad Pakistan N/A CRF CRF
Aga Khan University Hospital Karachi Pakistan TED N/A TED
National Institute of Blood Diseases and Karachi Pakistan CRF CRF CRF Bone Marrow Transplantation
Page | 78 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO AFBMTC Rawalpindi Pakistan N/A CRF CRF
Instituto Oncológico Nacional Panama City Panama CRF CRF CRF
Hospital Nacional Guillermo Almenara Lima Peru N/A CRF CRF Irigoyen Hospital Rebagliati Lima Peru CRF CRF CRF
Instituto Nacional Salud Del Niño San San Borja Peru N/A TED N/A Borja Medical University of Silesia Katowice Poland CRF CRF N/A
Poznan University of Medical Sciences Poznan Poland TED TED N/A
Poznan University of Medical Sciences ‐ 2 Poznan Poland MED‐A MED‐A N/A
Medical University of Warsaw, Central Warsaw Poland CRF CRF N/A Clinical Hospital Lower Silesian Center for Cellular Wroclaw Poland CRF CRF N/A Transplantation Instituto Portugues de Oncologia, BMT Lisbon Portugal CRF CRF N/A Unit Instituto Portugues de Oncologia Centro Porto Portugal MED‐A MED‐A MED‐A do Porto Russian Central Children's Hospital Moscow Russian MED‐A MED‐A N/A Federation Comprehensive Cancer Center, Bone Riyadh Saudi Arabia CRF CRF CRF Marrow Transplant Unit, King Fahad Medical City King Abdullah International Medical Riyadh Saudi Arabia N/A CRF CRF Research Center (KAIMRC) King Abdullah Specialist Children's Riyadh Saudi Arabia CRF CRF CRF Hospital (KASCH) King Faisal Specialist Hospital & Research Riyadh Saudi Arabia CRF CRF N/A Center King Faisal Specialist Hospital & Research Riyadh Saudi Arabia CRF CRF N/A Center ‐ Adults Riyadh Military Hospital (RKH) Riyadh Saudi Arabia TED TED N/A
KK Women's and Children's Hospital Singapore Singapore CRF CRF CRF
Page | 79 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO National Cancer Centre Singapore Singapore Singapore N/A N/A CRF
National University Hospital Singapore Singapore CRF CRF CRF
National University Hospital ‐ 2 Singapore Singapore CRF CRF CRF
Parkway Cancer Centre Singapore Singapore CRF CRF CRF
Singapore General Hospital Singapore Singapore CRF CRF N/A
University Hospital ‐ 2 Bratislava Slovak TED TED N/A Republic Slovenija Donor Ljubljana Slovenia CRF N/A N/A
Constantiaberg Medi‐Clinic Cape Town South Africa CRF CRF CRF
University of Cape Town Medical School Cape Town South Africa CRF CRF N/A
University of Witwatersrand, Parktown South Africa CRF CRF CRF Johannesburg Hospital Albert Alberts Stem Cell Transplant Unit Pretoria South Africa TED TED TED
Hospital de la Santa Creu i Sant Pau Barcelona Spain CRF CRF MED‐A
Hospital Infantil Vall d'Hebron Barcelona Spain MED‐A MED‐A MED‐A
Hospital Vall D'Hebron Barcelona Spain CRF CRF CRF
University of Barcelona Hospital Clinic Barcelona Spain TED TED N/A
Institut Català d'Oncologia‐ Hospital L'Hospitalet Spain CRF CRF MED‐A Duran I Reynals Clinica Puerta de Hierro Madrid Spain MED‐A MED‐A N/A
Hospital General Universitario Gregorio Madrid Spain CRF CRF CRF Maranon Hospital Infantil La Paz Madrid Spain CRF CRF CRF
Hospital Infantil Universitario Niño Jesús Madrid Spain CRF CRF MED‐A
Hospital Universitario Son Espases Palma Spain CRF CRF N/A Mallorca
Page | 80 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO Hospital Universitario La Fe Valencia Spain CRF CRF N/A
Sahlgrenska University Hospital Gothenborg Sweden CRF CRF N/A
University Hospital of Lund Lund Sweden MED‐A MED‐A N/A
Karolinska University Hospital, Centre for Stockholm Sweden CRF CRF N/A Allogeneic Stem Cell Transplantation Hematology, Cancer Center, University Umeå Sweden CRF MED‐A MED‐A Hospital, Umeå University Hospital ‐ Uppsala Uppsala Sweden CRF CRF N/A
University Hospital Basel Basel Switzerland CRF CRF N/A
Hopital Universite Geneva Switzerland CRF CRF N/A
Universitaetsspital Zurich Zurich Switzerland MED‐A MED‐A N/A
Taipei Veterans General Hospital Taipei Taiwan, CRF CRF N/A Province of China Chang Gung Children's Hospital Taoyuan Taiwan, CRF CRF CRF Province of China King Chulalongkorn Memorial Hospital Bangkok Thailand TED TED TED
Ankara University Medical School / Ibni Ankara Turkey TED TED N/A Sina Hospital Gulhane Military Medical Academy Ankara Turkey CRF CRF CRF
Hacettepe Universite ‐ Ihsan Dogramaci Ankara Turkey TED TED TED Cocuk Hastanesi Medical Park Hospital, Antalya‐Turkey Antalya Turkey CRF CRF CRF
Istanbul Medical Faculty Bone Marrow Istanbul Turkey TED TED TED Bank Capadocia Transplant Center Kayseri Turkey CRF CRF CRF
Birmingham Children's Hospital Birmingham United TED TED N/A Kingdom Birmingham Heartlands Hospital Birmingham United TED TED TED Kingdom
Page | 81 CIBMTR 2017 Annual Report APPENDIX A2: INTERNATIONAL CENTERS
Participating Center City Country MUD RELATED AUTO Queen Elizabeth Hospital Birmingham United CRF CRF N/A Kingdom Bristol Children's Hospital Bristol United CRF CRF N/A Kingdom Addenbrooke's Hospital ‐ Cambridge Cambridge United CRF CRF N/A University Kingdom Western General Hospitals Edinburgh United TED TED TED Kingdom Glasgow Royal Infirmary ‐ 2 Glasgow United MED‐A MED‐A N/A Kingdom Royal Hospital for Sick Children Glasgow United CRF CRF N/A Kingdom St. James's University Hospital Leeds United TED TED N/A Kingdom Great Ormond Street Hospital for London United CRF CRF N/A Children Kingdom Imperial College London London United CRF CRF N/A Kingdom Imperial College London, St. Mary's London United CRF CRF N/A Hospital Kingdom St. George's Hospital Medical School London United CRF CRF N/A Kingdom The London Clinic London United TED TED N/A Kingdom The Royal Free Hampstead NHS Trust London United MED‐A MED‐A N/A Kingdom Newcastle General Hospital / The Royal Newcastle United CRF CRF TED Victoria Infirmary Kingdom Swansea ‐ Haematology Swansea United N/A N/A MED‐A Kingdom Asociacion Española lo de Socorros Montevideo Uruguay CRF CRF CRF Mutuos British Hospital & Faculty of Medicine Montevideo Uruguay N/A CRF CRF
Centro de Trasplante del Servicio Medico Montevideo Uruguay CRF CRF CRF Integral (SMI) Hospital Maciel Montevideo Uruguay CRF CRF CRF
Hospital de Clínicas Caracas Caracas Venezuela CRF CRF CRF
Cuidad Hospitalaria Dr. Enrique Tejera Valencia Venezuela TED TED TED
Page | 82 APPENDIX B: COORDINATING CENTER CIBMTR 2017 Annual Report ORGANIZATIONAL STRUCTURE AND LEADERSHIP APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP
The CIBMTR Coordinating Center resides on two campuses. One is located at MCW in Milwaukee, WI, and the other is located at NMDP/Be The Match in Minneapolis, MN. The Coordinating Center provides administrative, statistical, data management, clinical trials, IT, and personnel support for CIBMTR activities, and it benefits from a unique, collegial partnership with the Division of Biostatistics of MCW. CIBMTR Milwaukee has approximately 80 employees and is an academic division of the MCW Department of Medicine. The Milwaukee office receives administrative support from the MCW departments of Grants and Contracts, Development, Public Affairs, Human Resources, and Office of Technology Development as well as the Department of Medicine Administration. CIBMTR Minneapolis has approximately 130 employees, and several NMDP/Be The Match departments provide support for CIBMTR activities, including Finance, Contracts, and Marketing and Communications.
Page | 83 CIBMTR 2017 Annual Report APPENDIX B: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS APPENDIX B1: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS
Chief Scientific Director Medical Faculty M Horowitz, MD, MS Senior Research Advisor D Weisdorf, MD1 Senior Scientific Administrator Director CIBMTR Milwaukee L Burns, MD M Eapen, MD, MS P Steinert, PhD, MBA K Flynn, PhD S Lee, MD, MPH2 M Pasquini, MD, MS Program Director, Advancement Program Director, JD Rizzo, MD, MS Director, IT B Shaw, MD, PhD Data Operations Director Statistics & Clinical E Bergman, MBA, MS Scientific Director J Brunner‐Grady, PA‐C S Fisher Outcomes Research M Arora, MD,MS1 W Pérez, MPH A D’Souza, MBBS, MD M Hamadani, MD P Hari, MD, MS Grants and Program Quality Business Manager, IS Manager M Riches, MD, MS3 Contracts Manager, Assurance Manager, Sr Clinical T Moerke W Saber, MD, MS P Vespalec Manager Research SCTOD Manager C Doleysh TBD S Lorenz S Meiers
Financial Clinical Research Program Medical Writer Senior Program IS Architect Programmer Bus Systems Associate Coordinator III Coordinator II J Gillis‐Smith Biostatistician Coordinator I P Gengler Analyst IV Analyst III S Banagis J Myers K Bhavsar TBD J Carreras M Geronime Database H Tian TBD Program Program Clinical Research Program M Chen Administrator Programmer Desktop Coordinator II Coordinator II Coordinator II Coordinator III H Wang B Liu Analyst III Support R Dunn T Hunt A Prentice A Kusch Biostatistician Sr. Data T Degen Technician M Williams Study Clinical LA Laczkowski M Simaytis K Bo‐Subait Integration J Gier T Skowronski Administrative Research Program TBD S Bo‐Subait Analyst Information Manager IS I Associate Coordinator Coordinator I Program O Dávila TBD Security (part time) K Mulligan A Benoit A Pope Coordinator II M Fei Database Analyst C Zhang Administrative Data Associate Clinical Research M Brey C Frethram Analyst III C Espinoza Assistant, Sr M Patel Coordinator I A Halfmann N He X Zhang DL Campbell Clinical Research A Hantke Program K Hebert Database J Claas Assistant III M Klein Coordinator I M Hemmer Analyst I K Jackson C Abel S Meyers TBD K Hu M Desai Administrative Data Entry C Litovich Metadata 1University of Minnesota Assistant A Pereles 2 H Millard Analyst Fred Hutchinson Cancer Research Ce nte r 3 A Wiedmeyer Clerk A St. Martin A Kummerow University of North Ca rol ina Hospitals L Stewart TBD = CIBMTR – MKE Campus
Page | 84 CIBMTR 2017 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS
APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 1)
Chief Scientific Director Associate Scientific Director Mary M Horowitz, MD, MS D Confer, MD Vice President, CIBMTR Minneapolis R King, MPH
Director, Immunobiology Senior Manager, Senior Manager, and Observational Research Monitoring & Auditing Bioinformatics Research S Spellman 1, MBS D Christianson C Kennedy, PhD
Supervisor, Genomic Services Y Bolon
Senior Principal Project Administrative Senior Clinical Bioinformatics Senior Biostatistician Immunobiology Coordinator Specialist Research Associate Scientists Bioinformatics P Chitphakdiathai Research Scientist H Severance A Carlson A Birch M Halagan Scientists M Haagenson A Howard M Proue Associated A Madbouly Biostatistician C Vierra‐Green L Wendland Bioinformatics Bioinformatics M Formanek Senior TBD Scientists Scientists J Sees Immunobiology Clinical Research W Wang S Fingerson Research Scientist Associate D Roe M Brown K Basa Bioinformatics Immunobiology E Eklund Software Research Scientist A Hendrickson Architect C Brady C Kunakom P Bashyal A Erickson J Peterson A Spahn C Vang S Waldvogel K Zaffran Clinical Trials Assistant J Hullerman‐Umar K Newport Clinical Research 1 Specialist Also serves as CIBMTR Assistant Scientific Director
E Eich = CIBMTR – MKE Campus
= CIBMTR – MSP Campus
Page | 85 CIBMTR 2017 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 2)
Chief Scientific Director Associate Scientific Director Mary M Horowitz, MD, MS D Confer, MD Vice President, CIBMTR Minneapolis R King, MPH
Director, Senior Manager, Data Management Prospective Research M Matlack E Leckrone
Survey Research Supervisor, Supervisor, Quality Supervisor, Clinical Group Clinical Research, Control Research, Supervisor Recipient Data B Levesque Donor Data BMT CTN D Mattila Management Management Project K Gardner A Hauck Manager A Foley Survey Senior Clinical Prospective Quality Control Clinical Clinical Research Clinical Research Research Research Research Specialist Research Coordinator III Coordinator III Associate Specialist Coordinator A Draxler K Kutzner Coordinator III Senior Human W Affield V Bagonza A Adams J Dworski Clinical Research Data Entry A Ewer Research S Logan C Jacox C Johnson Coordinator II Coordinator II Clinical Protection J Tenney TBD H Kobusingye E Mitchem L Horne Research Specialist ‐ IRB TBD A Mitsch Data Entry Coordinator II J Tkachenko TBD K Nutter Coordinator I R Krunkkala Training & Senior Human Clinical Research C Olson P Lee S Tasky Development Research Specialist P Wallace B Schlicht T Thole Specialist Protection A Mussetter Clinical Research Imaging L Colt Specialist M Tierney Coordinator I Assistant II C Jobe Sr Research J Vogel C Erickson T Winder Developer Clinical Research J Wallace TBD M Unekis Assistant N Voit Imaging Assistant Administrative K Christianson Administrative J Khang Specialist Assistant M Young M Ammi
= CIBMTR – MKE Campus = CIBMTR – MSP Campus
Page | 86 CIBMTR 2017 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 3)
Chief Scientific Director Associate Scientific Director Mary M Horowitz, MD, MS D Confer, MD Vice President, CIBMTR Minneapolis R King, MPH
Director, CIBMTR IT Systems M Prestegaard
Senior Manager, Manager, Quality Manager, Data Solutions Manager Program Management Assurance Solutions E Chan K Gee V Yarra J Pollack
Business Architect Principal Research Solutions Principal Data Senior System Principal Data Administrative B Burgess* Scientist Architect (BI) Architect Administrator Architect Specialist Senior Project B Milius* R Fritsch* R Renner* C Yang E Zink* TBD Manager Senior Data Analyst Senior Software Senior System Senior Software S Freeman K Schaper Engineer (ETL) Bioinformatics Administrator Engineer Senior Business Senior Bioinformatics S Stagg Scientist A Westin A Gomez Systems Analyst Programmer / Analyst Software E Williams Data Storage N Hood L Moberg J Schneider Engineer (BI) Senior Data Analyst R Kumar Project Manager Senior Quality Z Ahmed Analyst J Brelsford J Smith A Pull Assurance Analyst L Gabrielson D McDonell A Zheng B Wakaruk B Samba T Wirth Software ScrumMaster Quality Assurance Data Analyst Engineer L Clements Analyst (ETL) D Turner C Jordahl S Ewer J Bloomquist Senior Metadata Project Coordinator Quality Assurance Analyst DM Campbell Analyst W Zhang V Murukurthy Metadata Analyst J Oakes M Nych S Sorensen *All Principals and Architects will have dotted line reporting to the Dir ector, CIBMTR IT MSP = CIBMTR – MKE Campus
= CIBMTR – MSP Campus
Page | 87 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
APPENDIX B3: COORDINATING CENTER LEADERSHIP
SENIOR LEADERSHIP
Mary M. Horowitz, MD, MS Chief Scientific Director for the CIBMTR Principal Investigator for the Data and Coordinating Center of the BMT CTN Research Director for the SCTOD Robert A. Uihlein Professor of Hematologic Research at MCW Associate Director for Cancer Genomics Attending physician in the MCW HCT program
C. Randy Mills, PhD Executive Director for the CIBMTR Chief Executive Officer of NMDP/Be The Match
Dennis Confer, MD Associate Scientific Director for CIBMTR Minneapolis Co‐PI of the Data and Coordinating Center of the BMT CTN Co‐Scientific Director of the RCI BMT Ex Officio Senior Advisor of the Donor Health and Safety Working Committee Chief Medical Officer of NMDP/Be The Match
Daniel Weisdorf, MD Senior Research Advisor for the CIBMTR Scientific Director of the Acute Leukemia Working Committee President‐Elect of the WBMT Professor of Medicine at the University of Minnesota Chief of the Division of Hematology, Oncology, and Transplantation at the University of Minnesota Associate Chair of Clinical Research in the Department of Medicine at the University of Minnesota Attending physician at the University of Minnesota Medical Program
Page | 88 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
Mei‐Jie Zhang, PhD Chief Statistical Director for the CIBMTR Biostatistician for the Acute Leukemia and Graft Sources and Manipulation Working Committees Professor of Biostatistics at MCW
Mary Eapen, MBBS, MS Senior Scientific Director of Research Operations for the CIBMTR Scientific Director for the Graft Sources and Manipulation; Pediatric Cancer; and Primary Immune Deficiencies, Inborn Errors of Metabolism and Other Non‐Malignant Marrow Disorders Working Committees Protocol Officer for several BMT CTN trials Professor of Medicine at MCW
Kathryn E. Flynn, PhD Senior Scientific Director of Patient‐Reported Outcomes for the CIBMTR Associate Professor of Medicine at MCW
J. Douglas Rizzo, MD, MS Senior Scientific Director and Principal Investigator of the SCTOD for the CIBMTR Professor of Medicine at MCW Associate Director of Clinical Operations for the Froedtert and MCW Cancer Center Attending physician in the MCW HCT program
Page | 89 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
Bronwen Shaw MD, PhD Senior Scientific Director of Data Operations for the CIBMTR Co‐Scientific Director of the RCI BMT Scientific Director of the Late Effects and Quality of Life Working Committee as well as the Donor Health and Safety Working Committee Professor of Medicine at MCW Attending physician in the MCW HCT program
Linda Burns, MD Senior Scientific Director of the Health Services Research Program for the CIBMTR Co‐Scientific Director of the RCI BMT Vice President and Medical Director, Health Services Research, NMDP/Be The Match Past President of ASH, 2014
Marcelo Pasquini, MD, MS Senior Scientific Director of CIBMTR Clinical Trials Support – BMT CTN for the CIBMTR Scientific Director for the Autoimmune Diseases and Cellular Therapies Working Committee and the Regimen‐Related Toxicity and Supportive Care Working Committee Protocol Officer and Director of Medical Monitors for the BMT CTN CIBMTR Representative to the WBMT Associate Professor of Medicine at MCW Attending physician in the MCW HCT program
Martin Maiers, MS Vice President of Biomedical Informatics for the CIBMTR Staff liaison to the NMDP/Be The Match Histocompatibility Advisory Group (also known as the CIBMTR Immunobiology Steering Committee) Co‐Chair of Informatics: International Histocompatibility and Immunogenetics Workshop Member of World Health Organization HLA Nomenclature Committee
Page | 90 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
Roberta King, MPH Vice President for CIBMTR Minneapolis Oversees the administrative, research administration, scientific, and statistical support activities of CIBMTR Minneapolis Directs research operations functional areas for human subject protection program, data management, auditing and monitoring, observational research, prospective research, and IT Staff Liaison to the NMDP/Be The Match Donor and Patient Safety Monitoring Advisory Group
Patricia Steinert, PhD, MBA Administrator for CIBMTR Milwaukee Oversees the administrative, scientific, and statistical support activities of CIBMTR Milwaukee Directs research operations functional areas for data operations, statistical operations, IT, and advancement
SCIENTIFIC DIRECTORS
Mukta Arora, MD, MBBS, MS Scientific Director of the Graft‐versus‐Host Disease Working Committee Associate Professor of Medicine at the University of Minnesota Attending physician in the University of Minnesota HCT program
Anita D’Souza, MD Assistant Scientific Director of the Plasma Cell Disorders and Adult Solid Tumors Working Committee Assistant Professor of Medicine at MCW Attending physician in the MCW HCT program
Page | 91 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
Parameswaran Hari, MD, MS Scientific Director of the Plasma Cell Disorders and Adult Solid Tumors Working Committee Armand J. Quick – William F. Stapp Professor of Hematology at MCW Associate Division Chief, Hematology and Oncology at MCW Attending physician in the MCW HCT program
Mehdi Hamadani, MD Scientific Director of the Lymphoma Working Committee Director of Adult Blood and Marrow Transplantation Program at MCW Associate Professor of Medicine at MCW Attending physician in the MCW HCT program
Stephanie J. Lee, MD, MPH Senior Scientific Director of Immunobiology Research for the CIBMTR Co‐Scientific Director of the Immunobiology Working Committee Professor of Medicine at the University of Washington Attending physician member in the Fred Hutchinson Cancer Research Center HCT program
Caleb Kennedy, PhD Senior Manager, Bioinformatics Research Manages bioinformatic algorithm activities Supports research related to next generation DNA sequencing, delivery of new histocompatibility biomarkers, machine learning, and data mining
Page | 92 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
Marcie Riches, MD, MS Scientific Director of the Infection and Immune Reconstitution Working Committee Protocol Officer and Medical Monitor for several BMT CTN trials Clinical Associate Professor of Medicine at the University of North Carolina at Chapel Hill Director of BMT Clinical Research and Data Quality at the University of North Carolina at Chapel Hill Attending physician in the University of North Carolina Lineberger Comprehensive Cancer Center HCT program
Wael Saber, MD, MS Scientific Director of the Chronic Leukemia and Health Services and International Studies Working Committees Assistant Scientific Director of the Acute Leukemia Working Committee Associate Professor of Medicine at MCW Attending physician in the MCW HCT program
Stephen Spellman, MBS Co‐Scientific Director of the Graft‐versus‐Host Disease and Immunobiology Working Committees Director of Immunobiology and Observational Research for the CIBMTR Principal Investigator for the Research Repository Staff liaison to the NMDP/Be The Match Cord Blood Advisory Group Research subcommittee and Histocompatibility Advisory Group (also serves as the CIBMTR Immunobiology Steering Committee) Program Manager for the NMDP/Be The Match Office of Naval Research Grant Graduate faculty in the University of Minnesota Bioinformatics and Computational Biology program
Page | 93 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP STATISTICAL DIRECTORS
Kwang Woo Ahn, PhD Biostatistician for the Chronic Leukemia, Lymphoma, and Pediatric Cancer Working Committees Associate Professor of Biostatistics at MCW
Ruta Brazauskas, PhD Biostatistician for the Autoimmune Diseases and Cellular Therapies, Health Services and International Studies, and Late Effects and Quality of Life Working Committees Associate Professor of Biostatistics at MCW
Raphael Fraser, PhD Biostatistician for the Plasma Cell Disorders Working Committees and the BMT CTN Assistant Professor of Biostatistics at MCW
Soyoung Kim, PhD Biostatistician for the Infection and Immune Deficiencies and the Primary Immune Deficiencies, Inborn Errors of Metabolism and Other Non‐Malignant Marrow Disorders Working Committees Assistant Professor of Biostatistics at MCW
Page | 94 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
Ying Liu, PhD Biostatistician for the Chronic Leukemia and Graft‐versus‐Host Disease Working Committees Assistant Professor of Biostatistics at MCW
Brent Logan, PhD Biostatistician for the Donor Health and Safety and the Regimen‐ Related Toxicity and Supportive Care Working Committees Lead Statistician for the BMT CTN and Statistical Consultant to the NMDP/Be The Match Professor of Biostatistics at MCW Director of the Division of Biostatistics at MCW
Tao Wang, PhD Biostatistician for the Graft‐versus‐Host Disease and Immunobiology Working Committees Associate Professor of Biostatistics at MCW
OTHER LEADERSHIP STAFF
Erik Bergman, MBA, MS Director of IT for the CIBMTR in Milwaukee Leads the IT staff in Milwaukee, which is organized in four teams: Database, Applications Development, Technology Services, and Project Management Oversees management of the Research Database, including extraction of data from source systems as well as their transformation and loading to the database Responsible for data retrievals from the Research Database as well as key solutions for sharing data with stakeholders
Page | 95 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
Janet Brunner‐Grady, PA‐C Program Director of Data Operations for the CIBMTR Manages the clinical research coordinators, develops training programs, and monitors center CPI Assists clinical research coordinators on both campuses with clinical transplant‐related questions
Debra Christianson Senior Manager of Auditing and Monitoring for the CIBMTR Oversees the monitoring program for RCI BMT clinical trials and the on‐site source document audits on data submitted to the Research Database Responsible for the FormsNet Instruction Manual
Sherry Fisher Director of Advancement for the CIBMTR Manages the advancement activities and the Corporate Program, which generates new revenue to create a continued source of non‐ federal financial support Oversees meetings and communications activities, including the annual BMT Tandem Meetings
Erin Leckrone Director of Prospective Research for the CIBMTR Manages the activities of the RCI BMT, including the Survey Research Group Oversees the administration of the Clinical Trials Advisory Committee
Page | 96 CIBMTR 2017 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP
Marie Matlack Senior Manager of Data Management for the CIBMTR Manages clinical research coordinators and senior research programmers as well as data entry and imaging staff Oversees form revision and development and the CPI program Collaborates with the Minneapolis IT staff to develop enhancements to FormsNet
Waleska S. Pérez, MPH Program Director of Statistics and Clinical Outcomes Research for the CIBMTR Oversees the Master’s‐level statisticians of CIBMTR Milwaukee Provides administrative oversight of the Clinical Outcomes Research Program
Matt Prestegaard Director of IT for the CIBMTR in Minneapolis Leads the IT staff in Minneapolis, including project managers, programmer analysts, business systems and data analysts, quality assurance analysts, metadata analysts, bioinformaticists, and managers Develops, implements, and supports CIBMTR electronic data management, capture, and messaging systems (FormsNet and AGNIS); data marts and data warehouses; infrastructure for these applications; and the curation of common data elements within the Cancer Data Standards Registry and Repository
Page | 97 CIBMTR 2017 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP
APPENDIX C: COMMITTEE MEMBERSHIP
CIBMTR committees provide input and advice to the leadership team, ensuring the continued support of both the needs and priorities of its scientific and medical communities. All committees and their functions are listed in Table 1.3. APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP
The Advisory Committee provides oversight for CIBMTR policies and scientific agenda and also partners with the Working Committees to prioritize scientific studies. Members are elected to three‐ year terms by the CIBMTR Assembly and must be from qualifying CRF centers. All Advisory Committee terms begin on March 1.
ELECTED MEMBERS Chair Rob Soiffer, MD, Dana Farber Cancer Institute, Boston, MA Past Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
VICE CHAIRS North America Paul Carpenter, MD, Fred Hutchinson Cancer Research Center, Seattle, WA Europe Charles Craddock, MD, PhD, Queen Elizabeth Hospital, NHS Trust, Birmingham, United Kingdom Central / South America Nelson Hamerschlak, MD, PhD, Albert Einstein Hospital, Sao Paolo, Brazil Asia / Africa / Australia Mahmoud Aljurf, MD, MPH, King Faisal Specialist Hospital Center & Research, Riyadh, Saudi Arabia
MEMBERS AT LARGE North America Karen Ballen, MD, Massachusetts General Hospitals, Boston, MA Navneet Majhail, MD, MS, Cleveland Clinic Foundation, Cleveland, OH Steven Pavletic, MD, MS, NIH‐NCI Experimental Transplantation and Immunology Branch, Bethesda, MD Miguel‐Angel Perales, MD, Memorial Sloan Kettering Cancer Center, New York, NY Marcel van den Brink, MD, PhD, Memorial Sloan Kettering Cancer Center, New York, NY Kirsten Williams, MD, Children’s National Medical Center, Washington, DC Non‐North America Jane Apperley, MBChB, MD, FRCP, Imperial College / Hammersmith Hospital, London, United Kingdom Hildegard Greinix, MD, Medical University of Vienna, Vienna, Austria William Hwang, MBBS, MRCP, Singapore General Hospital, Singapore
Page | 98 CIBMTR 2017 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP Alok Srivastava, MD, Christian Medical College, Vellore, India Jeffrey Szer, MD, Royal Melbourne Hospital City Campus, Victoria, Australia Afonso Vigorito, MD, PhD, Unicamp – Hemocentro, Campinas, Brazil
APPOINTED MEMBERS ASBMT Representative Krishna Komanduri, MD, University of Miami, Miami, FL Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA Business Representative TBD Collection Center Representative James Mason, MD, Scripps Blood and Marrow Transplant Program, La Jolla, CA Cord Blood Bank Representative Andromachi Scaradavou, MD, New York Blood Center, Long Island City, NY Donor Center Representative Jason Gangewere, NMDP/Be The Match, Minneapolis, MN Patient / Family Representatives Jeffrey Haertling, Tierra Verde, FL (Co‐Chairs, Consumer Advocacy Hillary Hall, Dana Farber Cancer Institute, Cambridge, MA Committee)
EX OFFICIO MEMBERS Executive Director Randy Mills, PhD, NMDP/Be The Match, Minneapolis, MN Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI Chief Statistical Director Mei‐Jie Zhang, PhD, CIBMTR, Milwaukee, WI Senior Scientific Director SCTOD J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI Associate Scientific Director CIBMTR Minneapolis Dennis Confer, MD, CIBMTR, Minneapolis, MN Vice President CIBMTR Minneapolis Roberta King, CIBMTR, Minneapolis, MN Research Administrator CIBMTR Milwaukee Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN NMDP / MCW / HRSA Contracting Officer Representative Shelley Grant, MHSA, Rockville, MD NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret) MCW / HRSA Contracting Officer Representative Christine Nishiguchi, MS, MPH, Rockville, MD MCW / NCI Project Officer William Merritt, PhD, Bethesda, MD
Page | 99 CIBMTR 2017 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD Nominating Committee Chair Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
Page | 100 CIBMTR 2017 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP
APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP
The Executive Committee, a subcommittee of the Advisory Committee, ensures that the organization carries out its mission and adheres to CIBMTR policies and procedures; it also provides advice and counsel to the Coordinating Center between meetings of the Advisory Committee. Specifically, the Executive Committee is responsible for providing scientific and policy advice to the Chief Scientific Director and Coordinating Center, reviewing audit results and making recommendations for improvement, and appointing a CIBMTR Co‐Chair and additional CIBMTR representatives to the BMT Tandem Meetings Scientific Organizing Committee for the annual BMT Tandem Meetings. All Executive Committee terms begin on March 1.
ELECTED MEMBERS Chair Rob Soiffer, MD, Dana Farber Cancer Institute, Boston, MA Past Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
VICE CHAIRS North America Paul Carpenter, MD, Fred Hutchinson Cancer Research Center, Seattle, WA Europe Charles Craddock, MD, PhD, Queen Elizabeth Hospital, NHS Trust, Birmingham, United Kingdom Central / South America Nelson Hamerschlak, MD, PhD, Albert Einstein Hospital, Sao Paolo, Brazil Asia / Africa / Australia Mahmoud Aljurf, MD, MPH, King Faisal Specialist Hospital Center & Research, Riyadh, Saudi Arabia
APPOINTED MEMBERS ASBMT Representative Krishna Komanduri, MD, University of Miami, Miami, FL Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA Business Representative TBD Collection Center Representative James Mason, MD, Scripps Blood and Marrow Transplant Program, La Jolla, CA
Cord Blood Bank Representative Andromachi Scaradavou, MD, New York Blood Center, Long Island City, NY Donor Center Representative Jason Gangewere, NMDP/Be The Match, Minneapolis, MN Patient / Family Representatives Jeffrey Haertling, Tierra Verde, FL (Co‐Chairs, Consumer Advocacy Hillary Hall, Dana Farber Cancer Institute, Cambridge, MA Committee)
EX OFFICIO MEMBERS Executive Director Randy Mills, PhD, NMDP/Be The Match, Minneapolis, MN Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI Page | 101 CIBMTR 2017 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP Chief Statistical Director Mei‐Jie Zhang, PhD, CIBMTR, Milwaukee, WI Senior Scientific Director SCTOD J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI Associate Scientific Director CIBMTR Minneapolis Dennis Confer, MD, CIBMTR, Minneapolis, MN Vice President CIBMTR Minneapolis Roberta King, CIBMTR, Minneapolis, MN Research Administrator CIBMTR Milwaukee Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN NMDP / MCW / HRSA Contracting Officer Representative Shelley Grant, MHSA, Rockville, MD NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret) MCW / HRSA Contracting Officer Representative Christine Nishiguchi, MS, MPH, Rockville, MD MCW / NCI Project Officer William Merritt, PhD, Bethesda, MD MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD Nominating Committee Chair Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
Page | 102 CIBMTR 2017 Annual Report APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP
APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP
The Consumer Advocacy Committee communicates research results and data to the non‐medical community and provides patient and donor perspectives during the development of the CIBMTR research agenda. Many committee members have personal experience as a donor, recipient, or family member of a recipient.
CHAIRS Jeffery Haertling, Tierra Verde, FL Hilary Hall, Dana Farber Cancer Institute, Cambridge, MA
MEMBERS Jack Aiello, MS, San Jose, CA Maureen Beaman, MBA, Milwaukee, WI James Omel, MD, (retired professional) Grand Island, NE Kristin Scheeler, Leukemia and Lymphoma Society, White Plains, NY Jennifer Wilder, National Institutes of Health, Bethesda, MD
SCIENTIFIC DIRECTOR J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI
EX OFFICIO MEMBERS Linda Burns, MD, NMDP/ Be the Match Jeffrey Chell, MD, NMDP/Be The Match, Minneapolis, MN Dennis Confer, MD, NMDP/Be The Match, Minneapolis, MN Ellen Denzen, MBA, MS, (NMDP/Be The Match liaison) NMDP/Be The Match, Minneapolis, MN Carol Doleysh, (CIBMTR liaison) CIBMTR, Milwaukee, WI Jessica Gillis‐Smith, MPH, CIBMTR, Milwaukee, WI Shelley Grant, MHSA, Health Resources and Services Administration, Rockville, MD Mary Horowitz, MD, MS, CIBMTR, Milwaukee, WI Elizabeth Murphy, EdD, RN, (NMDP/Be The Match liaison) NMDP/Be The Match, Minneapolis, MN Christine Nishiguchi, MS, MPH, Health Resources and Services Administration, Rockville, MD Bronwen Shaw, MD, PhD, CIBMTR, Milwaukee, WI Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI
Page | 103 CIBMTR 2017 Annual Report APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP
APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP
The Nominating Committee consists of five members elected by the CIBMTR Assembly. Following an annual call for nominations, the Nominating Committee prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees. Elections are held annually by confidential electronic ballot. The Nominating Committee also makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments after seeking recommendations from the CIBMTR Assembly, Advisory Committee, and incumbent Working Committee Chairs. All terms begin on March 1.
CHAIR Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
MEMBERS Corey Cutler, MD, MPH, Dana Farber Cancer Institute, Boston, MA Ephraim Fuchs, MD, Johns Hopkins University Sidney Kimmel Cancer Center, Baltimore, MD Richard Maziarz, MD, Oregon Health and Science University, Portland, OR David Vesole, MD, PhD, Hackensack University Medical Center, Hackensack, NJ
Page | 104 CIBMTR 2017 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP
APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP
For information on Scientific Working Committee structure and organization, see Section 1.3.1. For information on Working Committee studies and their accomplishments, see Section 2.1.1.
ACUTE LEUKEMIA WORKING COMMITTEE Chairs Marcos de Lima, MD, University Hospitals Case Medical Center Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center Scientific Director Daniel Weisdorf, MD, CIBMTR Asst Scientific Dir Wael Saber, MD, MS, CIBMTR Statistical Director Mei‐Jie Zhang, PhD, CIBMTR Statistician Hai‐Lin Wang, MPH, CIBMTR
AUTOIMMUNE DISEASES AND CELLULAR THERAPIES WORKING COMMITTEE Chairs David McKenna, MD, University of Minnesota Medical Center, Fairview Stefanie Sarantopoulos, MD, PhD, Duke University Medical Center Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Consumer Adv Rep Hillary Hall, Dana Farber Cancer Institute Statistician Kyle Bo‐Subait, MPH, CIBMTR
CHRONIC LEUKEMIA WORKING COMMITTEE Chairs Edwin Alyea, MD, Dana Farber Cancer Institute Uday Popat, MD, MD Anderson Cancer Center Ronald Sobecks, MD, Cleveland Clinic Foundation Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Directors Kwang Woo Ahn, PhD, CIBMTR Ying Liu, PhD, CIBMTR Statistician Zhenhuan Hu, MPH, CIBMTR
DONOR HEALTH AND SAFETY WORKING COMMITTEE Chairs Galen Switzer, PhD, University of Pittsburgh Medical Center Michael Pulsipher, MD, Children’s Hospital of Los Angeles Nirali Shah, MD, MHSc, National Cancer Institute – NIH Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Ex Officio Sr Advisor Dennis Confer, MD, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Consumer Adv Reps Jeffrey Haertling Hillary Hall, Dana Farber Cancer Institute Statisticians Jennifer Sees, MPH, CIBMTR Pintip Chitphakdithai, PhD, CIBMTR
Page | 105 CIBMTR 2017 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE Chairs Vanderson Rocha, MD, PhD, Churchill Hospital Asad Bashey, MD, PhD, The Blood and Marrow Transplant Program at Northside Hospital Ian McNiece, PhD, MD Anderson Cancer Center Scientific Director Mary Eapen, MD, MS, CIBMTR Statistical Director Mei‐Jie Zhang, PhD, CIBMTR Statistician Andrew St. Martin, MS, CIBMTR
GRAFT‐VERSUS‐HOST DISEASE WORKING COMMITTEE Chairs Daniel Couriel, MD, University of Michigan Amin Alousi, MD, MD Anderson Cancer Center Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute Scientific Directors Mukta Arora, MD, MS, CIBMTR Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Ying Liu, PhD, CIBMTR Consumer Adv Reps Kristin Scheeler, Leukemia and Lymphoma Society Jennifer Wilder, BSN, RN, National Institutes of Health Statistician Michael Hemmer, MS, CIBMTR
HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE Chairs Jignesh Dalal, MD, Seidman Cancer Center‐University Hospitals Cleveland Medical Center Theresa Hahn, PhD, Roswell Park Cancer Institute Nandita Khera, MD, Mayo Clinic Arizona and Phoenix Children's Hospital William Wood, MD, MPH, University of North Carolina Hospitals Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Consumer Adv Rep Jack Aiello Statistician Naya He, MPH, CIBMTR
IMMUNOBIOLOGY WORKING COMMITTEE Chairs Michael Verneris, MD, University of Colorado Children’s Hospital Katharina Fleischhauer, MD, Universitätsklinikum Essen KMT Katharine Hsu, MD, PhD, Memorial Sloan Kettering Cancer Center Scientific Directors Stephanie J. Lee, MD, MPH, CIBMTR, Fred Hutchinson Cancer Research Center Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Statistician Michael Haagenson, MS, CIBMTR
Page | 106 CIBMTR 2017 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE Chairs Jeffery Auletta, MD, Nationwide Children’s Hospital Caroline Lindemans, MD, PhD, University Medical Center Utrecht Krishna Komanduri, MD, University of Miami Scientific Director Marcie Riches, MD, MS, CIBMTR, University of North Carolina Hospitals Statistical Directors Soyoung Kim, PhD, CIBMTR Statistician Min Chen, MS, CIBMTR
LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE Chairs Bipin Savani, MD, Vanderbilt University Medical Center Mary Flowers, MD, Fred Hutchinson Cancer Research Center Minoo Battiwalla, MD, MS, National Heart Lung and Blood Institute ‐ NIH Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Consumer Adv Reps Hillary Hall, Dana Farber Cancer Institute Jim Omel Statistician Heather Millard, MPH, CIBMTR
LYMPHOMA WORKING COMMITTEE Chairs Sonali Smith, MD, University of Chicago Hospitals Anna Sureda, MD, PhD, Institut Catala d'Oncologia Timothy Fenske, MD, MS, Froedtert Memorial Lutheran Hospital Scientific Director Mehdi Hamadani, MD, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Carlos Litovich, MPH, CIBMTR
PEDIATRIC CANCER WORKING COMMITTEE Chairs Gregory Hale, MD, Johns Hopkins All Children’s Hospital Parinda Mehta, MD, Cincinnati Children's Hospital Medical Center Angela Smith, MD, MS, University of Minnesota Medical Center Scientific Director Mary Eapen, MD, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Heather Millard, MPH, CIBMTR
Page | 107 CIBMTR 2017 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE Chairs Cristina Gasparetto, MD, Duke University Medical Center Yago Nieto, MD, PhD, M.D. Anderson Cancer Center Tomer Mark, MD, University of Colorado Hospital Shaji Kumar, MD, Mayo Clinic Rochester Scientific Director Parameswaran Hari, MD, MS, CIBMTR Asst Scientific Dir Anita D’Souza, MD, CIBMTR Statistical Directors Raphael Fraser, PhD, CIBMTR Statistician Omar Davila, MPH, CIBMTR
PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND OTHER NON‐MALIGNANT MARROW DISORDERS WORKING COMMITTEE Chairs Paolo Anderlini, MD, M.D. Anderson Cancer Center Neena Kapoor, MD, Children’s Hospital of Los Angeles Jaap‐Jan Boelens, MD, PhD, University Medical Center Utrecht Vikram Mathews, MD, Christian Medical College Hospital Scientific Director Mary Eapen, MD, CIBMTR Statistical Director Soyoung Kim, PhD, CIBMTR Statistician Kyle Hebert, MS
REGIMEN‐RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE Chairs Andrew Artz, MD, MS, University of Chicago Hospitals Alison Loren, MD, MS, Abramson Cancer Center University of Pennsylvania Medical Center Shin Mineishi, MD, Penn State Hershey Medical Center Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Statistician Caitrin Fretham, MPH, CIBMTR
Page | 108 CIBMTR 2017 Annual Report APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP
APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP
The NMDP/Be The Match Histocompatibility Advisory Group also serves as the CIBMTR Immunobiology Steering Committee. This committee reviews and approves the use of donor‐ recipient specimens from the Research Repository in CIBMTR studies.
CHAIR Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center, Tampa, FL
ADVISORY GROUP MEMBERS Robyn Ashton, RN, MSN, HRSA Division of Transplantation, Rockville, MD Juliet Barker, MD, Memorial Sloan Kettering Cancer Center, New York, NY Sarah Cooley, MD, University of Minnesota, Minneapolis, MN Mary Eapen, MD, MS, Medical College of Wisconsin, Milwaukee, WI Marcelo Fernandez‐Viña, PhD, Stanford Hospital and Clinics, Palo Alto, CA Robert Hartzman, MD, Capt. MC, USN (Ret.), Navy Representative, C.W. Bill Young Marrow Donor Recruitment and Research Program, Rockville, MD Carolyn K. Hurley, PhD, Diplomate ABHI, Georgetown University Hospital, Washington DC Brent Logan, PhD, Medical College of Wisconsin, Milwaukee, WI Carlheinz Mueller, MD, PhD, German National Bone Marrow Donor Registry (ZKRD), Ulm, Germany Miguel Angel Perales, MD, Memorial Sloan Kettering Cancer Center Raja Rajalingham, PhD, University of California, San Francisco, CA Bronwen Shaw, MD, PhD, Medical College of Wisconsin, Milwaukee, WI
NATIONAL MARROW DONOR PROGRAM (MNDP) STAFF Dennis Confer, MD, CIBMTR Jason Dehn, MPH, CIBMTR Karen Dodson, CIBMTR Martin Maiers, MS, CIBMTR Stephen Spellman, MS, CIBMTR
Page | 109 CIBMTR 2017 Annual Report APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP
APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP
The Clinical Trials Advisory Committee assists in the review, approval, and oversight of proposals and protocols for Phase I and Phase II clinical trials submitted to the RCI BMT (Section 2.3.2).
CHAIR John Koreth, MBBS, DPhil, Dana Farber Cancer Institute, Boston, MA
PAST CHAIR John Levine, MD, MS, Mount Sinai Medical Center, New York, NY
MEMBERS Natalie Callander, MD, University of Wisconsin Hospital and Clinics, Madison, WI Stephan Grupp, MD, PhD, Children’s Hospital of Philadelphia, Philadelphia, PA Amrita Krishnan, MD, City of Hope, Duarte, CA Marco Mielcarek, MD, Fred Hutchinson Cancer Research Center, Seattle, WA Sophie Paczesny, MD, PhD, Indiana University Hospital/Riley Hospital for Children, Indianapolis, IN Katayoun Rezvani, MD, PhD, M.D. Anderson Cancer Center, Houston, TX Bipin Savani, MD, Vanderbilt University Medical Center, Nashville, TN
APPOINTED MEMBERS Jeffrey Haertling, Tierra Verde, FL Hilary Hall, Dana Farber Cancer Institute, Cambridge, MA
EX OFFICIO MEMBERS Linda Burns, MD, NMDP/Be The Match Operations, Minneapolis, MN Dennis Confer, MD, CIBMTR, Minneapolis, MN Robert Hartzman, MD, Capt MC, USN (Ret.), Department of Defense, Rockville, MD Mary Horowitz, MD, MS, CIBMTR, Milwaukee, WI Roberta King, CIBMTR, Minneapolis, MN Erin Leckrone, CIBMTR, Minneapolis, MN Brent Logan, PhD, CIBMTR, Milwaukee, WI Nancy Poland, MA, NMDP/Be The Match Operations, Minneapolis, MN Marcie Riches, MD, MS, University of North Carolina Hospitals, Chapel Hill, NC Bronwen Shaw, MD, PhD, CIBMTR, Milwaukee, WI Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN
Page | 110 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
APPENDIX D: PUBLICATIONS
The PMCID number is assigned by PubMed Central, the NIH’s free digital archive of biomedical and life sciences journal literature, and is in compliance with the NIH policy on public access. APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS
The following publications were generated by Scientific Working Committees within the Clinical Outcomes Research Program. For more information about the Working Committees, see Section 2.1.1. SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID El‐Jawahri A, Chen Y‐B, Impact of pre‐transplant Cancer. 2017 May 15; PMC5419891 Brazauskas R, He N, Lee SJ, depression on outcomes of 123(10):1828‐1838. Knight JM, Majhail N, allogeneic and autologous doi:10.1002/cncr.30546. Buchbinder D, Schears RM, Wirk hematopoietic stem cell Epub 2017 Jan 19 BM, Wood WA, Ahmed I, Aljurf transplantation M, Szer J, Beattie SM, Battiwalla M, Dandoy C, Diaz M‐A, D'Souza A, Freytes CO, Gajewski J, Gergis U, Hashmi SK, Jakubowski A, Kamble RT, Kindwall‐Keller T, Lazarus HM, Malone AK, Marks DI, Meehan K, Savani BN, Olsson RF, Rizzieri D, Steinberg A, Speckhart D, Szwajcer D, Schoemans H, Seo S, Ustun C, Atsuta Y, Dalal J, Sales‐Bonfim C, Khera N, Hahn T, Saber W Liu HD, Ahn KW, Hu Z‐H, Allogeneic hematopoietic Biology of Blood and PMC5590102 Hamadani M, Nishihori T, Wirk cell transplantation for adult Marrow Transplantation: B, Beitinjaneh A, Rizzieri D, chronic myelomonocytic Journal of the American Grunwald MR, Sabloff M, Olsson leukemia Society for Blood and RF, Bajel A, Bredeson C, Daly A, Marrow Transplantation. Inamoto Y, Majhail N, Saad A, 2017 May 1; 23(5):767‐ Gupta V, Gerds A, Malone A, 775. doi:10.1016 Tallman M, Reshef R, Marks DI, /j.bbmt.2017.01.078. Copelan E, Gergis U, Savoie ML, Epub 2017 Jan 20 Ustun C, Litzow MR, Cahn J‐Y, Kindwall‐Keller T, Akpek G, Savani BN, Aljurf M, Rowe JM, Wiernik PH, Hsu JW, Cortes J, Kalaycio M, Maziarz R, Sobecks R, Popat U, Alyea E, Saber W.
Page | 111 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Michelis FV, Gupta V, Zhang M‐J, Cytogenetic risk determines Cancer. 2017 Jun 1; PMC5445018 Wang H‐L, Aljurf M, Bacher U, outcomes after allogeneic 123(11):2035‐2042. Beitinjaneh A, Chen Y‐B, DeFilipp transplantation in older doi:10.1002/cncr.30567. Z, Gale RP, Kebriaei P, Kharfan‐ patients with acute myeloid Epub 2017 Jan 24 Dabaja M, Lazarus HM, Nishihori leukemia in their second T, Olsson RF, Oran B, Rashidi A, complete remission: A Rizzieri DA, Tallman MS, de Lima Center for International M, Khoury HJ, Sandmaier BM, Blood and Marrow Weisdorf D, Saber W Transplant Research cohort analysis Weisdorf DJ, Millard HR, Allogeneic transplantation Cancer. 2017 Jun 1; PMC5445001 Horowitz MM, Hyare PS, for advanced acute myeloid 123(11):2025‐2034. Champlin R, Ho V, Mielcarek M, leukemia: The value of doi:10.1002/cncr.30536. Rezvani A, Stockerl‐Goldstein K, complete remission Epub 2017 Jan 24 Khoury HJ, De Lima M, Saber W, Sandmaier B, Zhang M‐J, Eapen M Brunstein C, Zhang M‐J, Barker J, The effect of inter‐unit HLA Haematologica. 2017 May PMC5477613 St Martin A, Bashey A, de Lima matching in double 1; 102(5):941‐947. M, Dehn J, Hematti P, Perales umbilical cord blood doi:10.3324/haematol.20 MA, Rocha V, Territo M, transplantation for acute 16.158584. Epub 2017 Jan Weisdorf D, Eapen M leukemia 25 Ustun C, Giannotti F, Zhang M‐J, Outcomes of UCB Leukemia. 2017 Jun 1; PMC5462854 Wang H‐L, Brunstein C, Labopin transplantation are 31(6):1408‐1414. M, Rocha V, de Lima M, Baron F, comparable in FLT3+ AML: doi:10.1038/leu.2017.42. Sandmaier BM, Eapen M, Results of CIBMTR, Epub 2017 Jan 25 Gluckman E, Nagler A, Weisdorf EUROCORD and EBMT DJ, Ruggeri A collaborative analysis Hamadani M, Kanate AS, DiGilio Allogeneic hematopoietic Biology of Blood and PMC5410937 A, Ahn KW, Smith SM, Lee JW, cell transplantation for Marrow Transplantation: Ayala E, Chao N, Hari P, Bolaños‐ aggressive NK cell leukemia. Journal of the American Meade J, Gress R, Smedegaard A Center for International Society for Blood and Anderson N, Chen Y‐B, Farooq U, Blood and Marrow Marrow Transplantation. Schiller G, Yared J, Sureda A, Transplant Research analysis 2017 May 1; 23(5):853‐ Fenske TS, Olteanu H 856. doi:10.1016 /j.bbmt.2017.01.082. Epub 2017 Feb 1 Lindsley RC, Saber W, Mar BG, Prognostic mutations in New England Journal of PMC5438571 Redd R, Wang T, Haagenson MD, myelodysplastic syndrome Medicine. 2017 Feb 9; Grauman PV, Hu Z‐H, Spellman after stem‐cell 376(6):536‐547. SR, Lee SJ, Verneris MR, Hsu K, transplantation doi:10.1056/NEJMoa1611 Fleischhauer K, Cutler C, Antin 604. Epub 2017 Feb 9 JH, Neuberg D, Ebert BL
Page | 112 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Muraro PA, Pasquini M, Atkins Long‐term outcomes after JAMA Neurology. 2017 PMC5744858 HL, Bowen JD, Farge D, Fassas A, autologous hematopoietic Apr 1; 74(4):459‐469. Freedman MS, Georges GE, stem cell transplantation for doi:10.1001/jamaneurol.2 Gualandi F, Hamerschlak N, multiple sclerosis 016.5867. Epub 2017 Feb Havrdova E, Kimiskidis VK, Kozak 20 T, Mancardi GL, Massacesi L, Moraes DA, Nash RA, Pavletic S, Ouyang J, Rovira M, Saiz A, Simoes B, Trnený M, Zhu L, Badoglio M, Zhong X, Sormani MP, Saccardi R Armstrong AE, Fonstad R, Current knowledge and Clinical Pediatrics. 2018 N/A Spellman S, Tullius Z, Chaudhury practice of pediatric Feb 1; 57(2):161‐167. S providers in umbilical cord doi:10.1177/0009922817 blood banking 692316. Epub 2017 Mar 1 Madbouly A, Wang T, Investigating the association Biology of Blood and PMC5541944 Haagenson M, Paunic V, Vierra‐ of genetic admixture and Marrow Transplantation: Green C, Fleischhauer K, Hsu KC, donor/recipient genetic Journal of the American Verneris MR, Majhail NS, Lee SJ, disparity with transplant Society for Blood and Maiers M outcomes Marrow Transplantation. 2017 Jun 1; 23(6):1029‐ 1037. doi:10.1016 /j.bbmt.2017.02.019. Epub 2017 Mar 2 Garderet L, D'Souza A, Jacobs P, Response assessment in Biology of Blood and N/A van Biezen A, Schönland S, myeloma: practical manual Marrow Transplantation: Kroeger N, Morris C, Hari P on consistent reporting in Journal of the American an era of dramatic Society for Blood and therapeutic advances Marrow Transplantation. 2017 Jul 1; 23(7):1193‐ 1202. doi:10.1016 /j.bbmt.2017.03.009. Epub 2017 Mar 8
Page | 113 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Gerds AT, Woo Ahn K, Hu ZH, Outcomes after umbilical Biology of Blood and PMC5474679 Abdel‐Azim H, Akpek G, Aljurf M, cord blood transplantation Marrow Transplantation: Ballen KK, Beitinjaneh A, Bacher for myelodysplastic Journal of the American U, Cahn J‐Y, Chhabra S, Cutler C, syndromes Society for Blood and Daly A, DeFilipp Z, Gale RP, Marrow Transplantation. Gergis U, Grunwald MR, Hale 2017 Jun 1; 23(6):971‐ GA, Hamilton BK, Jagasia M, 979. doi:10.1016/j.bbmt. Kamble RT, Kindwall‐Keller T, 2017.03.014. Epub 2017 Nishihori T, Olsson RF, Mar 10 Ramanathan M, Saad AA, Solh M, Ustun C, Valcárcel D, Warlick E, Wirk BM, Kalaycio M, Alyea E, Popat U, Sobecks R, Saber W Khoury HJ, Wang T, Hemmer Improved survival after Haematologica. 2017 May PMC5477615 MT, Couriel D, Alousi A, Cutler C, acute graft‐versus‐host 1; 102(5):958‐966. Aljurf M, Antin JH, Ayas M, disease diagnosis in the doi:10.3324/haematol.20 Battiwalla M, Cahn J‐Y, Cairo M, modern era 16.156356. Epub 2017 Chen Y‐B, Gale RP, Hashmi S, Mar 16 Hayashi RJ, Jagasia M, Juckett M, Kamble RT, Kharfan‐Dabaja M, Litzow M, Majhail N, Miller A, Nishihori T, Qayed M, Schoemans H, Schouten HC, Socie G, Storek J, Verdonck L, Vij R, Wood WA, Yu L, Martino R, Carabasi M, Dandoy C, Gergis U, Hematti P, Solh M, Jamani K, Lehmann L, Savani B, Schultz KR, Wirk BM, Spellman S, Arora M, Pidala J Kekre N, Zhang Y, Zhang M‐J, Effect of antithymocyte Haematologica. 2017 Jul PMC5566045 Carreras J, Ahmed P, Anderlini P, globulin source on 1; 102(7):1291‐1298. Atta EH, Ayas M, Boelens JJ, outcomes of bone marrow doi:10.3324/haematol.20 Bonfim C, Deeg HJ, Kapoor N, transplantation for severe 17.164459. Epub 2017 Lee J‐W, Nakamura R, Pulsipher aplastic anemia Mar 24 MA, Eapen M, Antin JH Cheng YC, Shi Y, Zhang M‐J, Long‐term outcome of Journal of Cancer. PMC5436253 Brazauskas R, Hemmer MT, inflammatory breast cancer 8(6):1009‐1017. Bishop MR, Nieto Y, Stadtmauer compared to non‐ doi:10.7150/jca.16870. E, Ayash L, Gale RP, Lazarus H, inflammatory breast cancer Epub 2017 Mar 25 Holmberg L, Lill M, Olsson RF, in the setting of high‐dose Wirk BM, Arora M, Hari P, Ueno chemotherapy with N autologous hematopoietic cell transplantation
Page | 114 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Wang Y, Wang T, Dagnall C, Relative telomere length Biology of Blood and PMC5657243 Haagenson M, Spellman SR, before hematopoietic cell Marrow Transplantation: Hicks B, Jones K, Lee SJ, Savage transplantation and Journal of the American SA, Gadalla SM outcome after unrelated Society for Blood and donor hematopoietic cell Marrow Transplantation. transplantation for acute 2017 Jul 1; 23(7):1054‐ leukemia 1058. doi:10.1016/j.bbmt. 2017.03.032. Epub 2017 Apr 4 Slack J, Albert MH, Balashov D, Outcome of hematopoietic Journal of Allergy and PMC5632132 Belohradsky BH, Bertaina A, cell transplantation for DNA Clinical Immunology. 2018 Bleesing J, Booth C, Buechner J, double‐strand break repair Jan 1; 141(1):322‐ Buckley RH, Ouachée‐Chardin M, disorders 328.e10. doi:10.1016/ Deripapa E, Drabko K, Eapen M, j.jaci.2017.02.036. Epub Feuchtinger T, Finocchi A, 2017 Apr 7 Gaspar HB, Ghosh S, Gillio A, Gonzalez‐Granado LI, Grunebaum E, Güngör T, Heilmann C, Helminen M, Higuchi K, Imai K, Kalwak K, Kanazawa N, Karasu G, Kucuk ZY, Laberko A, Lange A, Mahlaoui N, Meisel R, Moshous D, Muramatsu H, Parikh S, Pasic S, Schmid I, Schuetz C, Schulz A, Schultz KR, Shaw PJ, Slatter MA, Sykora K‐W, Tamura S, Taskinen M, Wawer A, Wolska‐Kunierz B, Cowan MJ, Fischer A, Gennery AR Gabriel M, Shaw BE, Brazauskas Risk factors for subsequent Biology of Blood and PMC5518678 R, Chen M, Margolis DA, central nervous system Marrow Transplantation: Sengelov H, Dahlberg A, Ahmed tumors in pediatric Journal of the American IA, Delgado D, Lazarus HM, allogeneic hematopoietic Society for Blood and Gibson B, Myers KC, Kamble RT, cell transplant: A study from Marrow Transplantation. Abdel‐Mageed A, Li C‐K, Flowers the Center for International 2017 Aug 1; 23(8):1320‐ MED, Battiwalla M, Savani BN, Blood and Marrow 1326. doi:10.1016 Majhail N, Shaw PJ Transplant Research /j.bbmt.2017.04.004. (CIBMTR) Epub 2017 Apr 12
Page | 115 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Khandelwal P, Millard HR, Thiel Hematopoietic stem cell Biology of Blood and PMC5669065 E, Abdel‐Azim H, Abraham AA, transplantation activity in Marrow Transplantation: Auletta JJ, Boulad F, Brown VI, pediatric cancer between Journal of the American Camitta BM, Chan KW, 2008 and 2014 in the United Society for Blood and Chaudhury S, Cowan MJ, Angel‐ States: A Center for Marrow Transplantation. Diaz M, Gadalla SM, Gale RP, International Blood and 2017 Aug 1; 23(8):1342‐ Hale G, Kasow KA, Keating AK, Marrow Transplant 1349. doi:10.1016 Kitko CL, MacMillan ML, Olsson Research report /j.bbmt.2017.04.018. RF, Page KM, Seber A, Smith AR, Epub 2017 Apr 24 Warwick AB, Wirk B, Mehta PA Segal E, Martens M, Wang HL, Comparing outcomes of Cancer. 2017 Sep 1; PMC5568918 Brazauskas R, Weisdorf D, matched related donor and 123(17):3346‐3355. Sandmaier BM, Khoury HJ, de matched unrelated donor doi:10.1002/cncr.30737. Lima M, Saber W hematopoietic cell Epub 2017 Apr 27 transplants in adults with B‐ Cell acute lymphoblastic leukemia Vrooman LM, Millard HR, Survival and late effects Biology of Blood and PMC5666571 Brazauskas R, Majhail NS, after allogeneic Marrow Transplantation: Battiwalla M, Flowers ME, hematopoietic cell Journal of the American Savani BN, Akpek G, Aljurf M, transplantation for Society for Blood and Bajwa R, Baker KS, Beitinjaneh A, hematologic malignancy at Marrow Transplantation. Bitan M, Buchbinder D, Chow E, less than three years of age 2017 Aug 1; 23(8):1327‐ Dandoy C, Dietz AC, Diller L, Gale 1334. doi:10.1016 RP, Hashmi SK, Hayashi RJ, /j.bbmt.2017.04.017. Hematti P, Kamble RT, Kasow Epub 2017 Apr 28 KA, Kletzel M, Lazarus HM, Malone AK, Marks DI, O'Brien TA, Olsson RF, Ringden O, Seo S, Steinberg A, Yu LC, Warwick A, Shaw B, Duncan C Schriber JR, Hari PN, Ahn KW, Fei Hispanics have the lowest Cancer. 2017 Aug 15; PMC5544566 M, Costa LJ, Kharfan‐Dabaja MA, stem cell transplant 123(16):3141‐3149. Angel‐Diaz M, Gale RP, Ganguly utilization rate for doi:10.1002/cncr.30747. S, Girnius SK, Hashmi S, autologous hematopoietic Epub 2017 May 4 Pawarode A, Vesole DH, Wiernik cell transplantation for PH, Wirk BM, Marks DI, Nishihori multiple myeloma in the T, Olsson RF, Usmani SZ, Mark United States: A CIBMTR TM, Nieto YL, D'Souza A report
Page | 116 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Krakow EF, Hemmer M, Wang T, Tools for the precision American Journal of [PMC Logan B, Arora M, Spellman S, medicine era: How to Epidemiology. 2017 Jul Journal – In Couriel D, Alousi A, Pidala J, Last develop highly personalized 15; 186(2):160‐172. Process] M, Lachance S, Moodie EEM treatment doi:10.1093/aje/kwx027. recommendations from Epub 2017 May 5 cohort and registry data using Q‐learning Bitan M, Ahn KW, Millard HR, Personalized prognostic risk Biology of Blood and PMC5683075 Pulsipher MA, Abdel‐Azim H, score for long‐term survival Marrow Transplantation: Auletta JJ, Brown V, Chan KW, for children with acute Journal of the American Diaz MA, Dietz A, Vincent MG, leukemia after allogeneic Society for Blood and Guilcher G, Hale GA, Hayashi RJ, transplantation Marrow Transplantation. Keating A, Mehta P, Myers K, 2017 Sep 1; 23(9):1523‐ Page K, Prestidge T, Shah NN, 1530. doi:10.1016 Smith AR, Woolfrey A, Thiel E, /j.bbmt.2017.05.011. Davies SM, Eapen M Epub 2017 May 17 Boudreau JE, Giglio F, Gooley TA, KIR3DL1/HLA‐B subtypes Journal of Clinical PMC5501362 Stevenson PA, Le Luduec J‐B, govern acute myelogenous Oncology. 2017 Jul 10; Shaffer BC, Rajalingam R, Hou L, leukemia relapse after 35(20):2268‐2278. Hurley CK, Noreen H, Reed EF, hematopoietic cell doi:10.1200/JCO.2016.70. Yu N, Vierra‐Green C, Haagenson transplantation 7059. Epub 2017 May 18 M, Malkki M, Petersdorf EW, Spellman S, Hsu K Fleischhauer K, Ahn KW, Wang Directionality of non‐ Bone Marrow [PMC HL, Zito L, Crivello P, Müller C, permissive HLA‐DPB1 T‐cell Transplantation. 2017 Sep Journal – In Verneris M, Shaw BE, Pidala J, epitope group mismatches 1; 52(9):1280‐1287. Process] Oudshorn M, Lee SJ, Spellman does not improve clinical doi:10.1038/bmt.2017.96. SR risk stratification in 8/8 Epub 2017 Jun 5 matched unrelated donor hematopoietic cell transplantation D'Souza A, Lee S, Zhu X, Pasquini Current use and trends in Biology of Blood and PMC5565685 M hematopoietic cell Marrow Transplantation: transplantation in the Journal of the American United States Society for Blood and Marrow Transplantation. 2017 Sep 1; 23(9):1417‐ 1421. doi:10.1016 /j.bbmt.2017.05.035. Epub 2017 Jun 9
Page | 117 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Epperla N, Ahn KW, Ahmed S, Rituximab‐containing Journal of Hematology & PMC5469142 Jagasia M, DiGilio A, Devine SM, reduced‐intensity Oncology. 2017 Jun 12; Jaglowski S, Kennedy V, Rezvani conditioning improves 10(1):117. doi:10.1186 AR, Smith SM, Sureda A, Fenske progression‐free survival /s13045‐017‐0487‐y. Epub TS, Kharfan‐Dabaja MA, Armand following allogeneic 2017 Jun 12 P, Hamadani M transplantation in B cell non‐Hodgkin lymphoma Eapen M, Wang T, Veys PA, Allele‐level HLA matching The Lancet Haematology. PMC5699478 Boelens JJ, St Martin A, Spellman for umbilical cord blood 2017 Jul 1; 4(7):e325‐ S, Bonfim CS, Brady C, Cant AJ, transplantation for non‐ e333. doi:10.1016/S2352‐ Dalle J‐H, Davies SM, Freeman J, malignant diseases in 3026(17)30104‐7. Epub Hsu KC, Fleischhauer K, Kenzey children: A retrospective 2017 Jun 13 C, Kurtzberg J, Michel G, Orchard analysis PJ, Paviglianiti A, Rocha V, Veneris MR, Volt F, Wynn R, Lee SJ, Horowitz MM, Gluckman E, Ruggeri A D'Souza A, Zhang M‐J, Huang J, Trends in pre‐ and post‐ Leukemia. 2017 Sep 1; PMC5587375 Fei M, Pasquini M, Hamadani M, transplant therapies with 31(9):1998‐2000. Hari P first autologous doi:10.1038/leu.2017.185. hematopoietic cell Epub 2017 Jun 13 transplantation among patients with multiple myeloma in the United States, 2004‐2014 Bashey A, Zhang M‐J, McCurdy Mobilized peripheral blood Journal of Clinical PMC5590802 SR, St Martin A, Argall T, stem cells versus Oncology. 2017 Sep 10; Anasetti C, Ciurea SO, Fasan O, unstimulated bone marrow 35(26):3002‐3009. Gaballa S, Hamadani M, Munshi as a graft source for T‐cell‐ doi:10.1200/JCO.2017.72. P, Al Malki MM, Nakamura R, replete haploidentical donor 8428. Epub 2017 Jun 23 O'Donnell PV, Perales M‐A, Raj transplantation using post‐ K, Romee R, Rowley S, Rocha V, transplant Salit RB, Solh M, Soiffer RJ, Fuchs cyclophosphamide EJ, Eapen M Switzer GE, Bruce J, Pastorek G, Parent versus child donor Bone Marrow [PMC Kiefer DM, Kobusingye H, perceptions of the bone Transplantation. 2017 Sep Journal – In Drexler R, Besser RAM, Confer marrow donation 1; 52(9):1338‐1341. Process] DL, Horowitz MM, King RJ, Shaw experience doi:10.1038/bmt.2017.12 BE, van Walraven SM, Wiener L, 4. Epub 2017 Jun 26 Packman W, Varni JW, Pulsipher MA
Page | 118 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Fleischhauer K, Shaw BE HLA‐DP in unrelated Blood. 2017 Aug 1; N/A hematopoietic cell 130(9):1089‐1096. transplantation revisited: doi:10.1182/blood‐2017‐ Challenges and 03‐742346. Epub 2017 Jun opportunities 30 Muffly L, Pasquini M, Martens Increasing use of allogeneic Blood. 2017 Aug 31; PMC5580273 M, Brazauskas R, Zhu X, Adekola hematopoietic cell 130(9):1156‐1164. K, Aljurf M, Ballen KK, Bajel A, transplantation in patients doi:10.1182/blood‐2017‐ Baron F, Battiwalla M, aged 70 years and older in 03‐772368. Epub 2017 Jul Beitinjaneh A, Cahn J‐Y, Carabasi the United States 3 M, Chen Y‐B, Chhabra S, Ciurea S, Copelan E, D'Souza A, Edwards J, Foran J, Freytes CO, Fung HC, Gale RP, Giralt S, Hashmi SK, Hildebrandt GC, Ho V, Jakubowski A, Lazarus H, Luskin MR, Martino R, Maziarz R, McCarthy P, Nishihori T, Olin R, Olsson RF, Pawarode A, Peres E, Rezvani AR, Rizzieri D, Savani BN, Schouten HC, Sabloff M, Seftel M, Seo S, Sorror M, Szer J, Wirk BM, Wood WA, Artz A Kanate AS, DiGilio A, Ahn KW, Al Allogeneic haematopoietic British Journal of [PMC Malki M, Jacobsen E, Steinberg cell transplantation for Haematology. Journal – In A, Hammerschlak N, Kharfan‐ extranodal natural killer/T‐ doi:10.1111/bjh.14879. Process] Dabaja M, Salit R, Ball E, Bashir cell lymphoma, nasal type: A Epub 2017 Aug 2 Q, Cashen A, Couriel D, Diez‐ CIBMTR analysis Martin J, Katsanis E, Linhares Y, Mori S, Nash R, Pawarode A, Perales M‐A, Phipps CD, Richman C, Savani BN, Shapira MY, Stiff P, Strair R, Fenske TS, Smith SM, Sureda A, Olteanu H, Hamadani M Karaesmen E, Rizvi AA, Preus Replication and validation of Blood. 2017 Sep 28; PMC5620418 LM, McCarthy PL, Pasquini MC, genetic polymorphisms 130(13):1585‐1596. Onel K, Zhu X, Spellman S, associated with survival doi:10.1182/blood‐2017‐ Haiman CA, Stram DO, Pooler L, after allogeneic blood or 05‐784637. Epub 2017 Sheng X, Zhu Q, Yan L, Liu Q, Hu marrow transplant Aug 15 Q, Webb A, Brock G, Clay‐ Gilmour AI, Battaglia S, Tritchler D, Liu S, Hahn T, Sucheston‐ Campbell LE
Page | 119 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Arnold SD, Brazauskas R, He N, Li Clinical risks and healthcare Haematologica. 2017 Nov PMC5664386 Y, Aplenc R, Jin Z, Hall M, Atsuta utilization of 1; 102(11):1823‐1832. Y, Dalal J, Hahn T, Khera N, haematopoietic cell doi:10.3324/haematol.20 Bonfim C, Majhail NS, Diaz MA, transplantation for sickle 17.169581. Epub 2017 Freytes CO, Wood WA, Savani cell disease in the U.S. using Aug 17 BM, Kamble RT, Parsons S, merged databases Ahmed I, Sullivan K, Beattie S, Dandoy C, Munker R, Marino S, Bitan M, Abdel‐Azim H, Aljurf M, Olsson RF, Joshi S, Buchbinder D, Eckrich MJ, Hashmi S, Lazarus H, Marks DI, Steinberg A, Saad A, Gergis U, Krishnamurti L, Abraham A, Rangarajan HG, Walters M, Lipscomb J, Saber W, Satwani P Shaw BE, Brazauskas R, Millard Centralized patient‐reported Cancer. 2017 Dec 1; PMC5693638 HR, Fonstad R, Flynn KE, outcome data collection in 123(23):4687‐4700. Abernethy A, Vogel J, Petroske transplantation is feasible doi:10.1002/cncr.30936. C, Mattila D, Drexler R, Lee SJ, and clinically meaningful Epub 2017 Aug 17 Horowitz MM, Rizzo JD Malogolowkin MH, Hemmer MT, Outcomes following Bone Marrow PMC5665725 Le‐Rademacher J, Hale GA, autologous hematopoietic Transplantation. 2017 Nov Mehta PA, Smith AR, Kitko C, stem cell transplant for 1; 52(11):1549‐1555. Abraham A, Abdel‐Azim H, patients with relapsed doi:10.1038/bmt.2017.17 Dandoy C, Angel Diaz M, Gale Wilms' tumor: A CIBMTR 8. Epub 2017 Sep 4 RP, Guilcher G, Hayashi R, Jodele retrospective analysis S, Kasow KA, MacMillian ML, Thakar M, Wirk BM, Woolfrey A, Thiel EL Abraham A, Hsieh M, Eapen M, Relationship between mixed Biology of Blood and [PMC Fitzhugh C, Carreras J, Keesler D, donor‐recipient chimerism Marrow Transplantation: Journal – In Guilcher G, Kamani N, Walters and disease recurrence after Journal of the American Process] MC, Boelens JJ, Tisdale J, Shenoy hematopoietic cell Society for Blood and S transplantation for sickle Marrow Transplantation. cell disease 2017 Dec 1; 23(12):2178‐ 2183. doi:10.1016 /j.bbmt.2017.08.038. Epub 2017 Sep 4
Page | 120 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Clay‐Gilmour AI, Hahn T, Preus Genetic association with B‐ Blood Advances. 2017 Sep PMC5728332 LM, Onel K, Skol A, Hungate E, cell acute lymphoblastic 12; 1(20):1717‐1728. Zhu Q, Haiman CA, Stram DO, leukemia in allogeneic doi:10.1182/bloodadvanc Pooler L, Sheng X, Yan L, Liu Q, transplant patients differs es.2017006023. Epub Hu Q, Liu S, Battaglia S, Zhu X, by age and sex 2017 Sep 8 Block AW, Sait SNJ, Karaesmen E, Rizvi A, Weisdorf DJ, Ambrosone CB, Tritchler D, Ellinghaus E, Ellinghaus D, Stanulla M, Clavel J, Orsi L, Spellman S, Pasquini MC, McCarthy PL, Sucheston‐ Campbell LE Mahindra A, Hari P, Fraser R, Fei Autologous hematopoietic Bone Marrow [PMC M, Huang J, Berdeja J, Callander cell transplantation for Transplantation. 2017 Dec Journal – In N, Costa L, Diaz MA, Freytes C, multiple myeloma patients 1; 52(12):1616‐1622. Process] Gale RP, Girnius S, Holmberg L, with renal insufficiency: A doi:10.1038/bmt.2017.19 Kharfan‐Dabaja M, Kumar S, Kyle Center for International 8. Epub 2017 Sep 18 R, Lazarus H, Lee C, Maiolino A, Blood and Marrow Moreb J, Nishihori T, Pawarode Transplant Research analysis A, Saad A, Savani BN, Schriber J, William B, Wirk BM, Krishnan A, Nieto Y, D'Souza A Kelly DL, Buchbinder D, Duarte Neurocognitive dysfunction Biology of Blood and [PMC RF, Auletta JJ, Bhatt N, Byrne M, in hematopoietic cell Marrow Transplantation: Journal – In Gabriel M, Mahindra A, Norkin transplant recipients: Expert Journal of the American Process] M, Schoemans H, Shah AJ, review from the Late Effects Society for Blood and Ahmed I, Atsuta Y, Basak GW, and Quality of Life Working Marrow Transplantation. Beattie S, Bhella S, Bredeson C, Committee of the CIBMTR doi:10.1016/j.bbmt.2017. Bunin N, Dalal J, Daly A, and Complications and 09.004. Epub 2017 Sep 19 Gajewski J, Gale RP, Galvin J, Quality of Life Working party Hamadani M, Hayashi RJ, of the EBMT Adekola K, Law J, Lee CJ, Liesveld J, Malone AK, Nagler A, Naik S, Nishihori T, Parsons SK, Scherwath A, Schofield H‐L, Szer J, Twist I, Warwick A, Wirk BM, Yi J, Battiwalla M, Flowers ME, Shaw BE
Page | 121 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Stroncek DF, Shaw BE, Logan BR, Donor experiences of Biology of Blood and PMC5743544 Kiefer DM, Savani BN, Anderlini second marrow or Marrow Transplantation: P, Bredeson CN, Hematti P, peripheral blood stem cell Journal of the American Ganguly S, Diaz MA, Abdel‐Azim collection mirror the first, Society for Blood and H, Ahmed I, Maharaj D, Seftel M, but CD34+ yields are less Marrow Transplantation. Beitinjaneh A, Seo S, Yared JA, 2018 Jan 1; 24(1):175‐184. Halter J, O'Donnell PV, Hale GA, doi:10.1016/j.bbmt.2017. Defilipp Z, Lazarus H, Liesveld JL, 09.013. Epub 2017 Sep 25 Zhou Z, Munshi P, Olsson RF, Kasow KA, Szer J, Switzer GE, Chitphakdithai P, Shah N, Confer DL, Pulsipher MA Heimall J, Logan BR, Cowan MJ, Immune reconstitution and Blood. 2017 Dec 21; PMC5746165 Notarangelo LD, Griffith LM, survival of 100 SCID patients 130(25):2718‐2727. Puck JM, Kohn DB, Pulsipher post‐hematopoietic cell doi:10.1182/blood‐2017‐ MA, Parikh S, Martinez C, transplant: a PIDTC natural 05‐781849. Epub 2017 Kapoor N, O'Reilly R, Boyer M, history study Oct 11 Pai SY, Goldman F, Burroughs L, Chandra S, Kletzel M, Thakar M, Connelly J, Cuvelier G, Davila Saldana BJ, Shereck E, Knutsen A, Sullivan KE, DeSantes K, Gillio A, Haddad E, Petrovic A, Quigg T, Smith AR, Stenger E, Yin Z, Shearer WT, Fleisher T, Buckley RH, Dvorak CC Hill BT, Ahn KW, Hu Z‐H, Aljurf Assessment of impact of Biology of Blood and [PMC M, Beitinjaneh A, Cahn J‐Y, human leukocyte antigen Marrow Transplantation: Journal – In Cerny J, Kharfan‐Dabaja MA, (HLA) type on outcomes of Journal of the American Process] Ganguly S, Ghosh N, Grunwald allogeneic hematopoietic Society for Blood and MR, Inamoto Y, Kindwall‐Keller stem cell transplant for Marrow Transplantation. T, Nishihori T, Olsson RF, Saad A, chronic lymphocytic doi:10.1016/j.bbmt.2017. Seftel M, Seo S, Szer J, Tallman leukemia (CLL) 10.015. Epub 2017 Oct 12 M, Ustun C, Wiernik PH, Maziarz RT, Kalaycio M, Alyea E, Popat U, Sobecks R, Saber W Epperla N, Ahn KW, Armand P, Fludarabine and busulfan Biology of Blood and PMC5743624 Jaglowski S, Ahmed S, Kenkre versus fludarabine, Marrow Transplantation: VP, Savani B, Jagasia M, Shah cyclophosphamide, and Journal of the American NN, Fenske TS, Sureda A, Smith rituximab as reduced‐ Society for Blood and SM, Hamadani M intensity conditioning for Marrow Transplantation. allogeneic transplantation in 2018 Jan 1; 24(1):78‐85. follicular lymphoma doi:10.1016/j.bbmt.2017. 10.011. Epub 2017 Oct 13
Page | 122 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Htut M, D'Souza A, Krishnan A, Autologous/allogeneic HCT Biology of Blood and [PMC Bruno B, Zhang M‐J, Fei M, Diaz versus tandem autologous Marrow Transplantation: Journal – In MA, Copelan E, Ganguly S, transplantation for multiple Journal of the American Process] Hamadani M, Kharfan‐Dabaja myeloma: Comparison of Society for Blood and MA, Lazarus H, Lee C, Meehan K, long term post relapse Marrow Transplantation. Nishihori T, Saad A, Seo S, survival doi:10.1016/j.bbmt.2017. Ramanathan M, Usmani SZ, 10.024. Epub 2017 Oct 24 Gasparetto C, Mark TM, Nieto Y, Hari P Shaw BE, Syrjala KL, Onstad LE, PROMIS measures can be Cancer. [PMC Flowers ME, Jim H, Baker KS, used to assess symptoms doi:10.1002/cncr.31089. Journal – In Buckley S, Fairclough DL, and function in long‐term Epub 2017 Oct 26 Process] Horowitz MM, Lee SJ hematopoietic cell transplantation survivors Myers RM, Hill BT, Shaw BE, Kim Long‐term outcomes among Cancer. [PMC S, Millard HR, Battiwalla M, 2‐year survivors of doi:10.1002/cncr.31114. Journal – In Majhail NS, Buchbinder D, autologous hematopoietic Epub 2017 Nov 10 Process] Lazarus HM, Savani BN, Flowers cell transplantation for MED, D'Souza A, Ehrhardt MJ, Hodgkin and diffuse large b‐ Langston A, Yared JA, Hayashi RJ, cell lymphoma Daly A, Olsson RF, Inamoto Y, Malone AK, DeFilipp Z, Margossian SP, Warwick AB, Jaglowski S, Beitinjaneh A, Fung H, Kasow KA, Marks DI, Reynolds J, Stockerl‐Goldstein K, Wirk B, Wood WA, Hamadani M, Satwani P Qayed M, Wang T, Hemmer MT, Influence of age on acute Biology of Blood and [PMC Spellman S, Arora M, Couriel D, and chronic GVHD in Marrow Transplantation: Journal – In Alousi A, Pidala J, Abdel‐Azim H, children receiving HLA‐ Journal of the American Process] Aljurf M, Ayas M, Bitan M, Cairo identical sibling BMT for Society for Blood and M, Choi SW, Dandoy C, Delgado acute leukemia: implications Marrow Transplantation. D, Gale RP, Hale G, Frangoul H, for prophylaxis doi:10.1016/j.bbmt.2017. Kamble RT, Kharfan‐Dabaja M, 11.004. Epub 2017 Nov 16 Lehmann L, Levine J, MacMillan M, Marks DI, Nishihori T, Olsson RF, Hematti P, Ringden O, Saad A, Satwani P, Savani BN, Schultz KR, Seo S, Shenoy S, Waller EK, Yu L, Horowitz MM, Horan J
Page | 123 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID William BM, Wang T, Haagenson Impact of human leukocyte Biology of Blood and [PMC M, Fleischhauer K, Verneris M, antigen (HLA) alleles on Marrow Transplantation: Journal – In Hsu KC, de Lima MJ, Fernandez‐ outcomes of allogeneic Journal of the American Process] Vina M, Spellman SR, Lee SJ, Hill transplantation for B‐cell Society for Blood and BT non‐Hodgkin lymphomas: A Marrow Transplantation. Center for International doi:10.1016/j.bbmt.2017. Blood and Marrow 11.003. Epub 2017 Nov 16 Transplant Research analysis Kumar SK, Dispenzieri A, Fraser Early relapse after Leukemia. doi:10.1038/ [PMC R, Mingwei F, Akpek G, Cornell autologous hematopoietic leu.2017.331. Epub 2017 Journal – In R, Kharfan‐Dabaja M, Freytes C, cell transplantation remains Nov 16 Process] Hashmi S, Hildebrandt G, a poor prognostic factor in Holmberg L, Kyle R, Lazarus H, multiple myeloma but Lee C, Mikhael J, Nishihori T, Tay outcomes have improved J, Usmani S, Vesole D, Vij R, Wirk over time B, Krishnan A, Gasparetto C, Mark T, Nieto Y, Hari P, D'Souza A Kebriaei P, Anasetti C, Zhang M‐ Intravenous busulfan Biology of Blood and [PMC J, Wang H‐L, Aldoss I, de Lima M, compared to total body Marrow Transplantation: Journal – In Khoury HJ, Sandmaier BM, irradiation pre‐transplant Journal of the American Process] Horowitz MM, Artz A, Bejanyan conditioning for adults with Society for Blood and N, Ciurea S, Lazarus HM, Gale acute lymphoblastic Marrow Transplantation. RP, Litzow M, Bredeson C, Seftel leukemia doi:10.1016/j.bbmt.2017. MD, Pulsipher MA, Boelens J‐J, 11.025. Epub 2017 Nov 29 Alvarnas J, Champlin R, Forman S, Pullarkat V, Weisdorf D, Marks DI Hamadani M, Horowitz MM Allogeneic transplantation Journal of Oncology PMC5728364 for follicular lymphoma: Practice. 2017 Dec 1; Does one size fit all? 13(12):798‐806. doi:10.1200/JOP.2017.026 336. Epub 2017 Dec 1
Page | 124 CIBMTR 2017 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Casulo C, Friedberg JW, Ahn KW, Autologous transplantation Biology of Blood and [PMC Flowers C, DiGilio A, Smith SM, in follicular lymphoma with Marrow Transplantation: Journal – In Ahmed S, Inwards D, Aljurf M, early therapy failure: A NLCS Journal of the American Process] Chen AI, Choe H, Cohen J, and CIBMTR analysis Society for Blood and Copelan E, Farooq U, Fenske TS, Marrow Transplantation. Freytes C, Gaballa S, Ganguly S, doi:10.1016/j.bbmt.2017. Jethava Y, Kamble RT, Kenkre 12.771. Epub 2017 Dec 11 VP, Lazarus H, Lazaryan A, Olsson RF, Rezvani AR, Rizzieri D, Seo S, Shah GL, Shah N, Solh M, Sureda A, William B, Cumpston A, Zelenetz AD, Link BK, Hamadani M Bejanyan N, Zhang M‐J, Wang H‐ Pre‐transplant consolidation Biology of Blood and [PMC L, Lazaryan A, de Lima M, Marks is not beneficial for adults Marrow Transplantation: Journal – In DI, Sandmaier BM, Bachanova V, with all undergoing Journal of the American Process] Rowe J, Tallman M, Kebriaei P, myeloablative allogeneic Society for Blood and Kharfan‐Dabaja M, Gale RP, transplantation Marrow Transplantation. Lazarus HM, Ustun C, Copelan E, doi:10.1016/j.bbmt.2017. Hamilton BK, Schiller G, Hogan 12.784. Epub 2017 Dec 21 W, Hashmi S, Seftel M, Kanakry C, Olsson RF, Martino R, Saber W, Khoury HJ, Weisdorf D Gadalla SM, Wang T, Loftus D, No association between Bone Marrow [PMC Friedman L, Dagnall C, donor telomere length and Transplantation. Journal – In Haagenson M, Spellman SR, outcomes after allogeneic doi:10.1038/s41409‐017‐ Process] Buturovic L, Blauwkamp M, unrelated hematopoietic 0029‐9. Epub 2017 Dec 21 Shelton J, Fleischhauer K, Hsu cell transplant in patients KC, Verneris MR, Krstajic D, with acute leukemia Hicks B, Jones K, Lee SJ, Savage SA
Page | 125 CIBMTR 2017 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS
APPENDIX D2: BMT CTN PUBLICATIONS
The following publications were generated by the BMT CTN, a component of the Clinical Trials Support Program, which conducts multi‐institutional Phase II and III trials focused on HCT. The BMT CTN Data and Coordinating Center maintains continuity of operations and facilitates effective communications. The Data and Coordinating Center effort is a collaboration of the CIBMTR, NMDP/Be The Match, and the Emmes Corporation. For more information, see Section 2.3.1. BMT CTN PUBLICATIONS Authors Title Citation PMCID Scott BL, Pasquini MC, Logan Myeloablative versus Journal of Clinical PMC5455603 BR, Wu J, Devine SM, Porter reduced‐intensity Oncology. 2017 Apr 10; DL, Maziarz RT, Warlick ED, hematopoietic cell 35(11):1154‐1161. Fernandez HF, Alyea EP, transplantation for acute doi:10.1200/JCO.2016.7 Hamadani M, Bashey A, Giralt myeloid leukemia and 0.7091. Epub 2017 Feb S, Geller NL, Leifer E, Le‐ myelodysplastic 13 Rademacher J, Mendizabal syndromes AM, Horowitz MM, Deeg HJ, Horwitz ME Arora M, Weisdorf DJ, Pharmacogenetics of Clinical Transplantation. PMC5413396 Shanley RM, Thyagarajan B steroid‐responsive acute 2017 May 2; graft‐versus‐host disease 31(5):e12949. doi:10.1111/ctr.12949. Epub 2017 Apr 4 Holtan SG, Newell LF, Cutler Low EGF in Bone Marrow PMC5699445 C, Verneris MR, DeFor TE, Wu myeloablative Transplantation. 2017 J, Howard A, MacMillan ML, allotransplantation: Sep 1; 52(9):1300‐1303. Antin JH, Blazar BR, Weisdorf Association with severe doi:10.1038/bmt.2017.8 DJ, Panoskaltsis‐Mortari A acute GvHD in BMT CTN 9. Epub 2017 Jun 5 0402 Eapen M, Kurtzberg J, Zhang Umbilical cord blood Biology of Blood and PMC5605440 MJ, Hattersely G, Fei M, transplantation in Marrow Mendizabal A, Chan KW, children with acute Transplantation: Journal Oliveria S, Schultz KR, Wall D, leukemia: Impact of of the American Society Horowitz MM, Wagner JE. conditioning on for Blood and Marrow transplantation Transplantation. 2017 outcomes Oct 2; 23(10):1714‐ 1721. doi:10.1016 /j.bbmt.2017.06.023. Epub 2017 Jul 3
Page | 126 CIBMTR 2017 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS BMT CTN PUBLICATIONS Authors Title Citation PMCID Holstein SA, Jung S‐H, Updated analysis of The Lancet PMC5718627 Richardson PG, Hofmeister CALGB (Alliance) 100104 Haematology. 2017 Sep CC, Hurd DD, Hassoun H, assessing lenalidomide 1; 4(9):e431‐e442. Giralt S, Stadtmauer EA, versus placebo doi:10.1016/S2352‐ Weisdorf DJ, Vij R, Moreb JS, maintenance after single 3026(17)30140‐0. Epub Callander NS, van Besien K, autologous stem‐cell 2017 Aug 17 Gentile TG, Isola L, Maziarz transplantation for RT, Bashey A, Landau H, multiple myeloma: a Martin T, Qazilbash MH, randomised, double‐ Rodriguez C, McClune B, blind, phase 3 trial Schlossman RL, Smith SE, Hars V, Owzar K, Jiang C, Boyd M, Schultz C, Wilson M, Hari P, Pasquini MC, Horowitz MM, Shea TC, Devine SM, Linker C, Anderson KC, McCarthy PL Turcotte LM, DeFor TE, Donor and recipient Bone Marrow PMC5752567 Newell LF, Cutler CS, Verneris plasma follistatin levels Transplantation. 2018 MR, Wu J, Howard A, are associated with Jan 1; 53(1):64‐68. MacMillan ML, Antin JH, acute GvHD in Blood and doi:10.1038/bmt.2017.2 Vercellotti GM, Slungaard A, Marrow Transplant 36. Epub 2017 Oct 23 Blazar BR, Weisdorf DJ, Clinical Trials Network Panoskaltsis‐Mortari A, 0402 Holtan SG Holstein SA, Avet‐Loiseau H, BMT CTN myeloma Biology of Blood and [PMC Journal Hahn T, Ho CM, Lohr JG, intergroup workshop on Marrow – In Process] Munshi NC, Pavia B, Pasquini minimal residual disease Transplantation: Journal MC, Tario JD Jr, Usmani, SZ, and immune profiling: of the American Society Wallace PK, Weisel K, summary and for Blood and Marrow McCarthy PL recommendations from Transplantation. the organizing doi:10.1016/j.bbmt.201 committee 7.12.774. Epub 2017 Dec 11
Page | 127 CIBMTR 2017 Annual Report APPENDIX D3: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS
APPENDIX D3: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS
The following publication was generated by the Health Services Research program, through which the CIBMTR conducts research in health disparities, health policy, and system capacity issues involving HCT. For more information, see Section 2.4. HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID Preussler JM, Meyer CL, Mau Healthcare costs and Biology of Blood and PMC5497312 L‐W, Majhail NS, Denzen EM, utilization for patients Marrow Edsall KC, Farnia SH, Saber W, age 50 to 64 years with Transplantation: Journal Burns LJ, Vanness DJ acute myeloid leukemia of the American Society treated with for Blood and Marrow chemotherapy or with Transplantation. 2017 chemotherapy and Jun 1; 23(6):1021‐1028. allogeneic hematopoietic doi:10.1016/j.bbmt.201 cell transplantation 7.02.017. Epub 2017 Mar 2 Neumann JL, Mau L‐W, Virani Burnout, Moral Distress, Biology of Blood and N/A S, Denzen EM, Boyle DA, Work‐Life Balance and Marrow Boyle NJ, Dabney J, De Career Satisfaction Transplantation: Journal KeselLofthus A, Kalbacker M, among Hematopoietic of the American Society Khan T, Majhail NS, Murphy Cell Transplantation for Blood and Marrow EA, Paplham P, Parran L, Professionals Transplantation. Perales M‐A, Rockwood TH, doi:10.1016/j.bbmt.201 Schmit‐Pokorny K, Shanafelt 7.11.015. Epub 2017 TD, Stenstrup E, Wood WA, Dec 2 Burns LJ
Page | 128 CIBMTR 2017 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS
APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS
The following publications were generated by the Bioinformatics Research Program, which provides expertise in, and conducts research on, translational and operational bioinformatics. For more information, see Section 2.5. BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID Kamoun M, McCullough KP, HLA amino acid Transplantation. N/A Maiers M, Fernandez Vina polymorphisms and 101(5):e170‐e177. MA, Li H, Teal V, Leichtman kidney allograft survival doi:10.1097/TP.000000 AB, Merion RM 0000001670. Epub 2017 Feb 18 Becnel LB, Hastak S, Ver Hoef BRIDG: A domain Journal of the American N/A W, Milius RP, Slack J, Wold D, information model for Medical Informatics Glickman ML, Brodsky B, Jaffe translational and clinical Association: JAMIA. C, Kush R, Helton E protocol‐driven 2017 Sep 1; 24(5):882‐ research. 890. doi:10.1093/jamia/ ocx004. Epub 2017 Feb 26 Halagan M, Manor S, Shriki N, East meets west ‐ impact Biology of Blood and N/A Yaniv I, Zisser B, Madbouly A, of ethnicity on donor Marrow Maiers M, Stein J match rates in the Ezer Transplantation: Journal Mizion Bone Marrow of the American Society Donor Registry for Blood and Marrow Transplantation. 2017 Aug 1; 23(8):1381‐1386. doi:10.1016/j.bbmt.201 7.04.005. Epub 2017 Apr 7 Pappas DJ, Lizee A, Paunic V, Significant variation The Pharmacogenomics PMC5656547 Beutner KR, Motyer A, between SNP‐based HLA Journal. doi:10.1038 Vukcevic D, Leslie S, Biesiada imputations in diverse /tpj.2017.7. Epub 2017 J, Meller J, Taylor KD, Zheng populations: the last Apr 25 X, Zhao LP, Gourraud P‐A, mile is the hardest Hollenbach JA, Mack SJ, Maiers M Roe D, Vierra‐Green C, Pyo C‐ Revealing complete Genes and Immunity. PMC5637231 W, Eng K, Hall R, Kuang R, complex KIR haplotypes 2017 Sep 1; 18(3):127‐ Spellman S, Ranade S, phased by long‐read 134. doi:10.1038 Geraghty DE, Maiers M sequencing technology /gene.2017.10. Epub 2017 Jun 1
Page | 129 CIBMTR 2017 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID Xie C, Yeo ZX, Wong M, Piper Fast and accurate HLA Proceedings of the PMC5544337 J, Long T, Kirkness EF, Biggs typing from short‐read National Academy of WH, Bloom K, Spellman S, next‐generation Sciences of the United Vierra‐Green C, Brady C, sequence data with xHLA States of America. 2017 Scheuermann RH, Telenti A, Jul 3; 114(30):8059‐ Howard S, Brewerton S, 8064. Turpaz Y, Venter JC doi:10.1073/pnas.17079 45114. Epub 2017 Jul 3 Alter I, Gragert L, Fingerson S, HLA class I haplotype PLOS Computational PMC5590998 Maiers M, Lousoun Y diversity is consistent Biology. with selection for 13(8):e1005693. frequent existing doi:10.1371/journal.pcb haplotypes i.1005693. Epub 2017 Aug 28 Louziun Y, Alter I, Gragert L, Modeling coverage gaps Immunogenetics. N/A Albrecht M, Maiers M in haplotype frequencies doi:10.1007/s00251‐ via Bayesian inference to 017‐1040‐4. Epub 2017 improve stem cell donor Nov 9 selection Chang C‐J, Osoegawa K, Milius Collection and storage of Human Immunology. [PMC Journal RP, Maiers M, Xiao W, HLA NGS genotyping doi:10.1016/j.humimm. – In Process] Fernandez‐Vina M, Mack SJ data for the 17th 2017.12.004. Epub 2017 International HLA and Dec 14 Immunogenetics Workshop Jones RB, Martinez C, Majhail Stem cell transplantation Biology of Blood and N/A NS, Prestegaard M, Maiers M, and informatics ‐ current Marrow Horwitz M, Komanduri K considerations Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.201 7.12.792. Epub 2017 Dec 27
Page | 130 CIBMTR 2017 Annual Report APPENDIX D5: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS APPENDIX D5: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS
The following publications were generated by the Statistical Methodology Research Program, which develops and evaluates the statistical models used in HCT. For more information, see Section 2.6. STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID Zheng C, Dai R, Hari PN, Instrumental variable with Statistics in Medicine. PMC5479873 Zhang M‐J competing risk model 2017 Apr 15; 36(8):1240‐1255. doi:10.1002/sim.7205. Epub 2017 Jan 8 Ahn KW, Banerjee A, Sahr N, Group and within‐group Lifetime Data Analysis. N/A Kim S variable selection for doi:10.1007/s10985‐ competing risks data 017‐9400‐9. Epub 2017 Aug 4 Liu Y, Logan BR, Liu N, Xu ZY, Deep reinforcement 2017 IEEE N/A tang J, Wang YZ learning for dynamic International treatment regimens on Conference on medical registry data Healthcare Informatics. 2017 Aug 23‐26. doi:10.1109 /ICHI.2017.45. Epub 2017 Sep 14 Logan BR, Sparapani R, Decision making and Statistical Methods in N/A McCulloch RE, Laud PW uncertainty quantification Medical Research. for individualized doi:10.1177/0962280 treatments using Bayesian 217746191. Epub Additive Regression Trees 2017 Dec 18
Page | 131 CIBMTR 2017 Annual Report APPENDIX D6: PUBLICATIONS NOT PREVIOUSLY REPORTED
APPENDIX D6: 2016 PUBLICATIONS NOT PREVIOUSLY REPORTED
The following publications were not presented in the 2016 CIBMTR Annual Report because they were published at the end of the year. 2016 PUBLICATIONS NOT PREVIOUSLY REPORTED Authors Title Citation PMCID (BMT CTN) Steroids versus steroids Biology of Blood and PMC5045896 Rashidi A, DiPersio JF, plus additional agent in Marrow Transplan‐ Sandmaier BM, Colditz GA, frontline treatment of tation: Journal of the Weisdorf DJ acute graft‐versus‐host American Society for disease: a systematic Blood and Marrow review and meta‐analysis Transplantation. 2016 of randomized trials Jun 1; 22(6):1133‐ 1137. Epub 2016 Mar 10 (Statistical Methodology Pseudo‐value approach The Annals of Applied PMC5656291 Research Program) for conditional quantile Statistics. 2016 Jun 1; Ahn KW, Logan BR residual lifetime analysis 10(2):618‐637. Epub for clustered survival and 2016 Jul 22 competing risks data (Bioinformatics Research Application of high‐ PLoS One. PMC5087893 Program) throughput next‐ 11(10):e0165810. Yin Y, Lan JH, Nguyen D, generation sequencing for Epub 2016 Oct 31 Valenzuela N, Takemura P, HLA typing on buccal Bolon Y‐T, Springer B, Saito extracted DNA: Results K, Zheng Y, Hague T, Pasztor from over 10,000 donor A, Horvath G, Rigo K, Reed recruitment samples EF, Zhang Q (BMT CTN) US intergroup study of Blood Advances. PMC5642915 Ravandi F, Othus M, O'Brien chemotherapy plus 1(3):250‐259. Epub S, Forman SJ, Ha CS, Wong dasatinib and allogeneic 2016 Dec 27 JYC, Tallman MS, Paietta E, stem cell transplant in Racevskis J, Uy GL, Horowitz Philadelphia chromosome M, Takebe N, Little R, Borate positive ALL U, Kebriaei P, Kingsbury L, Kantarjian H, Radich JP, Erba HP, Appelbaum FR (Chronic Leukemia Working Transplantation for Hematology / the N/A Committee) myelodysplastic Education Program of Saber W, Horowitz MM syndromes: Who, when, the American Society and which conditioning of Hematology. 2016 regimens Dec 2; 2016(1):478‐ 484
Page | 132 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
APPENDIX E: PRESENTATIONS
2017 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI 13‐SCP Individualized treatment summaries and survivorship Oral N Majhail care plans for hematopoietic cell transplant survivors reduces cancer treatment distress in a randomized multicenter study Bio‐ MIHAIP: Comprehensive pipeline to discover Oral Wei W informatics immunogenic minor histocompatibility antigens via whole genome sequences of HLA‐matched donor‐ recipient pairs Bio‐ Selecting optimal cord blood units: The limitations of Poster M Halagan informatics cell dose Bio‐ GFE ACT: A community resource for calling HLA allele Poster M Halagan informatics names from consensus sequences with gene feature enumeration notation Bio‐ Machine learning model that predicts binding between Poster M Maiers informatics KIR3DL1 and HLA Class I allotypes BMT CTN Efficacy of early post‐transplant vaccination using a Poster E Waller 0201 prime and boost strategy is independent of a prior history of GVHD: Results from BMT CTN 0201 BMT CTN Day 100 levels of blood immune cells are prognostic Oral S Fernando 0201 biomarkers for allogeneic hematopoietic stem cell transplant outcomes: Results from BMT CTN 0201 BMT CTN Amphiregulin improves stratification of the refined Oral S Holtan 0302 / 0802 Minnesota acute graft‐vs‐host disease risk score: Results from BMT CTN 0302/0802 BMT CTN Easy to read informed consent form for hematopoietic Poster N Majhail 1205/HSR12‐ cell transplantation clinical trials: Results from BMT CTN 02 1205 study CK12‐01 Optimal timing of allogeneic stem cell transplantation Poster B Hu for chronic myeloid leukemia patients in the tyrosine kinase inhibitor era CK14‐02 Prognostic score and cytogenetic risk classification for Oral J Brown chronic lymphocytic leukemia patients who underwent reduced intensity conditioning allogeneic HCT: A CIBMTR report
Page | 133 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI CK15‐02 Comparison of outcomes after myeloablative versus Oral S Chhabra reduced intensity conditioning allogeneic hematopoietic cell transplantation for chronic myeloid leukemia Corporate Nicord single unit expanded umbilical cord blood Oral M Horwitz transplantation: Final results of a multicenter Phase I / II trial DS13‐01 Bone marrow transplant product quality has decreased Poster N over time. A retrospective examination of NMDP Prokopishyn collected bone marrow products from 1994‐2016 GS16‐03 Selecting between HLA‐matched siblings and HLA‐ Oral E Fuchs haploidentical related donors for acute leukemia in the era of post‐transplant cyclophosphamide: The Center for International Blood and Marrow Transplant Registry and the Acute Leukemia working party of the European Society for Blood and Marrow Transplant GV16‐01(1) Graft‐vs‐host disease free relapse‐free survival and Poster R Mehta chronic GVHD in alternative donor hematopoietic cell transplantation in pediatric patients GV16‐02(2) Graft‐versus‐host disease free relapse‐free survival and Oral R Mehta chronic GVHD in alternative donor hematopoietic cell transplantation in adults HSR13‐02 Individualized treatment summaries and survivorship Oral E Murphy care plans for hematopoietic cell transplant survivors reduces cancer treatment distress in a randomized multicenter study HSR15‐01 Healthcare reimbursement and service utilization for Poster L Mau one year of post‐alloHCT care for Medicare beneficiaries age 65 and older with acute myeloid leukemia HSR16‐03 A national survey of transplant physicians’ attitudes Poster A El‐Jawahri about palliative care IB10‐01c Chromosome 6 loss of heterozygosity in pre‐transplant Poster S Gadalla blood samples of patients with severe aplastic anemia is associated with lower risk of acute graft‐versus‐host disease
Page | 134 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI IB10‐01d Donor lymphocyte cell‐specific telomere length and Poster S Gadalla causes of death after unrelated hematopoietic cell transplant in patients with marrow failure IB14‐08 Analysis of 10,462 8/8 HLA‐matched unrelated donor Oral B Shaw transplants could not identify a donor selection score, as younger age is the only significant donor characteristic associated with survival LE16‐01(1) Late fatal infections remain higher than expected in Oral M Norkin adults receiving allogeneic stem cell transplant LE16‐02(2) Late fatal infections remains frequent cause of Oral M Norkin mortality in pediatric allogeneic stem cell transplant recipients LK13‐02 Prognostic significance of cytogenetic abnormalities in Poster A Lazaryan patients with Philadelphia‐negative ALL undergoing allogeneic hematopoietic stem cell transplantation in complete remissions: A CIBMTR analysis LK15‐02 Graft‐vs‐Leukemia effect in acute lymphoblastic Oral M Yeshurun leukemia: Mild acute graft‐vs‐host disease protects against relapse and improves survival after allogeneic transplantation: A CIBMTR analysis MM16‐01(2) Revised‐international staging system is independently Poster S Kumar predictive and prognostic for early relapse (<24 months) after upfront autologous hematopoietc cell transplantation for newly diagnosed multiple myeloma MM16‐01(2) A comparison between 3 staging systems in multiple Poster E Scott myeloma using the CIBMTR database NM16‐01 Allogeneic hematopoietic stem cell transplantation in Poster R Eikema older patients aged 50 years or older with severe aplastic anemia: Results from the European Society for Blood and Marrow Transplant and the CIBMTR RT16‐01 The effect of conditioning regimen dose reduction in Poster C Brunstein obese patients undergoing autologous transplantation SC17‐01 Allogeneic hematopoietic cell transplantation for acute Oral M Eapen myeloid leukemia and myelodysplastic syndrome: Effect of transplant conditioning regimen intensity on outcomes
Page | 135 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 BMT TANDEM MEETINGS Study Title Type PI 10‐CBA Excellent outcomes in 1589 patients receiving umbilical Poster K Ballen cord blood transplantation using unlicensed units from a centralized cord blood registry BMT CTN Multi‐state modeling identifies significant determinants Oral J Pidala 0201 / 0402 of immune suppression discontinuation and risk for subsequent graft vs. host disease: A comprehensive secondary data analysis of BMT CTN 0201 and 0402 trials BMT CTN Follistatin and Endoglin: Potential biomarkers of Oral S Holtan 0402 endothelial damage and non‐relapse mortality after myeloablative allogeneic hematopoietic cell transplantation in BMT CTN 0402 HSR16‐01 Understanding the physician’s perspectives about Oral N Khera translating research into clinical practice: Example of BMT CTN 0201 results HSR16‐02 Lessons learned from merging CIBMTR data and CMS Poster E Denzen Medicare claims data L Mau IB14‐07 Analysis of predicted indirectly recognizable HLA Poster E Spierings epitopes (PIRCHE) in mismatched unrelated donor HCT: A CIBMTR cohort study IB15‐06 Recipient Pre‐HCT telomere length and outcomes after Poster Y Wang unrelated donor HCT for acute leukemia IN07‐01 / Impact of early bloodstream infection (BSI) by Poster C Ustun IN11‐01 vancomycin‐resistant enterococci (VRE) on long‐term transplant outcomes LE14‐01 Risks of new myeloid neoplasms after autologous Oral S Hashmi transplant for plasma cell myeloma and lymphomas: R Dean Effects of drug and ionizing radiation exposures LE15‐01 Long‐term outcomes among two‐year survivors of Oral P Satwani autologous hematopoietic cell transplant for Hodgkin and diffuse large B‐cell lymphoma LY15‐03 AutoHCT is associated with improved overall survival Oral C Casulo (OS) in follicular lymphoma patients (pts) experiencing J Friedberg early therapy failure after frontline chemo‐ immunotherapy: A NLCS & CIBMTR analysis LY16‐01 AlloHCT for extranodal NK/T‐cell lymphoma, nasal type Oral A Kanate (ENKL): A CIBMTR analysis
Page | 136 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 BMT TANDEM MEETINGS Study Title Type PI LY16‐05 Rituximab versus non‐rituximab containing reduced Oral N Epperla intensity conditioning (RIC) regimens for alloHCT in b‐ cell non‐Hodgkin lymphomas (B‐NHL): A CIBMTR analysis MM14‐03 Trends in survival outcomes among patients (pts) Oral A Dispenzieri relapsing early after autologous stem cell S Kumar transplantation (ASCT) for MM RT09‐04/IB09‐ Novel genetic variants associated with death due to Oral T Hahn 06d acute lymphoblastic leukemia within one year after HLA‐matched unrelated donor blood and marrow transplantation (DISCOVeRY‐BMT study) RT09‐04 / Functional genetic variants on 14q32 associate with Oral L Sucheston‐ IB09‐06e death due to AML and MDS within one year after HLA‐ Campbell matched unrelated donor blood and marrow transplantation (DISCOVeRY BMT Study) RT13‐02 Impact of higher dose total body irradiation Oral M Sabloff conditioning on outcome of an allogeneic hematopoietic cell transplant in the modern era SC15‐03 Stratification of allogeneic hematopoietic cell Poster W Saber transplant patients by risk of developing veno‐occlusive disease: A model for assigning a risk score SC16‐04 Results of a Phase II clinical trial of tocilizumab, Oral W Drobyski tacrolimus and methotrexate (TOC/TAC/MTX) for the prevention of acute GVHD and matched case control comparison to TAC/MTX after allogeneic stem cell transplantation
Page | 137 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 AMERICAN SOCIETY OF CLINICAL ONCOLOGY (ASCO) ANNUAL MEETING Study Title Type PI BMT CTN 0702 Response status as predictor of survival after Poster G Somlo autologous hematopoietic cell transplant (AHCT), without or with consolidation (with bortezomib, lenalidomide (Len) and dexamethasone) and len maintenance (AM vs. ACM) versus tandem AHCT and len maintenance (TAM) for up‐front treatment of patients (pts) with MM. BMT CTN 0702‐STaMINA (NCT01109004) BMT CTN 0903 AlloHCT for hematologic malignancies in human Oral RF Ambinder immunodeficiency virus infected (HIV) patients (pts): BMT CTN 0903/AIDS malignancy consortium (AMC‐ 080) trial
LY16‐04 Autologous (auto) versus matched sibling donor (MSD) Oral J Godrey or MUD allogeneic (allo) HCT in follicular lymphoma (FL) patients (pts) with early chemoimmunotherapy failure (ECF): A CIBMTR analysis
2017 AI GENOMICS HACKATHON Study Title Type PI Bio‐ ML based Prediction of Immunogenic Neoantigens for Oral A Yao informatics Immunotherapy
2017 AMERICAN SOCIETY OF PEDIATRIC HEMATOLOGY / ONCOLOGY (ASPHO) Study Title Type PI RCI BMT Female gender and malignancy are associated with Oral N Bhatt 09‐SQOL low PEDSQL scores at 12 months post‐hematopoietic cell transplantation
Page | 138 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 AMERICAN STATISTICAL ASSOCIATION JOINT STATISTICAL MEETING Study Title Type PI BMT CTN Phase II trials and the use of registry controls Oral B Logan
2017 BIOSTATISTICS IN THE MODERN COMPUTING ERA CONFERENCE Study Title Type PI BMT CTN Group sequential tests of treatment effect on survival Oral M Martens 0402 and cumulative incidence at a fixed time point
2017 EUROPEAN FEDERATION FOR IMMUNOGENETICS (EFI) Study Title Type PI Bio‐ Community resources for automated annotation of Poster M Maiers informatics HLA, KIR and beyond Bio‐ HLA and the EMR: Developing HL7 FHIR tools for Poster RP Milius informatics exchanging NGS‐based HLA genotyping
2017 EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION (EBMT) ANNUAL MEETING
Study Title Type PI
RCI BMT Minimal Residual Disease (MRD) pre‐ and post‐ HCT for Poster D Jacobsohn 09‐MRD children with AML is highly predictive of event‐free survival: A Pediatric Blood and Marrow Transplant Consortium Study
2017 INDIVIDUALIZING MEDICINE CONFERENCE ADVANCING CARE THROUGH GENOMICS
Study Title Type PI
Bio‐ Predicting structural haplotypes of human KIR from Poster D Roe informatics whole genome sequences and its application to GoNL
Page | 139 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 INTERNATIONAL CANCER EDUCATION CONFERENCE
Study Title Type PI
HSR13‐02 Development of patient‐centered treatment summary Poster E Murphy and survivorship care plans: patient and provider perspectives
2017 INTERNATIONAL CHINESE STATISTICAL ASSOCIATION MEETING
Study Title Type PI
BMT CTN Randomized Phase II trials vs. single arm Phase II trials Oral B Logan with a registry control in rare diseases
2017 INTERNATIONAL CONFERENCE ON LONG‐TERM COMPLICATIONS OF TREATMENT OF CHILDREN AND ADOLESCENTS FOR CANCER
Study Title Type PI
RCI BMT 09‐ Variations in self‐reported and parent proxy PedsQL Poster N Bhatt SQOL scores in pediatric patients undergoing hematopoietic cell transplantation
Page | 140 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 INTERNATIONAL MYELOMA WORKSHOP
Study Title Type PI
BMT CTN Post autoHCT therapies in high risk MM. Subgroup Oral A Krishnan 0702 analysis of Phase III BMT CTN 0702‐STaMINA: AutoHCT followed by lenalidomide maintenance (Len) (AM) versus auto HCT and len and bortezomib (BZ) and dexamethasone consolidation len maintenance (ACM) vs tandem autoHCT len maintenance BMT CTN Lenalidomide (LEN) maintenance following high‐dose Oral P McCarthy 0704 / CALGB melphalan and ASCT in patients (Pts) with newly 100104 / diagnosed MM: A meta‐analysis of overall survival (OS) ECOG 100104 BMT CTN Overall survival (OS) and progression‐free survival (PFS) Oral P McCarthy 0704 / CALGB adjusted for treatment crossover in the CALGB/ECOG 100104 / 100104 (Alliance) study of lenalidomide (LEN) versus ECOG 100104 placebo (PBO) maintenance after stem cell transplant (SCT) for patients with newly diagnosed multiple myeloma
2017 INTERNATIONAL SOCIETY FOR CELLULAR THERAPY
Study Title Type PI
BMT CTN Development and management of a multi‐center, Poster C Nelson 1401 center‐specific cellular therapy manufacturing approach: The experience of the BMT CTN Protocol #1401
2017 PATIENT‐CENTERED OUTCOMES RESEARCH INSTITUTE (PCORI) ANNUAL MEETING
Study Title Type PI
HSR15‐04 Engaging patients in developing a patient‐centered HCT Poster L Burns outcomes research agenda
Page | 141 CIBMTR 2017 Annual Report APPENDIX E: PRESENTATIONS
2017 SOCIETY FOR CLINICAL TRIALS
Study Title Type PI
BMT CTN Translation of a single‐center cellular therapy Poster C Nelson 1401 manufacturing approach to a multi‐center, center‐ specific manufacturing platform: The experience of the BMT CTN Protocol #1401
Page | 142 CIBMTR 2017 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS
APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS
This study development and management process pertains to studies for which the CIBMTR provides data, scientific, and statistical support. Data sets are also made available to investigators who have their own statistical resources. Final analyses and manuscripts resulting from these analyses are reviewed and approved by the CIBMTR prior to journal submission.
STUDY DEVELOPMENT AND MANAGEMENT PROCESS
Protocol pending. Proposals remain in this preliminary stage until the PI creates a Planned draft protocol.
Draft protocol received. When a PI submits a draft protocol, Coordinating Center staff review it.
Protocol development. During the development process, the Working Committee biostatisticians, Scientific Director, and Chairs refine the submission into a comprehensive study protocol. They add a table with a preliminary description of the proposed study population and present the draft protocol for discussion at a weekly Coordinating Center statistical meeting. When a protocol is approved, Coordinating Center personnel invite Working Committee members to participate in a Writing Committee.
In Progress Sample typing. If applicable, the PIs perform laboratory tests (e.g., genotyping) on samples from the CIBMTR Research Repository. The testing data will be used in the analysis to determine any correlation with clinical outcome.
Supplemental forms / data collection. Most studies use routinely‐collected data. If necessary, Coordinating Center staff, in collaboration with the PI and relevant Working Committee Chairs, develop a supplemental form, which is approved prior to soliciting centers for additional data. Use of supplemental data (e.g., data not collected on standard CIBMTR data collection forms) is discouraged unless it will result in a particularly meaningful publication and/or external funding can support the extra burden placed on centers and supplement forms reimbursement costs.
Page | 143 CIBMTR 2017 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS
STUDY DEVELOPMENT AND MANAGEMENT PROCESS
Data file preparation. The objective of data file preparation is to create a file of eligible subjects who are consecutively treated at participating centers with adequate follow‐up, with minimal missing data fields, and in large enough numbers to give the analysis sufficient statistical power to meet the stated study objectives. This process involves a series of steps by the MS‐level statistician, working with the Scientific Director, PI(s), and sometimes the Clinical Research Coordinator, to ensure data quality: Verifying selection criteria Including and excluding patients so that the investigators can determine whether the final study population is representative of the target population Assessing follow‐up Determining the extent and nature of missing values and their potential effect on the study In Progress Resolving and reconciling data discrepancies / outliers by examining data (continued) collection forms and communicating with centers and the PI Analysis in progress. Analysis proceeds in several phases. The first generally includes a detailed description of the patient population and univariate and multivariate analyses of study endpoints. Study PI(s) and associated Working Committee Chairs present these data for discussion at a weekly Coordinating Center statistical meeting and then distribute them to Writing Committee members for suggestions and comments. The PI works with Coordinating Center staff in an iterative process to review comments from the Writing Committee. The process repeats until final analysis, which serves as the basis for the manuscript.
Ongoing. A study in ongoing status is long‐term and often involves multiple grants and/or renewals outside of the CIBMTR in order to reach its objectives. The study typically has its own Statistical Director for analysis, but it requires data from the CIBMTR, usually each year.
Manuscript preparation. The PI is primarily responsible for manuscript preparation and is expected to prepare a draft manuscript within 30 days of receiving analysis results. Study Leadership reviews and revises the document, ensuring that the description and interpretation of the statistical analyses are Preliminary accurate and contribute to the fundamental message of the manuscript. The Results Coordinating Center then distributes the approved first draft to the Writing Committee and solicits feedback. The PI incorporates comments from the Writing Committee and creates a revised draft, which is reviewed in an iterative process by the Writing Committee until reaching a reasonable consensus on a final manuscript.
Page | 144 CIBMTR 2017 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS
STUDY DEVELOPMENT AND MANAGEMENT PROCESS
Submitted. The Coordinating Center staff is responsible for submitting the manuscript and corresponding with the chosen journal. The Working Committee Scientific Director often serves as corresponding author, and the study statistician Preliminary forwards all editor and reviewer comments to the PI and Statistical Director. The Results PI is expected to prepare a response, working with Study Leadership who provide additional analyses of data, as needed. Coordinating Center personnel (continued) communicate with the journal, including re‐submissions, in most cases.
In press. A publication is in press when it has been approved but does not yet have a citation.
Published. A manuscript is considered published when a citation is available, Completed including a PMCID number, if applicable. For a list of 2016 publications, see Appendix D.
Page | 145 CIBMTR 2017 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS
APPENDIX G: CLINICAL STUDIES AND TRIALS
Through the Clinical Trials Support Program, the Coordinating Center supports clinical trial planning and interpretation; data collection, including long‐term follow‐up data; and real‐time accrual assessment. See Section 2.3 for more information. APPENDIX G1: BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT
The BMT CTN (Section 2.3.1) is the US national trials group charged with developing and conducting multicenter Phase II and III clinical trials focused on HCT. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation. A status of BMT CTN trials open for enrollment is included in this appendix and is available on the BMT CTN website. For additional information on completed Network trials, see the annual progress report on the BMT CTN website.
BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT Protocol Title Status to Date Number BMT CTN 07LT Continued, long‐term follow‐up and Opened in Jan 2015 to patients lenalidomide maintenance therapy for enrolled on BMT CTN 0702 without patients who were enrolled on BMT CTN disease progression 0702 Anticipated accrual completion in 2018 BMT CTN Phase III study comparing HLA‐ Opened to accrual Jun 2012 1101 haploidentical related donor bone 354 of 410 patients enrolled marrow versus double umbilical cord Anticipated accrual completion in blood with reduced‐intensity conditioning 2018 for patients with hematologic malignancies BMT CTN A multi‐center biologic assignment trial Opened to accrual Dec 2013 1102 comparing reduced intensity allogeneic 314 of 338‐400 patients enrolled hematopoietic cell transplant to Anticipated accrual completion in hypomethylating therapy or best early 2018 supportive care in patients aged 50‐75 with intermediate‐2 and high risk myelodysplastic syndrome BMT CTN A randomized double‐blind Phase III study Opened to accrual Jul 2016 1201 of ibrutinib during and following 10 of 80 patients enrolled (Alliance autologous stem cell transplantation Anticipated accrual completion in A051301) versus placebo in patients with relapsed mid 2018 or refractory diffuse large B‐cell lymphoma of the activated B‐cell subtype
Page | 146 CIBMTR 2017 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS
BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT Protocol Title Status to Date Number BMT CTN Phase III trial of calcineurin inhibitor‐free Opened to accrual Aug 2015 1301 interventions for prevention of graft‐ 298 of 345 patients enrolled versus host‐disease Anticipated accrual completion in mid 2018 BMT CTN Phase II, double‐blind placebo controlled Opened to accrual Aug 2015 1302 trial of maintenance ixazomib after 39 of 138 patients enrolled allogeneic HCT for high risk multiple Anticipated accrual completion in myeloma mid 2019 BMT CTN A randomized Phase III study comparing Opened to accrual by BMT CTN 1304 conventional dose treatment using a Nov 2013 (DFCI 10‐106) combination of lenalidomide, bortezomib, 717 of 720 patients enrolled and dexamethasone (RVD) to high‐dose Anticipated accrual completion in treatment with peripheral stem cell early 2018 transplant in the initial management of myeloma in patients up to 65 years of age BMT CTN A Phase II multicenter trial of single Opened to accrual Jul 2016 1401 autologous HCT followed by lenalidomide 102 of 188 patients enrolled maintenance for multiple myeloma with Anticipated accrual completion in or without vaccination with dendritic early 2019 cell/myeloma fusions BMT CTN A randomized, Phase II, multicenter, open Opened to accrual Oct 2016 1501 label, study evaluating sirolimus and 107 of 150 patients enrolled prednisone in patients with refined Anticipated accrual completion in Minnesota standard risk, Ann Arbor 1/2 early 2018 confirmed acute GVHD BMT CTN Optimizing cord blood and haploidentical Opened to accrual May 2017 1502 aplastic anemia transplantation (CHAMP) 5 of 60 patients enrolled Anticipated accrual completion in mid 2020 BMT CTN A study to compare bone marrow Opened to accrual Nov 2016 1503 transplantation to standard care in 34 of 200 patients enrolled adolescents and young adults with severe Anticipated accrual completion in sickle cell disease late 2019 BMT CTN A multi‐center, randomized, double‐blind, Opened to accrual May 2017 1506 placebo‐controlled Phase III Trial of the 28 of 346 patients enrolled FLT3 inhibitor gilteritinib administered as Anticipated accrual completion in maintenance therapy following allogeneic mid 2019 transplant for patients with FLT3/ITD AML
Page | 147 CIBMTR 2017 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS
BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT Protocol Title Status to Date Number BMT CTN Reduced intensity conditioning for Opened to accrual Oct 2017 1507 haploidentical bone marrow 2 of 80 patients enrolled transplantation in patients with Anticipated accrual completion in symptomatic sickle cell disease late 2021 BMT CTN A randomized Phase III trial of Opened to accrual Aug 2017 1601 consolidation with autologous HCT No patients enrolled yet (ECOG‐ACRIN followed by maintenance rituximab vs. Anticipated accrual completion in EA4151) maintenance rituximab alone for patients mid 2021 with mantle cell lymphoma in minimal residual disease‐negative first complete remission
Page | 148 CIBMTR 2017 Annual Report APPENDIX G2: RCI BMT CLINICAL STUDIES
APPENDIX G2: RCI BMT CLINICAL STUDIES
The RCI BMT (Section 2.3.2) provides researchers in the field of cellular therapy with infrastructure and expertise in clinical trial conduct and analysis. The program’s goal is to help investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting into such groups as the BMT CTN or the national cancer cooperative groups. It also facilitates large survey and cohort studies. A status of its projects is included in this appendix. RCI BMT CLINICAL STUDIES Protocol Title Status to Date Number 09‐SQOL Pilot study to assess the feasibility of Closed to accrual Sep 2013 collecting quality of life data in 301 adults and 89 pediatric recipients collaboration with the SCTOD enrolled Manuscript published Aug 2017
11‐TREO Multi‐center study evaluating Closed to accrual April 2014 treosulfan, fludarabine, and low‐dose 40 pediatric recipients enrolled TBI in children with AML / MDS Abstracts presented at 2016 EBMT; 2016 undergoing allogeneic HCT BMT Tandem Meetings Data file preparation complete; manuscript draft in process 06‐DON RDSafe: A multi‐institutional study of Closed to accrual July 2014 hematopoietic stem cell donor safety 1,812 donors enrolled and quality of life Follow up assessments completed July 2015 Ancillary study manuscript published June 2017; primary manuscript submitted for publication Dec 2017 09‐MRD A multi‐center study to determine Closed to accrual Oct 2014 the role of minimal residual disease 150 pediatric recipients enrolled testing before and after HCT for Data file preparation complete; pediatric acute myeloid leukemia manuscript draft in process 09‐PLEX A Phase II study evaluating the safety Closed to accrual Dec 2014 and efficacy of intravenous plerixafor 128 enrolled (64 recipient‐donor pairs) for the mobilization and Follow‐up completed Feb 2016 transplantation of HLA‐matched Data file preparation complete; sibling donor hematopoietic stem manuscript draft in process cells in recipients with hematological malignancies
Page | 149 CIBMTR 2017 Annual Report APPENDIX G2: RCI BMT CLINICAL STUDIES RCI BMT CLINICAL STUDIES Protocol Title Status to Date Number rHuG‐CSF Long‐term follow‐up study evaluating Closed to accrual Oct 2015 hematologic and non‐hematologic 21,794 unrelated donors enrolled cancers, thrombotic events, and Follow‐up continues through 2020 autoimmune disorders in unrelated Interim analysis completed; reviewed by donors undergoing bone marrow NMDP DPSM in Nov 2016 harvest versus peripheral blood stem cell mobilization with recombinant human granulocyte colony‐ stimulating factor PBSC Filgrastim‐mobilized peripheral blood Opened to accrual Apr 1996 stem cells for allogeneic >36,000 unrelated donors enrolled transplantation with unrelated Will close to accrual upon FDA license of donors product; another protocol will open for all unlicensed product 10‐CBA A multi‐center access and distribution Opened to accrual Oct 2011 protocol for unlicensed 3,717 recipients enrolled; 449 enrolled cryopreserved cord blood units for in 2017 transplantation in pediatric and adult Open indefinitely to allow access and patients with hematologic distribution of unlicensed cord blood malignancies and other indications units 10 CMS‐ Assessment of allogeneic HCT in Opened to accrual Dec 2010 MDS‐1 Medicare beneficiaries with MDS and 1,697 patients enrolled related disorders KIR‐DS A multi‐center study looking at the Closed to accrual Aug 2016 selection of a favorable KIR donor Unrelated donor sample management 2,099 unrelated donors approached; 920 provided KIR sample at confirmatory typing timepoint Study closed with the NMD IRB Nov 2017 Statin Impact of donor statin use on graft‐ Closed to accrual Oct 2016 versus‐host disease after unrelated 6,673 of 7,000 donors enrolled donor HCT; URD data collection Data file preparation in process BMT CTN A study comparing reduced intensity Survey Research Group performing QOL 1102‐QOL allogeneic hematopoietic cell assessments transplant to hypomethylating 203 total assessments completed in 2017 therapy or best supportive care in patients aged 50‐75 with intermediate‐2 and high risk myelodysplastic syndrome
Page | 150 CIBMTR 2017 Annual Report APPENDIX G2: RCI BMT CLINICAL STUDIES RCI BMT CLINICAL STUDIES Protocol Title Status to Date Number BMT CTN A cost effectiveness ancillary study to Collaboration with Fred Hutchinson 1102‐ parent study 1102‐QOL above Cancer Research Center Ancillary CEA Survey Research Group performing Cost study Effectiveness Analysis (CEA) survey collection First subject contacted in Oct 2015 73 surveys completed in 2017 13‐TLEC Prospective non‐therapeutic study, Opened to accrual Mar 2015 assessing the long‐term toxicity of 305 of 340 recipients enrolled; 143 HCT for childhood leukemia enrolled in 2017 13‐SCP A randomized study to evaluate the Collaboration with Health Services impact of survivorship care planning Research Program on cancer survivors’ self‐ Closed to accrual June 2016 management and adherence to care 495 recipients enrolled recommendations and utilization of Data file preparation completed and follow‐up care manuscript draft in process HPD High priority donor project: Survey First subjects contacted by the Survey Research Group supporting Be The Research Group in Sep 2015 Match Operations Final outreach completed in Nov 2016 253 donors identified as potential participants; SRG reached 194; 170 agreed to participate MMP Millennial member project: Survey First subject contacted by the Survey Research Group supporting Be The Research Group in July 2016 Match Operations project 1,446 registry members; 1,160 agreed to have contact information shared with study partner University of Pittsburgh Mesoscale Clinical evaluation of the MSD® point Open to accrual Aug 2016 of care biodosimetry test 20 of 20 recipients enrolled; 4 enrolled in 2017 Closed to accrual Feb 2017 Pending closure with NMDP IRB 15‐MMUD HLA‐mismatched unrelated donor Opened to accrual Aug 2016 bone marrow transplantation with 41 of 80 recipients enrolled; 40 enrolled post‐transplantation in 2017 cyclophosphamide for patients with hematologic malignancies
Page | 151 CIBMTR 2017 Annual Report APPENDIX G2: RCI BMT CLINICAL STUDIES RCI BMT CLINICAL STUDIES Protocol Title Status to Date Number State Street Generation of an induced pluripotent First donors contacted Jan 2017 stem cell bank immune matched to a 6 of 12 whole blood products collected majority of the US population 16‐NTCD Naïve T cell depletion for prevention Initial protocol team meeting Oct 2016 of chronic GVHD in pediatric Initial DSMB approval July 2017 population Initial NMDP IRB approval Nov 2017 PBMTC‐ Phase 1 study of CD33‐redirected Protocol team established June 2017 CAR‐T chimeric antigen receptor T cell Initial protocol team meeting Aug 2017 immunotherapy in children and young adults with relapsed or refractory acute myeloid leukemia INSPIRE‐SCP Integrating health informatics in a Collaboration with Fred Hutchinson scalable stepped care self‐ Cancer Research Center management program for HCT Initial protocol team meeting Feb 2017 survivors RCI BMT will prepare individualized care plans and contact patients for electronic surveys 17‐PRO Investigate quality of life in older vs A companion study to the CIBMTR CMS‐ younger patients receiving approved expanded access study transplants for myelodysplasia Serves as a pilot study for new ePRO system Protocol development began Nov 2017
Page | 152 CIBMTR 2017 Annual Report APPENDIX H: FORMS SUBMISSION PROCESS
APPENDIX H: FORMS SUBMISSION PROCESS
Center submits CRID Assignment Form (Form 2804), and CRID is generated Indication for CRID Assignment Form (Form 2814) is added to Forms Due list Center completes Indication Form and reports indication as HCT Pre‐TED (Form 2400) is added to Forms Due list Center completes and submits Pre‐TED Pre‐TED data are processed through the selection algorithm resulting in CRF or TED track o If autologous recipient declines consent for research, stop here. Otherwise, follow the appropriate track below CRF Track TED Track Forms 2004, 2005, and 2006 are added, depending on Forms 2004, 2005, and 2006 are added, depending 1 donor type and if the donor has been used for a prior on donor type, consent for sample repository, and if transplant.* the donor has been used for a prior transplant.* Baseline form 2000, disease specific inserts, and Follow‐ Post‐TED Follow‐up Form 2450 is added to Forms 2 up Forms are added to Forms Due list. Due list. Center completes designated Post‐TED Forms at 3 Center completes Baseline form after infusion. appropriate time points. Is recipient alive? If yes, go to Step 5. If no, report the Center completes designated CRF Follow‐up Forms at 4 death on the follow‐up form, and go to Reporting appropriate time points. Recipient Death. Is recipient alive? If yes, go to Step 6. If no, report the Did recipient have a subsequent transplant? If yes, go 5 death on the follow‐up form, and go to Reporting to Step 6. If no, continue reporting at next time point Recipient Death. (Step 3). Did recipient have subsequent transplant? If yes, go to Subsequent transplant is reported on the next 6 Step 7. If no, continue reporting at next time point (Step available follow‐up form. 4). When the form reporting the subsequent transplant When the form reporting the subsequent transplant is in is in complete status, future forms for the prior 7 complete status, future forms for the prior transplant will transplant will be automatically deleted from be automatically deleted from FormsNet. FormsNet. Center completes and submits Pre‐TED (Form 2400) for Center completes and submits Pre‐TED (Form 2400) 8 subsequent transplant. Go to Step 2 for subsequent for subsequent transplant. Go to Step 2 for transplant. subsequent transplant. Reporting Recipient Death The recipient’s death is reported on the Post TED. A Death Form 2900 is completed to report the recipient’s 2900 Death Form should not be completed for death.** patients on the TED track.
* For more details regarding when Forms 2004, 2005, and 2006 are required, see “How Forms Come Due (2004, 2005, and 2006)”. **Complete Death Form 2900 even if autopsy is pending. Another death form will be requested to confirm cause of death if autopsy was pending.
Page | 153 CIBMTR 2017 Annual Report APPENDIX I: WEBSITES APPENDIX I: WEBSITES
Throughout this report, electronic links to webpages and documents are provided; they are underlined and italicized for identification. If you are unable to access items using the links provided, enter the underlined and italicized words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org). URLs for the websites mentioned in this report are provided here.
Name URL Be The Match bethematch.org Be The Match Clinical bethematchclinical.org BMT CTN bmtctn.net CIBMTR cibmtr.org CIBMTR Collaborate collaborate.cibmtr.org CIBMTR Portal portal.cibmtr.org HRSA Blood Cell Transplant bloodcell.transplant.hrsa.gov
Page | 154 CIBMTR 20176 Annual Report APPENDIX J: GLOSSARY APPENDIX J: GLOSSARY
Abbreviation/ Meaning Acronym AGNIS A Growable Network Information System AIDS acquired immunodeficiency syndrome ALL acute lymphoblastic leukemia alloHCT allogeneic hematopoietic cell transplantation AMC AIDS Malignancy Consortium AML acute myeloid (myelogenous) leukemia ASBMT American Society of Blood and Marrow Transplantation ASCT autologous stem cell transplantation ASH American Society of Hematology autoHCT autologous hematopoietic cell transplantation BMT CTN Blood and Marrow Transplant Clinical Trials Network BRIDG Biomedical Research Integrated Domain Group BSI bloodstream infection CALGB Cancer and Leukemia Group B CDE common data elements CDISC Clinical Data Interchange Standards Consortium CED Coverage with Evidence Determination CIBMTR Center for International Blood and Marrow Transplant Research CITI Collaborative IRB Training Initiative CMS Centers for Medicare and Medicaid Services CPA center performance analytics CPI Continuous Process Improvement CRF Comprehensive Report Form CRID CIBMTR Recipient Identification Number CTED Cellular Therapy Essential Data DBtC Data Back to Centers application DFCI Dana Farber Cancer Institute DISCO Data and Information for Statistical Center Operations DRI Disease Risk Index EAIN Engagement Award Initiative Notice EBMT European Society for Blood and Marrow Transplantation eDBtC enhanced Data Back to Centers application EGF epidermal growth factor ENKL extranodal natural killer (NK) / T‐cell lymphoma, nasal type ePRO electronic patient‐reported outcomes FACT Foundation for the Accreditation of Cellular Therapy FDA Food and Drug Administration FHIR Fast Healthcare Interoperability Resources GFE ACT gene feature enumeration allele calling tool GVHD graft‐versus‐host disease
Page | 155 CIBMTR 20176 Annual Report APPENDIX J: GLOSSARY Abbreviation/ Meaning Acronym HCT hematopoietic cell transplantation HIPAA Health Insurance Portability and Accountability Act HIV human immunodeficiency virus HLA human leukocyte antigen HRSA Health Resources and Services Administration IBMTR International Bone Marrow Transplant Registry IND Investigational new drug IRB Institutional Review Board IT Information Technology KIR killer‐cell immunoglobulin‐like receptors MCW Medical College of Wisconsin MDS myelodysplastic syndrome MF myelofibrosis MIHA mediated by immunogenic minor histocompatibility antigens MIHAIP MiHA identification pipeline MM multiple myeloma MRD matched related donor MUD matched unrelated donor N/A not applicable NCI National Cancer Institute NK natural killer NGS next‐generation sequencing NHLBI National Heart, Lung, and Blood Institute NIAID National Institute of Allergy and Infectious Disease NIH National Institutes of Health NLCS national lymphocare study NMDP National Marrow Donor Program OS overall survival PBMTC Pediatric Blood and Marrow Transplant Consortium PBSC peripheral blood stem cell PCORI Patient‐Centered Outcomes Research Institute PI principal investigator PIRCHE predicted indirectly recognizable HLA epitopes PMCID PubMed Central unique identifier PNH paroxysmal nocturnal hemoglobinuria QOL quality of life RCI BMT Resource for Clinical Investigations in Blood and Marrow Transplantation RDSafe Related Donor Safety Study SAA severe aplastic anemia SCTOD Stem Cell Therapeutic Outcomes Database SNP single nucleotide polymorphisms SOS sinusoidal obstructive syndrome TED Transplant Essential Data
Page | 156 CIBMTR 20176 Annual Report APPENDIX J: GLOSSARY Abbreviation/ Meaning Acronym URD unrelated donor US United States VOD veno‐occlusive disease VRE vancomycin‐resistant enterococci vs versus WBMT Worldwide Network for Blood and Marrow Transplantation WMDA World Marrow Donor Association
Page | 157 The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID).
CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and Medical College of Wisconsin.
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