CIBMTR Scientific July , Working Committee Research Portfolio

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CIBMTR Scientific Working Committee Research Portfolio: July 1, 2017

TABLE OF CONTENTS 1.0 OVERVIEW ...... 1 1.1 Membership ...... 2 1.2 Leadership ...... 2 1.3 Productivity ...... 3 1.4 How to Get Involved ...... 3 2.0 ACUTE WORKING COMMITTEE ...... 6 2.1 Leadership ...... 6 2.2 Recent Publications ...... 6 2.3 Current Studies ...... 10 3.0 AUTOIMMUNE DISEASES AND CELLULAR THERAPIES WORKING COMMITTEE ...... 14 3.1 Leadership ...... 14 3.2 Recent Publications ...... 14 3.3 Current Studies ...... 14 4.0 CHRONIC LEUKEMIA WORKING COMMITTEE ...... 16 4.1 Leadership ...... 16 4.2 Recent Publications ...... 16 4.3 Current Studies ...... 19 5.0 DONOR HEALTH AND SAFETY WORKING COMMITTEE ...... 22 5.1 Leadership ...... 22 5.2 Recent Publications ...... 22 5.3 Current Studies ...... 24 6.0 SOURCES AND MANIPULATION WORKING COMMITTEE ...... 27 6.1 Leadership ...... 27 6.2 Recent Publications ...... 27 6.3 Current Studies ...... 30 7.0 GRAFT‐VS‐HOST DISEASE WORKING COMMITTEE ...... 32 7.1 Leadership ...... 32 7.2 Recent Publications ...... 32 7.3 Current Studies ...... 34 8.0 HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE ...... 38 8.1 Leadership ...... 38 8.2 Recent Publications ...... 38 8.3 Current Studies ...... 41 9.0 IMMUNOBIOLOGY WORKING COMMITTEE ...... 44 9.1 Leadership ...... 44 9.2 Recent Publications ...... 44 9.3 Current Studies ...... 51 2017 Scientific Working Committee Research Portfolio TABLE OF CONTENTS

10.0 INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE ...... 59 10.1 Leadership ...... 59 10.2 Recent Publications ...... 59 10.3 Current Studies ...... 60 11.0 LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE ...... 62 11.1 Leadership ...... 62 11.2 Recent Publications ...... 62 11.3 Current Studies ...... 66 12.0 LYMPHOMA WORKING COMMITTEE ...... 70 12.1 Leadership ...... 70 12.2 Recent Publications ...... 70 12.3 Current Studies ...... 75 13.0 PEDIATRIC CANCER WORKING COMMITTEE ...... 77 13.1 Leadership ...... 77 13.2 Recent Publications ...... 77 13.3 Current Studies ...... 79 14.0 PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE ...... 80 14.1 Leadership ...... 80 14.2 Recent Publications ...... 80 14.3 Current Studies ...... 84 15.0 PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND NON‐ MALIGNANT MARROW DISORDERS WORKING COMMITTEE ...... 87 15.1 Leadership ...... 87 15.2 Recent Publications ...... 87 15.3 Current Studies ...... 90 16.0 REGIMEN‐RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE ...... 93 16.1 Leadership ...... 93 16.2 Recent Publications ...... 93 16.3 Current Studies ...... 96 APPENDIX A: COLLABORATIVE STUDIES ...... 99 APPENDIX B: STUDY DEVELOPMENT AND MANAGEMENT PROCESS ...... 104 APPENDIX C: GUIDELINES FOR STUDY PRINCIPAL INVESTIGATORS ...... 107 APPENDIX D: GLOSSARY ...... 112 2017 Scientific Working Committee Research Portfolio 1.0 OVERVIEW

1.0 OVERVIEW The CIBMTR® (Center for International Blood and Marrow Transplant Research®) collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy research worldwide to increase survival and enrich quality of life for patients. A research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin (MCW), the CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of transplant centers, and a unique and extensive clinical database. Fifteen international Scientific Working Committees oversee most of the CIBMTR’s clinical outcomes research. Each committee focuses on a specific disease, use of HCT, or complication of transplant therapy (Table 1). This report details the Scientific Working Committee Research Portfolio as of July 1, 2017. Table 1. Working Committee Focus Areas

Working Committee Scientific Focus Acute Leukemia HCT for acute leukemia and pre‐leukemia Autoimmune Diseases and HCT for autoimmune disorders and non‐transplant uses Cellular Therapies of hematopoietic stem cells Chronic Leukemia HCT for chronic , myelodysplastic disorders, and myeloproliferative disorders Donor Health and Safety Donor safety and outcomes Graft Sources and Manipulation Graft types, composition, and manipulation techniques Graft‐versus‐Host Disease Biology, prevention, and treatment of graft‐versus‐host disease and its complications Health Services and Social and economic barriers to HCT access, including International Studies quality of care and the influence of psychosocial factors on transplant outcomes, as well as international issues and differences in HCT Immunobiology Histocompatibility and other genetic and immunologic issues related to HCT Infection and Immune Prevention and treatment of post‐transplant infections Reconstitution and issues related to recovery of immune function Late Effects and Long‐term survival after HCT, including clinical and Quality of Life psychosocial effects of transplantation Lymphoma HCT for Hodgkin and non‐Hodgkin lymphoma Pediatric Cancer HCT for childhood leukemias and other issues related to use of HCT in children Plasma Cell Disorders and HCT for multiple myeloma and other plasma cell Adult Solid Tumors disorders as well as solid tumors in adults

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Working Committee Scientific Focus Primary Immune Deficiencies, HCT for congenital and acquired immune deficiencies, Inborn Errors of Metabolism, inborn errors of metabolism, aplastic anemia, congenital and Other Non‐Malignant disorders of hematopoiesis, and other non‐malignant Marrow Disorders hematopoietic disorders Regimen‐Related Toxicity and Preparative regimens, prevention, and treatment of early Supportive Care non‐graft‐versus‐host disease toxicities; supportive care in the early post‐transplant period

1.1 Membership

Total Working Committee membership exceeds 2,600 researchers. Membership is open to any researcher willing to take an active role in developing and conducting studies that use CIBMTR data and / or resources. While most of these individuals are HCT clinicians, statisticians and basic scientists also participate. PhD statistical faculty and Master’s‐level statisticians from the CIBMTR Coordinating Center provide their unique expertise in data analysis. Basic scientists investigating human leukocyte antigen (HLA), immunogenetics, pharmacogenetics, stem cell biology, and other areas related to HCT provide essential expertise in their respective research areas. The Working Committee structure encourages a collaborative but rigorous methodological approach to all CIBMTR activities.

1.2 Leadership

Each Working Committee is staffed by at least one MS‐level Statistician, a PhD Statistical Director, and an MD Scientific Director from the CIBMTR Coordinating Center. Each also typically has three to four Chairs who are appointed by the Advisory Committee.

1.2.1 Committee Chairs Working Committee Chairs are appointed by the Advisory Committee to non‐renewable five‐ year terms. Appointments are made each fall, with terms commencing on March 1 of the following year. Terms are staggered to facilitate succession and maintain continuity. Individuals may serve as Chair more than once but not consecutively for the same committee. Chairs participate in the nomination process for replacement positions and give special consideration to promising junior investigators, thus promoting ongoing leadership for the work of the CIBMTR. Working Committee Chairs provide subject matter expertise in autologous and allogeneic transplantation as well as understanding of CIBMTR organization and procedures. They must be members of CIBMTR centers that submit comprehensive report form (CRF)‐level data and are compliant with Continuous Process Improvement standards for data submission, unless an exception is granted by the Advisory Committee. Chairs are occasionally selected from outside these guidelines for their specific scientific expertise, for example, a scientist who directs a histocompatibility laboratory, apheresis center, or donor registry, who is committed to the CIBMTR and to the field of HCT.

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Chairs monitor and facilitate the progress of studies in their Working Committee’s portfolio. They communicate with Principal Investigators (PIs) to address barriers and / or delays and participate in weekly CIBMTR Coordinating Center study critiques when studies in their portfolios are being discussed. In addition to chairing annual Working Committee meetings, Chairs meet by teleconference every four to six weeks with their committee’s Scientific Director and biostatisticians to review the progress of study proposals and ongoing studies. Chairs lead the annual Working Committee meeting, and, using input from that meeting, they prioritize studies and establish the research agenda for the following year.

1.3 Productivity

There are currently >175 studies in progress, 60 of which are collaborations with other organizations (Appendix A). At the 2017 BMT Tandem Meetings, 33 new study proposals were approved. The prioritization and selection process (Figure 1) ensures the most important issues can be addressed in a timely manner. During the past year, Working Committee study investigators published 48 manuscripts in peer‐ reviewed journals, about half of the total number of CIBMTR publications. An additional five manuscripts are in press. In each committee’s section of this report, publications since July 1, 2012, are listed (Sections 2.2‐16.2). For a complete list of CIBMTR publications, visit the CIBMTR Publication List webpage. Working Committee study investigators presented 35 abstracts (26 oral and 9 poster) at national and international conferences this year. These presentations include 22 (16 oral and 6poster) at the 2016 American Society of Hematology Annual Meeting and 12 (9 oral and 3 poster) at the 2017 BMT Tandem Meetings.

1.4 How to Get Involved

Working Committees are collaborative in nature, and all interested individuals are encouraged to participate:  Join a Working Committee. Learn more about each committee on the CIBMTR Working Committee webpage. To join a Working Committee, email [email protected], contact the Working Committee leadership listed on the individual committee’s webpage, or attend a Working Committee Meeting at the BMT Tandem Meetings.  Attend a Working Committee Meeting at the BMT Tandem Meetings. All BMT Tandem Meeting attendees may attend to learn more about the committee, its recent publications and current studies, and have the opportunity to learn about and provide feedback on new study proposals.  Participate in a Writing Committee. When a draft protocol is approved by the Working Committee leadership and Coordinating Center, all Working Committee members on record are invited to participate in the study Writing Committee.  Propose a Study. Anyone willing to follow the study development and management process (Appendix B) is eligible to propose a study to the Working Committees (Figure

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1). Guidelines for CIBMTR study PIs, including hints and tips to make the study process as successful as possible, are provided in Appendix C. For more information regarding participation in a Working Committee, access the “Learn more about how to get involved in a Working Committee” section on the CIBMTR Working Committee webpage.

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Figure 1. Working Committee Study Proposal Review Process

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2.0 ACUTE LEUKEMIA WORKING COMMITTEE

2.1 Leadership

Chair: Marcos de Lima, MD, University Hospitals Case Medical Center Email: [email protected] Chair: Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center Email: [email protected] Scientific Director: Daniel Weisdorf, MD, CIBMTR Minneapolis Email: [email protected] Asst Sci Director: Wael Saber, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Mei‐Jie Zhang, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Hai‐Lin Wang, MPH, CIBMTR Milwaukee Email: [email protected]

2.2 Recent Publications

2017 Segal E, Martens M, Wang HL, Brazauskas R, Weisdorf D, Sandmaier BM, Khoury HJ, de Lima M, Saber W. Comparing outcomes of matched related donor and matched unrelated donor hematopoietic cell transplants in adults with B‐Cell acute lymphoblastic leukemia. Cancer. doi:10.1002/cncr.30737. Epub 2017 Apr 27. ncbi.nlm.nih.gov/pubmed/28452054 Ustun C, Giannotti F, Zhang M‐J, Wang H‐L, Brunstein C, Labopin M, Rocha V, de Lima M, Baron F, Sandmaier B, Eapen M, Gluckman E, Nagler A, Weisdorf DJ, Ruggeri A. Outcomes of UCB transplantation are comparable in FLT3+ AML: Results of CIBMTR, EUROCORD and EBMT collaborative analysis. Leukemia. 2017 Jun 1; 31(6):1408‐1414. doi:10.1038/leu.2017.42. Epub 2017 Jan 25. PMC5462854. ncbi.nlm.nih.gov/pubmed/28119528 Weisdorf DJ, Millard HR, Horowitz MM, Hyare PS, Champlin R, Ho V, Mielcarek M, Rezvani A, Stockerl‐Goldstein K, Khoury HJ, De Lima M, Saber W, Sandmaier B, Zhang M‐J, Eapen M. Allogeneic transplantation for advanced acute myeloid leukemia: The value of complete remission. Cancer. 2017 Jun 1; 123(11):2025‐2034. doi:10.1002/cncr.30536. Epub 2017 Jan 24. PMC5445001. ncbi.nlm.nih.gov/pubmed/28117884

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Michelis FV, Gupta V, Zhang M‐J, Wang H‐L, Aljurf M, Bacher U, Beitinjaneh A, Chen Y‐B, DeFilipp Z, Gale RP, Kebriaei P, Kharfan‐Dabaja M, Lazarus HM, Nishihori T, Olsson RF, Oran B, Rashidi A, Rizzieri DA, Tallman MS, de Lima M, Khoury HJ, Sandmaier BM, Weisdorf D, Saber W. Cytogenetic risk determines outcomes after allogeneic transplantation in older patients with acute myeloid leukemia in their second complete remission: A Center for International Blood and Marrow Transplant Research cohort analysis. Cancer. 2017 Jun 1; 123(11):2035‐2042. doi:10.1002/cncr.30567. Epub 2017 Jan 24. PMC5445018. ncbi.nlm.nih.gov/pubmed/28117898

2016 Rosko AE, Wang H‐L, de Lima M, Sandmaier B, Khoury HJ, Artz A, Brammer J, Bredeson C, Farag S, Kharfan‐Dabaja M, Lazarus HM, Marks DI, Martino Bufarull R, McGuirk J, Mohty M, Nishihori T, Nivison‐Smith I, Rashidi A, Ringden O, Seftel M, Weisdorf D, Bachanova V, Saber W. Reduced intensity conditioned allograft yields favorable survival for older adults with B‐cell acute lymphoblastic leukemia. American Journal of Hematology. 2017 Jan 1; 92(1):42‐49. doi:10.1002/ajh.24575. Epub 2016 Nov 12. PMC5167625. ncbi.nlm.nih.gov/pubmed/27712033 Deol A, Sengsayadeth S, Ahn KW, Wang H‐L, Aljurf M, Antin JH, Battiwalla M, Bornhauser M, Cahn J‐Y, Camitta B, Chen Y‐B, Cutler CS, Gale RP, Ganguly S, Hamadani M, Inamoto Y, Jagasia M, Kamble R, Koreth J, Lazarus HM, Liesveld J, Litzow MR, Marks DI, Nishihori T, Olsson RF, Reshef R, Rowe JM, Saad AA, Sabloff M, Schouten HC, Shea TC, Soiffer RJ, Uy GL, Waller EK, Wiernik PH, Wirk B, Woolfrey AE, Bunjes D, Devine S, de Lima M, Sandmaier BM, Weisdorf D, Khoury HJ, Saber W. Does FLT3 mutation impact survival after hematopoietic stem cell transplantation for acute myeloid leukemia? A Center for International Blood and Marrow Transplant Research (CIBMTR) analysis. Cancer. doi:10.1002/cncr.30140. Epub 2016 Jun 17. PMC5030151. ncbi.nlm.nih.gov/pubmed/27315441 Ganzel C, Mathews V, Alimoghaddam K, Ghavamzadeh A, Kuk D, Devlin S, Wang H, Zhang M‐J, Weisdorf D, Douer D, Rowe JM, Polge E, Esteve J, Nagler A, Mohty M, Tallman M. Autologous transplant remains the preferred therapy for relapsed APL in CR2. Marrow Transplantation. doi:10.1038/bmt.2016.96. Epub 2016 Apr 18. PMC5014591. ncbi.nlm.nih.gov/pubmed/27088379 Seftel MD, Neuberg D, Zhang M‐J, Wang H‐L, Ballen KK, Bergeron J, Couban S, Freytes CO, Hamadani M, Kharfan‐Dabaja MA, Lazarus HM, Nishihori T, Paulson K, Saber W, Sallan SE, Soiffer R, Tallman MS, Woolfrey AE, DeAngelo DJ, Weisdorf DJ. Pediatric‐inspired therapy compared to allografting for Philadelphia chromosome‐negative adult ALL in first complete remission. American Journal of Hematology. 2016 Mar 1; 91(3):322‐329. doi:10.1002/ ajh.24285. Epub 2016 Feb 22. PMC4764423. ncbi.nlm.nih.gov/pubmed/26701142 Munker R, Brazauskas R, Wang HL, de Lima M, Khoury HJ, Gale RP, Maziarz RT, Sandmaier BM, Weisdorf D, Saber W. Allogeneic hematopoietic cell transplantation for patients with mixed phenotype acute leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Jun 1; 22(6):1024‐1029. doi:10.1016/j.bbmt.2016.02.013. Epub 2016 Feb 21. PMC4867266. ncbi.nlm.nih.gov/pubmed/ 26903380

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2015 Pasquini MC, Zhang M‐J, Medeiros BC, Armand P, Hu Z‐H, Nishihori T, Aljurf MD, Akpek G, Cahn J‐ Y, Cairo MS, Cerny J, Copelan EA, Deol A, Freytes CO, Gale RP, Ganguly S, George B, Gupta V, Hale GA, Kamble RT, Klumpp TR, Lazarus HM, Luger SM, Liesveld JL, Litzow MR, Marks DI, Martino R, Norkin M, Olsson RF, Oran B, Pawarode A, Pulsipher MA, Ramanathan M, Reshef R, Saad AA, Saber W, Savani BN, Schouten HC, Ringdén O, Tallman MS, Uy GL, Wood WA Jr, Wirk B, Pérez WS, Batiwalla M, Weisdorf DJ. Hematopoietic cell transplantation outcomes in monosomal karyotype myeloid malignancies. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Feb 1; 22(2):248‐257. doi:10.1016/j.bbmt.2015.08.024. Epub 2015 Aug 29. PMC4716890. ncbi.nlm.nih.gov/pubmed/ 26327629 Goyal SD, Zhang MJ, Wang HL, Akpek G, Copelan EA, Freytes C, Gale RP, Hamadani M, Inamoto Y, Kamble RT, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Reshef R, Ritchie DS, Saber W, Savani BN, Seber A, Shea TC, Tallman MS, Wirk B, Bunjes DW, Devine SM, de Lima M, Weisdorf DJ, Uy GL. Allogeneic hematopoietic cell transplant for AML: No impact of pre‐transplant extramedullary disease on outcome. Bone Marrow Transplantation. 2015 Aug 1; 50(8):1057‐ 1062. doi:10.1038/bmt.2015.82. Epub 2015 Apr 27. PMC4527880. ncbi.nlm.nih.gov/pubmed/ 25915806

2014 Bejanyan N, Weisdorf DJ, Logan BR, Wang H‐L, Devine SM, de Lima M, Bunjes DW, Zhang M‐J. Survival of patients with acute myeloid leukemia relapsing after allogeneic hematopoietic cell transplantation: A Center for International Blood and Marrow Transplant Research study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Mar 1; 21(3):454‐459. doi:10.1016/j.bbmt.2014.11.007. Epub 2014 Nov 15. PMC4329076. ncbi.nlm.nih.gov/pubmed/25460355 Holter Chakrabarty JL, Rubinger M, Le‐Rademacher J, Wang H‐L, Grigg A, Selby GB, Szer J, Rowe JM, Weisdorf DJ, Tallman MS. Autologous is superior to allogeneic hematopoietic cell transplantation for acute promyelocytic leukemia in second complete remission. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Jul 1; 20(7):1021‐1025. doi:10.1016/j.bbmt.2014.03.025. Epub 2014 Mar 30. PMC4097890. ncbi.nlm.nih.gov/pubmed/24691221 McClune BL, Ahn KW, Wang H‐L, Antin JH, Artz AS, Cahn J‐Y, Deol A, Freytes CO, Hamadani M, Holmberg LA, Jagasia MH, Jakubowski AA, Kharfan‐Dabaja MA, Lazarus HM, Miller AM, Olsson R, Pedersen TL, Pidala J, Pulsipher MA, Rowe JM, Saber W, van Besien KW, Waller EK, Aljurf MD, Akpek G, Bacher U, Chao NJ, Chen Y‐B, Cooper BW, Dehn J, de Lima MJ, Hsu JW, Lewis ID, Marks DI, McGuirk J, Cairo MS, Schouten HC, Szer J, Ramanathan M, Savani BN, Seftel M, Socie G, Vij R, Warlick ED, Weisdorf DJ. Allotransplantation for patients age ≥40 years with non‐Hodgkin lymphoma: Encouraging progression‐free survival. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Jul 1; 20(7):960‐968. doi:10.1016/j.bbmt.2014.03.013. Epub 2014 Mar 15. PMC4057955. ncbi.nlm.nih.gov/pubmed/24641829

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2013 Warlick ED, Paulson K, Brazauskas R, Zhong X, Miller AM, Camitta BM, George B, Savani BN, Ustun C, Marks DI, Waller EK, Baron F, Freytes CO, Socie G, Akpek G, Schouten HC, Lazarus HM, Horwitz EM, Koreth J, Cahn J‐Y, Bornhauser M, Seftel M, Cairo MS, Laughlin MJ, Sabloff M, Ringdén O, Gale RP, Kamble RT, Vij R, Gergis U, Mathews V, Saber W, Chen Y‐B, Liesveld JL, Cutler CS, Ghobadi A, Uy GL, Eapen M, Weisdorf DJ, Litzow MR. Effect of postremission therapy before reduced‐intensity conditioning allogeneic transplantation for acute myeloid leukemia in first complete remission. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Feb 1; 20(2):202‐208. doi:10.1016/j.bbmt.2013.10.023. Epub 2013 Nov 1. PMC3924751. ncbi.nlm.nih.gov/pubmed/ 24184335 Bachanova V, Marks DI, Zhang M‐J, Wang H, de Lima M, Aljurf MD, Arellano M, Artz AS, Bacher U, Cahn J‐Y, Chen Y‐B, Copelan EA, Drobyski WR, Gale RP, Greer JP, Gupta V, Hale GA, Kebriaei P, Lazarus HM, Lewis ID, Lewis VA, Liesveld JL, Litzow MR, Loren AW, Miller AM, Norkin M, Oran B, Pidala J, Rowe JM, Savani BN, Saber W, Vij R, Waller EK, Wiernik PH, Weisdorf DJ. Ph+ ALL patients in first complete remission have similar survival after reduced intensity and myeloablative allogeneic transplantation: Impact of tyrosine kinase inhibitor and minimal residual disease. Leukemia. 2014 Mar 1; 28(3):658‐665. doi:10.1038/leu.2013.253. Epub 2013 Sep 27. PMC3951192. ncbi.nlm.nih.gov/pubmed/23989431 Lee SJ, Storer B, Wang H, Lazarus HM, Waller EK, Isola LM, Klumpp TR, Umejiego JB, Savani BN, Loren AW, Cairo MS, Camitta BM, Cutler CS, George B, Khoury HJ, Marks DI, Rizzieri DA, Copelan EA, Gupta V, Liesveld JL, Litzow MR, Miller AM, Schouten HC, Gale RP, Cahn J‐Y, Weisdorf DJ. Providing personalized prognostic information for adult leukemia survivors. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Nov 1; 19(11):1600‐1607. doi:10.1016/j.bbmt.2013.08.013. Epub 2013 Sep 6. PMC3832127. ncbi.nlm.nih.gov/pubmed/24018394 Koreth J, Pidala J, Perez WS, Deeg HJ, Garcia‐Manero G, Malcovati L, Cazzola M, Park S, Itzykson R, Ades L, Fenaux P, Jadersten M, Hellstrom‐Lindberg E, Gale RP, Beach CL, Lee SJ, Horowitz MM, Greenberg PL, Tallman MS, DiPersio JF, Bunjes D, Weisdorf DJ, Cutler C. Role of reduced‐ intensity conditioning allogeneic hematopoietic stem‐cell transplantation in older patients with de novo myelodysplastic syndromes: An international collaborative decision analysis. Journal of Clinical Oncology. 2013 Jul 20; 31(21):2662‐2670. doi:10.1200/ JCO.2012.46.8652. Epub 2013 Jun 24. PMC3825320. ncbi.nlm.nih.gov/pubmed/23797000 Foran JM, Pavletic SZ, Logan BR, Agovi‐Johnson MA, Pérez WS, Bolwell BJ, Bornhäuser M, Bredeson CN, Cairo MS, Camitta BM, Copelan EA, Dehn J, Gale RP, George B, Gupta V, Hale GA, Lazarus HM, Litzow MR, Maharaj D, Marks DI, Martino R, Maziarz RT, Rowe JM, Rowlings PA, Savani BN, Savoie ML, Szer J, Waller EK, Wiernik PH, Weisdorf DJ. Unrelated donor allogeneic transplantation after failure of autologous transplantation for acute myelogenous leukemia: A study from the Center for International Blood and Marrow Transplantation Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Jul 1; 19(7):1102‐1108. doi:10.1016/j.bbmt. 2013.04.022. Epub 2013 Apr 28. PMC3691352. ncbi.nlm.nih.gov/pubmed/23632091

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2012 Keating A, DaSilva G, Pérez WS, Gupta V, Cutler CS, Ballen KK, Cairo MS, Camitta BM, Champlin RE, Gajewski JL, Lazarus HM, Lill M, Marks DI, Nabhan C, Schiller GJ, Socie G, Szer J, Tallman MS, Weisdorf DJ. Autologous blood cell transplantation versus HLA‐identical sibling transplantation for acute myeloid leukemia in first complete remission: A registry study from the Center for International Blood and Marrow Transplantation Research. Haematologica. 2013 Feb 1; 98(2):185‐192. doi:10.3324/haematol.2012.062059. Epub 2012 Sep 14. PMC3561424. ncbi.nlm.nih.gov/pubmed/22983587

2.3 Current Studies

LK13‐02 Title: Prognostic significance of cytogenetic abnormalities in patients with Philadelphia‐negative acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation in complete remission PIs: Aleksandr Lazaryan (University of Minnesota Blood and Marrow Transplant Program) Veronika Bachanova (University of Minnesota Blood and Marrow Transplant Program) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

LK14‐01 Title: Effect of post‐remission consolidation chemotherapy prior to allogeneic transplantation for acute lymphocytic leukemia in first complete remission: A Center for International Blood and Marrow Transplant Research Study PIs: Nelli Bejanyan (University of Minnesota Blood and Marrow Transplant Program) Aleksandr Lazaryan (University of Minnesota Blood and Marrow Transplant Program) Daniel Weisdorf (University of Minnesota Blood and Marrow Transplant Program) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

LK15‐01 Title: Allogeneic transplants vs other consolidation in elderly AML PIs: Andrw Artz (University of Chicago Medicine) Celestatin Ustun (University of Minnesota Blood and Marrow Transplant Program) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with Alliance, Eastern Cooperative Oncology Group (ECOG) and Southwest Oncology Group (SWOG)

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LK15‐02 Title: Impact of GVHD on outcome after allogeneic hematopoietic cell transplantation for acute lymphocytic leukemia: A retrospective registry study PIs: Moshe Yeshurun (Davidoff Cancer Center, Rabin Medical Center, Beilinson Hospital) Jacob Rowe (Rambam Medical Center) Martin Tallman (Memorial Sloan Kettering Cancer Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

LK15‐03 Title: Comparison of outcomes of older adolescents and young adults with Philadelphia‐ chromosome/BCR‐ABL1‐negative acute lymphoblastic leukemia receiving post‐ remission consolidation chemotherapy with pediatric‐inspired chemotherapy on CALGB 10403 or myeloablative allogeneic hematopoietic cell transplantation PIs: Matthew Wieduwilt (University of California, San Diego Medical Center) Wendy Stock (University of Chicago Medicine) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with Cancer and Leukemia Group B 10403 (member Alliance for Clinical Trials in Oncology) (CALGB)

LK15‐04 Title: Outcome of hematopoietic stem cell transplantation using ‐ based versus chemotherapy‐based full intensity conditioning regimens for adults with acute lymphoblastic leukemia PIs: Ibrahim Aldoss (City of Hope) Vinod Pullarkat (City of Hope) Stephen Forman (City of Hope) Joseph Alvarnase (City of Hope) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

LK16‐01 Title: Reduced intensity conditioning regimens for acute myeloid leukemia: A comparison of busulfan and melphalan based regimens from the CIBMTR database PIs: Zartash Gul (University of Cincinnati Medical Center) Hassan Alkhateeb (Mayo Clinic Rochester) Rajneesh Nath (Banner MD Anderson Cancer Center) Status: Protocol Development (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

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LK16‐02 Title: DRI‐guided choice of conditioning intensity for allogeneic hematopoietic cell transplantation in adults with acute myeloid leukemia and myelodysplastic syndromes PIs: Nelli Bejanyan (University of Minnesota Blood and Marrow Transplant Program) Erica Warlick (University of Minnesota Blood and Marrow Transplant Program) Claudio Brunstein (University of Minnesota Blood and Marrow Transplant Program) Daniel Weisdorf (University of Minnesota Blood and Marrow Transplant Program) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

LK16‐03 Title: Allogeneic transplantation to treat therapy related acute myeloid leukemia and myelodysplastic syndromes PIs: Natalie Callander (University of Wisconsin Hospital and Clinics) Leland Metheny (Seidman Cancer Center‐University Hospitals Cleveland Medical Center) Marcos de Lima (Seidman Cancer Center‐University Hospitals Cleveland Medical Center) Aric Hall (University of Wisconsin Hospital and Clinics) Status: Data File Preparation (as of July 1, 2017) Analysis (expected by June 30, 2018)

LK16‐04 Title: Comparing outcomes between HLA‐haploidentical and matched sibling allogeneic transplants in patients with acute myeloid leukemia PIs: Rizwan Romee (Barnes Jewish Hospital) Armin Rashidi (University of Minnesota) Mehdi Hamadani (CIBMTR ‐ Milwaukee) Wael Saber (CIBMTR ‐ Milwaukee) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

LK17‐01 Title: Outcomes of acute myeloid leukemia patients who undergo allogeneic transplant stratified by depth of clinical response PIs: Mary‐Elizabeth Percival (University of Washington) Brenda Sandmaier (Fred Hutchinson Cancer Research Center) Eli Estey (M.D. Anderson Cancer Center) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

Page | 12 2017 Scientific Working Committee Research Portfolio 2.0 ACUTE LEUKEMIA WORKING COMMITTEE

LK17‐02 Title: Allogeneic Hematopoietic Transplant Outcomes in Adult Patients with MLL‐ rearranged Acute Myeloid Leukemia PIs: Kamal Menghrajani (Memorial Sloan Kettering Cancer Center) Martin Tallman (Memorial Sloan Kettering Cancer Center) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

LK17‐03 Title: Impact of post‐transplant maintenance therapy with BCR‐ABL tyrosine kinase inhibitors on outcomes of Philadelphia chromosome‐positive acute lymphoblastic leukemia PIs: Zack DeFilipp (Massachusetts General Hospital) Yi‐Bin Chen (Massachusetts General Hospital) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

Page | 13 3.0 AUTOIMMUNE DISEASES AND CELLULAR 2017 Scientific Working Committee Research Portfolio THERAPIES WORKING COMMITTEE

3.0 AUTOIMMUNE DISEASES AND CELLULAR THERAPIES WORKING COMMITTEE

3.1 Leadership

Chair: David McKenna, MD, University of Minnesota Medical Center, Fairview Email: [email protected] Chair: Stefanie Sarantopoulos, MD, PhD, Duke University Medical Center Email: [email protected] Chari: Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute Email: [email protected] Scientific Director: Marcelo Pasquini, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Ruta Brazauskas, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Khalid (Kyle) Bo‐Subait, MPH, CIBMTR Milwaukee Email: [email protected] CAC Representative: Hilary Hall, Dana Farber Cancer Institute

3.2 Recent Publications

2017 Muraro PA, Pasquini M, Atkins HL, Bowen JD, Farge D, Fassas A, Freedman MS, Georges GE, Gualandi F, Hamerschlak N, Havrdova E, Kimiskidis VK, Kozak T, Mancardi GL, Massacesi L, Moraes DA, Nash RA, Pavletic S, Ouyang J, Rovira M, Saiz A, Simoes B, Trnný M, Zhu L, Badoglio M, Zhong X, Sormani MP, Saccardi R. Long‐term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurology. doi:10.1001/jamaneurol. 2016.5867. Epub 2017 Feb 20. ncbi.nlm.nih.gov/pubmed/28241268

3.3 Current Studies

CT10‐01 Title: Donor leukocyte infusion versus second allogeneic HCT for disease relapse after first allogeneic HCT PIs: Noelle Frey (Abramson Cancer Center University of Pennsylvania Medical Center) Alison Loren (Abramson Cancer Center University of Pennsylvania Medical Center) David Porter (Abramson Cancer Center University of Pennsylvania Medical Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

Page | 14 3.0 AUTOIMMUNE DISEASES AND CELLULAR 2017 Scientific Working Committee Research Portfolio THERAPIES WORKING COMMITTEE

CT13‐01 Title: Utility of donor leukocyte infusion for the treatment of drug‐resistant viral or fungal infections in allogeneic HCT recipients: A CIBMTR analysis PIs: Gorgun Akpek (Rush University Medical Center) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

AC14‐01 Title: Long term outcomes after AutoHCT for rapidely progressive systemic scleroderma PIs: Dominique Farge (St. Louis Hospital) Maria Carolina Oliveira (University of São Paulo) George Georges (Fred Hutchinson Cancer Research Center) Status: Data Collection / Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with European Society for Blood and Marrow Transplantation (EBMT)

AC16‐01 Title: Pattern of use and outcomes with donor lymphocyte infusion after HLA‐ haploidentical allogeneic hematopoietic stem cell transplant PIs: Eva Gupta (Mayo Clinic Florida) James Foran (Mayo Clinic Florida) Vivek Roy (Mayo Clinic Florida) Status: Data Collection / Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

AC17‐01 Title: CD‐19 chimeric antigen receptor T Cells with or without hematopoietic cell transplantation for treatment of refractory Acute Lymphocytic Leukemia PIs: Miguel‐Angel Perales (Memorial Sloan Kettering Cancer Center) Jae Park (Memorial Sloan Kettering Cancer Center) Sarah Nikiforow (Dana Farber Cancer Institute) Status: Draft Protocol Received (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

Page | 15 2017 Scientific Working Committee Research Portfolio 4.0 CHRONIC LEUKEMIA WORKING COMMITTEE

4.0 CHRONIC LEUKEMIA WORKING COMMITTEE

4.1 Leadership

Chair: Edwin Alyea, MD, Dana Farber Cancer Institute Email: [email protected] Chair: Uday Popat, MD, MD Anderson Cancer Center Email: [email protected] Chair: Ronald Sobecks, MD, Cleveland Clinic Foundation Email: [email protected] Scientific Director: Wael Saber, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Kwang Woo Ahn, PhD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Ying Liu, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Zhenhuan Hu, MPH, CIBMTR Milwaukee Email: [email protected]

4.2 Recent Publications

2017 Saber W, Horowitz MM. Transplantation for myelodysplastic syndromes: Who, when, and which conditioning regimens. Hematology / the Education Program of the American Society of Hematology. 2016 Dec 2; 2016(1):478‐484. doi:10.1182/asheducation‐2016.1.478. ncbi.nlm.nih.gov/pubmed/27913519 Gerds AT, Woo Ahn K, Hu ZH, Abdel‐Azim H, Akpek G, Aljurf M, Ballen KK, Beitinjaneh A, Bacher U, Cahn J‐Y, Chharbra S, Cutler C, Daly A, DeFilipp Z, Gale RP, Gergis U, Grunwald MR, Hale GA, Hamilton BK, Jagasia M, Kamble RT, Kindwell‐Keller T, Nishihori T, Olsson RF, Ramanathan M, Saad AA, Solh M, Ustun C, Valcárcel D, Warlick E, Wirk BM, Kalaycio M, Alyea E, Popat U, Sobecks R, Saber W. Outcomes after umbilical cord blood transplantation for myelodysplastic syndromes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jun 1; 23(6):971‐979. doi:10.1016/j.bbmt. 2017.03.014. Epub 2017 Mar 10. PMC5474679. ncbi.nlm.nih.gov/pubmed/28288952 Liu HD, Ahn KW, Hu Z‐H, Hamadani M, Nishihori T, Wirk B, Beitinjaneh A, Rizzieri D, Grunwald MR, Sabloff M, Olsson RF, Bajel A, Bredeson C, Daly A, Inamoto Y, Majhail N, Saad A, Gupta V, Gerds A, Malone A, Tallman M, Reshef R, Aljurf M, Marks DI, Copelan E, Gergis U, Savoie ML, Ustun C, Litzow MR, Cahn JY, Kindwell‐Keller T, Akpek G, Savani BN, Rowe JM, Wiernik PH, Hsu JW, Cortes J, Kalaycio M, Maziarz R, Sobecks R, Popat U, Alyea E, Saber W. Allogeneic hematopoietic cell transplant for adult chronic myelomonocytic leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2017.01.078. Epub 2017 Jan 20. ncbi.nlm.nih.gov/ pubmed/28115276

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2016 Kharfan‐Dabaja MA, Kumar A, Hamadani M, Stilgenbauer S, Ghia P, Anasetti C, Dreger P, Montserrat E, Perales M‐A, Alyea EP, Awan FT, Ayala E, Barrientos JC, Brown JR, Castro JE, Furman RR, Gribben J, Hill BT, Mohty M, Moreno C, O'Brien S, Pavletic SZ, Pinilla‐Ibarz J, Reddy NM, Sorror M, Bredeson C, Carpenter P, Savani BN. Clinical practice recommendations for use of allogeneic hematopoietic cell transplantation in chronic lymphocytic leukemia on behalf of the Guidelines Committee of the American Society for Blood and Marrow Transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Dec 1; 22(12):2117‐2125. doi:10.1016/ j.bbmt.2016.09.013. Epub 2016 Sep 19. PMC5116249. ncbi.nlm.nih.gov/pubmed/27660167 Ustun C, Gotlib J, Popat U, Artz A, Litzow M, Reiter A, Nakamura R, Kluin‐Nelemans HC, Verstovsek S, Gajewski J, Perales M‐A, George T, Shore T, Sperr W, Saber W, Kota V, Yavuz AS, Pullarkat V, Rogosheske J, Hogan W, Van Besien K, Hagglund H, Damaj G, Arock M, Horny H‐P, Metcalfe DD, Deeg HJ, Devine S, Weisdorf D, Akin C, Valent P. Consensus opinion on allogeneic hematopoietic cell transplantation in advanced systemic mastocytosis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug 1; 22(8):1348‐1356. doi:10.1016/j.bbmt.2016.04.018. Epub 2016 Apr 27. ncbi.nlm.nih.gov/pubmed/27131865 Shaffer BC, Ahn KW, Hu Z‐H, Nishihori T, Malone AK, Valcárcel D, Grunwald MR, Bacher U, Hamilton B, Kharfan‐Dabaja MA, Saad A, Cutler C, Warlick E, Reshef R, Wirk BM, Sabloff M, Fasan O, Gerds A, Marks D, Olsson R, Wood WA, Costa LJ, Miller AM, Cortes J, Daly A, Kindwall‐Keller TL, Kamble RT, Rizzieri DA, Cahn J‐Y, Gale RP, William B, Litzow M, Wiernik PH, Liesveld J, Savani BN, Vij R, Ustun C, Copelan E, Popat U, Kalaycio M, Maziarz R, Alyea E, Sobecks R, Pavletic S, Tallman M, Saber W. Scoring system prognostic of outcome in patients undergoing allogeneic hematopoietic cell transplantation for . Journal of Clinical Oncology. doi:10.1200/JCO.2015.65.0515. Epub 2016 Apr 4. ncbi.nlm.nih.gov/pubmed/ 27044940 Chaudhury S, Sparapani R, Hu ZH, Nishihori T, Abdel‐Azim H, Malone A, Olsson R, Hamadani M, Daly A, Bacher U, Wirk BM, Kamble RT, Gale RP, Wood WA, Hale G, Wiernik PH, Hashmi SK, Marks DI, Ustun C, Munker R, Savani BN, Alyea E, Popat U, Sobecks R, Kalaycio M, Maziarz R, Burlington, Wisconsin 53105Hijiya N, Saber W. Outcomes of allogeneic hematopoietic cell transplantation in children (<18y) and young adults (18‐29y) with chronic myeloid leukemia: A CIBMTR cohort analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Jun 1; 22(6):1056‐1064. doi:10.1016/j.bbmt.2016.02.015. Epub 2016 Mar 8. PMC4877686. ncbi.nlm.nih.gov/ pubmed/26964698

Page | 17 2017 Scientific Working Committee Research Portfolio 4.0 CHRONIC LEUKEMIA WORKING COMMITTEE

2014 Copelan EA, Avalos BR, Ahn KW, Zhu X, Gale RP, Grunwald MR, Hamadani M, Hamilton BK, Hale GA, Marks DI, Waller EK, Savani BN, Costa LJ, Ramanathan M, Cahn J‐Y, Khoury HJ, Weisdorf DJ, Inamoto Y, Kamble RT, Schouten HC, Wirk B, Litzow MR, Aljurf MD, van Besien KW, Ustun C, Bolwell BJ, Bredeson CN, Fasan O, Ghosh N, Horowitz MM, Arora M, Szer J, Loren AW, Alyea EP, Cortes J, Maziarz RT, Kalaycio ME, Saber W. Comparison of outcomes of allogeneic transplantation for chronic myeloid leukemia with cyclophosphamide in combination with intravenous busulfan, oral busulfan, or total body irradiation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Mar 1; 21(3):552‐558. doi:10.1016/j.bbmt.2014.12.010. Epub 2014 Dec 17. PMC4329042. ncbi.nlm.nih.gov/pubmed/25528388 Sobecks RM, Leis JF, Gale RP, Ahn KW, Zhu X, Sabloff M, de Lima M, Brown JR, Inamoto Y, Hale GA, Aljurf MD, Kamble RT, Hsu JW, Pavletic SZ, Wirk B, Seftel MD, Lewis ID, Alyea EP, Cortes J, Kalaycio ME, Maziarz RT, Saber W. Outcomes of human leukocyte antigen‐matched sibling donor hematopoietic cell transplantation in chronic lymphocytic leukemia: Myeloablative versus reduced‐intensity conditioning regimens. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Sep 1; 20(9):1390‐1398. doi:10.1016/j.bbmt.2014.05.020. Epub 2014 May 28. PMC4174349. ncbi.nlm.nih.gov/pubmed/24880021 Armand P, Kim HT, Logan BR, Wang Z, Alyea EP, Kalaycio ME, Maziarz RT, Antin JH, Soiffer RJ, Weisdorf DJ, Rizzo JD, Horowitz MM, Saber W. Validation and refinement of the disease risk index for allogeneic stem cell transplantation. Blood. 2014 Jun 5; 123(23):3664‐3671. doi:10.1182/blood‐2014‐01‐552984. Epub 2014 Apr 17. PMC4047501. ncbi.nlm.nih.gov/ pubmed/24744269 Atallah E, Bylow K, Troy J, Saber W. Treatment of older patients with high‐risk myelodysplastic syndromes (MDS): The emerging role of allogeneic hematopoietic stem cell transplantation (Allo HSCT). Current Hematologic Malignancy Reports. 2014 Mar 1; 9(1):57‐65. doi:10.1007/s11899‐013‐0195‐9. Epub 2014 Jan 8. PMC4031643. ncbi.nlm.nih.gov/ pubmed/24398726

2013 Sabloff M, Sobecks RM, Ahn KW, Zhu X, de Lima M, Brown JR, Inamoto Y, Holland HK, Aljurf MD, Laughlin MJ, Kamble RT, Hsu JW, Wirk BM, Seftel M, Lewis ID, Arora M, Alyea EP, Kalaycio ME, Cortes J, Maziarz RT, Gale RP, Saber W. Does total body irradiation conditioning improve outcomes of myeloablative human leukocyte antigen‐identical sibling transplantations for chronic lymphocytic leukemia? Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Mar 1; 20(3):421‐424. doi:10.1016/j.bbmt.2013.11.032. Epub 2013 Dec 7. PMC4026353. ncbi.nlm.nih.gov/pubmed/ 24321745

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Gupta V, Malone AK, Hari PN, Ahn KW, Hu Z‐H, Gale RP, Ballen KK, Hamadani M, Olavarria E, Gerds AT, Waller EK, Costa LJ, Antin JH, Kamble RT, van Besien KM, Savani BN, Schouten HC, Szer J, Cahn J‐Y, de Lima MJ, Wirk B, Aljurf MD, Popat U, Bejanyan N, Litzow MR, Norkin M, Lewis ID, Hale GA, Woolfrey AE, Miller AM, Ustun C, Jagasia MH, Lill M, Maziarz RT, Cortes J, Kalaycio ME, Saber W. Reduced‐intensity hematopoietic cell transplantation for patients with primary myelofibrosis: A cohort analysis from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Jan 1; 20(1):89‐97. doi:10.1016/ j.bbmt.2013.10.018. Epub 2013 Oct 23. PMC3886623. ncbi.nlm.nih.gov/pubmed/24161923 Copelan EA, Hamilton BK, Avalos B, Ahn KW, Bolwell BJ, Zhu X, Aljurf M, van Besien K, Bredeson C, Cahn J‐Y, Costa LJ, de Lima M, Gale RP, Hale GA, Halter J, Hamadani M, Inamoto Y, Kamble RT, Litzow MR, Loren AW, Marks DI, Olavarria E, Roy V, Sabloff M, Savani BN, Seftel M, Schouten HC, Ustun C, Waller EK, Weisdorf DJ, Wirk B, Horowitz MM, Arora M, Szer J, Cortes J, Kalaycio ME, Maziarz RT, Saber W. Better leukemia‐free and overall survival in AML in first remission following cyclophosphamide in combination with busulfan compared with TBI. Blood. 2013 Dec 5; 122(24):3863‐3870. doi:10.1182/blood‐2013‐07‐514448. Epub 2013 Sep 24. PMC3854108. ncbi.nlm.nih.gov/pubmed/24065243 Saber W, Cutler CS, Nakamura R, Zhang MJ, Atallah E, Rizzo JD, Maziarz RT, Cortes J, Kalaycio ME, Horowitz MM. Impact of donor source on hematopoietic cell transplantation outcomes for patients with myelodysplastic syndromes (MDS). Blood. 2013 Sep 12; 122(11):1974‐1982. doi:10.1182/blood‐2013‐04‐496778. Epub 2013 Jul 11. PMC3772501. ncbi.nlm.nih.gov/ pubmed/23847196

4.3 Current Studies

CK12‐01 Title: A decision analysis of the optimal timing of allogeneic hematopoietic stem cell transplantation in chronic myeloid leukemia in the era of Tyrosine Kinase Inhibitors PIs: Hans Lee (M.D. Anderson Cancer Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with MD Anderson Cancer Center

CK12‐02b Title: A retrospective assessment of outcomes of patients who have undergone allogeneic stem cell transplant for Chronic Lymphocytic Leukemia based on histocompatibility leukocyte antigen type PIs: Brian Hill (Cleveland Clinic Foundation) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018) * Collaborative study with National Institutes of Health (NIH)

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CK14‐02 Title: Validation of DFCI prognostic score for previously treated chronic lymphocytic leukemia patients who underwent reduced intensity conditioning allogeneic HSCT PIs: Jennifer Brown (Dana Farber Cancer Institute) Haesook Kim (Dana Farber Cancer Institute) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with Dana‐Farber Cancer Institute (DFCI)

CK15‐01 Title: Comparison of Transplant vs non Transplant Therapies for Myelofibrosis PIs: Karen Ballen (University of Virginia Health System) Ruben Mesa (Mayo Clinic Arizona and Phoenix Children's Hospital) Krisstina Gowin (Mayo Clinic Arizona and Phoenix Children's Hospital) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with Mayo Arizona, Duke, Mass General, MD Anderson, Vanderbilt

CK15‐02 Title: Comparison of outcomes after myeloablative versus reduced intensity conditioning for allogeneic hematopoietic stem cell transplant for chronic myeloid leukemia PIs: Saurabh Chhabra (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

CK15‐03 Title: Outcome of allogeneic hematopoietic cell transplantation in patients with acute myeloid leukemia with antecedent history of Philadelphia‐negative myeloproliferative Neoplasm PIs: Vikas Gupta (Princess Margaret Hospital) Status: Data File Preparation (as of July 1, 2017) Analysis (expected by June 30, 2018)

CK16‐01 Title: Identification of germline predisposition mutations in young MDS patients PIs: Lucy Godley (University of Chicago Medicine) Status: Protocol Development (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with University of Chicago

Page | 20 2017 Scientific Working Committee Research Portfolio 4.0 CHRONIC LEUKEMIA WORKING COMMITTEE

CK16‐02a Title: Contemporary role of tyrosine kinase inhibitors post allogeneic hematopoietic stem cell transplantation for advanced phase chronic myeloid leukemia PIs: Zach DeFilipp (Massachusetts General Hospital) Richard Ancheta (Scripps Blood & Marrow Transplant Program) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

CK16‐02b Title: Donor lymphocyte infusion vs. tyrosine kinase inhibitors in chronic myeloid leukemia post allogeneic hematopoietic cell transplantation PIs: Sara Schmidt (HCA Health Services of Oklahoma, Inc., Univ of OK) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

CK17‐01 Title: Development of a Prognostic Scoring System Predictive of outcomes in patients with Myelofibrosis after Allogeneic Hematopoietic Cell Transplantation PIs: Roni Tamari (Memorial Sloan Kettering Cancer Center) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018) * Collaborative study with EBMT

CK17‐02 Title: Reduced‐intensity conditioning transplantation in older MDS: The effect of specific conditioning regimens on transplant outcomes PIs: Betul Oran (M.D. Anderson Cancer Center) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

SC11‐06/10‐CMSMDS‐1 Title: Assessment of allogeneic hematopoietic stem cell transplantation in Medicare beneficiaries with myelodysplastic syndrome and related disorders PIs: Ehab Atallah (Froedtert Memorial Lutheran Hospital) J. Douglas Rizzo (CIBMTR ‐ Milwaukee) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

Page | 21 5.0 DONOR HEALTH AND 2017 Scientific Working Committee Research Portfolio SAFETY WORKING COMMITTEE

5.0 DONOR HEALTH AND SAFETY WORKING COMMITTEE

5.1 Leadership

Chair: Galen Switzer, PhD, University of Pittsburgh Medical Center Email: [email protected] Chair: Michael Pulsipher, MD, Primary Children's Hospital Email: [email protected] Chair: Nirali Shah, MD, MHSc, National Cancer Institute ‐ NIH Email: [email protected] Scientific Director: Bronwen Shaw, MBChB, MRCP, PhD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Brent Logan, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Deidre Kiefer, MPH, CIBMTR Minneapolis Email: [email protected] MS Statistician: Pintip Chitphakdithai, PhD, CIBMTR Minneapolis Email: [email protected] Ex Officio Sr Advisor: Dennis Confer, MD, CIBMTR Minneapolis Email: [email protected] CAC Representative: Jeffrey Haertling, Tierra Verde, FL CAC Representative: Hilary Hall, Dana Farber Cancer Institute

5.2 Recent Publications

2017 Switzer GE, Bruce J, Pastorek G, Kiefer DM, Kobusingye H, Drexler R, Besser RAM, Confer DL, Horowitz MM, King RJ, Shaw BE, van Walraven SM, Weiner L, Packman W, Varni JW, Pulsipher MA. Parent versus Child Donor Perceptions of the Bone Marrow Donation Experience. Bone Marrow Transplantation. doi:10.1038/bmt.2017.124. Epub 2017 Jun 26. ncbi.nlm.nih.gov/ pubmed/28650449

2016 Switzer GE, Bruce J, Kiefer DM, Kobusingye H, Drexler R, Besser RM, Confer DL, Horowitz MM, King RJ, Shaw BE, Riches M, Hayes‐Lattin B, Linenberger M, Bolwell B, Rowley SD, Litzow MR, Pulsipher MA. Health‐related quality of life among older related hematopoietic stem cell donors (>60 years) is equivalent to that of younger related donors (18 to 60 years): A related donor safety study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jan 1; 23(1):165‐171. doi:10.1016/j.bbmt.2016.10.008. Epub 2016 Oct 14. PMC5182103. ncbi.nlm.nih.gov/ pubmed/27751935

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Switzer GE, Bruce J, Kiefer DM, Kobusingye H, Drexler R, Besser RM, Confer DL, Horowitz MM, King RJ, Shaw BE, van Walraven SM, Wiener L, Packman W, Varni JW, Pulsipher MA. Health‐ related quality of life among pediatric hematopoietic stem cell donors. The Journal of Pediatrics. 2016 Nov 1; 178:164‐170.e1. doi:10.1016/j.jpeds.2016.07.009. Epub 2016 Aug 10. PMC5085860. ncbi.nlm.nih.gov/pubmed/27522440

2015 Anthias C, O'Donnell PV, Kiefer DM, Yared J, Norkin M, Anderlini P, Savani BN, Diaz MA, Bitan M, Halter JP, Logan BR, Switzer GE, Pulsipher MA, Confer DL, Shaw BE. European Group for Blood and Marrow Transplantation centers with FACT‐JACIE accreditation have significantly better compliance with related donor care standards. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Mar 1; 22(3):514‐519. doi:10.1016/j.bbmt.2015.11.009. Epub 2015 Nov 18. PMC4760354. ncbi.nlm.nih.gov/pubmed/26597079 Anthias C, Shaw BE, Kiefer DM, Liesveld JL, Yared J, Kamble RT, D'Souza A, Hematti P, Seftel MD, Norkin M, DeFilipp Z, Kasow KA, Abidi MH, Savani BN, Shah NN, Anderlini P, Diaz MA, Malone AK, Halter JP, Lazarus HM, Logan BR, Switzer GE, Pulsipher MA, Confer DL, O'Donnell PV. Significant improvements in the practice patterns of adult related donor care in US transplantation centers. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Mar 1; 22(3):520‐527. doi:10.1016/j.bbmt.2015. 11.008. Epub 2015 Nov 18. PMC4760616. ncbi.nlm.nih.gov/pubmed/26597080 Shaw BE, Logan BR, Kiefer DM, Chitphakdithai P, Pedersen TL, Abdel‐Azim H, Abidi MH, Akpek G, Diaz MA, Artz AS, Dandoy C, Gajewski JL, Hematti P, Kamble RT, Kasow KA, Lazarus HM, Liesveld JL, Majhail NS, O'Donnell PV, Olsson RF, Savani BN, Schears RM, Stroncek DF, Switzer GE, Williams EP, Wingard JR, Wirk BM, Confer DL, Pulsipher MA. Analysis of the effect of race, socioeconomic status, and center size on unrelated National Marrow Donor Program donor outcomes: donor toxicities are more common at low‐volume bone marrow collection centers. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1830‐1838. doi:10.1016/ j.bbmt.2015.06.013. Epub 2015 Jun 23. PMC4568129. ncbi.nlm.nih.gov/pubmed/26116089

2014 Hsu JW, Wingard JR, Logan BR, Chitphakdithai P, Akpek G, Anderlini P, Artz AS, Bredeson C, Goldstein S, Hale G, Hematti P, Joshi S, Kamble RT, Lazarus HM, O'Donnell PV, Pulsipher MA, Savani BN, Schears RM, Shaw BE, Confer DL. Race and ethnicity influences collection of granulocyte colony‐stimulating factor‐mobilized peripheral blood progenitor cells from unrelated donors, a Center for International Blood and Marrow Transplant Research analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jan 1; 21(1):165‐171. doi:10.1016/j.bbmt.2014. 10.007. Epub 2014 Oct 12. PMC4272878. ncbi.nlm.nih.gov/pubmed/25316111 Pulsipher MA, Chitphakdithai P, Logan BR, Navarro WH, Levine JE, Miller JP, Shaw BE, O'Donnell PV, Majhail NS, Confer DL. Lower risk for serious adverse events and no increased risk for cancer after PBSC vs BM donation. Blood. 2014 Jun 5; 123(23):3655‐3663. doi:10.1182/blood‐ 2013‐12‐542464. Epub 2014 Apr 15. PMC4047500. ncbi.nlm.nih.gov/pubmed/24735965

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2012 Pulsipher MA, Chitphakdithai P, Logan BR, Shaw BE, Wingard JR, Lazarus HM, Waller EK, Seftel M, Stroncek DF, Lopez AM, Maharaj D, Hematti P, O'Donnell PV, Loren AW, Leitman SF, Anderlini P, Goldstein SC, Levine JE, Navarro WH, Miller JP, Confer DL. Acute toxicities of unrelated bone marrow versus peripheral blood stem cell donation: Results of a prospective trial from the National Marrow Donor Program. Blood. 2013 Jan 3; 121(1):197‐206. doi:10.1182/blood‐ 2012‐03‐417667. Epub 2012 Oct 29. PMC3538330. ncbi.nlm.nih.gov/pubmed/23109243

5.3 Current Studies

DS05‐02a Title: Older adult related donors compared to adult related donors PIs: Michael Pulsipher (Children's Hospital of Los Angeles) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with University of Utah

DS05‐02c Title: Acute toxicities of related adult donors compared to unrelated adult PIs: Michael Pulsipher (Children's Hospital of Los Angeles) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with University of Utah

DS05‐02d Title: QoL for related adult donors compared to unrelated adult donors PIs: Galen Switzer (University of Pittsburgh Medical Center ‐ Cancer Center) Michael Pulsipher (Children's Hospital of Los Angeles) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with University of Utah

DS05‐02e Title: Acute toxicities for pediatric related donors compared to adult related donors PIs: Michael Pulsipher (Children's Hospital of Los Angeles) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with University of Utah

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DS05‐02g Title: Late toxicities and SAE for related donors PIs: Michael Pulsipher (Children's Hospital of Los Angeles) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with University of Utah

DS09‐03 Title: Effects of second donation on marrow/PBSC donors PIs: David Stroncek (National Heart Lung and Blood Institute) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

DS13‐01 Title: Retrospective examination of the role quantity of bone marrow harvests performed by a harvest center has on the overall quality of the harvested product: Assessment of the potential impacts bone marrow product quality has on utilization of bone marrow as a cell source for transplant PIs: Nicole Prokopishyn (University of Calgary) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018)

DS13‐02 Title: A retrospective analysis to understand the potential mechanisms underlying the clinical impact of ABO incompatibility on allogeneic transplant outcomes PIs: Bronwen Shaw (CIBMTR ‐ Milwaukee) Status: Protocol Development (as of July 1, 2017) Submitted (expected by June 30, 2018)

DS16‐01 Title: Comparison between one and two day apheresis in unrelated donors PIs: Jack W. Hsu (Shands HealthCare & University of Florida) John W. Wingard (Shands HealthCare & University of Florida) Status: Protocol Development (as of July 1, 2017) Analysis (expected by June 30, 2018)

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DS16‐S1 Title: Evaluation of practice guidelines for the assessment and surveillance follow‐up of pediatric hematopoietic stem cell donors PIs: Lori Wiener (National Cancer Institute) Galen Switzer (University of Pittsburgh Medical Center ‐ Cancer Center) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with NIH and University of Pittsburgh

DS16‐S2 Title: Survey of the screening and management for clonal disorders of hematopoiesis in related allogeneic donors PIs: Matthew Seftel (CancerCare Manitoba/University of Manitoba) Status: Data Collection / Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

DS17‐01 Title: The impact of donor body mass index on collection of G‐CSF mobilized peripheral blood progenotor cells from unrelated donors PIs: Nosha Farhadfar (Shands HealthCare & University of Florida) Jack W. Hsu (Shands HealthCare & University of Florida) John Wingard (Shands HealthCare & University of Florida) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

DS17‐02 Title: Impact of pre‐operative collection of auto blood for BM harvest on donor health and outcome PIs: Nosha Farhadfar (Shands HealthCare & University of Florida) John Wingard (Shands HealthCare & University of Florida) Hemant Murthy (H. Lee Moffitt Cancer Center and Research Institute) Status: Protocol Development (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with American Association of Blood Banks (AABB) and American Society for Blood and Marrow Transplantation (ASBMT)

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6.0 GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE

6.1 Leadership

Chair: Vanderson Rocha, MD, PhD, Churchill Hospital Email: [email protected] Chair: Asad Bashey, MD, PhD, The Blood and Marrow Transplant Program at Northside Hospital Email: [email protected] Chair: Ian McNiece, PhD, M.D. Anderson Cancer Center Email: [email protected] Scientific Director: Mary Eapen, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Mei‐Jie Zhang, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Andrew St. Martin, MS, CIBMTR Milwaukee Email: [email protected]

6.2 Recent Publications

2017 Bashey A, Zhang M‐J, McCurdy SR, St, Martin A, Argall T, Anasetti C, Ciurea SO, Fasan O, Gaballa S, Hamadani M, Munshi P, Al Malki MM, Nakamura R, O'Donnell PV, Perales M‐A, Raj K, Romee R, Rowley S, Rocha V, Salit RB, Solh M, Soiffer RJ, Fuchs EJ, Eapen M. Mobilized peripheral blood stem cells versus unstimulated bone marrow as a graft source for T‐cell replete haploidentical donor transplantation using post‐transplant cyclophosphamide. Journal of Clinical Oncology. doi:10.1200/JCO.2017.72.8428. Epub 2017 Jun 23. ncbi.nlm.nih.gov/ pubmed/28644773 Brunstein C, Zhang M‐J, Barker J, St, Martin A, Bashey A, de Lima M, Dehn J, Hematti P, Perales MA, Rocha V, Territo M, Weisdorf D, Eapen M. The effect of inter‐unit HLA matching in double umbilical cord blood transplantation for acute leukemia. Haematologica. 2017 May 1; 102(5):941‐947. doi:10.3324/haematol.2016.158584. Epub 2017 Jan 25. PMC5477613. ncbi.nlm.nih.gov/pubmed/28126967

2016 Brady C, Armitage S, Freed B, Duffy M, Gass A, Spellman S, Kurtzberg J, Regan D. How transplant centers deal with the dextran shortage: Recommendations for comparing alternatives. Transfusion. 2016 Nov 1; 56(11):2657‐2661. doi:10.1111/trf.13783. Epub 2016 Sep 5. ncbi.nlm.nih.gov/pubmed/27596242

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Kim HT, Zhang M‐J, Woolfrey AE, St, Martin A, Chen J, Saber W, Perales M‐A, Armand P, Eapen M. Donor and recipient sex in allogeneic stem cell transplantation: What really matters. Haematologica. 2016 Oct 1; 101(10):1260‐1266. doi:10.3324/haematol.2016.147645. Epub 2016 Jun 27. PMC5046656. ncbi.nlm.nih.gov/pubmed/27354023 Rafii H, Ruggeri A, Volt F, Brunstein CG, Carreras J, Eapen M, Gluckman E, Weisdorf DJ. Changing trends of unrelated umbilical cord blood transplantation for hematologic diseases in patients older than fifty years: A Eurocord‐Center for International Blood and Marrow Transplant research survey. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Sep 1; 22(9):1717‐1720. doi:10.1016/j.bbmt.2016.05.015. Epub 2016 May 21. ncbi.nlm.nih.gov/pubmed/27220265 Van Besien K, Hari P, Zhang M‐J, Liu H‐T, Stock W, Godley L, Odenike O, Larson R, Bishop M, Wickrema A, Gergis U, Mayer S, Shore T, Tsai S, Rhodes J, Cushing MM, Korman S, Artz A. Reduced intensity haplo plus single cord transplant compared to double cord transplant: improved engraftment and graft‐versus‐host disease‐free, relapse‐free survival. Haematologica. 2016 May 1; 101(5):634‐643. doi:10.3324/haematol.2015.138594. Epub 2016 Feb 11. PMC5004373. ncbi.nlm.nih.gov/pubmed/26869630 Al Malki MM, Horowitz M, Handgretinger R, Leung W, Roy D‐C, Huang X‐J, Fuchs E, Locatelli F, Blaise D, Mineishi S, Martelli M, Miller J, June C, Ai H‐S, Luznik L, Mavilio D, Lugli E, van den Brink MRM, Champlin RE, Ciurea SO. Proceedings from the Second Haploidentical Stem Cell Transplantation Symposium ‐ Haplo2014, San Francisco, California, December 4, 2014. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Apr 1; 22(4):594‐604. doi:10.1016/j.bbmt.2016.01.001. Epub 2016 Jan 13. ncbi.nlm.nih.gov/pubmed/26806585

2015 Kollman C, Spellman SR, Zhang M‐J, Hassebroek A, Anasetti C, Antin JH, Champlin RE, Confer DL, DiPersio JF, Fernandez‐Viña M, Hartzman RJ, Horowitz MM, Hurley CK, Karanes C, Maiers M, Mueller CR, Perales M‐A, Setterholm M, Woolfrey AE, Yu N, Eapen M. The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy. Blood. 2016 Jan 14; 127(2):260‐267. doi:10.1182/blood‐2015‐08‐663823. Epub 2015 Nov 2. PMC4713163. ncbi.nlm.nih.gov/pubmed/26527675 Ponce DM, Eapen M, Sparapani R, O'Brien TA, Chan KW, Chen J, Craddock J, Schultz KR, Wagner JE, Perales M‐A, Barker JN. In vivo T cell depletion with myeloablative regimens on outcomes after cord blood transplantation for acute lymphoblastic leukemia in children. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2173‐2179. doi:10.1016/j.bbmt.2015.08.022. Epub 2015 Aug 29. PMC4639413. ncbi.nlm.nih.gov/pubmed/26327630

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Ciurea SO, Zhang M‐J, Bacigalupo AA, Bashey A, Appelbaum FR, Aljitawi OS, Armand P, Antin JH, Chen J, Devine SM, Fowler DH, Luznik L, Nakamura R, O'Donnell PV, Perales M‐A, Pingali SR, Porter DL, Riches MR, Ringdén OTH, Rocha V, Vij R, Weisdorf DJ, Champlin RE, Horowitz MM, Fuchs EJ, Eapen M. Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood. 2015 Aug 20; 126(8):1033‐1040. doi:10.1182/blood‐2015‐04‐639831. Epub 2015 Jun 30. PMC4543223. ncbi.nlm.nih.gov/pubmed/26130705

2014 Ballen KK, Logan BR, Laughlin MJ, He W, Ambruso DR, Armitage SE, Beddard RL, Bhatla D, Hwang WYK, Kiss JE, Koegler G, Kurtzberg J, Nagler A, Oh D, Petz LD, Price TH, Quinones RR, Ratanatharathorn V, Rizzo JD, Sazama K, Scaradavou A, Schuster MW, Sender LS, Shpall EJ, Spellman SR, Sutton M, Weitekamp LA, Wingard JR, Eapen M. Effect of cord blood processing on transplantation outcomes after single myeloablative umbilical cord blood transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):688‐695. doi:10.1016/j.bbmt.2014. 12.017. Epub 2014 Dec 24. PMC4359657. ncbi.nlm.nih.gov/pubmed/25543094 Eapen M, Logan BR, Horowitz MM, Zhong X, Perales M‐A, Lee SJ, Rocha V, Soiffer RJ, Champlin RE. Bone marrow or peripheral blood for reduced‐intensity conditioning unrelated donor transplantation. Journal of Clinical Oncology. 2015 Feb 1; 33(4):364‐369. doi:10.1200/JCO. 2014.57.2446. Epub 2014 Dec 22. PMC4302216. ncbi.nlm.nih.gov/pubmed/25534391 Eapen M, Logan BR, Appelbaum FR, Antin JH, Anasetti C, Couriel DR, Chen J, Maziarz RT, McCarthy PL, Nakamura R, Ratanatharathorn V, Vij R, Champlin RE. Long‐term survival after transplantation of unrelated donor peripheral blood or bone marrow hematopoietic cells for hematologic malignancy. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jan 1; 21(1):55‐59. doi:10.1016/ j.bbmt.2014.09.006. Epub 2014 Sep 22. PMC4272909. ncbi.nlm.nih.gov/pubmed/25255165 Törlén J, Ringdén O, Le Rademacher J, Batiwalla M, Chen J, Erkers T, Ho V, Kebriaei P, Keever‐ Taylor C, Kindwall‐Keller T, Lazarus HM, Laughlin MJ, Lill M, O'Brien T, Perales M‐A, Rocha V, Savani BN, Szwajcer D, Valcarcel D, Eapen M. Low CD34 dose is associated with poor survival after reduced‐intensity conditioning allogeneic transplantation for acute myeloid leukemia and myelodysplastic syndrome. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Sep 1; 20(9):1418‐ 1425. doi:10.1016/j.bbmt.2014.05.021. Epub 2014 Jun 2. PMC4127369. ncbi.nlm.nih.gov/ pubmed/24892261 Weisdorf D, Eapen M, Ruggeri A, Zhang M‐J, Zhong X, Brunstein C, Ustun C, Rocha V, Gluckman E. Alternative donor transplantation for older patients with acute myeloid leukemia in first complete remission: A Center for International Blood and Marrow Transplant Research‐ Eurocord analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Jun 1; 20(6):816‐822. doi:10.1016/j.bbmt. 2014.02.020. Epub 2014 Feb 26. PMC4085692. ncbi.nlm.nih.gov/pubmed/24582782

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2013 Marks DI, Woo KA, Zhong X, Appelbaum FR, Bachanova V, Barker JN, Brunstein CG, Gibson J, Kebriaei P, Lazarus HM, Olsson R, Perales M‐A, Pidala J, Savani B, Rocha V, Eapen M. Unrelated umbilical cord blood transplant for adult acute lymphoblastic leukemia in first and second complete remission: A comparison with allografts from adult unrelated donors. Haematologica. 2014 Feb 1; 99(2):322‐328. doi:10.3324/haematol.2013.094193. Epub 2013 Sep 20. PMC3912963. ncbi.nlm.nih.gov/pubmed/24056817 Alousi AM, Le‐Rademacher J, Saliba RM, Appelbaum FR, Artz A, Benjamin J, Devine SM, Kan F, Laughlin MJ, Lazarus HM, Liesveld J, Perales M‐A, Maziarz RT, Sabloff M, Waller EK, Eapen M, Champlin RE. Who is the better donor for older hematopoietic transplant recipients: An older‐aged sibling or a young, matched unrelated volunteer? Blood. 2013 Mar 28; 121(13):2567‐2573. doi:10.1182/blood‐2012‐08‐453860. Epub 2013 Jan 29. PMC3612864. ncbi.nlm.nih.gov/pubmed/23361908

2012 Scaradavou A, Brunstein CG, Eapen M, LeRademacher J, Barker JN, Chao N, Cutler C, Delaney C, Kan F, Isola L, Karanes C, Laughlin MJ, Wagner JE, Shpall EJ. Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia. Blood. 2013 Jan 31; 121(5):752‐758. doi:10.1182/blood‐2012‐08‐449108. Epub 2012 Dec 9. PMC3563363. ncbi.nlm.nih.gov/pubmed/23223509

6.3 Current Studies

GS15‐01 Title: Optimal graft and donor selection for patients undergoing T‐cell replete haploidentical donor transplantation using post‐transplant cyclophosphamide PIs: Shannon McCurdy (The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins) Ephraim Fuchs (The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

GS15‐02 Title: Long term outcomes of 100‐day survivors of hematopoietic stem cell transplant by cell source PIs: William Hwang (Singapore General Hospital) Claudio Brunstein (University of Minnesota Blood and Marrow Transplant Program) Status: Protocol Development (as of July 1, 2017) Submitted (expected by June 30, 2018)

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GS16‐02 Title: Outcomes in haploidentical vs matched unrelated donor transplants in older patients PIs: Miguel‐Angel Perales (Memorial Sloan Kettering Cancer Center) Benjamin Tomlinson (Seidman Cancer Center‐University Hospitals Cleveland Medical Center) Marcos de Lima (Seidman Cancer Center‐University Hospitals Cleveland Medical Center) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

GS16‐03 Title: Donor selection for allogeneic stem cell transplantation: A case‐control comparison of children (using post‐transplantation cyclophosphamide in GVHD prophylaxis) versus HLA‐matched siblings PIs: Ephraim Fuchs (The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with EMBT

GS17‐01 Title: Comparing outcomes between cord blood and haploidentical transplants in patients with acute myeloid leukemia PIs: Rizwan Romee (Barnes Jewish Hospital) Status: Protocol Pending (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with Pediatric Health Information System® (PHIS)

GS17‐02 Title: Myeloablative vs reduced intensity conditioning in haploidentical transplantation PIs: Scott Solomon (The Blood and Marrow Transplant Program at Northside Hospital) Nirav Shah (Medical College of Wisconsin) Giancarlo Fatobene (Seattle Cancer Care Alliance) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

GS17‐03 Title: Unrelated donor bone marrow vs. peripheral blood transplants for AML and ALL in children PIs: Daniel Keesler (CIBMTR ‐ Milwaukee) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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7.0 GRAFT‐VS‐HOST DISEASE WORKING COMMITTEE

7.1 Leadership

Chair: Daniel Couriel, MD, Utah Blood and Marrow Transplant Program Email: [email protected] Chair: Amin Alousi, MD, MD Anderson Cancer Center Email: [email protected] Chair: Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute Email: [email protected] Scientific Director: Mukta Arora, MD, MS, CIBMTR Minneapolis Email: [email protected] Scientific Director: Stephen Spellman, MBS, CIBMTR Minneapolis Email: [email protected] Statistical Director: Tao Wang, PhD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Ying Liu, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Michael Hemmer, MS, CIBMTR Milwaukee Email: [email protected] CAC Representative: Kristin Scheeler, Leukemia and Lymphoma Society CAC Representative: Jennifer Wilder, BSN, RN, National Institutes of Health

7.2 Recent Publications

2017 Krakow EF, Hemmer M, Wang T, Logan B, Arora M, Spellman S, Couriel D, Alousi A, Pidala J, Last M, Lachance S, Moodie EEM. Tools for the precision medicine era: How to develop highly personalized treatment recommendations from cohort and registry data using Q‐learning. American Journal of Epidemiology. doi:10.1093/aje/kwx027. Epub 2017 May 3. ncbi.nlm.nih.gov /pubmed/28472335 Khoury HJ, Wang T, Hemmer MT, Couriel D, Alousi A, Cutler C, Aljurf M, Antin JH, Ayas M, Battiwalla M, Cahn J‐Y, Cairo M, Chen Y‐B, Gale RP, Hashmi S, 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, Socié G, Storek J, Verdonck L, Vij R, Wood WA, Yu L, Martino R, Carabasi M, Dandoy C, Gergis U, Hermatti P, Solh M, Jamani K, Lehmann L, Savani B, Schultz KR, Wirk BM, Spellman S, Arora M, Pidala J. Improved survival after acute graft vs. host disease diagnosis in the modern era. Haematologica. 2017 May 1; 102(5):958‐966. doi:10.3324/haematol.2016.156356. Epub 2017 Mar 16. PMC5477615. ncbi.nlm.nih.gov/pubmed/28302712

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2016 Chen Y‐B, Wang T, Hemmer MT, Brady C, Couriel DR, Alousi A, Pidala J, Urbano‐Isizua A, Choi SW, Nishihori T, Teshima T, Inamoto Y, Wirk B, Marks DI, Abdel‐Azim H, Lehmann L, Yu L, Bitan M, Cairo MS, Qayed M, Salit R, Gale RP, Martino R, Jaglowski S, Bajel A, Savani B, Frangoul H, Lewis ID, Storek J, Askar M, Kharfan‐Dabaja MA, Aljurf M, Ringen O, Reshef R, Olsson RF, Hashmi S, Seo S, Spitzer TR, MacMillan ML, Lazaryan A, Spellman SR, Arora M, Cutler CS. GVHD after umbilical cord blood transplantation for acute leukemia: An analysis of risk factors and effect on outcomes. Bone Marrow Transplantation. 2017 Mar 1; 52(3):400‐408. doi:10.1038/bmt. 2016.265. Epub 2016 Dec 12. PMC5332289. ncbi.nlm.nih.gov/pubmed/27941764

2015 Inamoto Y, Flowers MED, Wang T, Urbano‐Ispizua A, Hemmer MT, Cutler CS, Couriel DR, Alousi AM, Antin JH, Gale RP, Gupta V, Hamilton BK, Kharfan‐Dabaja MA, Marks DI, Ringdén OTH, Socié G, Solh MM, Akpek G, Cairo MS, Chao NJ, Hayashi RJ, Nishihori T, Reshef R, Saad A, Shah A, Teshima T, Tallman MS, Wirk B, Spellman SR, Arora M, Martin PJ. versus cyclosporine after hematopoietic cell transplantation for acquired aplastic anemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1776‐1782. doi:10.1016/j.bbmt.2015.05.023. Epub 2015 May 30. PMC4568149. ncbi.nlm.nih.gov/pubmed/26033280

2014 Arora M, Hemmer MT, Ahn KW, Klein JP, Cutler CS, Urbano‐Ispizua A, Couriel DR, Alousi AM, Gale RP, Inamoto Y, Weisdorf DJ, Li P, Antin JH, Bolwell BJ, Boyiadzis M, Cahn J‐Y, Cairo MS, Isola LM, Jacobsohn DA, Jagasia M, Klumpp TR, Petersdorf EW, Santarone S, Schouten HC, Wingard JR, Spellman SR, Pavletic SZ, Lee SJ, Horowitz MM, Flowers ME. Center for International Blood and Marrow Transplant Research chronic graft‐versus‐host disease risk score predicts mortality in an independent validation cohort. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):640‐ 645. doi:10.1016/j.bbmt.2014.10.022. Epub 2014 Dec 18. PMC4359642. ncbi.nlm.nih.gov/ pubmed/25528390 Arai S, Arora M, Wang T, Spellman SR, He W, Couriel DR, Urbano‐Ispizua A, Cutler CS, Bacigalupo AA, Battiwalla M, Flowers ME, Juckett MB, Lee SJ, Loren AW, Klumpp TR, Prockup SE, Ringdén OT, Savani BN, Socié G, Schultz KR, Spitzer T, Teshima T, Bredeson CN, Jacobsohn DA, Hayashi RJ, Drobyski WR, Frangoul HA, Akpek G, Ho VT, Lewis VA, Gale RP, Koreth J, Chao NJ, Aljurf MD, Cooper BW, Laughlin MJ, Hsu JW, Hematti P, Verdonck LF, Solh MM, Norkin M, Reddy V, Martino R, Gadalla S, Goldberg JD, McCarthy PL, Pérez‐Simón JA, Khera N, Lewis ID, Atsuta Y, Olsson RF, Saber W, Waller EK, Blaise D, Pidala JA, Martin PJ, Satwani P, Bornhäuser M, Inamoto Y, Weisdorf DJ, Horowitz MM, Pavletic SZ. Increasing incidence of chronic graft‐versus‐host disease in allogeneic transplantation: A report from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Feb 1; 21(2):266‐274. doi:10.1016/ j.bbmt.2014.10.021. Epub 2014 Oct 30. PMC4326247. ncbi.nlm.nih.gov/pubmed/25445023

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Boyiadzis M, Arora M, Klein JP, Hassebroek A, Hemmer M, Urbano‐Ispizua A, Antin JH, Bolwell BJ, Cahn J‐YY, Cairo MS, Cutler CS, Flowers ME, Gale RP, Herzig R, Isola LM, Jacobsohn DA, Jagasia MH, Klumpp TR, Lee SJ, Petersdorf EW, Santarone S, Spellman SR, Schouten HC, Verdonck LF, Wingard JR, Weisdorf DJ, Horowitz MM, Pavletic SZ. Impact of chronic graft‐versus‐host disease on late relapse and survival on 7,489 patients after myeloablative allogeneic hematopoietic cell transplantation for leukemia. Clinical Cancer Research. 2015 May 1; 21(9):2020‐2028. doi:10.1158/1078‐0432.CCR‐14‐0586. Epub 2014 Oct 27. PMC4411210. ncbi.nlm.nih.gov/pubmed/25348512

2012 Weisdorf D, Zhang M‐J, Arora M, Horowitz MM, Rizzo JD, Eapen M. Graft‐versus‐host disease induced graft‐versus‐leukemia effect: Greater impact on relapse and disease‐free survival after reduced intensity conditioning. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2012 Nov 1; 18(11):1727‐1733. doi:10.1016/j.bbmt.2012.06.014. Epub 2012 Jul 2. PMC3472079. ncbi.nlm.nih.gov/pubmed/ 22766220

7.3 Current Studies

GV12‐02 Title: Prognostic Implications of Acute Upper Gastrointestinal Graft‐versus‐Host Disease in Patients undergoing Myeloablative Hematopoietic Cell Transplantation PIs: Corey Cutler (Dana Farber Cancer Institute) Sarah Nikiforow (Dana Farber Cancer Institute) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

GV13‐01 Title: Impact of donor parity and donor type on outcomes of allogeneic hematopoietic stem cell transplantation PIs: Anita Kumar (Tufts Medical Center) Alison Loren (Abramson Cancer Center University of Pennsylvania Medical Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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GV14‐01 Title: Comparison of mycophenolate versus methotrexate in combination with a calcineurin inhibitor for graft‐versus‐host disease prophylaxis in allogeneic HCT PIs: Betty Hamilton (Cleveland Clinic Foundation) Saurabh Chhabra (Medical University of South Carolina) Navneet Majhail (Cleveland Clinic Foundation) Luciano Costa (University of Alabama at Birmingham) Robert Stuart (Medical University of South Carolina) Dennis Kim (Princess Margaret Mary Hospital) Oll Ringden (Karolinska Insitutet) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018)

GV14‐02 Title: Influence of recipient age on risk for acute and chronic graft versus host disease in children receiving HLA‐identical sibling bone marrow transplantation PIs: Muna Qayed (Children's Healthcare of Atlanta at Egleston) John T. Horan (Children's Healthcare of Atlanta at Egleston) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

GV15‐01a Title: Impact of donor obesity and inflammation on acute and chronic graft versus host disease among hematopoietic cell transplantation recipients PIs: Lucie Turcotte (University of Minnesota Blood and Marrow Transplant Program) Michael Verneris (University of Colorado ‐ Children's Hospital) Jennifer Knight (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

GV15‐01b Title: Impact of donor obesity and inflammation on acute and chronic graft versus host disease among hematopoietic cell transplantation recipients PIs: Lucie Turcotte (University of Minnesota Blood and Marrow Transplant Program) Michael Verneris (University of Colorado ‐ Children's Hospital) Jennifer Knight (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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GV15‐02 Title: Do bone marrow grafts result in improved GRFS when compared to peripheral blood grafts in adults receiving allogeneic HCT from matched unrelated donors? PIs: Amin Alousi (M.D. Anderson Cancer Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

GV16‐01 Title: GVHD‐free relapse‐free survival in alternative donor hematopoietic cell transplantation PIs: Rohtesh S. Mehta (University of Arkansas for Medical Sciences) Status: Analysis (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

GV16‐02 Title: The impact of the graft T cell dose on the outcome of allogeneic HLA‐matched peripheral blood stem cell transplantation. PIs: Ayman Saad (University of Alabama at Birmingham) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

GV17‐01 Title: Investigating antibiotic exposure and risk of acute graft versus host disease in children undergoing hematopoietic stem cell transplantation for acute leukemia PIs: Caitlin Elgarten (Children's Hospital of Philadelphia) Brian Fisher (Perelman School of Medicine at the University of Pennsylvania) Richard Aplenc (Children's Hospital of Philadelphia) Status: Protocol Development (as of July 1, 2017) Analysis (expected by June 30, 2018)

GV17‐02 Title: Risk factors of acute and chronic GVHD in T‐replete HLA haploidnetical HCT using post‐transplantation Cyclophosphamide PIs: Annie Im (University of Pittsburgh Medical Center ‐ Cancer Center) Betty Hamilton (Cleveland Clinic Foundation) Armin Rashidi (University of Minnesota) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

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GV17‐03 Title: Alterations in the characteristics and outcomes of acute and chronic GVHD following post‐transplant high dose Cytoxan prophylaxis for haploidentical transplantation and in patients over 60 at high risk for GVHD PIs: Rima Saliba (M.D. Anderson Cancer Center) Stefan Ciurea (M.D. Anderson Cancer Center) Jeff Schriber (Cancer Transplant Institute at Virginia G. Piper Cancer Center) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

GV17‐04 Title: Analysis of semi‐competing risks data from a nested case‐control study, applied to acute graft‐versus‐host disease and death after hematopoietic stem cell transplantation PIs: Ina Jazic (Harvard T.H. Chan School of Public Health) Sebastien Haneuse (Harvard T.H. Chan School of Public Health) Status: Data File Preparation (as of July 1, 2017) Analysis (expected by June 30, 2018) * Collaborative study with Harvard School of Public Health

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8.0 HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE

8.1 Leadership

Chair: Jignesh Dalal, MD, University Hospitals Case Medical Center Email: [email protected] Chair: Theresa Hahn, PhD, Roswell Park Cancer Institute Email: [email protected] Chair: Nandita Khera, MD, Mayo Clinic Arizona and Phoenix Children's Hospital Email: [email protected] Chair: William Wood, MD, MPH, University of North Carolina Hospitals Email: [email protected] Scientific Director: Wael Saber, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Ruta Brazauskas, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Naya He, MPH, CIBMTR Milwaukee Email: [email protected] CAC Representative: Jack Aiello, San Jose, CA

8.2 Recent Publications

2017 El‐Jawahri A, Chen Y‐B, Brazauskas R, He N, Lee SJ, Knight JM, Majhail N, Buchbinder D, Schears RM, Wirk BM, Wood WA, Ahmed I, Aljurf 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. Impact of pre‐transplant depression on outcomes of allogeneic and autologous hematopoietic stem cell transplantation. Cancer. 2017 May 15; 123(10): 1828‐1838. doi:10.1002/cncr.30546. Epub 2017 Jan 19. PMC5419891. ncbi.nlm.nih.gov /pubmed/28102896

2016 Wood WA, McGinn MK, Wilson D, Deal AM, Khera N, Shea TC, Devine SM, Appelbaum FR, Horowitz MM, Lee SJ. Practice patterns and preferences among hematopoietic cell transplantation clinicians. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Nov 1; 22(11):2092‐2099. doi:10.1016/j.bbmt.2016.07.014. Epub 2016 Jul 29. PMC5067220. ncbi.nlm.nih.gov/ pubmed/27481447

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Denzen EM, Thao V, Hahn T, Lee SJ, McCarthy PL, Rizzo JD, Ammi M, Drexler R, Flesch S, James H, Omondi N, Murphy E, Pederson K, Majhail NS. Financial impact of allogeneic hematopoietic cell transplantation on patients and families over 2 years: Results from a multicenter pilot study. Bone Marrow Transplantation. doi:10.1038/bmt.2016.103. Epub 2016 Apr 18. ncbi.nlm.nih.gov/pubmed/27088381 Gale RP, Seber A, Bonfim C, Pasquini M. Haematopoietic cell transplants in Latin America. Bone Marrow Transplantation. 2016 Jul 1; 51(7):898‐905. doi:10.1038/bmt.2016.35. Epub 2016 Mar 21. PMC4935585. ncbi.nlm.nih.gov/pubmed/26999468 Niederwieser D, Baldomero H, Szer J, Gratwohl M, Aljurf M, Atsuta Y, Bouzas LF, Confer D, Greinix H, Horowitz M, Iida M, Lipton J, Mohty M, Novitzky N, Nunez J, Passweg J, Pasquini MC, Kodera Y, Apperley J, Seber A, Gratwohl A. Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey. Bone Marrow Transplantation. 2016 Jun 1; 51(6):778‐785. doi:10.1038/bmt.2016.18. Epub 2016 Feb 22. PMC4889523. ncbi.nlm.nih.gov /pubmed/26901703 Kumar R, Kimura F, Ahn KW, Hu Z‐H, Kuwatsuka Y, Klein JP, Pasquini M, Miyamura K, Kato K, Yoshimi A, Inamoto Y, Ichinohe T, Wood WA Jr, Wirk B, Seftel M, Rowlings P, Marks DI, Schultz KR, Gupta V, Dedeken L, George B, Cahn J‐Y, Szer J, Lee JW, Ho AY, Fasth A, Hahn T, Khera N, Dalal J, Bonfim C, Aljurf M, Atsuta Y, Saber W. Comparing outcomes with bone marrow or peripheral blood stem cells as graft source for matched sibling transplants in severe aplastic anemia across different economic regions. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 May 1; 22(5):932‐940. doi:10.1016/j.bbmt.2016.01.012. Epub 2016 Jan 18. PMC4826285. ncbi.nlm.nih.gov/pubmed/26797402 Yoshimi A, Baldomero H, Horowitz M, Szer J, Niederwieser D, Gratwohl A, Kodera Y. Global use of peripheral blood vs bone marrow as source of stem cells for allogeneic transplantation in patients with bone marrow failure. JAMA: The Journal of the American Medical Association. 2016 Jan 12; 315(2):198‐200. doi:10.1001/jama.2015.13706. Epub 2016 Jan 12. ncbi.nlm.nih.gov/pubmed/26757470 Kanda J, Brazauskas R, Hu ZH, Kuwatsuka Y, Nagafuji K, Kanamori H, Kanda Y, Miyamura K, Murata M, Fukuda T, Sakamaki H, Kimura F, Seo S, Aljurf M, Yoshimi A, Milone G, Wood WA, Ustun C, Hashmi S, Pasquini M, Bonfim C, Dalal J, Hahn T, Atsuta Y, Saber W. GVHD after HLA‐ matched sibling BMT or PBSCT: Comparison of North American Caucasian and Japanese populations. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Apr 1; 22(4):744‐751. doi:10.1016/j.bbmt.2015. 12.027. Epub 2016 Jan 4. PMC4801761. ncbi.nlm.nih.gov/pubmed/26762681

2015 Knight JM, Rizzo JD, Logan BR, Wang T, Arevalo JMG, Ma J, Cole SW. Low socioeconomic status, adverse gene expression profiles, and clinical outcomes in hematopoietic stem cell transplant recipients. Clinical Cancer Research. 2016 Jan 1; 22(1):69‐78. doi:10.1158/1078‐ 0432.CCR‐15‐1344. Epub 2015 Aug 18. PMC4703514. ncbi.nlm.nih.gov/pubmed/26286914

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Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ. Comparison of characteristics and outcomes of trial participants and nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 trial. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1815‐1822. doi:10.1016/j.bbmt.2015.06.004. Epub 2015 Jun 11. PMC4568172. ncbi.nlm.nih.gov/pubmed/26071866

2014 Ballen KK, Joffe S, Brazauskas R, Wang Z, Aljurf MD, Akpek G, Dandoy C, Frangoul HA, Freytes CO, Khera N, Lazarus HM, LeMaistre CF, Mehta P, Parsons SK, Szwajcer D, Ustun C, Wood WA, Majhail NS. Hospital length of stay in the first 100 days after allogeneic hematopoietic cell transplantation for acute leukemia in remission: Comparison among alternative graft sources. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1819‐1827. doi:10.1016/j.bbmt. 2014.07.021. Epub 2014 Jul 23. PMC4194253. ncbi.nlm.nih.gov/pubmed/25064747 Wood WA, Lee SJ, Brazauskas R, Wang Z, Aljurf MD, Ballen KK, Buchbinder DK, Dehn J, Freytes CO, Lazarus HM, LeMaistre CF, Mehta P, Szwajcer D, Joffe S, Majhail NS. Survival improvements in adolescents and young adults after myeloablative allogeneic transplantation for acute lymphoblastic leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Jun 1; 20(6):829‐836. doi:10.1016/j.bbmt.2014.02.021. Epub 2014 Mar 7. PMC4019683. ncbi.nlm.nih.gov/pubmed/ 24607554 Kuwatsuka Y, Atsuta Y, Horowitz MM, Inagaki J, Kanda J, Kato K, Koh K, Zhang M‐J, Eapen M. Graft‐versus‐host disease and survival after cord blood transplantation for acute leukemia: A comparison of Japanese versus White populations. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 May 1; 20(5):662‐667. doi:10.1016/j.bbmt.2014.01.020. Epub 2014 Feb 10. PMC4071962. ncbi.nlm.nih.gov/pubmed/24525277

2013 Majhail NS, Tao L, Bredeson C, Davies S, Dehn J, Gajewski JL, Hahn T, Jakubowski A, Joffe S, Lazarus HM, Parsons SK, Robien K, Lee SJ, Kuntz KM. Prevalence of hematopoietic cell transplant survivors in the United States. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Oct 1; 19(10): 1498‐1501. doi:10.1016/j.bbmt.2013.07.020. Epub 2013 Jul 29. PMC3779514. ncbi.nlm.nih.gov/ pubmed/23906634 Hahn T, McCarthy PL Jr, Hassebroek A, Bredeson C, Gajewski JL, Hale GA, Isola LM, Lazarus HM, Lee SJ, Lemaistre CF, Loberiza F, Maziarz RT, Rizzo JD, Joffe S, Parsons S, Majhail NS. Significant improvement in survival after allogeneic hematopoietic cell transplantation during a period of significantly increased use, older recipient age, and use of unrelated donors. Journal of Clinical Oncology. 2013 Jul 1; 31(19):2437‐2449. doi:10.1200/JCO.2012.46.6193. Epub 2013 May 28. PMC3691359. ncbi.nlm.nih.gov/pubmed/23715573

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McCarthy PL Jr., Hahn T, Hassebroek A, Bredeson C, Gajewski J, Hale G, Isola L, Lazarus HM, Lee SJ, Lemaistre CF, Loberiza F, Maziarz RT, Rizzo JD, Joffe S, Parsons S, Majhail NS. Trends in use of and survival after autologous hematopoietic cell transplantation in North America, 1995‐ 2005: Significant improvement in survival for lymphoma and myeloma during a period of increasing recipient age. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Jul 1; 19(7):1116‐1123. doi:10.1016/j.bbmt.2013.04.027. Epub 2013 May 6. PMC3694566. ncbi.nlm.nih.gov/pubmed/ 23660172

2012 Majhail NS, Rizzo JD, Hahn T, Lee SJ, McCarthy PL, Ammi M, Denzen E, Drexler R, Flesch S, James H, Omondi N, Pedersen TL, Murphy E, Pederson K. Pilot study of patient and caregiver out‐of‐ pocket costs of allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation. 2013 Jun 1; 48(6):865‐871. doi:10.1038/bmt.2012.248. Epub 2012 Dec 10. PMC3596484. ncbi.nlm.nih.gov/pubmed/23222378

8.3 Current Studies

IS10‐01 Title: Outcomes of HCT for ALL: An international comparative analysis PIs: William Wood (University of North Carolina Hospitals) Navneet Majhail (Cleveland Clinic Foundation) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

HS12‐02 Title: Rates of transplantation in urban vs rural patients: Are rural patients less likely to receive an allogeneic transplant? PIs: Kristjan Paulson (CancerCare Manitoba/University of Manitoba) Matthew Seftel (CancerCare Manitoba/University of Manitoba) David Szwajcer (CancerCare Manitoba/University of Manitoba) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

HS13‐02 Title: Investigating inpatient health care utilization of matched sibling donor hematopoietic cell transplantation for children with sickle cell disease PIs: Staci Arnold (Emory University Hospital) Prakash Satwani (NYPH/ Columbia University Medical Center) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018) * Collaborative study with PHIS

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HS14‐01 Title: Investigating clinical outcomes and inpatient health care resource utilization of hematopoietic cell transplantation for children with acute leukemia PIs: Staci Arnold (Emory University Hospital) Richard Aplenc (Children's Hospital of Philadelphia) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with PHIS

HS15‐01 Title: Who is lost to follow‐up in the Center for International Blood and Marrow Transplant Research (CIBMTR) registry? PIs: David Buchbinder (Children's Hospital of Orange County) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

HS15‐02 Title: Impact of socioeconomic status on pediatric stem cell transplant outcomes PIs: Kira Bona (Dana Farber Cancer Institute) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

HS16‐01 Title: Trends in utilization and outcomes of autologous and allogeneic hematopoietic cell transplantation in racial and ethnic minorities PIs: Nandita Khera (Mayo Clinic Arizona and Phoenix Children's Hospital) Wael Saber (CIBMTR ‐ Milwaukee) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

HS16‐02 Title: The impact of marital status on hematopoietic stem cell transplant recipient outcomes: A surrogate for consistent caregiver PIs: Sara Beattie (Tom Baker Cancer Centre) Status: Data File Preparation (as of July 1, 2017) Analysis (expected by June 30, 2018)

HS16‐03 Title: Relationship of race/ethnicity and survival after single and double umbilical cord blood transplantation PIs: Karen Ballen (University of Virginia Health System) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

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HS17‐01 Title: Association of community health status and center survival for allogeneic hematopoietic cell transplantation PIs: Sanghee Hong (University of Washington) Navneet Majhail (Cleveland Clinic Foundation) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

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9.0 IMMUNOBIOLOGY WORKING COMMITTEE

9.1 Leadership

Chair: Michael Verneris, MD, University of Minnesota Medical Center, Fairview Email: [email protected] Chair: Katharina Fleischhauer, MD, Universitätsklinikum Essen KMT Email: katharina.fleischhauer@uk‐essen.de Chair: Katharine Hsu, MD, PhD, Memorial Sloan Kettering Cancer Center Email: [email protected] Scientific Director: Stephanie J. Lee, MD, MPH, CIBMTR, Fred Hutchinson Cancer Research Center Email: [email protected] Scientific Director: Stephen Spellman, MBS, CIBMTR Minneapolis Email: [email protected] Statistical Director: Tao Wang, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Mike Haagenson, MS, CIBMTR Minneapolis Email: [email protected]

9.2 Recent Publications

2017 Eapen M, Wang T, Veys PA, Boelens JJ, St, Martin A, Spellman S, Bonfim C, Brady C, Cant AJ, Dalle J‐H, Davies SM, Freeman J, Hsu KC, Fleischhauer K, Kenzey C, Kurtzberg J, Michel G, Orchard PJ, Paviglianiti A, Rocha V, Veneris M, Volt F, Wynn R, Lee SJ, Horowitz MM, Gluckman E, Ruggeri A. Allele‐level HLA matching for umbilical cord blood transplantation for non‐malignant diseases in children: A retrospective analysis. The Lancet Haematology. doi:10.1016/S2352‐ 3026(17)30104‐7. Epub 2017 Jun 13. ncbi.nlm.nih.gov/pubmed/28623181 Fleischhauer K, Ahn KW, Wang HL, Zito L, Crivello P, Müller C, Verneris M, Shaw BE, Pidala J, Oudshorn M, Lee SJ, Spellman SR. Directionality of non‐permissive HLA‐DPB1 T‐cell epitope group mismatches does not improve clinical risk stratification in 8/8 matched unrelated donor hematopoietic cell transplantation. Bone Marrow Transplantation. doi:10.1038/bmt.2017.96. Epub 2017 Jun 5. ncbi.nlm.nih.gov/pubmed/28581467 Boudreau JE, Giglio F, Gooley TA, Stevenson PA, Le LuduecJ‐B, Shaffer BC, Rajalingam R, Hou L, Hurley CK, Noreen H, Reed EF, Yu N, Vierra‐Green C, Haagenson M, Malkki M, Petersdorf EW, Spellman S, Hsu K. KIR3DL1/HLA‐B subtypes govern acute myelogenous leukemia relapse after hematopoietic cell transplantation. Journal of Clinical Oncology. 2017 Jul 1; 35(20):2268‐2278. doi:10.1200/JCO.2016.70.7059. Epub 2017 May 18. PMC5501362. ncbi.nlm.nih.gov/pubmed/28520526

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Wang Y, Wang T, Dagnall C, Haagenson M, Spellman SR, Hicks B, Jones K, Lee SJ, Savage SA, Gadalla SM. Relative telomere length prior to hematopoietic cell transplantation (HCT) and outcome after unrelated donor HCT for acute leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:DOI: 10.1016/j.bbmt.2017.03.032. Epub 2017 Apr 4. ncbi.nlm.nih.gov/pubmed/28389255 Madbouly A, Wang T, Haagenson M, Paunic V, Vierra‐Green C, Fleischhauer K, Hsu KC, Verneris MR. Investigating the association of genetic admixture and donor/recipient genetic disparity with transplant outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2017.02.019. Epub 2017 Mar 2. ncbi.nlm.nih.gov/pubmed/28263917 Lindsley RC, Saber W, Mar BG, Redd R, Wang T, Haagenson MD, Grauman PV, Hu Z‐H, Spellman SR, Lee SJ, Verneris MR, Hsu K, Fleischhauer K, Cutler C, Antin JH, Neuberg D, Ebert BL. Prognostic mutations in myelodysplastic syndrome after stem‐cell transplantation. New England Journal of Medicine. 2017 Feb 9; 376(6):536‐547. doi:10.1056/NEJMoa1611604. Epub 2017 Feb 9. PMC5438571. ncbi.nlm.nih.gov/pubmed/28177873

2016 Hou L, Vierra‐Green C, Lazaro A, Brady C, Haagenson M, Spellman S, Hurley CK. Limited HLA sequence variation outside of antigen recognition domain exons of 360 10 of 10 matched unrelated hematopoietic stem cell transplant donor‐recipient pairs. HLA. 2017 Jan 1; 89(1):39‐46. doi:10.1111/tan.12942. Epub 2016 Dec 15. PMC5425813. ncbi.nlm.nih.gov/ pubmed/27976839 Askar M, Sobecks R, Wang T, Haagenson M, Majhail N, Madbouly A, Thomas D, Zhang A, Fleischhauer K, Hsu K, Verneris M, Lee SJ, Spellman SR, Fernández‐Viña M. MHC class I chain‐ related gene A (MICA) donor‐recipient mismatches and MICA‐129 polymorphism in unrelated donor hematopoietic cell transplantations has no impact on outcomes in acute lymphoblastic leukemia, acute myeloid leukemia, or myelodysplastic syndrome: A Center for International Blood and Marrow Transplant Research study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Mar 1; 23(3):436‐444. doi:10.1016/j.bbmt.2016.11.021. Epub 2016 Dec 14. PMC5370205. ncbi.nlm.nih.gov/pubmed/27987385 Hoff GA, Fischer JC, Hsu K, Cooley S, Miller JS, Wang T, Haagenson M, Spellman S, Lee SJ, Uhrberg M, Venstrom JM, Verneris MR. Recipient HLA‐C haplotypes and microRNA 148a/b binding sites have no impact on allogeneic hematopoietic cell transplantation outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jan 1; 23(1):153‐160. doi:10.1016/j.bbmt.2016.09.028. Epub 2016 Oct 13. PMC5198579. ncbi.nlm.nih.gov/pubmed/27746218 Goyal RK, Lee SJ, Wang T, Trucco M, Haagenson M, Spellman SR, Verneris M, Ferrell RE. Novel HLA‐DP region susceptibility loci associated with severe acute GvHD. Bone Marrow Transplantation. 2017 Jan 1; 52(1):95‐100. doi:10.1038/bmt.2016.210. Epub 2016 Sep 5. PMC5215038. ncbi.nlm.nih.gov/pubmed/27595289

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Kornblit B, Wang T, Lee SJ, Spellman SR, Zhu X, Fleischhauer K, Müller C, Verneris MR, Müller K, Johansen JS, Vindelov L, Garred P. YKL‐40 in allogeneic hematopoietic cell transplantation after AML and myelodysplastic syndrome. Bone Marrow Transplantation. 2016 Dec 1; 51(12):1556‐1560. doi:10.1038/bmt.2016.192. Epub 2016 Jul 18. PMC5143177. ncbi.nlm.nih.gov/pubmed/27427920 Lazaryan A, Wang T, Spellman SR, Wang H‐L, Pidala J, Nishihori T, Askar M, Olsson R, Oudshoorn M, Abdel‐Azim H, Yong A, Gandhi M, Dandoy C, Savani B, Hale G, Page K, Bitan M, Reshef R, Drobyski W, Marsh SGE, Schultz K, Müller CR, Fernandez‐Viña M, Verneris MR, Horowitz MM, Arora M, Weisdorf DJ, Lee SJ. Human leukocyte antigen supertype matching after myeloablative hematopoietic cell transplantation with 7/8 matched unrelated donor allografts: A report from the Center for International Blood and Marrow Transplant Research. Haematologica. doi:10.3324/haematol.2016.143271. Epub 2016 May 31. ncbi.nlm.nih.gov/ pubmed/27247320 Marino SR, Lee SM, Binkowski TA, Wang T, Haagenson M, Wang H‐L, Maiers M, Spellman S, van Besien K, Lee SJ, Karrison T, Artz A. Identification of high‐risk amino‐acid substitutions in hematopoietic cell transplantation: A challenging task. Bone Marrow Transplantation. doi:10.1038/bmt.2016.142. Epub 2016 May 23. ncbi.nlm.nih.gov/pubmed/27214075 Bachanova V, Weisdorf DJ, Wang T, Marsh SGE, Trachtenberg E, Haagenson MD, Spellman SR, Ladner M, Guethlein LA, Parham P, Miller JS, Cooley SA. Donor KIR B genotype improves progression free survival of non‐Hodgkin lymphoma patients receiving unrelated donor transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.05.016. Epub 2016 May 21. ncbi.nlm.nih.gov/pubmed/27220262 Arora M, Lee SJ, Spellman SR, Weisdorf DJ, Guan W, Haagenson M, Wang T, Horowitz MH, Verneris MR, Fleischhauer K, Hsu K, Thyagarajan B. A validation study failed to confirm association between genetic variants in the base excision repair pathway and TRM and relapse post HCT. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.04.020. Epub 2016 May 4. ncbi.nlm.nih.gov/pubmed/27155583 Rocha V, Ruggeri A, Spellman S, Wang T, Sobecks R, Locatelli F, Askar M, Michel G, Arcese W, Iori AP, Purtill D, Danby R, Sanz GF, Gluckman E, Eapen M. Killer cell immunoglobulin‐like receptor ligand matching and outcomes after unrelated cord blood transplantation acute myeloid leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.04.007. Epub 2016 Apr 16. ncbi.nlm.nih.gov/pubmed/27090957 Gadalla SM, Khincha PP, Katki HA, Giri N, Wong JYY, Spellman S, Yanovski JA, Han JC, De Vivo, Alter BP, Savage SA. The limitations of qPCR telomere length measurement in diagnosing dyskeratosis congenita. Molecular Genetics & Genomic Medicine. 2016 Jul 1; 4(4):475‐479. doi:10.1002/mgg3.220. Epub 2016 Mar 20. PMC4947866. ncbi.nlm.nih.gov/pubmed/27468421 Nakasone H, Sahaf B, Tian L, Wang T, Haagenson MD, Schoenrock K, Perloff S, Ryan CE, Wu F, Spellman SR, Lee SJ, Ritz J, Miklos DB. Presensitization to HY antigens in female donors prior to transplant is not associated with male recipient post‐transplant HY antibody development nor with clinical outcomes. Haematologica. 101(1):e30‐e33. doi:10.3324/ haematol.2015.134551. Epub 2016 Jan 1. PMC4697905. ncbi.nlm.nih.gov/pubmed/26494841

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2015 Jindra PT, Conway SE, Ricklefs SM, Porcella SF, Anzick SL, Haagenson M, Wang T, Spellman S, Milford E, Kraft P, McDermott DH, Abdi R. Analysis of a genetic plymorphism in the costimulatory molecule TNFSF4 with hematopoietic stem cell transplant outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Jan 1; 22(1):27‐36. doi:10.1016/j.bbmt.2015.08.037. Epub 2015 Sep 5. PMC4743880. ncbi.nlm.nih.gov/pubmed/26348892 Petersdorf EW, Malkki M, O'hUigin C, Carrington M, Gooley T, Haagenson MD, Horowitz MM, Spellman SR, Wang T, Stevenson P. High HLA‐DP expression and graft‐versus‐host disease. New England Journal of Medicine. 2015 Aug 13; 373(7):599‐609. doi:10.1056/NEJMoa1500140. Epub 2015 Aug 13. PMC4560117. ncbi.nlm.nih.gov/pubmed/26267621 Verneris MR, Lee SJ, Ahn KW, Wang H‐L, Battiwalla M, Inamoto Y, Fernandez‐Vina MA, Gajewski J, Pidala J, Munker R, Aljurf M, Saber W, Spellman S, Koreth J. HLA mismatch is associated with worse outcomes after unrelated donor reduced‐intensity conditioning hematopoietic cell transplantation: An analysis from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1783‐1789. doi:10.1016/ j.bbmt.2015.05.028. Epub 2015 Jun 6. PMC4568127. ncbi.nlm.nih.gov/pubmed/26055300 Sobecks RM, Wang T, Askar M, Gallagher MM, Haagenson M, Spellman S, Fernandez‐Vina M, Malmberg K‐J, Müller C, Battiwalla M, Gajewski J, Verneris MR, Ringdén O, Marino S, Davies S, Dehn J, Bornhäuser M, Inamoto Y, Woolfrey A, Shaw P, Pollack M, Weisdorf D, Milller J, Hurley C, Lee SJ, Hsu K. Impact of KIR and HLA genotypes on outcomes after reduced‐intensity conditioning hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1589‐1596. doi:10.1016/j.bbmt.2015.05.002. Epub 2015 May 8. PMC4537837. ncbi.nlm.nih.gov/pubmed/25960307 Gadalla SM, Wang T, Haagenson M, Spellman SR, Lee SJ, Williams KM, Wong JY, De Vivo I, Savage SA. Association between donor leukocyte telomere length and survival after unrelated allogeneic hematopoietic cell transplantation for severe aplastic anemia. JAMA: The Journal of the American Medical Association. 2015 Feb 10; 313(6):594‐602. doi:10.1001/jama.2015.7. Epub 2015 Feb 10. PMC4388056. ncbi.nlm.nih.gov/pubmed/25668263 Logan AC, Wang Z, Alimoghaddam K, Wong RM, Lai T, Negrin RS, Grumet C, Logan BR, Zhang M‐J, Spellman SR, Lee SJ, Miklos DB. ABO mismatch is associated with increased nonrelapse mortality after allogeneic hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):746‐754. doi:10.1016/j.bbmt.2014.12.036. Epub 2015 Jan 5. PMC4363312. ncbi.nlm.nih.gov/pubmed/25572032

2014 Kornblit B, Enevold C, Wang T, Spellman S, Haagenson M, Lee SJ, Müller K. Toll‐like receptor polymorphisms in allogeneic hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Feb 1; 21(2):259‐265. doi:10.1016/j.bbmt.2014.09.016. Epub 2014 Nov 20. PMC4297590. ncbi.nlm.nih.gov/pubmed/25464115

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Venstrom JM, Pittari G, Gooley TA, Chewning JH, Spellman S, Haagenson M, Gallagher MM, Malkki M, Petersdorf E, Dupont B, Hsu KC. Donor activating KIR2DS1 in leukemia. New England Journal of Medicine. 2014 Nov 20; 371(21):2042. doi:10.1056/NEJMc1411443. Epub 2014 Nov 20. ncbi.nlm.nih.gov/pubmed/25409391 Broux B, Shamim Z, Wang T, Spellman S, Haagenson M, Stinissen P, Ryder LP, Müller K, Hellings N. The influence of interleukin‐7 receptor α‐chain haplotypes on outcome after allogeneic hematopoietic cell transplantation. International Journal of Immunogenetics. 2014 Dec 1; 41(6):521‐527. doi:10.1111/iji.12158. Epub 2014 Oct 29. PMC4238034. ncbi.nlm.nih.gov/ pubmed/25352021 Petersdorf EW, Gooley TA, Malkki M, Bacigalupo AP, Cesbron A, Du Toit E, Ehninger G, Egeland T, Fischer GF, Gervais T, Haagenson MD, Horowitz MM, Hsu K, Jindra P, Madrigal A, Oudshoorn M, Ringdén O, Schroeder ML, Spellman SR, Tiercy JM, Velardi A, Witt CS, O'Huigin C, Apps R, Carrington M. HLA‐C expression levels define permissible mismatches in hematopoietic cell transplantation. Blood. 2014 Dec 18; 124(26):3996‐4003. doi:10.1182/blood‐2014‐09‐ 599969. Epub 2014 Oct 16. PMC4271183. ncbi.nlm.nih.gov/pubmed/25323824 Pidala J, Lee SJ, Ahn KW, Spellman S, Wang H‐L, Aljurf M, Askar M, Dehn J, Fernandez Viña M, Gratwohl A, Gupta V, Hanna R, Horowitz MM, Hurley CK, Inamoto Y, Kassim AA, Nishihori T, Mueller C, Oudshoorn M, Petersdorf EW, Prasad V, Robinson J, Saber W, Schultz KR, Shaw B, Storek J, Wood WA, Woolfrey AE, Anasetti C. Nonpermissive HLA‐DPB1 mismatch increases mortality after myeloablative unrelated allogeneic hematopoietic cell transplantation. Blood. 2014 Oct 16; 124(16):2596‐2606. doi:10.1182/blood‐2014‐05‐576041. Epub 2014 Aug 26. PMC4199961. ncbi.nlm.nih.gov/pubmed/25161269 Fleischhauer K, Fernandez‐Viña MA, Wang T, Haagenson M, Battiwalla M, Baxter‐Lowe LA, Ciceri F, Dehn J, Gajewski J, Hale GA, Heemskerk MBA, Marino SR, McCarthy PL, Miklos D, Oudshoorn M, Pollack MS, Reddy V, Senitzer D, Shaw BE, Waller EK, Lee SJ, Spellman SR. Risk associations between HLA‐DPB1 T‐cell epitope matching and outcome of unrelated hematopoietic cell transplantation are independent of HLA‐DPA1. Bone Marrow Transplantation. 2014 Sep 1; 49(9):1176‐1183. doi:10.1038/bmt.2014.122. Epub 2014 Jun 23. PMC4154997. ncbi.nlm.nih.gov /pubmed/24955785 Cooley S, Weisdorf DJ, Guethlein LA, Klein JP, Wang T, Marsh SGE, Spellman S, Haagenson MD, Saeturn K, Ladner M, Trachtenberg E, Parham P, Miller JS. Donor killer cell Ig‐like receptor B haplotypes, recipient HLA‐C1, and HLA‐C mismatch enhance the clinical benefit of unrelated transplantation for acute myelogenous leukemia. Journal of . 2014 May 15; 192(10):4592‐4600. doi:10.4049/jimmunol.1302517. Epub 2014 Apr 18. PMC4031316. ncbi.nlm.nih.gov/pubmed/24748496 Gleason MK, Ross JA, Warlick ED, Lund TC, Verneris MR, Wiernik A, Spellman S, Haagenson MD, Lenvik AJ, Litzow MR, Epling‐Burnette PK, Blazar BR, Weiner LM, Weisdorf DJ, Vallera DA, Miller JS. CD16xCD33 bispecific killer cell engager (BiKE) activates NK cells against primary MDS and MDSC CD33+ targets. Blood. 2014 May 8; 123(19):3016‐3026. doi:10.1182/blood‐2013‐ 10‐533398. Epub 2014 Mar 20. PMC4014844. ncbi.nlm.nih.gov/pubmed/24652987

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Sengsayadeth S, Wang T, Lee SJ, Haagenson MD, Spellman S, Fernandez Viña MA, Muller CR, Verneris MR, Savani BN, Jagasia M. Cytotoxic T‐lymphocyte antigen‐4 single nucleotide polymorphisms are not associated with outcomes after unrelated donor transplantation: A Center for International Blood and Marrow Transplant Research analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Jun 1; 20(6):900‐903. doi:10.1016/j.bbmt.2014.03.005. Epub 2014 Mar 14. PMC4034271. ncbi.nlm.nih.gov/pubmed/24631737 Fernandez‐Viña MA, Wang T, Lee SJ, Haagenson M, Aljurf M, Askar M, Battiwalla M, Baxter‐Lowe L‐A, Gajewski J, Jakubowski AA, Marino S, Oudshoorn M, Marsh SGE, Petersdorf EW, Schultz K, Turner EV, Waller EK, Woolfrey A, Umejiego J, Spellman SR, Setterholm M. Identification of a permissible HLA mismatch in hematopoietic stem cell transplantation. Blood. 2014 Feb 20; 123(8):1270‐1278. doi:10.1182/blood‐2013‐10‐532671. Epub 2014 Jan 9. PMC3931192. ncbi.nlm.nih.gov/pubmed/24408320

2013 Eapen M, Klein JP, Ruggeri A, Spellman S, Lee SJ, Anasetti C, Arcese W, Barker JN, Baxter‐Lowe LA, Brown M, Fernandez‐Vina MA, Freeman J, He W, Iori AP, Horowitz MM, Locatelli F, Marino S, Maiers M, Michel G, Sanz GF, Gluckman E, Rocha V. Impact of allele‐level HLA matching on outcomes after myeloablative single unit umbilical cord blood transplantation for hematologic malignancy. Blood. 2014 Jan 2; 123(1):133‐140. doi:10.1182/blood‐2013‐05‐ 506253. Epub 2013 Oct 18. PMC3879902. ncbi.nlm.nih.gov/pubmed/24141369 Pidala J, Wang T, Haagenson M, Spellman SR, Askar M, Battiwalla M, Baxter‐Lowe LA, Bitan M, Fernandez‐Viña M, Gandhi M, Jakubowski AA, Maiers M, Marino SR, Marsh SGE, Oudshoorn M, Palmer J, Prasad VK, Reddy V, Ringden O, Saber W, Santarone S, Schultz KR, Setterholm M, Trachtenberg E, Turner EV, Woolfrey AE, Lee SJ, Anasetti C. Amino acid substitution at peptide‐ binding pockets of HLA class I molecules increases risk of severe acute GVHD and mortality. Blood. 2013 Nov 21; 122(22):3651‐3658. doi:10.1182/blood‐2013‐05‐501510. Epub 2013 Aug 27. PMC3837514. ncbi.nlm.nih.gov/pubmed/23982174 Shamim Z, Spellman S, Haagenson M, Wang T, Lee SJ, Ryder LP, Müller K. Polymorphism in the interleukin‐7 receptor‐alpha and outcome after allogeneic hematopoietic cell transplantation with matched unrelated donor. Scandinavian Journal of Immunology. 2013 Aug 1; 78(2):214‐220. doi:10.1111/sji.12077. Epub 2013 Jul 21. PMC3982186. ncbi.nlm.nih.gov/ pubmed/23692589 Morishima Y, Kawase T, Malkki M, Morishima S, Spellman S, Kashiwase K, Kato S, Cesbron A, Tiercy J‐M, Senitzer D, Velardi A, Petersdorf EW. Significance of ethnicity in the risk of acute graft‐versus‐host disease and leukemia relapse after unrelated donor hematopoietic stem cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Aug 1; 19(8):1197‐1203. doi:10.1016/ j.bbmt.2013.05.020. Epub 2013 Jun 6. PMC3972059. ncbi.nlm.nih.gov/pubmed/23747601 Hurley CK, Woolfrey A, Wang T, Haagenson M, Umejiego J, Aljurf M, Askar M, Battiwalla M, Dehn J, Horan J, Oudshoorn M, Pidala J, Saber W, Turner V, Lee SJ, Spellman SR. The impact of HLA unidirectional mismatches on the outcome of myeloablative hematopoietic stem cell transplantation with unrelated donors. Blood. 2013 Jun 6; 121(23):4800‐4806. doi:10.1182/ blood‐2013‐01‐480343. Epub 2013 May 1. PMC3674677. ncbi.nlm.nih.gov/pubmed/23637130

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Fernández‐Viña MA, Klein JP, Haagenson M, Spellman SR, Anasetti C, Noreen H, Baxter‐Lowe LA, Cano P, Flomenberg N, Confer DL, Horowitz MM, Oudshoorn M, Petersdorf EW, Setterholm M, Champlin R, Lee SJ, de Lima M. Multiple mismatches at the low expression HLA loci DP, DQ, and DRB3/4/5 associate with adverse outcomes in hematopoietic stem cell transplantation. Blood. 2013 May 30; 121(22):4603‐4610. doi:10.1182/blood‐2013‐02‐ 481945. Epub 2013 Apr 17. PMC3668493. ncbi.nlm.nih.gov/pubmed/23596045 Dobbelstein C, Ahn KW, Haagenson M, Hale GA, van Rood JJ, Miklos D, Waller EK, Spellman SR, Fernandez‐Vina M, Ganser A, Aljurf M, Bornhaeuser M, Gupta V, Marino SR, Pollack MS, Reddy V, Eder M, Lee SJ. Birth order and transplantation outcome in HLA‐identical sibling stem cell transplantation: An analysis on behalf of the Center for International Blood and Marrow Transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 May 1; 19(5):741‐745. doi:10.1016/j.bbmt. 2013.01.020. Epub 2013 Feb 1. PMC3864760. ncbi.nlm.nih.gov/pubmed/23380341 Petersdorf EW, Malkki M, Horowitz MM, Spellman SR, Haagenson MD, Wang T. Mapping MHC haplotype effects in unrelated donor hematopoietic cell transplantation. Blood. 2013 Mar 7; 121(10):1896‐1905. doi:10.1182/blood‐2012‐11‐465161. Epub 2013 Jan 10. PMC3591807. ncbi.nlm.nih.gov/pubmed/23305741

2012 Horan J, Wang T, Haagenson M, Spellman SR, Dehn J, Eapen M, Frangoul H, Gupta V, Hale GA, Hurley CK, Marino S, Oudshoorn M, Reddy V, Shaw P, Lee SJ, Woolfrey A. Evaluation of HLA matching in unrelated hematopoietic stem cell transplantation for nonmalignant disorders. Blood. 2012 Oct 4; 120(14):2918‐2924. doi:10.1182/blood‐2012‐03‐417758. Epub 2012 Jul 24. PMC3466972. ncbi.nlm.nih.gov/pubmed/22829628 Rocha V, Spellman S, Zhang M‐J, Ruggeri A, Purtill D, Brady C, Baxter‐Lowe LA, Baudoux E, Bergamaschi P, Chow R, Freed B, Koegler G, Kurtzberg J, Larghero J, Lecchi L, Nagler A, Navarrette C, Prasad V, Pouthier F, Price T, Ratanatharathorn V, van Rood JJ, Horowitz MM, Gluckman E, Eapen M. Effect of HLA‐matching recipients to donor noninherited maternal antigens on outcomes after mismatched umbilical cord blood transplantation for hematologic malignancy. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2012 Dec 1; 18(12):1890‐1896. doi:10.1016/j.bbmt.2012.07.010. Epub 2012 Jul 16. PMC3826155. ncbi.nlm.nih.gov/pubmed/ 22814031 Petersdorf EW, Malkki M, Gooley TA, Spellman SR, Haagenson MD, Horowitz MM, Wang T. MHC‐ resident variation affects risks after unrelated donor hematopoietic cell transplantation. Science Translational Medicine. 2012 Jul 25; 4(144):144ra101. doi:10.1126/scitranslmed. 3003974. PMC3633562. ncbi.nlm.nih.gov/pubmed/22837536

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9.3 Current Studies

R02‐40 / R03‐63d Title: Acquisition of natural killer cell receptors in recipients of unrelated transplant PIs: Jeffrey Miller (University of Minnesota Blood and Marrow Transplant Program) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with University of Minnesota

R04‐74d Title: Functional significance of killer cell immunoglobulin‐like receptor genes in HLA‐ matched and mismatched unrelated HCT PIs: Katharine Hsu (Memorial Sloan Kettering Cancer Center) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

IB06‐05 Title: Use of high‐resolution HLA data from the NMDP for the International Histocompatibility Working Group in HCT PIs: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

IB07‐04 Title: Employing advanced bioinformatics methods for predicting peptide specificities of HLA molecules in the characterization of permissible mismatches in hematopoietic cell transfer PIs: Soren Buus (University of Copenhagen) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

IB09‐01 Title: Clinical importance of minor histocompatibility complex haplotypes in umbilical cord blood transplantation PIs: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

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IB09‐03 Title: Clinical relevance of cytokine/immune response gene polymorphisms in umbilical cord blood transplantation PIs: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

IB09‐05 Title: Identification of functional single nucleotide polymorphisms in umbilical cord blood transplantation PIs: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

IB09‐06 / RT09‐04b Title: Genetic susceptibility to transplant‐related mortality after unrelated donor stem cell transplant PIs: Theresa Hahn (Roswell Park Cancer Institute) Status: Manuscript Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

IB09‐07 Title: Clinical significance of genome‐wide variation in unrelated HCT PIs: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

IB10‐01d Title: Flow Cytometry using FISH techniques in a Severe Aplastic Anemia population PIs: Shahinaz Gadalla (National Cancer Institute) Sharon Savage (National Cancer Institute) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with National Cancer Institute

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IB11‐01a Title: Analysis of the NIMA effect on the outcome of unrelated PBSC/BM transplantation PIs: Gerhard Ehninger (Universitaetsklinikum Dresden) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with Dynamic Kernel Module Support (DKMS)

IB11‐01b Title: Analysis of the IPA effect on the outcome of unrelated PBSC/BM transplantation PIs: Gerhard Ehninger (Universitaetsklinikum Dresden) Status: Data Collection / Data File Preparation (as of July 1, 2017) Data File Preparation (expected by June 30, 2018) * Collaborative study with DKMS

IB11‐08 Title: Synergism between minor and major histocompatibility antigens PIs: Eric Spierings (University Medical Center Utrecht) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

IB12‐02c Title: Prospective Assignment of HLA‐DPB1 T Cell Epitope Group Mismatches by Functional Distance Scores is Significantly Associated with Outcome of 8/8 Matched Unrelated Donor HCT PIs: Katharina Fleischhauer (Universitatsklinikum Essen KMT) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IB13‐08 Title: Short and long term survival assessment of post‐HSCT transplantation using predictive modeling on a Bayesian network framework PIs: Reza Abdi (Dana Farber Cancer Institute) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018) * Collaborative study with DFCI

IB13‐09 Title: The development of machine learning based classifiers to define the alloreactivity of HLA mismatches in unrelated donor hematopoietic stem cell transplantation PIs: Yoram Louzoun (Bar‐Ilan University) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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IB14‐01 Title: Impact of human leukocyte antigen haplotypes on outcomes of allogeneic transplantation for B‐cell non‐Hodgkin lymphomas PIs: Basem William (Ohio State Medical Center, James Cancer Center) Marcos de Lima (Seidman Cancer Center‐University Hospitals Cleveland Medical Center) Marcelo Fernandez‐Viña (Stanford Health Care) Brian Hill (Cleveland Clinic Foundation) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IB14‐03a Title: The prognostic impact of levels of CXC chemokine ligands on post hematopoietic cell transplantation outcomes in patients with myelodysplastic syndromes PIs: Wael Saber (CIBMTR ‐ Milwaukee) Binod Dhakal (Medical College of Wisconsin) Status: Analysis (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

IB14‐04 Title: Assessing the similarity of the T cell receptor repertoire in allogeneic hematopoietic stem cell recipients with the same single human leukocyte mismatches PIs: Everett Meyer (Stanford Health Care) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IB14‐05 Title: mtDNA haplotypes and unrelated donor transplant outcomes PIs: Michael Verneris (University of Colorado ‐ Children's Hospital) Logan Spector (University of Minnesota) Status: Analysis (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with University of Minnesota

IB14‐06 Title: Donor‐specific anit HLA antibodies, allele and antigen level HLA mismatches in the outcomes of transplantation of non‐malignant diseases with unrelated donors PIs: Marcelo Fernandez‐ Viña (Stanford Health Care) Ann Woolfrey (Fred Hutchinson Cancer Research Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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IB14‐07 Title: Indirectly recognizable HLA epitopes (PIRCHES): A retrospective validation study on the role of indirect recognition of mismatched HLA in hematopoietic stem cell transplantation outcome PIs: Eric Spierings (University Medical Center Utrecht) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with International Histocompatibility Working Group

IB14‐08 Title: Development and validation of a clinical unrelated donor selection score PIs: Bronwen Shaw (CIBMTR ‐ Milwaukee) Stephanie Lee (Fred Hutchinson Cancer Research Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IB15‐01 Title: The Impact of Single Nucleotide Gene Polymorphisms (SNP) in the Gamma Block of the Major Histocompatibility Complex (MHC) on Unrelated Donor Hematopoietic Cell Transplants (HCT) For Hematological Malignancies PIs: Medhat Askar (Baylor University Medical Center) Ronald Sobecks (Cleveland Clinic Foundation) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

IB15‐02 Title: Natural killer cell genomics and outcomes after allogeneic transplantation for chronic lymphocytic leukemia PIs: Veronika Bachanova (University of Minnesota Blood and Marrow Transplant Program) Jeffrey Miller (University of Minnesota Blood and Marrow Transplant Program) Daniel Weisdorf (University of Minnesota Blood and Marrow Transplant Program) Sarah Cooley (University of Minnesota Blood and Marrow Transplant Program) Status: Analysis (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with University of Minnesota

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IB15‐03 Title: Killer Immunoglobulin Receptor (KIR) Gene Content and Pediatric Acute Leukemia Transplant Outcomes PIs: Michael Verneris (University of Colorado ‐ Children's Hospital) Jeffrey Miller (University of Minnesota Blood and Marrow Transplant Program) Sarah Cooley (University of Minnesota Blood and Marrow Transplant Program) Status: Protocol Development (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with University of Minnesota

IB15‐04 Title: Clinical outcomes among hematopoietic stem cell transplant recipients as a function of socioeconomic status and related transcriptome differences PIs: Jennifer Knight (Medical College of Wisconsin) J. Douglas Rizzo (CIBMTR ‐ Milwaukee) Steve Cole (UCLA School of Medicine) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with Medical College of Wisconsin (MCW)

IB15‐05 Title: Secondary Findings in Exome Sequencing Data PIs: Sharon Savage (National Cancer Institute) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with National Cancer Institute (NCI)

IB15‐06a Title: Donor Telomere Length and Outcomes After Hematopoietic Stem Cell Transplantation for Acute Leukemia PIs: Shahinaz Gadalla (National Cancer Institute) David Loftus (Telomere Diagnostics) Evangelos Hytopoulos (Organization/Center information not available) Sharon Savage (National Cancer Institute) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018) * Collaborative study with NCI and Telomere Diagnostics, Inc.

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IB15‐06b Title: Using Telomatch Testing on Donor Telomere Length and Outcomes After Hematopoietic Stem Cell Transplantation for Acute Leukemia PIs: David Loftus (Telomere Diagnostics) Evangelos Hytopoulos (Organization/Center information not available) Ljubomir Buturovic (Telomere Diagnostics) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with Telomere Diagnositics, Inc.

IB15‐07 Title: Functional genetic variants of the ST2 gene in pairs of recipient and donors for risk stratification of GVHD and TRM outcomes PIs: Sophie Paczesny (Indiana University Hospital/Riley Hospital for Children) Stephen Spellman (CIBMTR ‐ Minneapolis) Jamie Renbarger (Indiana University School of Medicine) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IB16‐01 Title: The role of HLA‐E compatibility in the prognosis of acute leukemia patients undergoing 10/10 HLA matched unrelated HSCT PIs: Chrysanthi Tsamadou (IKT Ulm) Daniel Furst (UCLA Health) Joannis Mytilineos (IKT Ulm) Status: Sample Typing (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

IB16‐02 Title: Use of HLA structure and function parameters to understand the relationship between HLA disparity and transplant outcomes PIs: Lee Ann Baxter Lowe (Children's Hospital of Los Angeles) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

IB16‐03 Title: Role of recipient and donor genetic polymorphisms in interferon lambda 4 (INFL4) on outcomes after unrelated allogeneic cell transplant PIs: Shahinaz Gadalla (National Cancer Institute) Ludmila Prokunina‐Olsson (National Cancer Institute) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with NCI

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IB17‐01 Title: The impact of HLA‐DPB1 level of expression on clinical outcomes of transplantation PIs: Medhat Askar (Baylor University Medical Center) Marcelo Fernandez‐Viña (Stanford Health Care) Status: Deferred (as of July 1, 2017) Deferred (expected by June 30, 2018)

IB17‐02 Title: Donor‐recipient NK cell determinants associated with survival in JMML after hematopoietic stem cell transplantation PIs: Dean Lee (Ohio State Medical Center, James Cancer Center) Hemalatha Rangarahan (Nationwide Children's Hospital) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

IB17‐03 Title: Identification of genomic markers of post hematopoietic cell transplantation (HCT) outcomes in patients with myelofibrosis: A pilot study PIs: Wael Saber (CIBMTR ‐ Milwaukee) Shahinaz Gadalla (National Cancer Institute) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018) * Collaborative study with NCI

IB17‐04 Title: Epigenetic profiling of unrelated donor‐recipient pairs to improve donor selection during HCT transplants PIs: Stephan Beck (University College London) Karl Peggs (University College London) Vardhman Rakyan (Barts and The London School of Medicine, Blizard Institute) Amy Webster (University College London) Status: Protocol Development (as of July 1, 2017) Analysis (expected by June 30, 2018)

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10.0 INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE

10.1 Leadership

Chair: Jeffery Auletta, MD, Nationwide Children’s Hospital Email: [email protected] Chair: Caroline Lindemans, MD PhD, University Medical Center Utrecht, Pediatrics Email: [email protected] Chair: Krishna Komanduri, MD, University of Miami Email: [email protected] Scientific Director: Marcie Riches, MD, MS, University of North Carolina Hospitals Email: [email protected] Statistical Director: Soyoung Kim, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Min Chen, MS, CIBMTR Milwaukee Email: [email protected]

10.2 Recent Publications

2016 Maziarz RT, Brazauskas R, Chen M, McLeod AA, Martino R, Wingard JR, Aljurf M, Battiwalla M, Dvorak CC, Geroge B, Guinan EC, Hale GA, Lazarus HM, Lee J‐W, Liesveld JL, Ramanathan M, Reddy V, Savani BN, Smith FO, Strasfeld L, Taplitz RA, Ustun C, Boeckh MJ, Gea‐Banacloche J, Lindemans CA, Auletta JJ, Riches ML. Pre‐existing invasive fungal infection is not a contraindication for allogeneic HSCT for patients with hematologic malignancies: A CIBMTR study. Bone Marrow Transplantation. 2017 Feb 1; 52(2):270‐278. doi:10.1038/ bmt.2016.259. Epub 2016 Dec 19. PMC5358320. ncbi.nlm.nih.gov/pubmed/27991895 Ballen K, Ahn KW, Chen M, Abdel‐Azim H, Ahmed I, Aljurf M, Antin J, Bhatt AS, Boeckh M, Chen G, Dandoy C, George B, Laughlin M, Lazarus HM, MacMillan ML, Margolis DA, Marks DI, Norkin M, Rosenthal J, Saad A, Savani B, Schouten HC, Storek J, Szabolcs P, Ustun C, Verneris MR, Waller EK, Weisdorf DJ, Williams KM, Wingard JR, Wirk B, Wolfs T, Young JH, Auletta J, Komanduri KV, Lindemans C, Riches ML. Infection rates among acute leukemia patients receiving alternative donor hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.06.012. Epub 2016 Jun 22. ncbi.nlm.nih.gov/pubmed/27343716 Ramanathan M, Teira P, Battiwalla M, Barrett J, Ahn KW, Chen M, Green J, Laughlin M, Lazarus HM, Marks D, Saad A, Seftel M, Saber W, Savani BN, Waller E, Wingard J, Auletta JJ, Lindemans CA, Boeckh M, Riches ML. Impact of early CMV reactivation in cord blood stem cell recipients in the current era. Bone Marrow Transplantation. doi:10.1038/bmt.2016.89. Epub 2016 Apr 4. ncbi.nlm.nih.gov/pubmed/27042847

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Teira P, Battiwalla M, Ramanathan M, Barrett AJ, Ahn KW, Chen M, Green JS, Saad A, Antin JH, Savani BN, Lazarus HM, Seftel M, Saber W, Marks D, Aljurf M, Norkin M, Wingard JR, Lindemans CA, Boeckh M, Riches ML, Auletta JJ. Early cytomegalovirus reactivation remains associated with increased transplant related mortality in the current era: A CIBMTR analysis. Blood. 2016 May 19; 127(20):2427‐2438. doi:10.1182/blood‐2015‐11‐679639. Epub 2016 Feb 16. ncbi.nlm.nih.gov/pubmed/26884374 Williams KM, Ahn KW, Chen M, Aljurf MD, Agwu AL, Chen AR, Walsh TJ, Szabolcs P, Boeckh MJ, Auletta JJ, Lindemans CA, Zanis‐Neto J, Malvezzi M, Lister J, de Toledo Codina JS, Sackey K, Chakrabarty JLH, Ljungman P, Wingard JR, Seftel MD, Seo S, Hale GA, Wirk B, Smith MS, Savani BN, Lazarus HM, Marks DI, Ustun C, Abdel‐Azim H, Dvorak CC, Szer J, Storek J, Yong A, Riches MR. The incidence, mortality and timing of Pneumocystis jiroveci pneumonia after hematopoietic cell transplantation: A CIBMTR analysis. Bone Marrow Transplantation. 2016 Apr 1; 51(4):573‐580. doi:10.1038/bmt.2015.316. Epub 2016 Jan 4. PMC4823157. ncbi.nlm.nih.gov/pubmed/26726945

2015 Riches ML, Trifilio S, Chen M, Ahn KW, Langston A, Lazarus HM, Marks DI, Martino R, Maziarz RT, Papanicolou GA, Wingard JR, Young J‐AH, Bennett CL. Risk factors and impact of non‐ Aspergillus mold infections following allogeneic HCT: A CIBMTR infection and immune reconstitution analysis. Bone Marrow Transplantation. 2016 Feb 1; doi:10.1038/bmt.2015. 318. Epub 2015 Nov 2. ncbi.nlm.nih.gov/pubmed/26838373 Riches ML, Trifilio S, Chen M, Ahn KW, Langston A, Lazarus HM, Marks DI, Martino R, Maziarz RT, Papanicolou GA, Wingard JR, Young J‐AH, Bennett CL. Risk factors and impact of non‐ Aspergillus mold infections following allogeneic HCT: A CIBMTR infection and immune reconstitution analysis. Bone Marrow Transplantation. 2016 Feb 1; 51(2):277‐282. doi:10. 1038/bmt.2015.263. Epub 2015 Nov 2. PMC4740251. ncbi.nlm.nih.gov/pubmed/26524262

10.3 Current Studies

IN07‐01/IN11‐01a Title: VEP vs LEP vs both vs No BSI in patients undergoing allogeneic HCT PIs: Celalettin Ustun (University of Minnesota Blood and Marrow Transplant Program) Jo‐Anne Young (University of Minnesota Blood and Marrow Transplant Program) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IN07‐01/IN11‐01b Title: VRE vs other vs no BSI in patients undergoing allogeneic HCT PIs: Genovefa A Papanicolaou (Memorial Sloan‐Kettering Cancer Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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IN13‐01 Title: Bacterial and fungal infections in patients undergoing allogeneic hematopoietic cell transplantation following nonmyeloablative and myeloablative regimens PIs: Celalettin Ustun (University of Minnesota Blood and Marrow Transplant Program) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IN14‐01 Title: Post allogeneic hematopoietic transplant Ebstein Barr Virus related lymphproliferative disorder following conditioning with antithymocyte globulin or alemtuzumab PIs: Rammurti T. Kamble (Baylor College of Medicine Center for Cell and ) Seema Naik (Penn State Hershey Medical Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IN16‐01 Title: Viral encephalitis in hematopoietic stem cell transplant recipients, 2007‐2013 PIs: Mhaeen Abidi (University of Colorado Hospital) Parameswaran Hari (CIBMTR ‐ Milwaukee) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

IN16‐02 Title: Determination of the burden of mucosal barrier injury‐laboratory confirmed bloodstream infections in the first 100 days after stem cell transplant PIs: Christopher Dandoy (Cincinnati Children's Hospital Medical Center) Paulina Daniels (Cincinnati Children's Hospital Medical Center) Status: Protocol Development (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

IN17‐01 Title: Incidence and Outcomes of individuals with and without viral infections in recipients of haploidentical versus other allogeneic hematopoietic stem cell transplantation for patients with hematologic malignancies PIs: Rizwan Romee (Barnes Jewish Hospital) Anurag Singh (University of Kansas Cancer Center) Randy Allison Taplitz (University of California, San Diego Medical Center) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

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11.0 LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE

11.1 Leadership

Chair: Bipin Savani, MD, Vanderbilt University Medical Center Email: [email protected] Chair: Mary Flowers, MD, Fred Hutchinson Cancer Research Center Email: [email protected] Chair: Minoo Battiwalla, MD, MS, National Heart Lung and Blood Institute ‐ NIH Email: [email protected] Scientific Director: Bronwen Shaw, MBChB, MRCP, PhD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Ruta Brazauskas, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Heather Millard, MPH, CIBMTR Milwaukee Email: [email protected] CAC Representative: Hilary Hall, Dana Farber Cancer Institute CAC Representative: Jim Omel, San Jose, CA

11.2 Recent Publications

2017 Vrooman LM, Millard HR, Brazauskas R, Majhail NS, Battiwalla M, Flowers ME, Savani BN, Akpek G, Aljurf M, Bajwa R, Baker KS, Beitinjaneh A, Bitan M, Buchbinder D, Chow E, Dietz AC, Diller L, Gale RP, Hashmi SK, Hayashi RJ, Hematti P, Kamble RT, Kasow 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. Survival and late effects after allogeneic hematopoietic cell transplantation for hematologic malignancy at less than three years of age. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2017.04.017. Epub 2017 Apr 28. ncbi.nlm.nih.gov/pubmed/28461213 Gabriel M, Shaw B, Brazauskas R, Chen M, Margolis DA, Sengelov H, Dahlberg A, Ahmed IA, Delgado D, Lazarus HM, Gibson B, Myers KC, Kamble RT, Abdel‐Mageed A, Li C‐K, Flowers MED, Battiwalla M, Savani BN, Majhail N, Shaw P. Risk factors for subsequent central nervous system tumors in pediatric allogeneic hematopoietic cell transplant: A study from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Aug 1; 23(8):1320‐1326. doi:10.1016/j.bbmt.2017.04.004. Epub 2017 Apr 12. PMC5518678. ncbi.nlm.nih.gov/pubmed/28411175

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2016 Hashmi SK, Bredeson C, Duarte RF, Farnia S, Ferrey S, Fitzhugh C, Flowers MED, Gajewski J, Gastineau D, Greenwald M, Jagasia M, Martin P, Rizzo JD, Schmit‐Pokorny K, Majhail NS. National Institutes of Health Blood and Marrow Transplant Late Effects Initiative: The Healthcare Delivery Working Group Report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 May 1; 23(5):717‐725. doi:10.1016/j.bbmt.2016.09.025. Epub 2016 Oct 3. PMC5378687. ncbi.nlm.nih.gov/ pubmed/27713091 Morton LM, Saber W, Baker KS, Barrett AJ, Bhatia S, Engels EA, Gadalla SM, Kleiner DE, Pavletic S, Burns LJ. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Subsequent Neoplasms Working Group Report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Mar 1; 23(3):367‐378. doi:10.1016/j.bbmt.2016.09.005. Epub 2016 Sep 12. PMC5285307. ncbi.nlm.nih.gov/pubmed/27634019 Shaw BE, Hahn T, Martin PJ, Mitchell SA, Petersdorf EW, Armstrong GT, Shelburne N, Storer BE, Bhatia S. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Research Methodology and Study Design Working Group report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jan 1; 23(1):10‐23. doi:10.1016/j.bbmt.2016.08.018. Epub 2016 Aug 30. PMC5182148. ncbi.nlm.nih.gov/pubmed/27590102 DeFilipp Z, Duarte RF, Snowden JA, Majhail NS, Greenfield DM, Miranda JL, Arat M, Baker KS, Burns LJ, Duncan CN, Gilleece M, Hale GA, Hamadani M, Hamilton BK, Hogan WJ, Hsu JW, Inamoto Y, Kamble RT, Lupo‐Stanghellini MT, Malone AK, McCarthy P, Mohty M, Norkin M, Paplham P, Ramanathan M, Richart JM, Salooja N, Schouten HC, Schoemans H, Seber A, Steinberg A, Wirk BM, Wood WA, Battiwalla M, Flowers MED, Savani BN, Shaw BE. Metabolic syndrome and cardiovascular disease following hematopoietic cell transplantation: Screening and preventive practice recommendations from CIBMTR and EBMT. Bone Marrow Transplantation. 2017 Feb 1; 52(2):173‐182. doi:10.1038/bmt.2016.203. Epub 2016 Aug 22. PMC5288134. ncbi.nlm.nih.gov/pubmed/27548466 DeFilipp Z, Duarte RF, Snowden JA, Majhail NS, Greenfield DM, Miranda JL, Arat M, Baker KS, Burns LJ, Duncan CN, Gilleece M, Hale GA, Hamadani M, Hamilton BK, Hogan WJ, Hsu JW, Inamoto Y, Kamble RT, Lupo‐Stanghellini MT, Malone AK, McCarthy P, Mohty M, Norkin M, Paplham P, Ramanathan M, Richart JM, Salooja N, Schouten HC, Schoemans H, Seber A, Steinberg A, Wirk BM, Wood WA, Battiwalla M, Flowers MED, Savani BN, Shaw BE. Metabolic syndrome and cardiovascular disease following hematopoietic cell transplantation: Screening and preventive practice recommendations from CIBMTR and EBMT. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.05.007. Epub 2016 May 14. ncbi.nlm.nih.gov/ pubmed/27184625 Shaw BE, Lee SJ, Horowitz MM, Wood WA, Rizzo JD, Flynn KE. Can we agree on patient‐ reported outcome measures for assessing hematopoietic cell transplantation patients? A study from the CIBMTR and BMT CTN. Bone Marrow Transplantation. 2016 Sep 1; 51(9):1173‐ 1179. doi:10.1038/bmt.2016.113. Epub 2016 May 9. ncbi.nlm.nih.gov/pubmed/27159181

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Saber W, Zhang M‐J, Steinert P, Chen M, Horowitz MM. The impact of palifermin use on hematopoietic cell transplant outcomes in children. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug 1; 22(8):1460‐1466. doi:10.1016/j.bbmt.2016.04.008. Epub 2016 May 7. PMC4949099. ncbi.nlm.nih.gov/pubmed/27090960 Li Z, Rubinstein SM, Thota R, Savani M, Brissot E, Shaw BE, Majhail NS, Mohty M, Savani BN. Immune‐mediated complications after hematopoietic stem cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug 1; 22(8):1368‐1375. doi:10.1016/j.bbmt.2016.04.005. Epub 2016 Apr 14. ncbi.nlm.nih.gov/pubmed/27095688

2015 Ehrhardt MJ, Brazauskas R, He W, Rizzo JD, Shaw BE. Survival of patients who develop solid tumors following hematopoietic stem cell transplantation. Bone Marrow Transplantation. 2016 Jan 1; 51(1):83‐88. doi:10.1038/bmt.2015.203. Epub 2015 Sep 14. PMC4570237. ncbi.nlm.nih.gov/pubmed/26367222 Inamoto Y, Shah NN, Savani BN, Shaw BE, Abraham AA, Ahmed IA, Akpek G, Atsuta Y, Baker KS, Basak GW, Bitan M, DeFilipp Z, Gregory TK, Greinix HT, Hamadani M, Hamilton BK, Hayashi RJ, Jacobsohn DA, Kamble RT, Kasow KA, Khera N, Lazarus HM, Malone AK, Lupo‐Stanghellini MT, Margossian SP, Muffly LS, Norkin M, Ramanathan M, Salooja N, Schoemans H, Wingard JR, Wirk B, Wood WA, Yong A, Duncan CN, Flowers MED, Majhail NS. Secondary solid cancer screening following hematopoietic cell transplantation. Bone Marrow Transplantation. 2015 Aug 1; 50(8):1013‐1023. doi:10.1038/bmt.2015.63. Epub 2015 Mar 30. ncbi.nlm.nih.gov/pubmed/ 25822223

2014 Duncan CN, Majhail NS, Brazauskas R, Wang Z, Cahn J‐Y, Frangoul HA, Hayashi RJ, Hsu JW, Kamble RT, Kasow KA, Khera N, Lazarus HM, Loren AW, Marks DI, Maziarz RT, Mehta P, Myers KC, Norkin M, Pidala JA, Porter DL, Reddy V, Saber W, Savani BN, Schouten HC, Steinberg A, Wall DA, Warwick AB, Wood WA, Yu LC, Jacobsohn DA, Sorror ML. Long‐term survival and late effects among one‐year survivors of second allogeneic hematopoietic cell transplantation for relapsed acute leukemia and myelodysplastic syndromes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jan 1; 21(1):151‐158. doi:10.1016/j.bbmt.2014.10.006. Epub 2014 Oct 12. PMC4272862. ncbi.nlm.nih.gov/pubmed/25316109

Ringdén O, Brazauskas R, Wang Z, Ahmed I, Atsuta Y, Buchbinder D, Burns LJ, Cahn J‐Y, Duncan C, Hale GA, Halter J, Hayashi RJ, Hsu JW, Jacobsohn DA, Kamble RT, Kamani NR, Kasow KA, Khera N, Lazarus HM, Loren AW, Marks DI, Myers KC, Ramanathan M, Saber W, Savani BN, Schouten HC, Socie G, Sorror ML, Steinberg A, Popat U, Wingard JR, Mattsson J, Majhail NS. Second solid cancers after allogeneic hematopoietic cell transplantation using reduced‐intensity conditioning. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1777‐1784. doi:10.1016/j.bbmt.2014. 07.009. Epub 2014 Jul 17. PMC4194257. ncbi.nlm.nih.gov/pubmed/25042734

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Majhail NS, Rizzo JD, Lee SJ, Aljurf M, Atsuta Y, Bonfim C, Burns LJ, Chaudhri N, Davies S, Okamoto S, Seber A, Socie G, Szer J, Van Lint MT, Wingard JR, Tichelli A. Recommended screening and preventive practices for long‐term survivors after hematopoietic cell transplantation. Rinsho Ketsueki. 2014 Jun 1; 55(6):607‐632. doi:10.11406/rinketsu.55.607. Epub 2014 Jun 27. ncbi.nlm.nih.gov/pubmed/22446607 Joshi S, Savani BN, Chow EJ, Gilleece MH, Halter J, Jacobsohn DA, Pidala J, Quinn GP, Cahn J‐Y, Jakubowski AA, Kamani NR, Lazarus HM, Rizzo JD, Schouten HC, Socie G, Stratton P, Sorror ML, Warwick AB, Wingard JR, Loren AW, Majhail NS. Clinical guide to fertility preservation in hematopoietic cell transplant recipients. Bone Marrow Transplantation. 2014 Apr 1; 49(4):477‐484. doi:10.1038/bmt.2013.211. Epub 2014 Jan 13. PMC4071767. ncbi.nlm.nih.gov/ pubmed/24419521 Li X, Brazauskas R, Wang Z, Al‐Seraihy A, Baker KS, Cahn J‐Y, Frangoul HA, Gajewski JL, Hale GA, Hsu JW, Kamble RT, Lazarus HM, Marks DI, Maziarz RT, Savani BN, Shah AJ, Shah N, Sorror ML, Wood WA, Majhail NS. Avascular necrosis of bone after allogeneic hematopoietic cell transplantation in children and adolescents. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Apr 1; 20(4):587‐ 592. doi:10.1016/j.bbmt.2013.12.567. Epub 2014 Jan 2. PMC3959243. ncbi.nlm.nih.gov/ pubmed/24388803

2013 Loren AW, Brazauskas R, Chow EJ, Gilleece M, Halter J, Jacobsohn DA, Joshi S, Pidala J, Quinn GP, Wang Z, Apperley JF, Burns LJ, Hale GA, Hayes‐Lattin BM, Kamble R, Lazarus H, McCarthy PL, Reddy V, Warwick AB, Bolwell BJ, Duncan C, Socie G, Sorror ML, Wingard JR, Majhail NS. Physician perceptions and practice patterns regarding fertility preservation in hematopoietic cell transplant recipients. Bone Marrow Transplantation. 2013 Aug 1; 48(8):1091‐1097. doi:10.1038/bmt.2013.13. Epub 2013 Feb 18. PMC3914209. ncbi.nlm.nih.gov/ pubmed/23419436

2012 Danner‐Koptik KE, Majhail NS, Brazauskas R, Wang Z, Buchbinder D, Cahn J‐Y, Dilley KJ, Frangoul HA, Gross TG, Hale GA, Hayashi RJ, Hijiya N, Kamble RT, Lazarus HM, Marks DI, Reddy V, Savani BN, Warwick AB, Wingard JR, Wood WA, Sorror ML, Jacobsohn DA. Second malignancies after autologous hematopoietic cell transplantation in children. Bone Marrow Transplantation. 2013 Mar 1; 48(3):363‐368. doi:10.1038/bmt.2012.166. Epub 2012 Sep 10. PMC3525761. ncbi.nlm.nih.gov/pubmed/22964594

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Buchbinder D, Nugent DJ, Brazauskas R, Wang Z, Aljurf MD, Cairo MS, Chow R, Duncan C, Eldjerou LK, Gupta V, Hale GA, Halter J, Hayes‐Lattin BM, Hsu JW, Jacobsohn DA, Kamble RT, Kasow KA, Lazarus HM, Mehta P, Myers KC, Parsons SK, Passweg JR, Pidala J, Reddy V, Sales‐ Bonfim CM, Savani BN, Seber A, Sorror ML, Steinberg A, Wood WA, Wall DA, Winiarski JH, Yu LC, Majhail NS. Late effects in hematopoietic cell transplant recipients with acquired severe aplastic anemia: A report from the Late Effects Working Committee of the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2012 Dec 1; 18(12):1776‐1784. doi:10.1016/j.bbmt.2012.06.018. Epub 2012 Jul 31. PMC3496823. ncbi.nlm.nih.gov/pubmed/22863842

11.3 Current Studies

SC09‐05a Title: SCTOD assessment of Quality of Life data PIs: J. Douglas Rizzo (CIBMTR ‐ Milwaukee) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

LE12‐03 Title: Solid transplantation after hematopoietic cell transplantation PIs: Meera Gupta (Abramson Cancer Center University of Pennsylvania Medical Center) Peter Abt (Abramson Cancer Center University of Pennsylvania Medical Center) Matthew Levine (Abramson Cancer Center University of Pennsylvania Medical Center) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with United Network for Organ Sharing (UNOS)

LE13‐01 Title: Late cardiovascular morbidity and mortality following pediatric allogeneic HSCT PIs: Christine Duncan (Dana Farber Cancer Institute) Scott Baker (Fred Hutchinson Cancer Research Center) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

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LE13‐02 Title: Risk factors for melanoma following allogeneic hematopoietic stem cell transplantation PIs: Megan Herr (National Cancer Institute) Lindsay Morton (National Cancer Institute) Eric Engels (National Cancer Institute) Margaret Tucker (National Cancer Institute) Rochelle Curtis (NCI Experimental Transplantation and Immunology Branch) Ruth Pfeiffer (National Cancer Institute) David A Jacobson (Children’s National Medical Center) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018)

LE14‐01 Title: Risks and outcomes of new myeloid cancers after autologous hematopoietic cell transplantation PIs: Shahrukh Hashmi (King Faisal Specialist Hospital & Research Center) Robert Dean (Cleveland Clinic Foundation) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

LE15‐01 Title: Non‐relapse mortality and late effects among survivors of autologous hematopoietic cell transplant for Hodgkin and aggressive non‐Hodgkin lymphoma PIs: Regina Myers (Children's Hospital of Philadelphia) Brian Hill (Cleveland Clinic Foundation) Prakash Satwani (NYPH/ Columbia University Medical Center) Mehdi Hamadani (CIBMTR ‐ Milwaukee) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

LE16‐01 Title: Analysis of late mortality from infections in allogeneic hematopoietic transplant recipients with hematologic malignances PIs: Maxim Norkin (Shands HealthCare & University of Florida) John Wingard (Shands HealthCare & University of Florida) Juan Gea‐Banacloche (NCI Experimental Transplantation and Immunology Branch) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018)

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LE16‐02 Title: An investigation of new malignant neoplasms in pediatric patients undergoing allogeneic hematopoietic stem cell transplantation for non‐malignant diseases PIs: Justine Kahn (Morgan Stanley Children's Hospital of New York‐Presbyterian ‐ Columbia University Medical Center) Prakash Satwani (NYPH/ Columbia University Medical Center) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

LE16‐03 Title: Post‐transplant employment status in adult survivors of childhood hematopoietic stem cell transplant PIs: Neel Bhatt (CIBMTR ‐ Milwaukee) Bronwen Shaw (CIBMTR ‐ Milwaukee) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

LE16‐G1 Title: Neurocognitive dysfunction following hematopoietic cell transplantation PIs: Debra Lynch Kelly (Shands HealthCare & University of Florida) David Buchbinder (Children's Hospital of Orange County) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

LE17‐01 Title: Long‐term follow up after hematopoietic stem cell transplantation for sickle cell disease PIs: Elizabeth Stenger (Children's Healthcare of Atlanta at Egleston) Rachel Phelan (Medical College of Wisconsin) Shalini Shenoy (Washington University/St. Louis Children's Hospital) Lakshmanan Krishnamurti (Children's Healthcare of Atlanta at Egleston) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018) * Collaborative study with Emory University

LE17‐02 Title: Comparison of late effects among allogeneic hematopoietic cell transplantation survivors conditioned with high dose total body irradiation (TBI) versus non‐TBI based ablative regimens in adolescents and young adults (15‐39yo) with acute leukemia PIs: Catherine Lee (Utah Blood and Marrow Transplant Program) Lori Muffly (Stanford Health Care) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

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LE17‐G1 Title: Ocular complications after hematopoietic cell transplantation PIs: Yoshi Inamoto (National Cancer Center Hospital) Igor Petricek (University Hospital Centre Zagreb) NuriaSanz (University Hospital Puerta de Hierro‐Majadahonda) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

LE99‐01 Title: Quality of life in late HCT survivors PIs: John Wingard (Shands HealthCare & University of Florida) Status: Ongoing (as of July 1, 2017) Ongoing (expected by June 30, 2018) * Collaborative study with Univeristy of Florida

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12.0 LYMPHOMA WORKING COMMITTEE

12.1 Leadership

Chair: Sonali Smith, MD, University of Chicago Hospitals Email: [email protected] Chair: Anna Sureda, MD, Institut Catala d'Oncologia ‐ IDIBELL Email: [email protected] Chair: Timothy Fenske, MD, MS, Froedtert Memorial Lutheran Hospital Email: [email protected] Scientific Director: Mehdi Hamadani, MD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Kwang Woo Ahan, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Carlos Litovich, CIBMTR Milwaukee Email: [email protected]

12.2 Recent Publications

2017 Epperla N, Ahn KW, Ahmed S, Jagasia M, DiGilio A, Devine SM, Jaglowski S, Kennedy V, Rezvani AR, Smith SM, Sureda A, Fenske TS, Kharfan‐Dabaja MA, Armand P, Hamadani M. Rituximab‐ containing reduced‐intensity conditioning improves progression‐free survival following allogeneic transplantation in B cell non‐Hodgkin lymphoma. Journal of Hematology & Oncology. 2017 Jun 12; 10(1):117. doi:10.1186/s13045‐017‐0487‐y. Epub 2017 Jun 12. PMC5469142. ncbi.nlm.nih.gov/pubmed/28606176 Hamadani M, Kanate AS, DiGilio A, Ahn KW, Smith SM, Lee JW, Ayala E, Chao N, Hari P, Bolaños‐ Meade J, Gress R, Smedegaard Anderson N, Chen Y‐B, Farooq U, Schiller G, Yared J, Sureda A, Fenske TS, Olteanu H. Allogeneic hematopoietic cell transplantation for aggressive NK cell leukemia. A Center for International Blood and Marrow Transplant Research analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 May 1; 23(5):853‐856. doi:10.1016/j.bbmt.2017.01.082. Epub 2017 Feb 1. PMC5410937. ncbi.nlm.nih.gov/pubmed/28161608

2016 Ghosh N, Karmali R, Rocha V, Ahn KW, DiGilio A, Hari PN, Bachanova V, Bacher U, Dahi P, de Lima M, D'Souza A, Fenske TS, Ganguly S, Kharfan‐Dabaja MA, Prestidge TD, Savani BN, Smith SM, Sureda A, Waller EK, Jaglowski S, Herrera AF, Armand P, Salit RB, Wagner‐Johnston ND, Fuchs E, Bolaños‐Meade J, Hamadani M. Reduced‐intensity transplantation for lymphomas using haploidentical related donors versus HLA‐matched sibling donors. Journal of Clinical Oncology. doi:10.1200/JCO.2015.66.3476. Epub 2016 Jun 6. PMC5012706. ncbi.nlm.nih.gov/ pubmed/27269951

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Fenske TS, Hamadani M, Cohen JB, Costa LJ, Kahl BS, Evens AM, Hamlin PA, Lazarus HM, Petersdorf E, Bredeson C. Allogeneic hematopoietic cell transplantation as curative therapy for patients with non‐Hodgkin lymphoma: Increasingly successful application to older patients. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Sep 1; 22(9):1543‐1551. doi:10.1016/ j.bbmt.2016.04.019. Epub 2016 Apr 27. PMC4981559. ncbi.nlm.nih.gov/pubmed/27131863 Flowers CR, Costa LJ, Pasquini MC, Le‐Rademacher J, Lill M, Shore TB, Vaughan W, Craig M, Freytes CO, Shea TC, Horwitz ME, Fay JW, Mineishi S, Rondelli D, Mason J, Braunschweig I, Ai W, Yeh RF, Rodriguez TE, Flinn I, Comeau T, Yeager AM, Pulsipher MA, Bence‐Bruckler I, Laneuville P, Bierman P, Chen AI, Kato K, Wang Y, Xu C, Smith AJ, Waller EK. Efficacy of pharmacokinetics‐ directed busulfan, cyclophosphamide and etoposide conditioning and autologous stem cell transplantation for lymphoma: Comparison of a multicenter Phase 2 study and CIBMTR outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.03.018. Epub 2016 Mar 31. PMC4914052. ncbi.nlm.nih.gov/pubmed/27040394 Fenske TS, Ahn KW, Graff TM, DiGilio A, Bashir Q, Kamble RT, Ayala E, Bacher U, Brammer JE, Cairo M, Chen A, Chen Y‐B, Chhabra S, D'Souza A, Farooq U, Freytes C, Ganguly S, Hertzberg M, Inwards D, Jaglowski S, Kharfan‐Dabaja MA, Lazarus HM, Nathan S, Pawarode A, Perales M‐A, Reddy N, Seo S, Sureda A, Smith SM, Hamadani M. Allogeneic transplantation provides durable remission in a subset of DLBCL patients relapsing after autologous transplantation. British Journal of Haematology. doi:10.1111/bjh.14046. Epub 2016 Mar 15. PMC4940282. ncbi.nlm.nih.gov/pubmed/26989808

2015 Kanate AS, Mussetti A, Kharfan‐Dabaja MA, Ahn KW, DiGilio A, Beitinjaneh A, Chhabra S, Fenske TS, Freytes C, Gale RP, Ganguly S, Hertzberg M, Klyuchnikov E, Lazarus HM, Olsson R, Perales M‐ A, Rezvani A, Riches M, Saad A, Slavin S, Smith SM, Sureda A, Yared J, Ciurea S, Armand P, Salit R, Bolaños‐Meade J, Hamadani M. Reduced‐intensity transplantation for lymphomas using haploidentical related donor’s vs HLA‐matched unrelated donors. Blood. 2016 Feb 18; 127(7):938‐947. doi:10.1182/blood‐2015‐09‐671834. Epub 2015 Dec 15. PMC4760094. ncbi.nlm.nih.gov/pubmed/26670632 Klyuchnikov E, Bacher U, Woo Ahn K, Carreras J, Kröger NM, Hari PN, Ku GH, Ayala E, Chen AI, Chen Y‐B, Cohen JB, Freytes CO, Gale RP, Kamble RT, Kharfan‐Dabaja MA, Lazarus HM, Martino R, Mussetti A, Savani BN, Schouten HC, Usmani SZ, Wiernik PH, Wirk B, Smith SM, Sureda A, Hamadani M. Long‐term survival outcomes of reduced‐intensity allogeneic or autologous transplantation in relapsed grade 3 follicular lymphoma. Bone Marrow Transplantation. 2016 Jan 1; 51(1):58‐66. doi:10.1038/bmt.2015.223. Epub 2015 Oct 5. PMC4703480. ncbi.nlm.nih.gov/pubmed/26437062

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Klyuchnikov E, Bacher U, Kröger NM, Hari PN, Ahn KW, Carreras J, Bachanova V, Bashey A, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Hertzberg MS, Holmberg LA, Kharfan‐Dabaja MA, Klein A, Ku GH, Laport GG, Lazarus HM, Miller AM, Mussetti A, Olsson RF, Slavin S, Usmani SZ, Vij R, Wood WA, Maloney DG, Sureda AM, Smith SM, Hamadani M. Reduced‐intensity allografting as first transplantation approach in relapsed/refractory grades one and two follicular lymphoma provides improved outcomes in long‐term survivors. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2091‐2099. doi:10.1016/j.bbmt.2015.07.028. Epub 2015 Aug 4. PMC4639453. ncbi.nlm.nih.gov/pubmed/26253007 Satwani P, Ahn KW, Carreras J, Abdel‐Azim H, Cairo MS, Cashen A, Chen AI, Cohen JB, Costa LJ, Dandoy C, Fenske TS, Freytes CO, Ganguly S, Gale RP, Ghosh N, Hertzberg MS, Hayashi RJ, Kamble RT, Kanate AS, Keating A, Kharfan‐Dabaja MA, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Prestidge TD, Rolon JM, Savani BN, Vose JM, Wood WA, Inwards DJ, Bachanova V, Smith SM, Maloney DG, Sureda A, Hamadani M. A prognostic model predicting autologous transplantation outcomes in children, adolescents and young adults with Hodgkin lymphoma. Bone Marrow Transplantation. 2015 Nov 1; 50(11):1416‐1423. doi:10.1038/bmt.2015.177. Epub 2015 Aug 3. PMC4633349. ncbi.nlm.nih.gov/pubmed/ 26237164 Urbano‐Ispizua A, Pavletic SZ, Flowers ME, Klein JP, Zhang M‐J, Carreras J, Montoto S, Perales M‐ A, Aljurf MD, Akpek G, Bredeson CN, Costa LJ, Dandoy C, Freytes CO, Fung HC, Gale RP, Gibson J, Hamadani M, Hayashi RJ, Inamoto Y, Inwards DJ, Lazarus HM, Maloney DG, Martino R, Munker R, Nishihori T, Olsson RF, Rizzieri DA, Reshef R, Saad A, Savani BN, Schouten HC, Smith SM, Socié G, Wirk B, Yu LC, Saber W. The impact of graft‐versus‐host disease on the relapse rate in patients with lymphoma depends on the histological subtype and the intensity of the conditioning regimen. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1746‐1753. doi:10.1016/ j.bbmt.2015.05.010. Epub 2015 May 15. PMC4568162. ncbi.nlm.nih.gov/pubmed/25981509 Bachanova V, Burns LJ, Ahn KW, Laport GG, Akpek G, Kharfan‐Dabaja MA, Nishihori T, Agura E, Armand P, Jaglowski SM, Cairo MS, Cashen AF, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Ghosh N, Holmberg LA, Inwards DJ, Kanate AS, Lazarus HM, Malone AK, Munker R, Mussetti A, Norkin M, Prestidge TD, Rowe JM, Satwani P, Siddiqi T, Stiff PJ, William BM, Wirk B, Maloney DG, Smith SM, Sureda AM, Carreras J, Hamadani M. Impact of pretransplantation 18F‐fluorodeoxy glucose‐positron emission tomography status on outcomes after allogeneic hematopoietic cell transplantation for non‐Hodgkin lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1605‐1611. doi:10.1016/j.bbmt.2015.05.007. Epub 2015 May 14. PMC4558181. ncbi.nlm.nih.gov/pubmed/25983043

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2014 Bachanova V, Burns LJ, Wang T, Carreras J, Gale RP, Wiernik PH, Ballen KK, Wirk B, Munker R, Rizzieri DA, Chen Y‐B, Gibson J, Akpek G, Costa LJ, Kamble RT, Aljurf MD, Hsu JW, Cairo MS, Schouten HC, Bacher U, Savani BN, Wingard JR, Lazarus HM, Laport GG, Montoto S, Maloney DG, Smith SM, Brunstein C, Saber W. Alternative donors extend transplantation for patients with lymphoma who lack an HLA matched donor. Bone Marrow Transplantation. 2015 Feb 7; 50(2):197‐203. doi:10.1038/bmt.2014.259. Epub 2014 Nov 17. PMC4336786. ncbi.nlm.nih.gov/ pubmed/25402415 Lechowicz MJ, Lazarus HM, Carreras J, Laport GG, Cutler CS, Wiernik PH, Hale GA, Maharaj D, Gale RP, Rowlings PA, Freytes CO, Miller AM, Vose JM, Maziarz RT, Montoto S, Maloney DG, Hari PN. Allogeneic hematopoietic cell transplantation for mycosis fungoides and Sézary syndrome. Bone Marrow Transplantation. 2014 Nov 1; 49(11):1360‐1365. doi:10.1038/bmt. 2014.161. Epub 2014 Jul 28. PMC4221526. ncbi.nlm.nih.gov/pubmed/25068422 Hamadani M, Hari PN, Zhang Y, Carreras J, Akpek G, Aljurf MD, Ayala E, Bachanova V, Chen AI, Chen Y‐B, Costa LJ, Fenske TS, Freytes CO, Ganguly S, Hertzberg MS, Holmberg LA, Inwards DJ, Kamble RT, Kanfer EJ, Lazarus HM, Marks DI, Nishihori T, Olsson R, Reddy NM, Rizzieri DA, Savani BN, Solh M, Vose JM, Wirk B, Maloney DG, Smith SM, Montoto S, Saber W. Early failure of frontline rituximab‐containing chemo‐immunotherapy in diffuse large B cell lymphoma does not predict futility of autologous hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1729‐1736. doi:10.1016/j.bbmt.2014.06.036. Epub 2014 Jul 5. PMC4194275. ncbi.nlm.nih.gov/pubmed/25008330 Wirk B, Fenske TS, Hamadani M, Zhang M‐J, Hu Z‐H, Akpek G, Aljurf MD, Armand P, Ayala E, Bachanova V, Bolwell B, Cairo MS, Cashen A, Chen Y‐B, Costa LJ, Farhan S, Freytes CO, Gajewski JL, Gibson J, Hale GA, Holmberg LA, Hsu JW, Inwards DJ, Kamble RT, Maharaj D, Maziarz RT, Munker R, Nath R, Reddy NM, Reeder CB, Rizzieri DA, Sauter CS, Savani BN, Schouten HC, Sureda A, Vose JM, Waller EK, Wiernik PH, Gale RP, Burns LJ, Saber W. Outcomes of hematopoietic cell transplantation for diffuse large B cell lymphoma transformed from follicular lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Jul 1; 20(7):951‐959. doi:10.1016/j.bbmt.2014.03.014. Epub 2014 Mar 15. PMC4060436. ncbi.nlm.nih.gov/pubmed/24641828

2013 Fenske TS, Zhang M‐J, Carreras J, Ayala E, Burns LJ, Cashen A, Costa LJ, Freytes CO, Gale RP, Hamadani M, Holmberg LA, Inwards DJ, Lazarus HM, Maziarz RT, Munker R, Perales M‐A, Rizzieri DA, Schouten HC, Smith SM, Waller EK, Wirk BM, Laport GG, Maloney DG, Montoto S, Hari PN. Autologous or reduced‐intensity conditioning allogeneic hematopoietic cell transplantation for chemotherapy‐sensitive mantle‐cell lymphoma: Analysis of transplantation timing and modality. Journal of Clinical Oncology. 2014 Feb 1; 32(4):273‐281. doi:10.1200/JCO.2013.49.2454. Epub 2013 Dec 16. PMC3897255. ncbi.nlm.nih.gov/pubmed/ 24344210

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Hahn T, McCarthy PL, Carreras J, Zhang M‐J, Lazarus HM, Laport GG, Montoto S, Hari PN. Simplified validated prognostic model for progression‐free survival after autologous transplantation for relapsed or refractory for Hodgkin lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Dec 1; 19(12):1740‐1744. doi:10.1016/j.bbmt.2013.09.018. Epub 2013 Oct 3. PMC3906436. ncbi.nlm.nih.gov/pubmed/24096096 Smith SM, Burns LJ, van Besien K, LeRademacher J, He W, Fenske TS, Suzuki R, Hsu JW, Schouten HC, Hale GA, Holmberg LA, Sureda A, Freytes CO, Maziarz RT, Inwards DJ, Gale RP, Gross TG, Cairo MS, Costa LJ, Lazarus HM, Wiernik PH, Maharaj D, Laport GG, Montoto S, Hari PN. Hematopoietic cell transplantation for systemic mature T‐cell non‐Hodgkin lymphoma. Journal of Clinical Oncology. 2013 Sep 1; 31(25):3100‐3109. doi:10.1200/JCO.2012.46.0188. Epub 2013 Jul 29. PMC3753702. ncbi.nlm.nih.gov/pubmed/23897963 Maziarz RT, Wang Z, Zhang M‐J, Bolwell BJ, Chen AI, Fenske TS, Freytes CO, Gale RP, Gibson J, Hayes‐Lattin BM, Holmberg L, Inwards DJ, Isola LM, Khoury HJ, Lewis VA, Maharaj D, Munker R, Phillips GL, Rizzieri DA, Rowlings PA, Saber W, Satwani P, Waller EK, Maloney DG, Montoto S, Laport GG, Vose JM, Lazarus HM, Hari PN. Autologous haematopoietic cell transplantation for non‐Hodgkin lymphoma with secondary CNS involvement. British Journal of Haematology. 2013 Sep 1; 162(5):648‐656. doi:10.1111/bjh.12451. Epub 2013 Jul 6. PMC3766698. ncbi.nlm.nih.gov/pubmed/23829536 Hamadani M, Saber W, Ahn KW, Carreras J, Cairo MS, Fenske TS, Gale RP, Gibson J, Hale GA, Hari PN, Hsu JW, Inwards DJ, Kamble RT, Klein A, Maharaj D, Marks DI, Rizzieri DA, Savani BN, Schouten HC, Waller EK, Wirk B, Laport GG, Montoto S, Maloney DG, Lazarus HM. Impact of pretransplantation conditioning regimens on outcomes of allogeneic transplantation for chemotherapy‐unresponsive diffuse large B cell lymphoma and grade III follicular lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 May 1; 19(5):746‐753. doi:10.1016/j.bbmt.2013. 01.024. Epub 2013 Feb 1. PMC3650915. ncbi.nlm.nih.gov/pubmed/23380340 Hamadani M, Saber W, Ahn KW, Carreras J, Cairo MS, Fenske TS, Gale RP, Gibson J, Hale GA, Hari PN, Hsu JW, Inwards DJ, Kamble RT, Klein A, Maharaj D, Marks DI, Rizzieri DA, Savani BN, Schouten HC, Waller EK, Wirk B, Lazarus HM. Allogeneic hematopoietic cell transplantation for chemotherapy‐unresponsive : A cohort analysis from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Apr 1; 19(4):625‐631. doi:10.1016/j.bbmt.2013.01.009. Epub 2013 Jan 17. PMC3640440. ncbi.nlm.nih.gov/pubmed/23333532

2012 Maramattom LV, Hari PN, Burns LJ, Carreras J, Arcese W, Cairo MS, Costa LJ, Fenske TS, Lill M, Freytes CO, Gale RP, Gross TG, Hale GA, Hamadani M, Holmberg LA, Hsu JW, Inwards DJ, Lazarus HM, Marks DI, Maloney DG, Maziarz RT, Montoto S, Rizzieri DA, Wirk B, Gajewski JL. Autologous and allogeneic transplantation for Burkitt lymphoma outcomes and changes in utilization: A report from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Feb 1; 19(2):173‐179. doi:10.1016/j.bbmt.2012.11.016. Epub 2012 Nov 27. PMC3553321. ncbi.nlm.nih.gov/pubmed/23200705

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Bacher U, Klyuchnikov E, Le‐Rademacher J, Carreras J, Armand P, Bishop MR, Bredeson CN, Cairo MS, Fenske TS, Freytes CO, Gale RP, Gibson J, Isola LM, Inwards DJ, Laport GG, Lazarus HM, Maziarz RT, Wiernik PH, Schouten HC, Slavin S, Smith SM, Vose JM, Waller EK, Hari PN. Conditioning regimens for allotransplants for diffuse large B‐cell lymphoma: myeloablative or reduced intensity? Blood. 2012 Nov 15; 120(20):4256‐4262. doi:10.1182/ blood‐2012‐06‐436725. Epub 2012 Sep 24. PMC3501720. ncbi.nlm.nih.gov/pubmed/23007405

12.3 Current Studies

LY06‐03 Title: HLA identical sibling transplantation versus HLA‐matched unrelated donor transplantation in patients with follicular lymphoma PIs: Anna Sureda (Institut Català d'Oncologia‐ Hospital Duran I Reynals) Harry Schouten (Academische Ziekenhuis Maastricht) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018) * Collaborative study with EBMT

LY15‐03 Title: Does Autologous Stem Cell Transplant Overcome the Increased Risk of Death in Patients with Follicular Lymphoma Relapsing Early after First Line Chemoimmunotherapy? PIs: Carla Casulo (Strong Memorial Hospital ‐ University of Rochester Medical Center) Jonathan Friedberg (Strong Memorial Hospital ‐ University of Rochester Medical Center) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

LY16‐01a Title: Allogeneic hematopoietic cell transplantation for extranodal NK/T cell lmphoma, nasal type: A CIBMTR Analysis PIs: Abraham Kanate (West Virginia University Hospitals, Inc.) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

LY16‐02 Title: Alternative donor source stem cell transplant vs matched donor stem cell transplant for Hodgkin lymphoma PIs: Sairah Ahmed (M.D. Anderson Cancer Center) Jennifer Kanakry (National Cancer Institute) Status: Deferred (as of July 1, 2017) Protocol Development (expected by June 30, 2018) * Collaborative study with EBMT

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LY16‐03 Title: Haploidentical transplantation in diffuse large B cell lymphoma: A CIBMTR and EBMT collaborative study PIs: Anna Sureda (Institut Català d'Oncologia‐ Hospital Duran I Reynals) Peter Dreger (Universitaetsklinikum Heidelberg) Status: Protocol Development (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with EBMT

LY16‐04 Title: Utility of autologous vs allogeneic transplant as first transplantation approach in FL patients with early chemoimmunotherapy failure PIs: James Godfrey (University of Chicago Medicine) Sonali Smith (University of Chicago Medicine) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

LY16‐05b Title: Reduced‐intensity conditioning for Follicular lymphoma PIs: Naren Epperla (Ohio State Medical Center, James Cancer Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

LY17‐01 Title: Clinical Outcomes of medicare Eligible Patients (Age >=65) undergoing Allogeneic Hematopoietic Cell Transplant for Relapsed/Refractory Non‐Hodgkin Lymphoma PIs: Nirav Shah (Medical College of Wisconsin) Status: Protocol Development (as of July 1, 2017) Submitted (expected by June 30, 2018)

LY17‐02 Title: Allografts following reduced intensity conditioning PIs: Nilanjan Ghosh (Levine Cancer Institute) Sairah Ahmed (M.D. Anderson Cancer Center) Status: Protocol Development (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

LY17‐03 Title: Impact of allogeneic hematopoietic cell transplantation on the outcomes of angioimmunoblastic T‐cell lymphoma PIs: Naren Epperla (Ohio State Medical Center, James Cancer Center) Status: Protocol Development (as of July 1, 2017) Submitted (expected by June 30, 2018)

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13.0 PEDIATRIC CANCER WORKING COMMITTEE

13.1 Leadership

Chair: Gregory Hale, MD, John Hopkins All Children’s Hospital Email: [email protected] Chair: Parinda Mehta, MD, Cincinnati Children’s Hospital Medical Center Email: [email protected] Chair: Angela Smith, MD, MS, University of Minnesota Medical Center, Fairview Email: [email protected] Scientific Director: Mary Eapen, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Kwang Woo Ahn, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Heather Millard, MPH, CIBMTR Milwaukee Email: [email protected]

13.2 Recent Publications

2017 Khandelwal P, Millard HR, Thiel E, Abdel‐Azim H, Abraham AA, Auletta JJ, Boulad F, Brown VI, Camitta BM, Chan KW, Chaudhury S, Cowan MJ, Angel‐Diaz M, Gadalla SM, Gale RP, Hale G, Kasow KA, Keating AK, Kitko CL, MacMillan ML, Olsson RF, Page KM, Seber A, Smith AR, Warwick AB, Wirk B, Mehta PA. Hematopoietic stem cell transplant activity in pediatric cancer between 2008 and 2014 in the United States: A CIBMTR report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2017.04.018. Epub 2017 Apr 24. ncbi.nlm.nih.gov/pubmed/28450183

2015 Burke MJ, Verneris MR, Le Rademacher J, He W, Abdel‐Azim H, Abraham AA, Auletta JJ, Ayas M, Brown VI, Cairo MS, Chan KW, Diaz Perez MA, Dvorak CC, Egeler RM, Eldjerou L, Frangoul H, Guilcher GMT, Hayashi RJ, Ibrahim A, Kasow KA, Leung WH, Olsson RF, Pulsipher MA, Shah N, Shah NN, Thiel E, Talano JA, Kitko CL. Transplant outcomes for children with T cell acute lymphoblastic leukemia in second remission: A report from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2154‐2159. doi:10.1016/j.bbmt.2015.08.023. Epub 2015 Aug 29. PMC4654112. ncbi.nlm.nih.gov/pubmed/26327632

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Mehta PA, Zhang M‐J, Eapen M, He W, Seber A, Gibson B, Camitta BM, Kitko CL, Dvorak CC, Nemecek ER, Frangoul HA, Abdel‐Azim H, Kasow KA, Lehmann L, Gonzalez Vicent M, Diaz Pérez MA, Ayas M, Qayed M, Carpenter PA, Jodele S, Lund TC, Leung WH, Davies SM. Transplant outcomes for children with hypodiploid acute lymphoblastic leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1273‐1277. doi:10.1016/j.bbmt.2015.04.008. Epub 2015 Apr 10. PMC4465998. ncbi.nlm.nih.gov/pubmed/25865650

2014 Bitan M, He W, Zhang M‐J, Abdel‐Azim H, Ayas MF, Bielorai B, Carpenter PA, Cairo MS, Diaz MA, Horan JT, Jodele S, Kitko CL, Schultz KR, Kletzel M, Kasow KA, Lehmann LE, Mehta PA, Shah N, Pulsipher MA, Prestidge T, Seber A, Shenoy S, Woolfrey AE, Yu LC, Davies SM. Transplantation for children with acute myeloid leukemia: A comparison of outcomes with reduced intensity and myeloablative regimens. Blood. 2014 Mar 6; 123(10):1615‐1620. doi:10.1182/blood‐2013‐10‐535716. Epub 2014 Jan 16. PMC3945869. ncbi.nlm.nih.gov/ pubmed/24435046 Hitzler JK, He W, Doyle J, Cairo M, Camitta BM, Chan KW, Diaz Perez MA, Fraser C, Gross TG, Horan JT, Kennedy‐Nasser AA, Kitko C, Kurtzberg J, Lehmann L, O'Brien T, Pulsipher MA, Smith FO, Zhang M‐J, Eapen M, Carpenter PA. Outcome of transplantation for acute lymphoblastic leukemia in children with Down syndrome. Pediatric Blood & Cancer. 2014 Jun 1; 61(6):1126‐1128. doi:10.1002/pbc.24918. Epub 2014 Jan 4. PMC4080799. ncbi.nlm.nih.gov/ pubmed/24391118

2013 Kelly MJ, Horan JT, Alonzo TA, Eapen M, Gerbing RB, He W, Lange BJ, Parsons SK, Woods WG. Comparable survival for pediatric acute myeloid leukemia with poor‐risk cytogenetics following chemotherapy, matched related donor, or unrelated donor transplantation. Pediatric Blood & Cancer. 2014 Feb 1; 61(2):269‐275. doi:10.1002/pbc.24739. Epub 2013 Aug 19. PMC3919967. ncbi.nlm.nih.gov/pubmed/23955900 Locatelli F, Crotta A, Ruggeri A, Eapen M, Wagner JE, MacMillan ML, Zecca M, Kurtzberg J, Bonfim C, Vora A, Díaz de Heredia C, Teague L, Stein J, O'Brien TA, Bittencourt H, Madureira A, Strahm B, Peters C, Niemeyer C, Gluckman E, Rocha V. Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia: A EUROCORD, EBMT, EWOG‐MDS, CIBMTR study. Blood. 2013 Sep 19; 122(12):2135‐2141. doi:10.1182/blood‐2013‐03‐491589. Epub 2013 Aug 7. PMC3836039. ncbi.nlm.nih.gov/ pubmed/23926304 Hitzler JK, He W, Doyle J, Cairo M, Camitta BM, Chan KW, Diaz Perez MA, Fraser C, Gross TG, Horan JT, Kennedy‐Nasser AA, Kitko C, Kurtzberg J, Lehmann L, O'Brien T, Pulsipher MA, Smith FO, Zhang M‐J, Eapen M, Carpenter PA. Outcome of transplantation for acute myelogenous leukemia in children with Down syndrome. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Jun 1; 19(6):893‐ 897. doi:10.1016/j.bbmt.2013.02.017. Epub 2013 Mar 1. PMC3707801. ncbi.nlm.nih.gov/ pubmed/23467128

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2012 Tracey J, Zhang M‐J, Thiel E, Sobocinski KA, Eapen M. Transplantation conditioning regimens and outcomes after allogeneic hematopoietic cell transplantation in children and adolescents with acute lymphoblastic leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Feb 1; 19(2):255‐259. doi:10.1016/j.bbmt.2012.09.019. Epub 2012 Oct 3. PMC3553255. ncbi.nlm.nih.gov/pubmed/23041605

13.3 Current Studies

PC16‐01 Title: Outcomes after second HCT for relapsed malignancy in pediatric patients PIs: Troy C. Lund (University of Minnesota Blood and Marrow Transplant Program) Mary Eapen (CIBMTR ‐ Milwaukee) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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14.0 PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE

14.1 Leadership

Chair: Cristina Gasparetto, MD, Duke University Medical Center Email: [email protected] Chair: Yago Nieto, MD, PhD, MD Anderson Cancer Center Email: [email protected] Chair: Tomer Mark, MD, New York Presbyterian Hospital at Cornell Email: [email protected] Chair: Shaji Kumar, Mayo Clinic Rochester Email: [email protected] Scientific Director: Parameswaran Hari, MD, MS, CIBMTR Milwaukee Email: [email protected] Asst Sci Director: Anita D’Souza, MD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Raphael Fraser, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Omar Dávila, MPH, CIBMTR Milwaukee Email: [email protected]

14.2 Recent Publications

2017 Schriber JR, Hari PN, Ahn KW, Fei M, Costa LJ, Kharfan‐Dabaja MA, Angel‐Diaz M, Gale RP, Ganguly S, Girnius SK, Hashmi S, Pawarode A, Vesole DH, Wiernik PH, Wirk BM, Marks DI, Nishihori T, Olsson RF, Usmani S, Mark TM, Nieto YL, D'Souza A. Hispanics have the lowest stem cell transplant utilization rate for autologous hematopoietic cell transplantation for multiple myeloma in the United States: A CIBMTR report. Cancer. doi:10.1002/cncr.30747z. Epub 2017 May 4. ncbi.nlm.nih.gov/pubmed/28472539 Cheng YC, Shi Y, Zhang M‐J, Brazauskas R, Hemmer MT, Bishop MR, Nieto Y, Stadtmauer E, Ayashi L, Gale RP, Lazarus H, Holmberg L, Lill M, Olsson RF, Wirk BM, Arora M, Hari P, Ueno N. Long‐ term outcome of inflammatory breast cancer compared to non‐inflammatory breast cancer in the setting of high‐dose chemotherapy with autologous hematopoietic cell transplantation. Journal of Cancer. 8(6):1009‐1017. doi:10.7150/jca.16870. Epub 2017 Mar 25. PMC5436253. ncbi.nlm.nih.gov/pubmed/28529613

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Garderet L, D'Souza A, Jacobs P, van Biezen A, Schönland S, Kroeger N, Morris C, Hari P. Response assessment in myeloma: Practical manual on consistent reporting in an era of dramatic therapeutic advances. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jul 1; 23(7):1193‐1202. doi:10.1016/j.bbmt.2017.03.009. Epub 2017 Mar 8. ncbi.nlm.nih.gov/pubmed/28285082

2016 Cornell RF, D'Souza A, Kassim AA, Costa LJ, Innis‐Shelton RD, Zhang M‐J, Huang J, Abidi M, Aiello J, Akpek G, Bashey A, Bashir Q, Cerny J, Comenzo R, Diaz MA, Freytes C, Gale RP, Ganguly S, Hamadani M, Hashmi S, Holmberg L, Hossain N, Kamble RT, Kharfan‐Dabaja M, Kindwall‐Keller T, Kyle R, Kumar S, Lazarus H, Lee C, Maiolino A, Marks DI, Meehan K, Mikhael J, Nath R, Nishihori T, Olsson RF, Ramanathan M, Saad A, Seo S, Usmani S, Vesole D, Vij R, Vogl D, Wirk BM, Yared J, Krishnan A, Mark T, Nieto Y, Hari P. Maintenance versus induction therapy choice on outcomes after autologous transplantation for multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Feb 1; 23(2):269‐277. doi:10.1016/j.bbmt.2016.11.011. Epub 2016 Nov 15. PMC5346183. ncbi.nlm.nih.gov/pubmed/27864161 Cornell RF, Bachanova V, D'Souza A, Woo‐Ahn K, Martens M, Huang J, Al‐Homsi AS, Chhabra S, Copelan E, Diaz M‐A, Freytes CO, Gale RP, Ganguly S, Hamadani M, Hildebrandt G, Kamble RT, Kharfan‐Dabaja M, Kindwall‐Keller T, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Saad A, Usmani S, Vesole DH, Yared J, Mark T, Nieto Y, Hari P. Allogeneic transplantation for relapsed Waldenström macroglobulinemia and lymphoplasmacytic lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jan 1; 23(1):60‐66. doi:10.1016/j.bbmt.2016.10.010. Epub 2016 Oct 24. PMC5182098. ncbi.nlm.nih.gov/pubmed/27789362 Scott EC, Hari P, Sharma M, Le‐Rademacher J, Huang J, Vogl D, Abidi M, Beitinjaneh A, Fung H, Ganguly S, Hildebrandt G, Holmberg L, Kalaycio M, Kumar S, Kyle R, Lazarus H, Lee C, Maziarz RT, Meehan K, Mikhael J, Nishihori T, Ramanathan M, Usmani S, Tay J, Vesole D, Wirk B, Yared J, Savani BN, Gasparetto C, Krishnan A, Mark T, Nieto Y, D'Souza A. Post‐transplant outcomes in high‐risk compared with non‐high‐risk multiple myeloma: A CIBMTR analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Oct 1; 22(10):1893‐1899. doi:10.1016/j.bbmt.2016.07.007. Epub 2016 Aug 2. PMC5090978. ncbi.nlm.nih.gov/pubmed/27496215 Beksac M, Gragert L, Fingerson S, Maiers M, Zhang M‐J, Albrecht M, Zhong X, Cozen W, Dispenzieri A, Lonial S, Hari P. HLA polymorphism and risk of multiple myeloma. Leukemia. 2016 Nov 1; 30(11):2260‐2264. doi:10.1038/leu.2016.199. Epub 2016 Jul 27. PMC5134914. ncbi.nlm.nih.gov/pubmed/27461064 Rodriguez TE, Hari P, Stiff PJ, Smith SE, Sterrenberg D, Vesole DH. Busulfan, melphalan, and bortezomib versus high‐dose melphalan as a conditioning regimen for autologous hematopoietic stem cell transplantation in multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug; 22(8):1391‐6. doi: 10.1016/j.bbmt.2016.03.021. Epub 2016 May 7. ncbi.nlm.nih.gov/ pubmed/27164062

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2015 D'Souza A, Huang J, Hari P. New light chain amyloid response criteria help risk stratification of patients by day 100 after autologous hematopoietic cell transplant. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Apr 1; 22(4):768‐770. doi:10.1016/j.bbmt.2015.12.021. Epub 2015 Dec 29. PMC4801777. ncbi.nlm.nih.gov/pubmed/26743343 D'Souza A, Dispenzieri A, Wirk B, Zhang M‐J, Huang J, Gertz MA, Kyle RA, Kumar S, Comenzo RL, Gale RP, Lazarus HM, Savani BN, Cornell RF, Weiss BM, Vogl DT, Freytes CO, Scott EC, Landau HJ, Moreb JS, Costa LJ, Ramanathan M, Callander NS, Kamble RT, Olsson RF, Ganguly S, Nishihori T, Kindwall‐Keller TL, Wood WA, Mark TM, Hari P. Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: A Center for International Blood and Marrow Transplant Research study. Journal of Clinical Oncology. 2015 Nov 10; 33(32):3741‐3749. doi:10.1200/JCO.2015.62.4015. Epub 2015 Sep 14. PMC4737858. ncbi.nlm.nih.gov/pubmed/26371138 Uy GL, Costa LJ, Hari PN, Zhang M‐J, Huang J‐X, Anderson KC, Bredeson CN, Callander NS, Cornell RF, Perez MAD, Dispenzieri A, Freytes CO, Gale RP, Garfall A, Gertz MA, Gibson J, Hamadani M, Lazarus HM, Kalaycio ME, Kamble RT, Kharfan‐Dabaja MA, Krishnan AY, Kumar SK, Kyle RA, Landau HJ, Lee CH, Maiolino A, Marks DI, Mark TM, Munker R, Nishihori T, Olsson RF, Ramanathan M, Rodriguez TE, Saad AA, Savani BN, Schiller GJ, Schouten HC, Schriber JR, Scott E, Seo S, Sharma M, Ganguly S, Stadtmauer EA, Tay J, To LB, Vesole DH, Vogl DT, Wagner JL, Wirk B, Wood WA, D'Souza A. Contribution of chemotherapy mobilization to disease control in multiple myeloma treated with autologous hematopoietic cell transplantation. Bone Marrow Transplantation. 2015 Dec 1; 50(12):1513‐1518. doi:10.1038/bmt.2015.190. Epub 2015 Aug 24. PMC4548821. ncbi.nlm.nih.gov/pubmed/26301967

2014 Mahindra A, Raval G, Mehta P, Brazauskas R, Zhang M‐J, Zhong X, Bird JM, Freytes CO, Hale GA, Herzig R, Holmberg LA, Kamble RT, Kumar S, Lazarus HM, Majhail NS, Marks DI, Moreb JS, Olsson R, Saber W, Savani BN, Schiller GJ, Tay J, Vogl DT, Waller EK, Wiernik PH, Wirk B, Lonial S, Krishnan AY, Dispenzieri A, Brandenburg NA, Gale RP, Hari PN. New cancers after autotransplantations for multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):738‐ 745. doi:10.1016/j.bbmt.2014.12.028. Epub 2014 Dec 31. PMC4359647. ncbi.nlm.nih.gov/ pubmed/25555448 Costa LJ, Huang J‐X, Hari PN. Disparities in utilization of autologous hematopoietic cell transplantation for treatment of multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):701‐706. doi:10.1016/j.bbmt.2014.12.024. Epub 2014 Dec 30. PMC4361014. ncbi.nlm.nih.gov/pubmed/25555447

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Vij R, Kumar S, Zhang M‐J, Zhong X, Huang J, Dispenzieri A, Abidi MH, Bird JM, Freytes CO, Gale RP, Kindwall‐Keller TL, Kyle RA, Landsburg DJ, Lazarus HM, Munker R, Roy V, Sharma M, Vogl DT, Wirk B, Hari PN. Impact of pretransplant therapy and depth of disease response before autologous transplantation for multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Feb 1; 21(2):335‐341. doi:10.1016/j.bbmt.2014.10.023. Epub 2014 Nov 1. PMC4297511. ncbi.nlm.nih.gov/pubmed/25445028 Sharma M, Zhang M‐J, Zhong X, Abidi MH, Akpek G, Bacher U, Callander NS, Dispenzieri A, Freytes CO, Fung HC, Gale RP, Gasparetto C, Gibson J, Holmberg LA, Kindwall‐Keller TL, Klumpp TR, Krishnan AY, Landau HJ, Lazarus HM, Lonial S, Maiolino A, Marks DI, Mehta P, Mikhael Med JR, Nishihori T, Olsson R, Ramanathan M, Roy V, Savani BN, Schouten HC, Scott E, Tay J, To LB, Vesole DH, Vogl DT, Hari P. Older patients with myeloma derive similar benefit from autologous transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1796‐1803. doi:10.1016/j.bbmt. 2014.07.013. Epub 2014 Jul 18. PMC4194262. ncbi.nlm.nih.gov/pubmed/25046833

2013 Saad A, Mahindra A, Zhang M‐J, Zhong X, Costa LJ, Dispenzieri A, Drobyski WR, Freytes CO, Gale RP, Gasparetto CJ, Holmberg LA, Kamble RT, Krishnan AY, Kyle RA, Marks D, Nishihori T, Pasquini MC, Ramanathan M, Lonial S, Savani BN, Saber W, Sharma M, Sorror ML, Wirk BM, Hari PN. Hematopoietic cell transplant comorbidity index is predictive of survival after autologous hematopoietic cell transplantation in multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Mar 1; 20(3):402‐408. doi:10.1016/j.bbmt.2013.12.557. Epub 2013 Dec 14. PMC3961011. ncbi.nlm.nih.gov/pubmed/24342394 Freytes CO, Vesole DH, LeRademacher J, Zhong X, Gale RP, Kyle RA, Reece DE, Gibson J, Schouten HC, McCarthy PL, Lonial S, Krishnan AY, Dispenzieri A, Hari PN. Second transplants for multiple myeloma relapsing after a previous autotransplant‐reduced‐intensity allogeneic vs autologous transplantation. Bone Marrow Transplantation. 2014 Mar 1; 49(3):416‐421. doi:10.1038/bmt.2013.187. Epub 2013 Nov 25. PMC3947725. ncbi.nlm.nih.gov/pubmed/ 24270389 Costa LJ, Zhang M‐J, Zhong X, Dispenzieri A, Lonial S, Krishnan A, Freytes C, Vesole D, Gale RP, Anderson K, Wirk B, Savani BN, Waller EK, Schouten H, Lazarus H, Meehan K, Sharma M, Kamble R, Vij R, Kumar S, Nishihori T, Kindwall‐Keller T, Saber W, Hari PN. Trends in utilization and outcomes of autologous transplantation as early therapy for multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Nov 1; 19(11):1615‐1624. doi:10.1016/j.bbmt.2013.08.002. Epub 2013 Aug 10. PMC3952066. ncbi.nlm.nih.gov/pubmed/23939198

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Hale GA, Arora M, Ahn KW, He W, Camitta B, Bishop MR, Bitan M, Cairo MS, Chan K, Childs RW, Copelan E, Davies SM, Perez MAD, Doyle JJ, Gale RP, Vicent MG, Horn BN, Hussein AA, Jodele S, Kamani NR, Kasow KA, Kletzel M, Lazarus HM, Lewis VA, Myers KC, Olsson R, Pulsipher M, Qayed M, Sanders JE, Shaw PJ, Soni S, Stiff PJ, Stadtmauer EA, Ueno NT, Wall DA, Grupp SA. Allogeneic hematopoietic cell transplantation for neuroblastoma: The CIBMTR experience. Bone Marrow Transplantation. 2013 Aug 1; 48(8):1056‐1064. doi:10.1038/bmt.2012.284. Epub 2013 Feb 18. PMC3661721. ncbi.nlm.nih.gov/pubmed/23419433 Michaelis LC, Saad A, Zhong X, Le‐Rademacher J, Freytes CO, Marks DI, Lazarus HM, Bird JM, Holmberg L, Kamble RT, Kumar S, Lill M, Meehan KR, Saber W, Schriber J, Tay J, Vogl DT, Wirk B, Savani BN, Gale RP, Vesole DH, Schiller GJ, Abidi M, Anderson KC, Nishihori T, Kalaycio ME, Vose JM, Moreb JS, Drobyski W, Munker R, Roy V, Ghobadi A, Holland HK, Nath R, To LB, Maiolino A, Kassim AA, Giralt SA, Landau H, Schouten HC, Maziarz RT, Michael J, Kindwall‐Keller T, Stiff PJ, Gibson J, Lonial S, Krishnan A, Dispenzieri A, Hari P. Salvage second hematopoietic cell transplantation in myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 May 1; 19(5):760‐766. doi:10.1016/j.bbmt.2013.01.004. Epub 2013 Jan 5. PMC3816739. ncbi.nlm.nih.gov/pubmed/ 23298856

14.3 Current Studies

MM14‐01 Title: High dose chemotherapy and autologous stem cell transplantation for germ cell tumors PIs: Muna Qayed (Children's Healthcare of Atlanta at Egleston) Thomas Olson (Children's Healthcare of Atlanta at Egleston) Kuang‐Yueh Chiang (Children's Healthcare of Atlanta at Egleston) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018)

MM14‐03 Title: Trends in survival outcomes among patients relapsing early after autologous stem cell transplantation for multiple myeloma PIs: Shaji K. Kumar (Mayo Clinic Rochester) Angela Dispenzieri (Mayo Clinic Rochester) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

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MM15‐02 Title: Post‐relapse Survival Rates after Tandem Auto‐HSCT vs. Auto/Allo‐HSCT in Multiple Myeloma PIs: Amrita Krishnan (City of Hope) Benedetto Bruno (San Giovanni Battista Hospital‐University of Torino) Manish Sharma (Thomas Jefferson University Hospital, Inc.) Myo Htut (City of Hope) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

MM16‐01 Title: Validation of R‐ISS in real world population of Multiple Myeloma undergoing HDM and evaluate outcomes of autologous hematopoietic cell transplantation in patients with high risk multiple myeloma using the international myeloma working group 2014 and 2015 criteria PIs: Sathish Kumar (Singapore General Hospital) Emma Scott (Oregon Health and Science University) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

MM16‐02 Title: Alternative donor allogeneic hematopoietic transplantation strategies for Multiple Myeloma in adult patients: Comparing umbilical cord blood versus haploidentical related donor transplantation PIs: Abraham Kanate (West Virginia University Hospitals, Inc.) Nirav Shah (Medical College of Wisconsin) Qaiser Bashir (M.D. Anderson Cancer Center) Stefan Ciurea (M.D. Anderson Cancer Center) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with EBMT

MM16‐03 Title: Third stem cell transplant for multiple myeloma: An analysis from the CIBMTR database PIs: Rajneesh Nath (Banner MD Anderson Cancer Center) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018)

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MM17‐01 Title: Hematopoietic Cell Transplantation for Primary Plasma Cell Leukemia in the Era of Novel Agents PIs: Saulius Girnius (University of Cincinnati) Sagar S. Patel (Cleveland Clinic Foundation) Lohith S. Bachegowda (M.D. Anderson Cancer Center) Binod Dhakal (Medical College of Wisconsin) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

MM17‐02 Title: The Impact of Bortezomib Based Induction Therapy vs No Induction Therapy on Outcomes for Light Chain Amyloidosis PIs: Robert F. Cornell (Vanderbilt University Medical Center) Luciano Costa (University of Alabama at Birmingham) Stacey A. Goodman (Vanderbilt University Medical Center) Status: Protocol Development (as of July 1, 2017) Submitted (expected by June 30, 2018)

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15.0 PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND NON‐MALIGNANT 2017 Scientific Working Committee Research Portfolio MARROW DISORDERS WORKING COMMITTEE

15.0 PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND NON‐MALIGNANT MARROW DISORDERS WORKING COMMITTEE

15.1 Leadership

Chair: Paolo Anderlini, MD, MD Anderson Cancer Center Email: [email protected] Chair: Neena Kapoor, MD, Children’s Hospital of Los Angeles Email: [email protected] Chair: Jaap‐Jan Boelens, MD, PhD, University Medical Center Utrecht, Pediatrics Email: [email protected] Chair: Vikram Mathews, MD, Christian Medical College Hospital Email: [email protected] Scientific Director: Mary Eapen, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Soyoung Kim, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Kyle Hebert, MS, CIBMTR Milwaukee Email: [email protected]

15.2 Recent Publications

2017 Slack J, Albert MH, Balashov D, Belohradsky BH, Bertaina A, Bleesing J, Booth C, Büchner J, Buckley RH, Ouachée‐Chardin M, Deripapa E, Drabko K, Eapen M, Feuchtinger T, Finocchi A, Gaspar HB, Ghosh S, Gillio A, Gonzalez‐Granado LI, Grunebaum E, Güngör T, Heilmann C, Heminen 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‐Ku?nierz B, Cowan MJ, Fischer A, Gennery AR. Outcome of hematopoietic cell transplantation for DNA‐ double strand breakage repair disorders. Journal of Allergy and Clinical Immunology. doi:10.1016/j.jaci.2017.02.036. Epub 2017 Apr 7. ncbi.nlm.nih.gov/pubmed/28392333

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15.0 PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND NON‐MALIGNANT 2017 Scientific Working Committee Research Portfolio MARROW DISORDERS WORKING COMMITTEE

2016 Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simões B, Ferster A, Dupont S, de la Fuente J, Dalle J‐H, Zecca M, Walters MC, Krishnamurti L, Bhatia M, Leung K, Yanik G, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier JP, Ayas M, Cavazzana M, Matthes‐Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M. Sickle cell disease: An international survey of results of HLA‐identical sibling hematopoietic stem cell transplantation. Blood. 2017 Mar 16; 129(11):1548‐1556. doi:10.1182/blood‐2016‐10‐745711. Epub 2016 Dec 13. PMC5356458. ncbi.nlm.nih.gov/ pubmed/27965196 Gadalla SM, Wang T, Dagnall C, Haagenson M, Spellman SR, Hicks B, Jones K, Katki HA, Lee SJ, Savage SA. Effect of recipient age and stem cell source on the association between donor telomere length and survival after allogeneic unrelated hematopoietic cell transplantation for severe aplastic anemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Dec 1; 22(12):2276‐2282. doi:10.1016/j.bbmt.2016.09.012. Epub 2016 Sep 15. PMC5116252. ncbi.nlm.nih.gov/pubmed/27641680

2015 Ayas M, Eapen M, Le‐Rademacher J, Carreras J, Abdel‐Azim H, Alter BP, Anderlini P, Battiwalla M, Bierings M, Buchbinder DK, Bonfim C, Camitta BM, Fasth AL, Gale RP, Lee MA, Lund TC, Myers KC, Olsson RF, Page KM, Prestidge TD, Radhi M, Shah AJ, Schultz KR, Wirk B, Wagner JE, Deeg HJ. Second allogeneic hematopoietic cell transplantation for patients with Fanconi anemia and bone marrow failure. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1790‐1795. doi:10.1016/j.bbmt.2015.06.012. Epub 2015 Jun 23. PMC4568139. ncbi.nlm.nih.gov/pubmed/ 26116087 Orchard PJ, Fasth AL, Le Rademacher J, He W, Boelens JJ, Horwitz EM, Al‐Seraihy A, Ayas M, Bonfim CM, Boulad F, Lund T, Buchbinder DK, Kapoor N, O'Brien TA, Diaz Perez MA, Veys PA, Eapen M. Hematopoietic stem cell transplantation for infantile osteopetrosis. Blood. 2015 Jul 9; 126(2):270‐276. doi:10.1182/blood‐2015‐01‐625541. Epub 2015 May 26. PMC4497967. ncbi.nlm.nih.gov/pubmed/26012570 Veys PA, Nanduri V, Baker KS, He W, Bandini G, Biondi A, Dalissier A, Davis JH, Eames GM, Egeler RM, Filipovich AH, Fischer A, Jürgens H, Krance R, Lanino E, Leung WH, Matthes S, Michel G, Orchard PJ, Pieczonka A, Ringdén O, Schlegel PG, Sirvent A, Vettenranta K, Eapen M. Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning. British Journal of Haematology. 2015 Jun 1; 169(5):711‐ 718. doi:10.1111/bjh.13347. Epub 2015 Mar 27. PMC4433436. ncbi.nlm.nih.gov/pubmed/ 25817915

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2014 Kim S‐Y, Le Rademacher J, Antin JH, Anderlini P, Ayas M, Battiwalla M, Carreras J, Kurtzberg J, Nakamura R, Eapen M, Deeg HJ. Myelodysplastic syndrome evolving from aplastic anemia treated with immunosuppressive therapy: Efficacy of hematopoietic stem cell transplantation. Haematologica. 2014 Dec 1; 99(12):1868‐1875. doi:10.3324/haematol. 2014.108977. Epub 2014 Aug 8. PMC4258748. ncbi.nlm.nih.gov/pubmed/25107891 Lund TC, Cathey SS, Miller WP, Eapen M, Andreansky M, Dvorak CC, Davis JH, Dalal JD, Devine SM, Eames GM, Ferguson WS, Giller RH, He W, Kurtzberg J, Krance R, Katsanis E, Lewis VA, Sahdev I, Orchard PJ. Outcomes after hematopoietic stem cell transplantation for children with I‐cell disease. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1847‐1851. doi:10.1016/j.bbmt.2014. 06.019. Epub 2014 Jul 10. PMC4194244. ncbi.nlm.nih.gov/pubmed/25016194

2013 Eckrich MJ, Ahn K‐W, Champlin RE, Coccia P, Godder K, Horan J, Margolis D, Deeg HJ, Eapen M. Effect of race on outcomes after allogeneic hematopoietic cell transplantation for severe aplastic anemia. American Journal of Hematology. 2014 Feb 1; 89(2):125‐129. doi:10.1002/ ajh.23594. Epub 2013 Nov 15. PMC4109804. ncbi.nlm.nih.gov/pubmed/24122901 Gadalla SM, Sales‐Bonfim C, Carreras J, Alter BP, Antin JH, Ayas M, Bodhi P, Davis J, Davies SM, Deconinck E, Deeg HJ, Duerst RE, Fasth A, Ghavamzadeh A, Giri N, Goldman FD, Kolb EA, Krance R, Kurtzberg J, Leung WH, Srivastava A, Or R, Richman CM, Rosenberg PS, Sanchez de Toledo Codina J, Shenoy S, Socié G, Tolar J, Williams KM, Eapen M, Savage SA. Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2013 Aug 1; 19(8):1238‐1243. doi:10.1016/j.bbmt.2013.05.021. Epub 2013 Jun 8. PMC3736557. ncbi.nlm.nih.gov/pubmed/23751955 Ayas M, Saber W, Davies SM, Harris RE, Hale GA, Socie G, LeRademacher J, Thakar M, Deeg HJJ, Al‐ Seraihy A, Battiwalla M, Camitta BM, Olsson R, Bajwa RS, Bonfim CM, Pasquini R, MacMillan ML, George B, Copelan EA, Wirk B, Al Jefri A, Fasth AL, Guinan EC, Horn BN, Lewis VA, Slavin S, Stepensky P, Bierings M, Gale RP. Allogeneic hematopoietic cell transplantation for Fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia. Journal of Clinical Oncology. 2013 May 1; 31(13):1669‐1676. doi:10.1200/JCO.2012.45.9719. Epub 2013 Apr 1. PMC3635221. ncbi.nlm.nih.gov/pubmed/ 23547077 Boelens JJ, Aldenhoven M, Purtill D, Ruggeri A, Defor T, Wynn R, Wraith E, Cavazzana‐Calvo M, Rovelli A, Fischer A, Tolar J, Prasad VK, Escolar M, Gluckman E, O'Meara A, Orchard PJ, Veys P, Eapen M, Kurtzberg J, Rocha V. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. Blood. 2013 May 9; 121(19):3981‐3987. doi:10.1182/blood‐2012‐09‐455238. Epub 2013 Mar 14. PMC3836041. ncbi.nlm.nih.gov/pubmed/23493783

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15.3 Current Studies

ID12‐01 Title: Allogeneic Hematopoietic Stem Cell Transplant for Combined Immunodeficiency and Common Variable Immunodeficiency PIs: Geoff Cuvelier (CancerCare Manitoba/University of Manitoba) Greg Guilcher (Alberta Children's Hospital) Nicola Wright (Alberta Children's Hospital) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

AA13‐01 Title: Correlation of levels of donor cell chimerism with hemoglobinopathy symptoms following allogeneic blood stem cell transplantation PIs: Allistar Abraham (Children's National Medical Center) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018)

ID13‐01 Title: Second and subsequent haematopoietic stem cell transplants for Congenital Neutropenia/Kostmann Agranulocytosis PIs: Steven Keogh (The Children's Hospital at Westmead) James Connelly (Vanderbilt University Medical Center) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with Severe Chronic Neutropenia International Registry

AA13‐02 Title: Malignancies in patients with FA PIs: John Wagner (University of Minnesota Blood and Marrow Transplant Program) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with NCI

NM14‐02 Title: Outcomes of allogeneic hematopoietic cell transplant in patients with Shwachman diamond syndrome PIs: Kasiani Myers (Cincinnati Children's Hospital Medical Center) Status: Protocol Development (as of July 1, 2017) Submitted (expected by June 30, 2018)

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15.0 PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND NON‐MALIGNANT 2017 Scientific Working Committee Research Portfolio MARROW DISORDERS WORKING COMMITTEE

NM15‐01 Title: Outcome of Allogeneic Hematopoietic Cell Transplant in Erythropoietic Porphyria PIs: Ayman Saad (University of Alabama at Birmingham) Hisham Abdel‐Azim (Children's Hospital of Los Angeles) Joseph Bloomer (University of Alabama at Birmingham) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with EBMT

NM16‐01 Title: Combined EBMT/CIBMTR retrospective study of allogeneic stem cell transplant outcomes in older patients (age > 50 years) with severe aplastic anemia PIs: Carmel Rice (King's College Hospital) Judith Marsh (King's College Hospital) Victoria Potter (King's College Hospital) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with EBMT

NM16‐02 Title: Allogeneic hematopoietic cell transplantation for primary immune deficiencies: Current patterns of practice and change over the last 10 years PIs: Rebecca Marsh (Cincinnati Children's Hospital Medical Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

NM16‐03 Title: Results of transplants from genetically‐identical twin donors in persons with aplastic anemia PIs: Robert Peter Gale (LifebankUSA) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

NM16‐04 Title: The effect of conditioning regimen on clinical outcomes of allogeneic hematopoietic cell transplantation in severe aplastic anemia PIs: Nelli Bejanyan (University of Minnesota Blood and Marrow Transplant Program) Natasha Kekre (The Ottawa Hospital Blood & Marrow Transplant Program) Daniel Weisdorf (University of Minnesota Blood and Marrow Transplant Program) Joseph Antin (Dana Farber Cancer Institute) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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15.0 PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND NON‐MALIGNANT 2017 Scientific Working Committee Research Portfolio MARROW DISORDERS WORKING COMMITTEE

NM17‐01 Title: Late effects after hematopoietic stem cell transplantation in patients with HLH PIs: AnnaCarin Horne (Karolinska Institutet) Scott Baker (Fred Hutchinson Cancer Research Center) Karin Beutel (Histiocyte Society) Status: Protocol Pending (as of July 1, 2017) Data File Preparation (expected by June 30, 2018) * Collaborative study with EBMT

NM17‐02 Title: Evaluation of the impact of changing clinical profile, transplant conditioning regimens and stem cell source on clinical outcome in patients with Thalassemia major PIs: Vikram Matthews (Christian Medical College Hospital) Chunfu Li (Nanfang Hospital) Suradej Hongeng (Ramathibodi Hospital) Status: Protocol Development (as of July 1, 2017) Analysis (expected by June 30, 2018) * Collaborative study with India, China, Thailand

NM17‐03 Title: Allogeneic hematopoietic stem cell transplantation in alternative donor for sickle cell disease PIs: Mary Eapen (CIBMTR ‐ Milwaukee) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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16.0 REGIMEN‐RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE

16.1 Leadership

Chair: Andrew Artz, MD, MS, University of Chicago Hospitals Email: [email protected] Chair: Alison Loren, MD, MS, Abramson Cancer Center University of Pennsylvania Medical Center Email: [email protected] Chair: Shin Mineishi, MD, University of Alabama Hospital at Birmingham Email: [email protected] Scientific Director: Marcelo Pasquini, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Brent Logan, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Caitrin Fretham, MPH, CIBMTR Milwaukee Email: [email protected]

16.2 Recent Publications

2017 Muffly L, Pasquini M, Martens M, Brazauskas R, Zhu X, Adekola K, Aljurf M, Ballen KK, Bajel A, Baron F, Battiwalla M, Beitinjaneh A, Cahn J‐Y, Carabasi 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. Increasing use of allogeneic hematopoietic cell transplantation in patients age 70 years and older in the United States. Blood. doi:10.1182/blood‐2017‐03‐772368. Epub 2017 Jul 3. ncbi.nlm.nih.gov/pubmed/ 28674027

2016 Martin PJ, Fan W, Storer BE, Levine DM, Zhao LP, Warren EH, Flowers MED, Lee SJ, Carpenter PA, Boeckh M, Hingorani S, Yan L, Hu Q, Preus L, Liu S, Spellman S, Zhu X, Pasquini M, McCarthy P, Stram D, Sheng X, Pooler L, Haiman CA, Sucheston‐Campbell L, Hahn T, Hansen JA. Replication of associations between genetic polymorphisms and chronic graft‐versus‐host disease. Blood. 2016 Nov 17; 128(20):2450‐2456. doi:10.1182/blood‐2016‐07‐728063. Epub 2016 Oct 6. PMC5114491. ncbi.nlm.nih.gov/pubmed/27758874

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Artz AS, Logan B, Zhu X, Akpek G, Bufarull RM, Gupta V, Lazarus HM, Litzow M, Loren A, Majhail NS, Maziarz RT, McCarthy P, Popat U, Saber W, Spellman S, Ringden O, Wickrema A, Pasquini MC, Cooke KR. The prognostic value of serum C‐reactive protein, ferritin, and albumin prior to allogeneic transplantation for acute myeloid leukemia and myelodysplastic syndromes. Haematologica. 2016 Nov 1; 101(11):1426‐1433. doi:10.3324/haematol.2016.145847. Epub 2016 Sep 4. PMC5394859. ncbi.nlm.nih.gov/pubmed/27662010 Magenau J, Westervelt P, Khaled S, McGuirk J, Hari P, Eapen M, Becker PS, Parkin B, Braun T, Logan B, Wang H, Jagasia M, Rowley SD, Kim DD, Schechter T, Frey N, Scott B, Churay T, Lieland S, Forman S, Mineishi S. A multicenter trial of myeloablative clofarabine and busulfan conditioning for relapsed or primary induction failure AML not in remission at the time of allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplantation. 2017 Jan 1; 52(1):59‐65. doi:10.1038/bmt.2016.188. Epub 2016 Jul 18. PMC5215562. ncbi.nlm.nih.gov/pubmed/27427921 Pasquini MC, Le‐Rademacher J, Zhu X, Artz A, DiPersio J, Fernandez HF, Mineishi S, Kamishohara M, Mehta J, Nakamura Y, Ratanatharathorn V, Sobecks R, Burkart J, Bredeson C. Intravenous busulfan‐based myeloablative conditioning regimens prior to hematopoietic cell transplantation for hematologic malignancies. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug 1; 22(8):1424‐ 1430. doi:DOI: 10.1016/j.bbmt.2016.04.013. Epub 2016 May 3. PMC4949158. ncbi.nlm.nih.gov/pubmed/27154848 Strouse C, Richardson P, Prentice G, Korman S, Hume R, Nejadnik B, Horowitz MM, Saber W. Defibrotide for Treatment of Severe Veno‐Occlusive Disease in Pediatrics and Adults: An Exploratory Analysis Using Data from the CIBMTR. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Jul 1; 22(7):1306‐1312. doi:10.1016/j.bbmt.2016.04.011. Epub 2016 Apr 19. PMC4914048. ncbi.nlm.nih.gov/pubmed/27108694

2015 Hahn T, Sucheston‐Campbell LE, Preus L, Zhu X, Hansen JA, Martin PJ, Yan L, Liu S, Spellman S, Tritchler D, Clay A, Onel K, Pasquini M, McCarthy PL. Establishment of definitions and review process for consistent adjudication of cause‐specific mortality after allogeneic unrelated‐ donor hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1679‐ 1686. doi:10.1016/j.bbmt.2015.05.019. Epub 2015 May 29. PMC4537799. ncbi.nlm.nih.gov/ pubmed/26028504 Olsson RF, Logan BR, Chaudhury S, Zhu X, Akpek G, Bolwell BJ, Bredeson CN, Dvorak CC, Gupta V, Ho VT, Lazarus HM, Marks DI, Ringdén OTH, Pasquini MC, Schriber JR, Cooke KR. Primary graft failure after myeloablative allogeneic hematopoietic cell transplantation for hematologic malignancies. Leukemia. 2015 Aug 1; 29(8):1754‐1762. doi:10.1038/leu.2015.75. Epub 2015 Apr 24. PMC4527886. ncbi.nlm.nih.gov/pubmed/25772027

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Sorror ML, Logan BR, Zhu X, Rizzo JD, Cooke KR, McCarthy PL, Ho VT, Horowitz MM, Pasquini MC. Prospective validation of the predictive power of the hematopoietic cell transplantation comorbidity index: A Center for International Blood and Marrow Transplant Research study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Aug 1; 21(8):1479‐1487. doi:10.1016/j.bbmt.2015.04.004. Epub 2015 Apr 7. PMC4512746. ncbi.nlm.nih.gov/pubmed/25862591 Holter‐Chakrabarty JL, Pierson N, Zhang M‐J, Zhu X, Akpek G, Aljurf MD, Artz AS, Baron F, Bredeson CN, Dvorak CC, Epstein RB, Lazarus HM, Olsson RF, Selby GB, Williams KM, Cooke KR, Pasquini MC, McCarthy PL. The sequence of cyclophosphamide and myeloablative total body irradiation in hematopoietic cell transplantation for patients with acute leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1251‐1257. doi:10.1016/j.bbmt.2015.03.017. Epub 2015 Mar 31. PMC4465990. ncbi.nlm.nih.gov/pubmed/25840335 Chen Y‐B, Lane AA, Logan BR, Zhu X, Akpek G, Aljurf MD, Artz AS, Bredeson CN, Cooke KR, Ho VT, Lazarus HM, Olsson RF, Saber W, McCarthy PL, Pasquini MC. Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high‐dose therapy with autologous hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1046‐1053. doi:10.1016/j.bbmt.2015.02.005. Epub 2015 Feb 13. PMC4426014. ncbi.nlm.nih.gov/pubmed/ 25687795

2014 Hong S, Le‐Rademacher J, Artz A, McCarthy PL, Logan BR, Pasquini MC. Comparison of non‐ myeloablative conditioning regimens for lymphoproliferative disorders. Bone Marrow Transplantation. 2015 Mar 1; 50(3):367‐374. doi:10.1038/bmt.2014.269. Epub 2014 Dec 1. PMCID4351124. ncbi.nlm.nih.gov/pubmed/25437248 Aplenc R, Zhang M‐J, Sung L, Zhu X, Ho VT, Cooke K, Dvorak C, Hale G, Isola LM, Lazarus HM, McCarthy PL, Olsson R, Pulsipher M, Pasquini MC, Bunin N. Effect of body mass in children with hematologic malignancies undergoing allogeneic bone marrow transplantation. Blood. 2014 May 29; 123(22):3504‐3511. doi:10.1182/blood‐2013‐03‐490334. Epub 2014 Apr 7. PMC4041168. ncbi.nlm.nih.gov/pubmed/24711663

2013 Bredeson C, LeRademacher J, Kato K, DiPersio JF, Agura E, Devine SM, Appelbaum FR, Tomblyn MR, Laport GG, Zhu X, McCarthy PL, Ho VT, Cooke KR, Armstrong E, Smith A, Rizzo JD, Burkart JM, Pasquini MC. Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation. Blood. 2013 Dec 5; 122(24):3871‐3878. doi:10.1182/blood‐2013‐08‐519009. Epub 2013 Sep 30. PMC3854109. ncbi.nlm.nih.gov/ pubmed/24081656

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2012 Akpek G, Pasquini MC, Logan B, Agovi M‐A, Lazarus HM, Marks DI, Bornhaeüser M, Ringdén O, Maziarz RT, Gupta V, Popat U, Maharaj D, Bolwell BJ, Rizzo JD, Ballen KK, Cooke KR, McCarthy PL, Ho VT. Effects of spleen status on early outcomes after hematopoietic cell transplantation. Bone Marrow Transplantation. 2013 Jun 1; 48(6):825‐831. doi:10.1038/bmt.2012.249. Epub 2012 Dec 10. PMC3606905. ncbi.nlm.nih.gov/pubmed/23222382

16.3 Current Studies

RT07‐01b Title: Prospective validation of the impacts of the HCT comorbidity index, alone and combined with aging on HCT outcomes for non‐malignant diseases PIs: Mohamed Sorror (Fred Hutchinson Cancer Research Center) Monica Thakar (Medical College of Wisconsin) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018)

RT09‐04/IB09‐06b Title: Genetic susceptibility to transplant‐related mortality after unrelated donor stem cell transplant PIs: Theresa Hahn (Roswell Park Cancer Institute) Status: Manuscript Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018) * Collaborative study with Roswell Park Cancer Institute

RT09‐04 / IB09‐06c Title: A case‐control genome‐wide association study (GWAS) of AML and MDS with the Genetics and Epidemiology of Myeloid Malignancies (GEMM) consortium PIs: Kenan Onel (Comer Children's Hospital/University of Chicago Medicine) Theresa Hahn (Roswell Park Cancer Institute) Lara Sucheston‐Campbell (Ohio State Medical Center, James Cancer Center) Status: Submitted (as of July 1, 2017) Published (expected by June 30, 2018) * Collaborative study with GEMM consortium

RT13‐02 Title: Safety of high‐dose TBI followed by an allogeneic stem cell transplant for hematologic malignancies PIs: Mitchell Sabloff (The Ottawa Hospital Blood & Marrow Transplant Program) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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RT14‐01 Title: Trends and risk factors for infant mortality following allogeneic hematopoietic cell transplant: Case‐Control study PIs: Prakash Satwani (NYPH/ Columbia University Medical Center) Suhag Parikh (Duke University Medical Center) Status: Data File Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

RT14‐02 Title: Endothelial injury complications after allogeneic hemotapoietic cell transplantation PIs: Sonata Jodele (Cincinnati Children's Hospital Medical Center) Benjamin Laskin (Cincinnati Children's Hospital Medical Center) Stella M Davies (Cincinnati Children's Hospital Medical Center) Muthalagu Ramanathan (UMass Memorial Medical Center) Wichai Chinratanalab (Vanderbilt University Medical Center) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

RT14‐03 Title: Multicenter cohort identification of transplant‐related risk‐factors for infection, organ failure, and mortality among pediatric hematopoietic stem cell transplant patients requiring intensive care unit admission PIs: Matt Zinter (University of California San Francisco Medical Center) Chris Dvorak (University of California San Francisco Medical Center) Anil Sapru (University of California San Francisco Medical Center) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018) * Collaborative study with Virtual PICU Systems

RT15‐01 Title: Comparison of outcomes for myeloablative conditioning regimens combining busulfan with either cyclophosphamide or fludarabine PIs: Andrew Harris (Utah Blood and Marrow Transplant Program) John Levine (Mount Sinai Medical Center) Status: Manuscript Preparation (as of July 1, 2017) Submitted (expected by June 30, 2018)

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RT15‐02 Title: Association of anti‐epileptic medication with outcomes after conditioning with targeted busulfan followed by cyclophosphamide before allogeneic hematopoietic cell transplantation PIs: Paul J. Martin (Fred Hutchinson Cancer Research Center) Jeannine S. McCune (City of Hope) Status: Data File Preparation (as of July 1, 2017) Manuscript Preparation (expected by June 30, 2018)

RT16‐01 Title: Effect of BEAM dose adjustments on the outcomes of patients with lymphoma or multiple myeloma PIs: Claudio Brunstein (University of Minnesota Blood and Marrow Transplant Program) John Rogosheske (University of Minnesota Blood and Marrow Transplant Program) Miguel‐Angel Perales (Memorial Sloan Kettering Cancer Center) Status: Analysis (as of July 1, 2017) Submitted (expected by June 30, 2018)

RT16‐02 Title: Evaluation of lung toxicity following allogeneic stem cell transplant with fludarabine/total body irradiation conditioning regimen PIs: Ayman Saad (University of Alabama at Birmingham) Kentaro Mingawa (Penn State Hershey Medical Center) Yoshinobu Kanda (Saitama Medical School) Shin Mineishi (Penn State Hershey Medical Center) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

RT17‐01 Title: Allogeneic hematopoietic stem cell transplant outcome for patients with end stage renal disease on dialysis PIs: Nosha Farhadfar (Shands HealthCare & University of Florida) John Wingard (Shands HealthCare & University of Florida) Hemant Murthy (H. Lee Moffitt Cancer Center and Research Institute) Status: Protocol Development (as of July 1, 2017) Data File Preparation (expected by June 30, 2018)

Page | 98 2017 Scientific Working Committee Research Portfolio APPENDIX A: COLLABORATIVE STUDIES

APPENDIX A: COLLABORATIVE STUDIES

Alliance  LK15‐01: Allogeneic transplants vs other consolidation in elderly AML o Also in collaboration with Eastern Cooperative Oncology Group (ECOG) and Southwest Oncology Group (SWOG)

American Association of Blood Banks (AABB)  DS17‐02: Impact of pre‐operative collection of auto blood for BM harvest on donor health and outcome o Also in collaboration with American Society for Blood and Marrow Transplantation (ASBMT)

American Society for Blood and Marrow Transplantation (ASBMT)  DS17‐02: Impact of pre‐operative collection of auto blood for BM harvest on donor health and outcome o Also in collaboration with American Association of Blood Banks

Cancer and Leukemia Group B  LK15‐03: Comparison of outcomes of older adolescents and young adults with Philadelphia‐ chromosome/BCR‐ABL1‐negative acute lymphoblastic leukemia receiving post‐remission consolidation chemotherapy with pediatric‐inspired chemotherapy on CALGB 10403 or myeloablative allogeneic hematopoietic cell transplantation

China  NM17‐02: Evaluation of the impact of changing clinical profile, transplant conditioning regimens and stem cell source on clinical outcome in patients with Thalassemia major o Also in collaboration with India and Thailand

Dana‐Farber Cancer Institute  CK14‐02: Validation of DFCI prognostic score for previously treated chronic lymphocytic leukemia patients who underwent reduced intensity conditioning allogeneic HSCT  IB13‐08: Short and long term survival assessment of post‐HSCT transplantation using predictive modeling on a Bayesian network framework

Deutsche Knochenmarkspenderdatei GmbH (DKMS; German Bone Marrow Donor Center)  IB11‐01a: Analysis of the NIMA effect on the outcome of unrelated PBSC/BM transplantation  IB11‐01b: Analysis of the IPA effect on the outcome of unrelated PBSC/BM transplantation

Page | 99 2017 Scientific Working Committee Research Portfolio APPENDIX A: COLLABORATIVE STUDIES Duke University Medical Center  CK15‐01: Comparison of Transplant vs non Transplant Therapies for Myelofibrosis o Also in collaboration with M.D. Anderson Cancer Center, Massachusetts General Hospital, Mayo Clinic Arizona and Phoenix Children's Hospital, and Vanderbilt University Medical Center

Eastern Cooperative Oncology Group (ECOG)  LK15‐01: Allogeneic transplants vs other consolidation in elderly AML o Also in collaboration with Alliance and Southwest Oncology Group (SWOG) Emory University  LE17‐01: Long‐term follow up after hematopoietic stem cell transplantation for sickle cell disease

European Society for Blood and Marrow Transplantation (EBMT)  AC14‐01: Long term outcomes after AutoHCT for rapidely progressive systemic scleroderma  CK17‐01: Development of a Prognostic Scoring System Predictive of outcomes in patients with Myelofibrosis after Allogeneic Hematopoietic Cell Transplantation  GS16‐03: Donor selection for allogeneic stem cell transplantation: A case‐control comparison of children (using post‐transplantation cyclophosphamide in GVHD prophylaxis) versus HLA‐ matched siblings  LY06‐03: HLA identical sibling transplantation versus HLA‐matched unrelated donor transplantation in patients with follicular lymphoma  LY16‐02: Alternative donor source stem cell transplant vs matched donor stem cell transplant for Hodgkin lymphoma  LY16‐03: Haploidentical transplantation in diffuse large B cell lymphoma: A CIBMTR and EBMT collaborative study  MM16‐02: Alternative donor allogeneic hematopoietic transplantation strategies for Multiple Myeloma in adult patients: Comparing umbilical cord blood versus haploidentical related donor transplantation  NM15‐01: Outcome of Allogeneic Hematopoietic Cell Transplant in Erythropoietic Porphyria  NM16‐01: Combined EBMT/CIBMTR retrospective study of allogeneic stem cell transplant outcomes in older patients (age > 50 years) with severe aplastic anemia  NM17‐01: Late effects after hematopoietic stem cell transplantation in patients with HLH

Genetics and Epidemiology of Myeloid Malignancies Consortium  RT09‐04 / IB09‐06c: A case‐control genome‐wide association study (GWAS) of AML and MDS with the Genetics and Epidemiology of Myeloid Malignancies (GEMM) consortium

Harvard School of Public Health  GV17‐04: Analysis of semi‐competing risks data from a nested case‐control study, applied to acute graft‐versus‐host disease and death after hematopoietic stem cell transplantation

India  NM17‐02: Evaluation of the impact of changing clinical profile, transplant conditioning regimens and stem cell source on clinical outcome in patients with Thalassemia major o Also in collaboration with China and Thailand Page | 100 2017 Scientific Working Committee Research Portfolio APPENDIX A: COLLABORATIVE STUDIES International Histocompatibility Working Group  IB06‐05: Use of high‐resolution HLA data from the NMDP for the International Histocompatibility Working Group in HCT  IB07‐04: Employing advanced bioinformatics methods for predicting peptide specificities of HLA molecules in the characterization of permissible mismatches in hematopoietic cell transfer  IB09‐01: Clinical importance of minor histocompatibility complex haplotypes in umbilical cord blood transplantation  IB09‐03: Clinical relevance of cytokine/immune response gene polymorphisms in umbilical cord blood transplantation  IB09‐05: Identification of functional single nucleotide polymorphisms in umbilical cord blood transplantation  IB09‐07: Clinical significance of genome‐wide variation in unrelated HCT  IB11‐08: Synergism between minor and major histocompatibility antigens  IB14‐07: Indirectly recognizable HLA epitopes (PIRCHES): A retrospective validation study on the role of indirect recognition of mismatched HLA in hematopoietic stem cell transplantation outcome  R04‐74d: Functional significance of killer cell immunoglobulin‐like receptor genes in HLA‐ matched and mismatched unrelated HCT

M.D. Anderson Cancer Center  CK12‐01: A decision analysis of the optimal timing of allogeneic hematopoietic stem cell transplantation in chronic myeloid leukemia in the era of Tyrosine Kinase Inhibitors  CK15‐01: Comparison of Transplant vs non Transplant Therapies for Myelofibrosis o Also in collaboration with Duke University Medical Center, Massachusetts General Hospital, Mayo Clinic Arizona and Phoenix Children's Hospital, and Vanderbilt University Medical Center

Massachusetts General Hospital  CK15‐01: Comparison of Transplant vs non Transplant Therapies for Myelofibrosis o Also in collaboration with Duke University Medical Center, M.D. Anderson Cancer Center, Mayo Clinic Arizona and Phoenix Children's Hospital, and Vanderbilt University Medical Center

Mayo Clinic Arizona and Phoenix Children's Hospital  CK15‐01: Comparison of Transplant vs non Transplant Therapies for Myelofibrosis o Also in collaboration with Duke University Medical Center, M.D. Anderson Cancer Center, Massachusetts General Hospital, and Vanderbilt University Medical Center

Medical College of Wisconsin  IB15‐04: Clinical outcomes among hematopoietic stem cell transplant recipients as a function of socioeconomic status and related transcriptome differences

Page | 101 2017 Scientific Working Committee Research Portfolio APPENDIX A: COLLABORATIVE STUDIES National Cancer Institute  IB10‐01d: Flow Cytometry using FISH techniques in a Severe Aplastic Anemia population  AA13‐02: Malignancies in patients with FA  IB15‐05: Secondary Findings in Exome Sequencing Data  IB15‐06a: Donor Telomere Length and Outcomes After Hematopoietic Stem Cell Transplantation for Acute Leukemia o Also in collaboration with Telomere Diagnostics, Inc.  IB16‐03: Role of recipient and donor genetic polymorphisms in interferon lambda 4 (INFL4) on outcomes after unrelated allogeneic cell transplant  IB17‐03: Identification of genomic markers of post hematopoietic cell transplantation (HCT) outcomes in patients with myelofibrosis: A pilot study

National Institutes of Health  CK12‐02b: A retrospective assessment of outcomes of patients who have undergone allogeneic stem cell transplant for Chronic Lymphocytic Leukemia based on histocompatibility leukocyte antigen type  DS16‐S1: Evaluation of practice guidelines for the assessment and surveillance follow‐up of pediatric hematopoietic stem cell donors o Also in collaboration with University of Pittsburgh

Pediatric Health Information System® (PHIS)  GS17‐01: Comparing outcomes between cord blood and haploidentical transplants in patients with acute myeloid leukemia  HS13‐02: Investigating inpatient health care utilization of matched sibling donor hematopoietic cell transplantation for children with sickle cell disease  HS14‐01: Investigating clinical outcomes and inpatient health care resource utilization of hematopoietic cell transplantation for children with acute leukemia

Roswell Park Cancer Institute  IB09‐06b / RT09‐04b: Genetic susceptibility to transplant‐related mortality after unrelated donor stem cell transplant

Severe Chronic Neutropenia International Registry  ID13‐01: Second and subsequent haematopoietic stem cell transplants for Congenital Neutropenia/Kostmann Agranulocytosis

Southwest Oncology Group (SWOG)  LK15‐01: Allogeneic transplants vs other consolidation in elderly AML o Also in collaboration with Alliance and Eastern Cooperative Oncology Group (ECOG)

Telomere Diagnostics, Inc.  IB15‐06a: Donor telomere length and outcomes after HCT for acute leukemia o Also in collaboration with National Cancer Institute  IB15‐06b: Using Telomatch Testing on Donor Telomere Length and Outcomes After Hematopoietic Stem Cell Transplantation for Acute Leukemia

Page | 102 2017 Scientific Working Committee Research Portfolio APPENDIX A: COLLABORATIVE STUDIES Thailand  NM17‐02: Evaluation of the impact of changing clinical profile, transplant conditioning regimens and stem cell source on clinical outcome in patients with Thalassemia major o Also in collaboration with China and India

University of Chicago  CK16‐01: Identification of germline predisposition mutations in young MDS patients

University of Florida  LE99‐01: Quality of life in late HCT survivors

University of Minnesota  IB14‐05: mtDNA haplotypes and unrelated donor transplant outcomes  IB15‐02: Natural killer cell genomics and outcomes after allogeneic transplantation for chronic lymphocytic leukemia  IB15‐03: Killer Immunoglobulin Receptor (KIR) Gene Content and Pediatric Acute Leukemia Transplant Outcomes  R02‐40 / R03‐63d: Acquisition of natural killer cell receptors in recipients of unrelated transplant

University of Pittsburgh  DS16‐S1: Evaluation of practice guidelines for the assessment and surveillance follow‐up of pediatric hematopoietic stem cell donors o Also in collaboration with Natoinal institutes of Health

University of Utah  DS05‐02a: Older adult related donors compared to adult related donors  DS05‐02c: Acute toxicities of related adult donors compared to unrelated adult  DS05‐02d: QoL for related adult donors compared to unrelated adult donors  DS05‐02e: Acute toxicities for pediatric related donors compared to adult related donors  DS05‐02g: Late toxicities and SAE for related donors

United Network for Organ Sharing  LE12‐03: Solid after hematopoietic cell transplantation

Vanderbilt University Medical Center  CK15‐01: Comparison of Transplant vs non Transplant Therapies for Myelofibrosis o Also in collaboration with Duke University Medical Center, M.D. Anderson Cancer Center, Massachusetts General Hospital, and Mayo Clinic Arizona and Phoenix Children's Hospital

Virtual PICU Systems  RT14‐03: Multicenter cohort identification of transplant‐related risk‐factors for infection, organ failure, and mortality among pediatric hematopoietic stem cell transplant patients requiring intensive care unit admission

Page | 103 APPENDIX B: STUDY DEVELOPMENT 2017 Scientific Working Committee Research Portfolio AND MANAGEMENT PROCESS APPENDIX B: STUDY DEVELOPMENT AND MANAGEMENT PROCESS This study development cycle pertains to studies for which CIBMTR provides data, scientific, and statistical support. Data sets are also made available to investigators who have their own statistical resources. 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 Planned creates a 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.

Sample typing. If applicable, the PIs perform laboratory tests (e.g., In Progress 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 transplant centers and supplement forms reimbursement costs.

Page | 104 APPENDIX B: STUDY DEVELOPMENT 2017 Scientific Working Committee Research Portfolio 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 Preliminary analyses are accurate and contribute to the fundamental message of the Results manuscript. The 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.

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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 forwards all editor and reviewer comments to the PI and Preliminary Statistical Director. The PI is expected to prepare a response, working with Results Study Leadership who provides additional analyses of data, as needed. (continued) Coordinating Center personnel 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.

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APPENDIX C: GUIDELINES FOR STUDY PRINCIPAL INVESTIGATORS The role of a Study Chair / PI is simple: always behave ethically and do whatever it takes to complete the study that answers your research question. It is easier to accomplish this task if you have an understanding of the CIBMTR study process, specifically where and when your efforts are most needed. The following document will explain the life‐cycle of a CIBMTR observational study and review the responsibilities of a PI. Hints and tips to make the study process as successful as possible are noted with an arrow (→).

STUDY PROPOSAL The PI is usually the first person who suggests the study and who prepares the Study Proposal (cibmtr.org/Studies/Observational/ProposeStudy/Pages/index.aspx). Ideally the PI presents his or her proposal in person to the appropriate Working Committee at a BMT Tandem Meetings. Some PIs view the CIBMTR presentation as a formality but, in reality, it is an important opportunity to convince the other Working Committee members that your study is more important, more feasible, and more likely to advance the field and be published in a high‐profile journal than other studies being proposed. CIBMTR Working Committee hours and resources are limited, and not all good studies proposed can be supported. → Review the data collection forms (cibmtr.org/DataManagement/DataCollectionForms/Pages/ Investigators.aspx) to ensure the data you wish to study are available for the timeframe you wish to study. Many people propose studies that require data not collected routinely by the CIBMTR. Studies that require additional data collection usually get greater scrutiny because of the extra time and effort required whenever centers have to be contacted for additional data. Additionally, the response rate to requests for supplemental data is often disappointing; ask your own data managers how difficult it is to go back and find data for patients transplanted years ago. The ability to collect supplemental data successfully depends on how complex and / or extensive the data are, the size of the study population, how far back in time the transplants were done, and whether you have resources (people or funds) to assist in the process. → Examine the latest accrual tables for your proposed Working Committee to ensure there are sufficient data in the CIBMTR Research Database to answer your study questions. Accrual tables are provided in the meeting agenda for each committee’s annual in person meeting at the BMT Tandem Meetings under Attachment 2. → Review this report and / or the CIBMTR Publication List and Working Committee Study Lists of planned, in progress, and recently published studies to ensure someone else has not already conducted the study you are proposing. → Note that study proposals may be submitted throughout the year. The vast majority are submitted just before the deadline, three months before the BMT Tandem Meetings. If you want your proposal to benefit from greater CIBMTR statistical and scientific input, then submitting your proposal far in advance is helpful. Proposals submitted throughout the year will be reviewed by the Working Committee Chairs and Scientific Director. They have the authority to approve a proposal based on the importance of the scientific question or they may elect to defer it until presentation at the Working Committee meeting.

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PRIORITIZATION AND DISTRIBUTION OF STATISTICAL HOURS After the BMT Tandem Meetings, the Working Committee Chairs, Scientific Directors, Statistical Director, and MS Statistician meet to discuss the results of the meeting and prioritize new and ongoing studies. Studies are assigned Coordinating Center hours according to their need and priority. In general, a study needs 40‐80 hours of Statistical Staff time to finish the protocol document, 80‐160 hours to prepare the data file (depending on whether additional data collection, follow‐up, or excessive data cleaning is necessary), 80 hours for the analysis phase, and 40‐60 hours for manuscript preparation. PIs are generally notified about Committee decisions (i.e., approval, prioritization, and assigned hours) regarding their proposals within one month after the meeting. At that time, PIs also learn which MS‐level Statistician is assigned to their studies; this MS‐level Statistician subsequently serves as the point person for communications regarding study issues. → PIs can increase the chance of their proposal being approved by carefully preparing the Proposal Form that is presented to the Working Committee. Discussion with Working Committee Leadership in advance of the Working Committee meeting may help clarify the study and address study design questions. Many great concepts fail because PIs do not consider available data, size of the available study population, power calculations, and other statistical issues. The Working Committee is much less receptive to studies that appear to have multiple unresolved issues at the meeting.

STUDY PROTOCOL The next step in the study’s life is generation of the study protocol. This is an important document that is first drafted by the PI and submitted to the MS‐level Statistician. The draft study protocol should be completed within two months of concept approval notification. In preparing this document, it is crucial to carefully review the applied study selection criteria and description of patient, disease, and transplant characteristics. The PI must also carefully consider the variables to be included in the analysis because the MS‐level Statisticians, Statistical Directors, and Scientific Directors use these documents to guide data collection and cleaning. Common pitfalls include failure to include important variables to address study hypotheses and failure to consider potentially confounding variables. After the initial draft is reviewed and approved by the Coordinating Center, it is circulated to the Working Committee for comment; at that time, Committee members may request to participate in the study and a Writing Committee is formed (see below). Individuals wishing to serve on the Writing Committee provide substantive comments on the study protocol. It is the PI’s responsibility to collate and address these comments by either modifying the protocol or providing an explanation for not incorporating suggested changes. Since Writing Committee members earn their authorship by reviewing the study protocol, analyses, and manuscripts, the CIBMTR also keeps track of comments and contributions. → Each study protocol is reviewed at the weekly Coordinating Center conference call / meeting (held on Tuesdays, 9:30‐10:30 am US Central Time) before distribution to the Working Committee; it is very helpful for the PI to join that meeting by phone and to participate in the discussion of the study’s design and implementation. (Studies are again discussed at a Coordinating Center weekly meeting as they reach significant milestones. PI participation in each of these discussions is strongly encouraged.)

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→ The most successful PIs respond to Writing Committee critiques as they do journal reviews — by carefully organizing them and responding to each. If a Writing Committee member brought up an issue, it is likely that a reviewer will also bring up the same points. It is expected that the PI will summarize and respond to these critiques within three weeks after the deadline for comments has passed. → PIs have a great deal of control over the interval between study proposal approval and the completion of a final study protocol. Timely submission of the draft protocol and response to Writing Committee comments can vault your study ahead of others in terms of Coordinating Center priority. If yours is ready to go and another is not, yours may be given priority, even if initially it was planned for the other study to be done first.

DATA COLLECTION If supplemental data collection is needed for the study, approval from the Chief Scientific Director is required. The PI needs to provide the following information for the approval: 1) number of questions, 2) types of questions, 3) number of cases and 4) the study calendar. Once the request has been approved, the Forms Development Clinical Research Coordinator will prepare a supplemental form for review within one week. This draft form will be a Word document listing all the supplemental questions that are relevant, as well as the most frequent response options. This form will have input from the Scientific Director, PI, Study Statistician, Metadata and Data Operations Staff for clarity, length, internal consistency of response options, and feasibility of data retrieval. The form will be formatted to be consistent with other CIBMTR forms, and a table will be created in the database to receive the data. This step is very important for any study collecting additional data. If the form is long or leaves out critical variables, the ultimate study results could be compromised by missing data. The supplemental form will go to the Chief Scientific Director, Scientific Director, and PI for final approval. The Scientific Director and PI will prepare a letter detailing the importance of the data needed for the study with a copy to the Medical Director. This letter will be sent with the study request. If terms or concepts on the supplemental data collection form are unfamiliar to the data management teams, an instruction manual that describes the variable and provides examples of how data managers should interpret primary data will have to be written. Each study is assigned a Clinical Research Coordinator who communicates with centers to facilitate data submission. Most, but not all, centers are very responsive to these requests. If some centers are lagging behind in submitting extra forms, PIs may need to make personal email or phone appeals. → Providing the initial draft form and content for the instruction manual is the responsibility of the PI. Delay in putting it together can significantly delay initiating the data collection process. If the process is inordinately delayed so that the data needed for a study is not available in a timely manner, the study may be deferred to the next year. → For smaller studies, where every patient counts, personal appeals from the PI to the Transplant Center Director can sometimes be very effective.

DATA FILE PREPARATION In this step, the MS‐level Statistician prepares a data file using the finalized study protocol as guidance. Data interpretation issues may arise here, especially if uncommon variables are necessary for the study. Values for common variables have probably already been reviewed and, if missing or out of range or inconsistent, already clarified (data “cleaning”) for other studies. If

Page | 109 2017 Scientific Working Committee Research Portfolio APPENDIX C: GUIDELINES FOR STUDY PIS your study is the first to examine a particular variable or study population, then expect to do a lot of data cleaning. → The PI can accelerate this process by being available to the MS‐level Statistician and Scientific Director as questions come up. The PI should also carefully review the frequencies of study variables for outliers and other clinical inconsistencies.

UNIVARIATE ANALYSIS Once the data file is prepared, the MS‐level Statistician performs as much of the analysis as possible before handing the data set to the Statistical Director assigned to the project. First, a table of study population characteristics and preliminary univariate analysis is prepared. This is reviewed by the PI and Scientific Director. When they are satisfied with the population, the study is scheduled for another Coordinating Center weekly meeting / conference call to confirm final composition of the population and study design and review the univariate analysis before multivariate analyses are performed. Relevant comments from the Coordinating Center review will be summarized by the MS‐level Statistician or the Scientific Director and relayed back to the PI for comment if the PI cannot participate personally in the meeting. → As noted above, the PI is invited to participate in the CIBMTR Coordinating Center Meeting (Tuesdays) when his or her study is discussed. It is worth repeating that it is very helpful for the PI to participate since they can often address questions as they arise so that the statistical input is most helpful.

MULTIVARIATE ANALYSIS Once the population characteristics and univariate analyses are approved, the data file is transmitted to the Statistical Directors for multivariate and more complex modeling. When completed, results are sent to the PI and Scientific Director who present them on a weekly Coordinating Center conference call. The PI and Scientific Director address comments provided at the meeting and then prepare a memo for circulation to the Writing Committee for comments. The comment period usually lasts two to three weeks. The PI summarizes the comments and prepares another memo for the Writing Committee within three weeks of the close of the comment period. If substantive issues arise, especially related to the study population or analyses, then a conference call involving the PI, Scientific Director, Statistical Director, and MS‐ level Statistician may need to be convened to plan an approach for addressing the comments. → The most successful PIs take advantage of the MS‐level Statisticians’ and Statistical Directors’ familiarity with the project and the data to finish their analyses quickly. If extended time passes between each phase of the analysis, the Statisticians will have to re‐familiarize themselves with the project and coding. A task that could take a couple of hours immediately after the initial results are completed may take much longer a month or two later (and the Statisticians understandably will be less excited about picking up the project again).

ABSTRACTS Many PIs hope to submit abstracts to national and international meetings. Multivariate analyses must be complete with enough time to allow generation of an abstract. These abstracts must be circulated to the Writing Committee and reviewed by the Coordinating Center faculty and staff prior to submission. Please allow enough time to complete these steps before the abstract deadline. If the abstract is accepted for oral presentation, the Coordinating Center staff will also

Page | 110 2017 Scientific Working Committee Research Portfolio APPENDIX C: GUIDELINES FOR STUDY PIS need to review the slides, primarily for accuracy but sometimes also to make suggestions for clarity. The CIBMTR has a template for format and background that is required for all presentations. → Planning for meeting abstracts for the American Society of Hematology and other meetings happens immediately after the BMT Tandem Meetings. If you would like to submit your abstract to one of these meetings, an early declaration of your intentions and demonstrable effort in moving towards that goal will result in your study getting higher priority. → In general, studies are only submitted to one meeting; once submitted in abstract form, priority should be placed on writing and submitting the manuscript.

MANUSCRIPT Once the analysis is completed, drafting the manuscript is the responsibility of the PI. A draft manuscript is expected within 30 days of the final analysis. The draft is circulated to the Writing Committee and comments are again summarized and incorporated. At least one round and sometimes up to three or more rounds are necessary to create a final manuscript. The CIBMTR will do the final formatting for journal submission, attach all the co‐authors’ information (such as institution and contact information), collect any necessary signatures, and submit the paper. The CIBMTR has a long list of acknowledgment for funding sources that are attached to the paper. → The initial manuscript draft usually causes the greatest delay in study progress and is the step most directly under control of the PI. The most successful PIs recognize that publishing their study results is a critical measure of success for all involved parties ‐ themselves, the CIBMTR and all the collaborators involved in the study. Working Committee Chairs have the authority to re‐assign a study to a different PI if the delay in manuscript preparation is too long (>60 days).

ACCEPTANCE Unless the paper is accepted on the first submission, it will need to be revised or resubmitted. If comments are straightforward, the PI can prepare a response to reviewers for circulation, along with the revised version. Some comments from reviewers require additional analyses or discussion at a Coordinating Center meeting prior to resubmission. The CIBMTR will assist with manuscript resubmission. Once the paper is accepted, the PI also handles proof review. → Unless a study is completed in record time, it will be “in progress” at the next BMT Tandem Meetings. PIs should plan to present a study update at the CIBMTR Working Committee meetings or designate another person on the Writing Committee to do this, as long as the study is active. → Any expected or unexpected deviations from the above timetable should be discussed between the PI and Working Committee Leadership. Sometimes unavoidable delays are due to either the CIBMTR or the PI. A proactive plan designed to keep the study moving forward should be devised. Generally, the CIBMTR expects studies to be completed within 18‐24 months.

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APPENDIX D: GLOSSARY Abbreviation/ Meaning Acronym AABB American Association of Blood Banks ABO Landsteiner's blood grouping system ALL acute lymphoblastic leukemia alloHCT allogeneic hematopoietic cell transplantation AML acute myeloid (myelogenous) leukemia APL acute promyelocytic leukemia ASBMT American Society for Blood and Marrow Transplantation autoHCT autologous hematopoietic cell transplantation BEAM carmustine (BCNU), etoposide, cytarabine, and melphalan BM bone marrow BMT bone or blood marrow transplant BMT CTN Blood and Marrow Transplant Clinical Trials Network BSI bacterial bloodstream infections CAC Consumer Advocacy Committee CALGB Cancer and Leukemia Group B (member Alliance for Clinical Trials in Oncology) CIBMTR Center for International Blood and Marrow Transplant Research CMV cytomegalovirus CNS central nervous system CR2 second complete remission CRF Comprehensive Report Form DFCI Dana Farber Cancer Institute DLBCL diffuse large B‐cell lymphoma DNA deoxyribonucleic acid DRI disease risk index EBMT European Society for Blood and Marrow Transplantation ECOG Eastern Cooperative Oncology Group EWOG‐MDS European Working Group of Myelodysplastic Syndromes in Childhood FA Fanconi anemia FACT Foundation for the Accreditation of Cellular Therapy FISH Flourescence in situ Hybridisation FL follicular lymphoma FLT3 FMS like tyrosine kinase 3 G‐CSF subcutaneous filgrastim GEMM Genetics and Epidemiology of Myeloid Malignancies GRFS relapse‐free survival GVHD graft‐versus‐host disease GWAS genome‐wide association study HCT or HSCT hematopoietic stem cell transplantation HLA human leukocyte antigen

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Abbreviation/ Meaning Acronym JACIE Joint Accreditation Committee – International Society for Cellular Therapy & European Society for Blood and Marrow Transplantation JMML Juvenile Myelomonocytic Leukemia KIR killer‐cell immunoglobulin‐like receptors KIR3DL1 killer cell immunoglobulin like receptor, three Ig domains and long cytoplasmic tail 1 LEP late early phase MCW Medical College of Wisconsin MDS myelodysplastic syndrome MDSC myeloid‐derived suppressor cells mtDNA mitochondrial DNA MHC major histocompatibility complex MLL mixed lineage leukemia MS Master of Science (level statistician) NCI National Cancer Institute NK natural killer (cell) NIH National Institutes of Health NIMA National Integrated Medical Association NMDP National Marrow Donor Program PBSC peripheral blood stem cell PBSCT peripheral blood stem cell transplantation Ph+ Philadelphia chromosome positive PHIS Pediatric Health Information System® PI principal investigator PICU pediatric intensive care unit PIRCHES predicted indirectly recognizable HLA epitopes PMCID PubMed Central unique identifier QoL quality of life SNP single nucleotide gene polymorphism SWOG Southwest Oncology Group SWOT strengths, weaknesses, opportunities, threats TBI total body irradiation TNFSF4 tumor necrosis factor superfamily member 4 TRM transplantation‐related mortality US United States VEP very early phase VRE vancomycin‐resistant enterococcus vs versus

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