ASBMT eNews AMERICAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION

September 2017 IN THIS ISSUE

1, 15 Clinical Research

2, 3 A Word From the President CLINICAL RESEARCH 3, 6-8, 13 Legislation & Regulation Effective Transplantation Option to Matched Donor Transplant

Children with acute leukemia (GVHD), but none of the patients 4-6 BMT Tandem Meetings who receive haploidentical developed extensive chronic hematopoietic cell transplantation GVHD. Approximately four 9-12 Association News (HCT) following αβ T- and B-cell years after HCT, the five-year depletion have similar survival probability of survival free of 13, 14 FACT Update outcomes as children who receive chronic GVHD and relapse was HLA-matched donor HCT, 71%. The researchers determined 16 Translational Science according to a study published in that the total body irradiation- Studies Blood. This clinical trial was containing preparative regimen was conducted with 80 children, who the only variable positively 17 Calendar of Events received a fully myeloablative influencing survival without preparative regimen. Three to five relapse or chronic GVHD. days prior to transplantation, Comparing these outcomes to a SEE ALSO patients were given anti-T- study of 91 children receiving

lymphocyte globulin to prevent transplantation from an HLA- Job & Fellowship Connections rejection and -versus-host identical sibling or a 10/10 allele- disease. Prophylaxis was not matched unrelated donor, BBMT Journal administered after transplantation. researchers concluded that the Two of the children experienced transplantation method used for this ASBMT Home primary graft failure; four of the study is a comparable alternative patients died; and 19 patients for children with acute leukemia in relapsed. In addition, 24 of the need of urgent allogeneic patients developed grade 1-2 acute transplantation. More... skin graft-versus-host disease

Upfront Autologous Transplant Better for Young Myeloma Patients Than Regimen Newly diagnosed myeloma outcomes of 268 patients randomly patients have a better chance of assigned to receive two courses of ASSOCIATIONsurvival NafterEWS upfront autologous melphalan 200 mg/m2 and cell transplantation than after oral autologous transplantation and 261 chemotherapy and lenalidomide, patients who received reports a study appearing in Leukemia. Researchers analyzed Continues on page 15

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A WORD FROM PRESIDENT KRISHNA KOMANDURI, M.D. Dear Colleagues: I hope this message finds you well. As I proposed policy change would not go into write this, August is fading, as are the memories effect. To me, this highlights perfectly the of time spent away from work with family over importance of a professional society, which can the summer. Our four kids are transitioning to rapidly mobilize and advocate for the needs of three new middle and high its members, centers and patients. This should schools, so chaos is reigning also provide some inspiration to those of us supreme at our house as we growing a little cynical about the power of our

settle into new schedules and collective voice. I’m also happy to report that routines. our ASBMT health policy staff, working with Last month I wrote about their NMDP colleagues, have organized our the possibility of late summer first Capitol Hill day next month. We expect stormy weather, and that this will further help us to find our political unfortunately , my column was voice to best advocate for all of you. a little too prophetic. Hurricane Harvey rolled For me it’s also grant season, which brings ashore in Corpus Christi, Texas, rapidly into focus the increasingly uncertain climate of intensifying from a mere tropical storm to a research funding. As many ASBMT colleagues

devastating hurricane, before evolving into a tweeted this last month, metrics of National significant rain and flooding event in the Institutes of Health (NIH) funding continue to Houston area. I still recall witnessing firsthand reach new and increasingly frustrating the surprising and devastating effects of landmarks. For example, the median age of Tropical Storm Allison in Houston in 2001 and investigators at the time of funding of their first how the city openly embraced refugees of NIH RO1 grant has now exceeded 46 years – a Hurricane Katrina in 2005. My thoughts are startling and depressing figure. As someone now with our ASBMT colleagues in Houston, who sits on a promotion and tenure committee their patients, families and communities as they that still holds to conventional views about the

still face the effects of this storm as I write, and importance of NIH funding as a PI for the as they eventually enter the long phase of award of tenure, I’m increasingly concerned recovery from a profound disaster. that the rift between expectations and reality With that somber caveat, I want to provide a will lead to an exodus of talented physician- positive update on the importance of ASBMT scientists from our academic ranks. I know the advocacy that I highlighted in my previous stresses felt by many of us with established column. Just as storms can directly threaten any careers are greatly magnified for the junior of us, I noted last month that changing financial investigators who are our future. I urge all of us, climates can influence the security and with our local and national representatives, to

sustainability of our transplant centers. This highlight how much more difficult this situation summer, the ASBMT and its partners became has become. Fortunately, the NIH has broad aware of proposed Centers for Medicare & bipartisan support in Congress, but the Medicaid Services (CMS) changes in challenge is proportionally increasing funding reimbursement that had the potential to severely in a climate of fiscal restraint. reduce inpatient payments for transplant This week, I had the pleasure of continuing a episodes (by as much as 70% or more). new tradition of regular conference calls with The ASBMT, the National Marrow Donor the chairs of our ASBMT Special Interest Program (NMDP) and many of you quickly and Groups (SIGs) and senior ASBMT leaders. The

strongly advocated against this change. Earlier goal of these calls, and our historical face-to- this month, we were delighted to hear that our collective voices were heard, and that the Continues on page 3

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PRESIDENT’S MESSAGE (CONTINUED FROM PAGE 2) face meeting at Tandem, is to increase the leadership, from the Board of Directors to crosstalk of our increasingly productive and leaders of our SIGs, committees and task forces,

complex society. We hope to use these we would also like to hear from our membership interactions to make a better Tandem meeting at large during this critical look in the mirror. In with an increased focus on our critical scientific the coming months, we will tell you how to plenaries, but also better communication so that submit your thoughts on our present and future members can attend sessions of interest across directions, as it is critical that our goals and disciplines and SIG-directed sessions. We hope directions directly reflect the aspirations and to dedicate improved infrastructure (e.g., using will of our members. informatics) to enhance communications within There is much more going on, and I’ll look

and between SIGs, committees and task forces forward to continuing to update you about our so involved members can better assimilate a many areas of progress in the coming months. growing number of parallel initiatives across At the midpoint of my term, I will note that it our Society. has been a great privilege to serve you thus far, I’ll close with noting that the ASBMT Board and I look forward to a very productive fall for of Directors will also convene for its fall the Society. meeting in Chicago in early October. The As always, please feel free to contact me or Society will use this opportunity to start our your other elected representatives directly, as process of self-reflection and strategic planning your feedback is essential for our collective

that we discussed earlier in the year. We are success! pleased that Michael Boo, J.D., who served as the chief strategy officer for the NMDP, will act All the best, to facilitate this strategic planning process. While we will formally survey our designated Krishna

LEGISLATION & REGULATION

ASBMT/NMDP Legislative Day in Washington, D.C. ASBMT will be hosting its first Legislative “This has been a year of tremendous Day in Washington, D.C., on Thursday, Sept. 7, upheaval in the health policy area,” said in partnership with the NMDP. The goal for the Stephanie Farnia, ASBMT Director of Health day will be to educate Congressional Policy and Strategic Relations. “Changes to representatives on issues that affect the field of Medicare, Medicaid, the Affordable Care Act, hematopoietic cell transplantation – Medicare and the NIH budget have all been near-constant reimbursement, physician payment and topics of conversation. This climate has decreasing opportunities for research funding. A underscored the need for ASBMT to have an group of approximately 10-15 individuals have active core set of volunteers willing to interact been chosen to attend, based on home locations with their legislators about important issues that that represent key Congressional or Senate will potentially affect the field and/or the districts. patients you all serve.”

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BMT TANDEM MEETINGS

Registration Now Open for the 2018 BMT Tandem Meetings Salt Palace Convention Center Early registration allows you to: Salt Lake City, Utah • take advantage of discounted Feb. 21–25, 2018 registration rates; • make reservations at your preferred Online registration, abstract submission and hotel; and housing reservations are now open for the 2018 • purchase your ticket to the BMT BMT Tandem Meetings. Innovative educational Tandem Meetings reception, as space is sessions and limited. related conferences will More information on these benefits can be provide tools to found on the Tandem Meetings home page. improve your skills, broaden your knowledge base and help you lead the future of blood and marrow transplantation (BMT). Recognition of the effectiveness of blood and marrow transplant and cellular therapies has never been greater. Dynamic growth in the field has introduced novel and exciting approaches to treatment and care across the country and around the world. The 2018 BMT Tandem Meetings harness the latest technologies, most influential research and the brightest minds to deliver this game-changing information to you. Join us!

Abstract Submission More than 100 abstracts will be selected for oral presentations during the general scientific sessions and peripheral conferences.

SUBMIT YOUR ABSTRACT

Deadline: Oct. 3, 2017 (11:59 PM PST) 5

BMT TANDEM MEETINGS (CONTINUED FROM PAGE 4)

ASBMT Awards to be Presented at the 2018 BMT Tandem Meetings The ASBMT is proud to announce the names A past president of ASBMT, Dr. Negrin is of the award recipients who will be honored at being honored for his exceptional contributions the 2018 BMT Tandem Meetings this February to the field of hematopoietic transplantation, in Salt Lake City. including his profound contribution to The 2018 ASBMT Lifetime Achievement understanding of innate and adaptive immunity Award will be presented to A. John Barrett, and its importance in determining outcomes of M.D., chief of the Bone allogeneic hematopoietic transplantation. His Marrow Stem Cell efforts to develop novel model systems and Section technologies, from bioluminescent imaging to of the Hematology Branch, novel sequencing approaches, and to apply them National Heart Lung Blood to studies of effector and regulatory T cells, B Institute. A past president cells, NK cells and other immune subsets have of the ASBMT, Dr. Barrett inspired a generation of scientists. More is an outstanding senior investigator with importantly, they are advancing the possibility numerous contributions to research in blood and that targeted therapies and adoptive marrow transplantation for more than 30 years. immunotherapy will improve outcomes related to Co-chair of this year’s annual ASBMT-ISCT both relapse and graft-versus-host disease. While Cell Therapy Training Course, Dr. Barrett has his scientific contributions are the primary reason been involved with the course since its inaugural for this award, the ASBMT also recognizes his session in 2015. exceptional leadership, and his service as “It is important to note that his application of associate editor of Blood and as the founding immune suppression to treat marrow failure editor of Blood Advances. disorders has been practice-changing,” said The 2018 ASBMT Public Service Awards Catherine Bollard, M.D., FRACP, FRCPA, of will be presented to Jeffrey W. Chell, M.D., and the Children’s National Health System in Michael Boo, J.D. Washington, D.C. “Furthermore, both his Until his retirement in April of this year, laboratory and clinical work in transplantation Michael Boo, J.D., served as chief strategy immunology have significantly contributed to officer of the National Marrow the stepwise improvements in outcome, and the Donor Program/Be The Match broadening application of allogeneic stem cell (NMDP), the national registry transplantation that we have witnessed for the United States, between particularly in the last 20 years.” 2001 and 2016. His Robert S. Negrin, M.D., professor of contributions included the medicine and chief of the Division of Blood and implementation of a new Marrow Transplantation at strategic planning process that Stanford Medicine, was helped guide substantial growth at NMDP, the selected for the 2018 development of the Center for Cord Blood in ASBMT E. Donnall 2003, and increasing the NMDP’s share of the Thomas Lecture. The cord blood market in the United States from 15% lecture recognizes an to over 90%. Boo also identified and launched eminent physician or new products and services that had significant scientist, either a clinician or investigator, who bottom-line revenue impact and developed new has contributed meritoriously to the relationships within the extensive NMDP advancement of knowledge in blood and marrow transplantation.

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BMT TANDEM MEETINGS (CONTINUED FROM PAGE 5)

ASBMT Awards (continued from page 5) network of national and international partners, “Dr. Chell has had transformative impact in which improved access to cell sources and the fields of blood and marrow transplantation markets worldwide. and cellular immunotherapy,” noted ASBMT As NMDP chief executive officer and President Krishna Komanduri, M.D. “Through executive director of the Center for International efforts including his leadership of the NMDP’s Blood and Marrow Transplant Research, Dr. System Capacity Initiative, which brought Chell led the NMDP through together diverse stakeholders to discuss how the a period of dramatic clinical academic framework could support the and academic growth. Under sustainable growth of transplant centers, he his leadership, the Be The helped foster the growth of academic transplant Match Registry has programs while always maintaining a patient- dramatically expanded, centered focus.” increasing the access of ASBMT and NMDP centers to unrelated donor products, including cord blood units, from around the world. l y , LEGISLATION & REGULATION (CONTINUED FROM PAGE 3) I Policy Perspectives by Stephanie Farnia, ASBMT Director of Health Policyw and Strategic Relations Eclipses & Ellipses… that bodesa well for collaboration and partnership It has been a much busier summer in the in healthn care. (At this point, I’ll grasp at any health policy world than usual – multiple straws tI can dream up!) There are numerous comment periods for proposed updates to share this month; I will divide them annual policy rules from the up betweent hematopoietic cell transplantation Centers for Medicare & (HCT)o and chimeric antigen receptor (CAR) T Medicaid (CMS), uncertainty for easy future reference. around the Affordable Care r Act repeal efforts and a couple Medicaree and HCT of “out of left field” issues I’m Thanksc to your outreach efforts, the HCT blaming on eclipse-driven communityo was able to prevent a proposed zaniness. In large part, changeg to the logic that CMS uses to assign summer in the policy area is always reactionary – claimsn to MS-DRGs for the Inpatient anticipating proposed rules and responding as Prospectivi e Payment System. The change was needed when they are released – but this year felt of a technicalz nature – correctly recategorizing particularly so. As we move into autumn, efforts HCT ase a “non-operating room” procedure – but traditionally shift to implementation of policy it would have greatly impacted the payment rates changes clarified in the final rules, as well as for autologoust and allogeneic HCT. CMS issued strategic planning efforts around new or the followingh response in the Final Rule issued in continued areas of concern. I welcome that shift August:e “We acknowledge the concerns of the this year, in particular, and hope that the eclipse marked the beginning of a new cosmic alignment e Continues on page 7 f w fa on rt 7

LEGISLATION & REGULATION (CONTINUED FROM PAGE 6)

Policy Perspectives (continued from page 6) commenters. We agree that it is important to Transplant and Cellular Therapy (HCTCT) maintain the current Pre-MDC logic for these physicians within the CMS system for purposes procedures while also appropriately designating of billing. This status essentially affords you them as non-O.R. procedures. After additional options for billing specialty care and consideration of the public comments we will be helpful in allowing CMS to compare a received, we are finalizing our proposal to true peer group when it begins evaluating cost change the designation for the 20 ICD 10–PCS and quality. We will issue more detail on the procedure codes listed in Table 6P.4o. specific benefits of using this designation in your associated with the proposed rule and this final billing practices in a future newsletter. In the rule from O.R. procedures to non-O.R. meantime, start work on signing yourself up as a procedures, effective October 1, 2017, and HCTCT physician prior to Oct. 1. A CMS maintaining their assignment to the Pre-MDC reference document can be found here and some MS–DRGs 014, 016, and 017 for FY 2018” specific advice from a CMS representative is as (page 92 of the FY2018 Final Rule). This will follows: sound like gibberish to many of you, but please feel good in knowing that it translates to no “The new specialty codes will be effective negative change in reimbursement. October 1, 2017 and can be utilized as valid While avoiding a loss on the MS-DRG primary specialty codes or a secondary payment was a successful demonstration of our specialty code. The Provider Enrollment Chain defensive skills, there is much more to do in and Ownership System (PECOS) shall make the terms of seeking improved reimbursement for necessary changes to recognize and use the new transplant. The National Marrow Donor physician specialty codes. You can submit the Program/Be The Match has been working on that CMS-855I and CMS-855O applications using issue for several years now and is advancing the ‘Undefined Physician Type’ option and legislation through Congress that asks for parity write in the specific specialty until the forms can between solid organ and HCT in terms of be updated with the new specialty. For any Medicare payment policies. In an era where “new” submissions for the C9, complete the almost no issues have bipartisan support, this application via PECOS or paper application. does. Please read the newsletter item on this For existing providers who are currently issue and share this page with HCT and practicing Hematopoietic Cell Transplantation Government Affairs teams. and Cellular Therapy, you can submit a change The Quality Payment Program (QPP), also of information for those providers. If a provider known as MIPS or MACRA, recently held its has more than one specialty, it can be listed as a open comment period for the second year of the secondary specialty. For anything related to the program. While most HCT clinician teams will application submittal process, please contact not feel a great impact from QPP in the next few your local Medicare Administrative years, quality-based payment programs will not Contractor.” be disappearing anytime soon. ASBMT submitted a brief comment letter in regard to key aspects of the proposed rule and welcomes your (I put this in a box to give you enough room individual thoughts on areas for improvement as to print it, cut it out and tape it to the monitor of you become more involved with the program. your program administrator’s computer – Finally, as we highlighted in a column a few advisably alongside a coffee gift card months ago, HCT physicians will now be able to formally identify as Hematopoietic Cell Continues on page 8

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LEGISLATION & REGULATION (CONTINUED FROM PAGE 7) ANDEM EETINGS BMTPolicyT PerspectivesM (continued from page 7) or candy bar of choice. This is where the first value” of the first two CAR T products. The ellipses come in, as the implementation of this process is clearly outlined on the ICER change sounds relatively straightforward, but website and there are several opportunities for only time will tell…) public comment. ASBMT has submitted a public comment letter, which can be read CAR T Reimbursement Policy Updates here. As I mentioned earlier, a significant amount Finally, the new ICD-10-PCS code for of my time and focus over the last few months CAR T will go into effect on Oct. 1. We has been shifting to CAR T – trying to will remind you when the time comes, but understand the reimbursement models and please feel free to share this code with your predicted impact on centers, working on billing teams now. Kite requested the applications for new codes your teams will need development of these codes from CMS last in the near future, and providing input into winter, but new ICD-10 codes are payer- and discussions being had by payers and other product-agnostic, meaning the new CAR T national organizations. While busy, it has been a codes can be used with all payers and for all tremendously exciting time and a chance to put CAR T products. Caveat: check with your many of our HCT lessons to good use. commercial payers in terms of their use and Approval Timing and Reimbursement acceptance of this code, as always. Concerns: As most of you know, the Food and XW033C3: New Technology, Introduction Drug Administration’s approval for Novartis’s via Peripheral Vein; Engineered Autologous CTL019 is expected sometime in September, and Chimeric Antigen Receptor T-cell approval for Kite’s AxiCel could come anytime Immunotherapy before December. The treatment populations are XW043C3: New Technology, Introduction very different between the products and have via Central Vein; Engineered Autologous accordingly dissimilar payer implications; Kite’s Chimeric Antigen Receptor T-cell product will face Medicare hurdles, and Novartis Immunotherapy will need to navigate Medicaid on a state-by- And with that, a hectic summer of policy state basis. We will be sending a letter into CMS initiatives comes to a close. Next month, look in a few weeks outlining our concerns with the for updates on ASBMT’s submitted comment expected reimbursement structure and asking letters to CMS regarding the proposed them to consider payment policy alternatives Physici an Fee Schedule and Outpatient where possible. The other major ellipses in this Payment System rules, as well as a recap of area is pricing of the products – estimates run our planned Sept. 7 ASBMT/NMDP from $250,000 to upwards of $650,000, all of Legislative Day. which will have substantial repercussions for the health care system… Questions? As some of you already know, the Institute for [email protected] Clinical and Economic Review (ICER) has @HCT_Policy announced that it will be conducting an analysis of the “comparative clinical effectiveness and 9

ASSOCIATION NEWS ASBMT New Investigator Awards The ASBMT is now accepting applications NIH format, a letter of recommendation from for the 2018 New Investigator Awards. the applicant's sponsor/mentor (two pages The ASBMT New Investigator Awards are maximum) and a description of the proposed designed to encourage clinical or laboratory research (four pages maximum). research by young investigators in the blood New Investigator Award applications will and marrow transplantation (BMT) field. The be evaluated for: award is $30,000 per year, typically for two • Scientific merit within the BMT field years, and is preferably used to support the • Significance and anticipated overall investigator’s salary for his or her research impact of the potential findings to the effort. Alternatively, the award may be used BMT field for direct support of research costs. • Institutional environment Applicants are required to submit the New • Training record of the sponsor/mentor Investigator Award application, a curriculum vitae that adheres to the National Institutes of Deadline to apply is Oct. 16. For full details Health (NIH) Biographical Sketch format, a including eligibility requirements, list of other support that also adheres to the please click here.

4th Annual ASBMT Fall BMT Clinical Education Conference for NPs, PAs and Fellows Registration is now open for the 4th Highlights Annual ASBMT Fall Clinical Education Best of Tandem” Session Conference for NPs, PAs and Fellows . . . and Led by ASBMT president Krishna space is filling up Komanduri, M.D., the "Best of Tandem" FAST! The session will cover key scientific content conference will be presented at the 2017 BMT Tandem held Oct. 26-28, Meetings. 2017, at the Pediatric Breakout Sessions Sheraton Hotel in For clinicians looking for the best new Seattle. information and trends in pediatrics, don't Click here to miss the pediatric breakout sessions including register. Additional information about " Transplantation for lodging, learning objectives, and the full Pediatric Nonmalignant Disorders" with course schedule is provided here. Kanwaldeep Mallhi, M.D., and "Survivorship This 2½ day program will provide clinical Care for Pediatric Hematopoietic Cell education for nurse practitioners, physician Transplant Survivors" with Eric Chow, M.D., assistants, fellows and junior faculty caring M.P.H. for blood and marrow transplant patients. At View the full course brochure by clicking the end of the course, attendees will leave here. with a comprehensive syllabus, a self- assessment, and a deeper understanding of the Interested in Sponsoring? diagnostic evaluation and therapeutic If you are interested in becoming a sponsor treatments for acute and chronic for this conference, please email Angie Dahl complications of blood and marrow at [email protected]. transplantation. 10

ASSOCIATION NEWS (CONTINUED FROM PAGE 9)

CRTC Through the Eyes of an Instructor by Carlos A. Ramos, M.D., Baylor College of Medicine This July, I was invited to participate as occasion to strike up a conversation with an instructor in the ASBMT Clinical people whom you have never met before. In Research Training Course (CRTC). This contrast, this course provided an incredible opportunity was unique opportunity for networking and sharing ideas. for me because I attended We are a small field, and I have continued to the first CRTC in 2007 as interact regularly with many of the faculty a scholar. (and scholars) whom I met during the course. This year, 12 scholars Many of them have contributed to my and 10 faculty members academic career progression. from across the transplant Second, I recall how reassuring the course spectrum gathered in was, especially for someone who was about Charlotte, North Carolina, to take his first independent steps in the field. to review the principles of translational and It was comforting to learn that grant clinical research. Each scholar brought a applications are not always successful, that clinical research project to present to the life can take you places other than those that group, and all participants discussed and may professionally make the most sense, or further dissected these projects in small that one’s professional path is not always as groups that met throughout the course, with linear as one might assume by reading a CV. the intent of enabling each scholar to use the Finally, free from being distracted by other feedback to refine the proposed clinical trial. duties, I was able to give my undivided Throughout the course, scholars also attended attention to lectures on exciting, current short talks on clinical and statistical aspects topics in BMT given by leaders in the field. pertaining to the field of hematopoietic stem In the process, I learned several important cell transplantation, including design of aspects of clinical trial design and statistics, clinical trials, the history of blood and which are subjects seldom addressed during marrow transplantation (BMT), choice of our clinical training in fellowship. conditioning regimens, challenges in graft- Being on the other side this time, I was versus-host treatment, new targeted therapies, struck by how remarkable this class of and recently developed immune cell scholars was. I was extremely impressed by therapies, the latter being the topic of my talk how they ran with our suggestions and this year. incorporated them along with their new ideas When I attended the first event in 2007, I into their initial projects. At the end of the was about to transition from being a fellow to course, the scholars presented revised becoming an attending physician and I had an versions of their clinical trials, and it was amazing experience, with a few aspects of the fantastic to see how much they had improved. course still standing out in my memory. First, I thoroughly enjoyed participating in the I remember the thrill of having unimpeded course both as a scholar and now as faculty. access to leaders of the field in a collegial Many good things have happened to me in the atmosphere. We are often told as trainees that 10 years since I attended the course for the meetings such as the American Society of first time. As I mentioned to the scholars this Hematology are major opportunities for year, I am sure this course was instrumental networking. While this may be true, a major in many aspects of that journey. national meeting is not necessarily an easy 11

ASSOCIATION NEWS (CONTINUED FROM PAGE 10) Pharmacy SIG Literature Updates by LeAnne Kennedy, Pharm.D., BCOP, CPP, FHOPA, Chair, ASBMT Pharmacy SIG For the past few years, the Pharmacy in the past however, the leadership of ASBMT Special Interest Group (SIG) has been feels that this is a great service that should be preparing a literature update which is shared shared with all ASBMT members. Starting with with our SIG members. Members of the this month’s ASBMT eNews, you will receive a Pharmacy SIG Communications Committee link to the monthly literature summaries: review key journals monthly to identify http://asbmt.org/about-us/special-interest- important articles related to stem cell groups/pharmacy-sig#Lit. transplantation. The pharmacists then We hope that you will enjoy reading them, summarize the article’s key highlights deemed and if you have any comments, feel free to important for the practice of stem cell contact the Pharmacy SIG by emailing transplantation. Your transplant pharmacist [email protected]. may have passed this monthly summary to you

SIG Spotlight: Transplant Nursing Special Interest Group by Chris Rimkus, APRN Nursing educational sessions have been participate in the events and activities. held during the Tandem Meetings since 1999. Co-chairs and Planning Committee members Membership: originated from what was previously known as • is made up of nurses and non-nurses the Oncology Nursing Society’s (ONS) Blood alike and and Marrow Stem Cell Transplant Special • is free with the ASBMT membership Interest Group (SIG) with strong fees- just email the membership link and administrative support from the national ONS. you will be made a member. At that time, many of the co-chairs and Planning Committee members were also Benefits of membership include: affiliate members of ASBMT. The nursing • Connecting with others within your presence at the ASBMT Tandem Meeting specialty grew exponentially over the years, causing the ASBMT leadership to recognize nursing as a • Participating in projects very important player in the transplant process. • Opportunities to participate in ASBMT In 2009, the ASBMT reached out to initiatives or committees representation from nursing across the country • Make your voice heard on changes and to see if they would be interested and willing needs assessment for blood and marrow to start a SIG focused on nursing issues. transplantation (BMT) nurses

Purpose of the Nursing SIG Planned goals for 2017-2018 include: The purpose of the ASMBT Nursing SIG is • Expand membership to nurses across the to give nurses a voice in the ASBMT, allowing country, particularly staff nurses them to be part of the organization and • Collaboration with the European Blood planning of events and activities within the and Marrow Transplant Group on special society. The Nursing SIG will be governed by projects a steering committee, but all members can Continues on page 12 12

ASSOCIATION NEWS (CONTINUED FROM PAGE 11)

SIG Spotlight (continued from page 11) Meet your leaders of the SIG Current ASBMT Co-Chair Suni Elgar Current ASBMT Nursing SIG Chair: Chris ([email protected]): I am proud to be in my Rimkus ([email protected]): I have 15th year of transplant nursing. I am the worked in BMT since 1988 and have been manager at the Seattle Cancer Care Alliance involved with the ASBMT Nursing SIG since outpatient BMT clinic. My vision for the SIG is its inception. I am very passionate about best to grow nursing representation in ASBMT by practices in BMT. My hope for this SIG is that highlighting the essential components of we can bring nursing together to improve partnership between nurses and other health practices, showing the BMT community what care professionals in the care of BMT patients nursing can bring to the table to improve the and their families day-to-day care of BMT patients. Nurses are the We welcome collaboration with other SIG’s. front line of care and have tremendous insight Feel free to connect with any of the nursing SIG that can help improve quality and even affect steering committee members via email – we outcomes. welcome your thoughts and ideas. Our website can be found here.

In Memoriam - Johannes Joseph (“Jon”) van Rood, Founder of Donor Associations Professor Johannes Joseph (“Jon”) van will increase if a donor is offered to a central Rood, the founding father of database of many patients in need for a Donors Worldwide, transplant. For this reason, van Rood founded died July 21 in 1967. in Leeuwarden, He also co-founded the World Marrow Netherlands. He was 91. Donor Association, Europdonor (now known as His scientific and Matchis), the European Society for Blood and managerial work helped to Marrow Transplantation and the European shape immunogenetics, Federation for Immunogenetics. transplantation “I have known Jon since 1983,” said immunology and global cooperation in ASBMT Board Member James Gajewski, M.D. unrelated hematopoietic stem cell “I always considered him a great friend and transplantation for 60 years. mentor to so many of the field. His work on Until the mid-1960s, transplant surgeons histocompatibility and establishing the Dutch matched kidney donor and patient mainly by donor registry was vital. We lost one of the blood type. Patients had to wait until a suitable great founding fathers of our field.” donor was found in the center where they were Van Rood received many awards including treated. Van Rood obtained evidence that the the Robert Koch Prize and the Wolf Prize in human leucocyte antigen (HLA) system plays Medicine, and he was a member of the Royal an import role in the outcome of renal Netherlands Academy of Arts and Sciences. transplantation. Matching of the HLA-type of To read more about his life’s work, click donor and recipients led to superior graft and here and here. patient survival. However, the number of HLA antigens was large, which made the probability of finding a donor with a matching tissue type for a particular patient low. This probability 13

LEGISLATION & REGULATION (CONTINUED FROM PAGE 8) Help Protect Access to Life-Saving Transplants for Medicare Patients The National Marrow Donor Program/Be centers who treat over 1,200 Medicare patients The Match, in partnership with ASBMT, has each year could result in serious access issues worked extensively with the Centers for for Medicare patients. Patients who are denied Medicare & Medicaid Services (CMS) to access to transplant will face expensive, likely address the serious patient access issue of futile alternative treatment options. And, in inadequate reimbursement for stem cell most cases, transplant is their only option for transplant (SCT). survival. The current reimbursement rate for This is where the transplant community allogeneic transplants (MS-DRG 014) does not needs your help. Congressional partners are cover the true cost of SCT. The fiscal year 2018 planning to propose legislation that would reimbursement rate for an allogeneic transplant require CMS to align payment policy for SCT is almost $65,000, which includes donor search with solid organ transplant and separate donor and cell acquisition costs (approximately search and acquisition costs from the MS-DRG $48,000 for bone marrow or peripheral blood payment. transplant, and $65,000 for cord blood Please contact your members of Congress transplant). This payment policy leaves today ! Urge them to support legislation to transplant centers with very few dollars reform Medicare payment policy for bone remaining to cover services, supplies, drugs, marrow, peripheral blood stem cell and cord and other items for patients throughout the blood transplants. Visit BeTheMatch.org/cms to average 27-day post-transplant hospital stay. submit a letter today. These financial losses incurred by transplant

FACT UPDATE

FACT Accreditation Used as Criterion in Best Hospitals List U.S. News and World Report released its transplants, and two points were given if 2017-2018 Best Hospitals List, and FACT accreditation was for allogeneic transplants. All accreditation was again used as a ranking 50 hospitals with rankings in the cancer criterion for the cancer specialty. Since 2007, specialty have programs that are FACT- accredited programs receive a point value based accredited. Congratulations to these programs! on their accreditation. One point was given if View the list of best hospitals in the cancer accreditation was only for autologous specialty.

FACT Webinar: Patient-Centered Care Coordination in HCT Want to learn more about patient-centered community. Presenters will highlight the care coordination in hematopoietic cell benefits and metrics of effectiveness of care transplantation (HCT)? Join FACT for a live coordination, introduce the care coordination webinar presentation on Sept. 20 at 11 a.m. ET. toolkit and identify the key components of the Nandita Khera, M.D., M.P.H., of the Mayo proposed framework. Clinic Arizona, and Patricia Martin, RN, BSN, View webinar details and register here. of WellPoint Inc., will explain why care coordination is important for the HCT Continues on page 14 14

FACT UPDATE (CONTINUED FROM PAGE 13)

FACT Webinar (continued from page 13)

Blood Advances, a journal of the American with HCT within the context of coordination Society of Hematology, published a manuscript issues in care delivery and stakeholders authored by the webinar presenters and others involved. The article also outlines challenges, titled “Patient-centered care coordination in ongoing efforts to raise awareness, and hematopoietic cell transplantation” (Khera, potential policy changes around appropriate Martin, et al, 2017). The article describes a care reimbursement to cover all aspects of care coordination framework for patients treated coordination. Read full article

FACT and JACIE Reviewing Public Comments on Draft 7th Edition Standards FACT and JACIE published the draft 7th confirming agreement with the requirements, edition of the FACT-JACIE International improving clarity, and providing helpful Standards for Hematopoietic Cellular Therapy guidance information. Product Collection, Processing, and The Standards Committee is currently Administration for inspection and public reviewing the comments and incorporating edits comment from May through July 2017. Over 70 as appropriate. The final version of the 7th respondents submitted more than 600 edition Standards will be published in March comments on the draft. This stakeholder 2018 and become effective at the end of May participation benefits the Standards by 2018.

FACT Allied Learning Center FACT’s Allied Learning Center, a collaborative online modules applicable to their operations effort between FACT, AABB, ASBMT, the and training needs. The Allied Learning Center American Society for Apheresis, American is updated quarterly with new events. Society of Gene and Cell Therapy, American FACT encourages accredited organizations Society for Histocompatibility and to utilize these educational activities to maintain Immunogenetics, College of American knowledge of industry practices and Pathologists, Cord Blood Association, Food and regulations. Many of these sessions may be Drug Administration, International Society for used to meet FACT requirements for continuing Cellular Therapy, National Marrow Donor education. The sessions listed in the Allied Program/Be The Match, PACT, World Marrow Learning Center are in addition to the FACT Donor Association-NetCord, Save the Cord educational program, including live events and Foundation, and Society for Immunotherapy of on-demand events. Cancer provides a centralized resource for Explore the Allied Learning Center. organizations to quickly and easily obtain

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CLINICAL RESEARCH (CONTINUED FROM PAGE 1)

Upfront Autologous Transplant (continued from page 1) chemotherapy and lenalidomide. Four years chemotherapy and lenalidomide. Only 53% of after treatment, survival was more likely for the the patients who relapsed after chemotherapy transplantation recipients than the received a salvage transplant. The researchers chemotherapy recipients, regardless of determined that upfront autologous prognosis. Progression-free survival 1 was 42 transplantation was more likely to reduce the months for transplant recipients vs. 24 months risk of death than salvage autologous for the chemotherapy group. In addition, transplantation. The researchers concluded that progression-free survival 2 was 71% for the these study results confirm that upfront transplant group vs. 54% for the chemotherapy autologous transplantation should be the cohort, and overall survival was 84% after standard approach for young myeloma patients. transplantation vs. 70% following More...

Relapsed Aggressive B-Cell Non-Hodgkin Lymphoma and Rituximab Dosing A long-term study comparing doses of statistically significant differences between the rituximab found that there were no survival two doses. Disease status and the number of differences between standard and high doses of prior treatments were the only factors that the drug when used with carmustine, played a role in worse survival outcomes. cytarabine, etoposide and melphalan (BEAM) Patients who had transplantation during and autologous cell transplantation for relapsed complete remission or who received less than aggressive B-cell non-Hodgkin lymphoma. The three prior treatments had better five-year phase 2 study published in the British Journal overall survival, regardless of the rituximab of Haematology looked at data from 93 patients dose. The researchers concluded that these randomly assigned to the high- or standard-dose study results indicate there is no advantage to group. The researchers discovered that the five- using a high dose of rituximab over a standard year disease-free survival was 40% and the dose. More... five-year overall survival was 48% with no 12 1416

TRANSLATIONAL SCIENCE STUDIES Single T-Cell Receptor Editing Treats Multiple Myeloma

A new study published in Blood indicates edited T cells killed NY-ESO-1pos targets. TCR that a NY-ESO-1 T-cell receptor (TCR) single transferred cells were highly alloreactive, but editing approach quickly produces high single edited cells had a better safety profile. As numbers of tumor reactive T cells, treating a result, when the single edited multiple myeloma without inducing graft- cells were infused into mice with multiple versus-host disease (GVHD). The researchers myeloma, tumor rejection occurred without based the single editing method on disruption of GVHD. This led to higher survival rates for the the endogenous TCR α chain, followed by the mice that received the edited cells vs. those that transfer of genes encoded for a tumor-specific were treated with TCR transferred cells. These TCR. Single editing allowed optimal TCR study results led researchers to conclude that expression and preferential stem memory and single TCR gene editing is a clinically feasible central memory phenotype. Similar to unedited approach that increases the safety and efficacy T cells redirected by TCR gene transfer, single of cancer adoptive immunotherapy. More...

Researchers Develop Genetically Accurate Models of CMML and JMML Researchers of a study appearing in Blood secondary transplantation, which demonstrated have developed genetically accurate preclinical the resilience of CMML grafts and confirmed models of chronic myelomonocytic leukemia that CD34+ cells harbor the disease-initiating (CMML) and juvenile myelomonocytic compartment in vivo. The researchers also leukemia (JMML). Using immunocompromised successfully engrafted JMML primary samples mice with transgenic expression of human into 12 mice. Engraftment of both types of granulocyte-macrophage colony-stimulating leukemia led to overt phenotypic abnormalities factor, interleukin-3 and stem cell factor in a and lethality in recipients, which facilitated NOD/SCID-IL2Rƴnull background, researchers evaluation of the JAD2/FLT3 inhibitor conducted of CD34+ cells, pacritinib in vivo. According to the researchers, unfractionated bone marrow or peripheral blood the data from this study demonstrate that mice mononuclear cells derived from 18 patients. support the development of CMML and JMML This resulted in engraftment of CMML in the disease-initiating and mature leukemic cells in bone marrow, spleens, livers and lungs of 82 vivo. More... mice. Comparable results were seen with

Innate Lymphoid Cell and Interleukin-22 Deficiency Associated With GVHD Mice with graft-versus host disease (GVHD) manner. They also found that the thymopoietic experience intrathymic group 3 innate lymphoid impairment in GVHD associated with loss of cell (ILC) loss, causing interleukin-22 (IL-22) ILCs could be improved by restoring IL-22 deficiency, which worsens thymic epithelial signaling. Despite uninhibited alloreactivity, damage. Therefore, administering IL-22 after exogenous IL-22 administration after allogeneic bone marrow transplantation can transplantation led to better recovery of enhance thymopoiesis, reports a study from thymopoiesis and development of new thymus- Blood. While mice with GVHD had depleted derived peripheral T cells. The researchers thymic ILCs and IL-22, mice without GVHD did concluded that manipulation of the ILC-IL-22- not. The researchers also discovered that TEC axis may be useful for improving immune preventing IL-21 receptor signaling in donor T reconstitution after transplantation and other T- cells and the elimination of thymic ILCs cell deficiencies. More... improved thymopoiesis in an IL-22-dependent

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CALENDAR OF EVENTS CALENDAR OF EVENTS •SEPTEMBER •OCTOBER •NOVEMBER European Society for Medical Oncology International Society of Paediatric Memorial Sloan Kettering ESMO 2017 Congress Oncology Clinical Application of CAR T Cells September 8-12 49th Congress November 16-17 Madrid, Spain October 12-15 New York, New York Washington, D.C. Canadian Blood and Marrow Transplant European Society for Medical Oncology Group European Society for Gene & Cell Therapy Asia Congress “Pre- and Post-Transplant Issues in BMT” Annual Congress November 17-19 Themed Meeting Series October 17-20 Singapore September 9-10 Berlin, Germany St. John’s, Newfoundland, Canada •DECEMBER Canadian Blood and Marrow Transplant American Society of Hematology American Society for Histocompatibility Group Annual Meeting & Immunogenetics “Ocular Chronic GVHD” webinar December 9-12 43rd Annual Meeting October 18 Atlanta, Georgia September 11-15 San Francisco, California Association of Community Cancer •JANUARY Centers Cell & Gene Therapy World European School of Haematology 34th National Oncology Conference January 22-25 3rd International Conference on New October 18-20 Miami, Florida Concepts in Lymphoid Malignancies Nashville, Tennessee September 15-17 •FEBRUARY Mandelieu, France European Association of Tissue Banks BMT Tandem Meetings 26th Congress Combined ASBMT and CIBMTR Annual Canadian Blood and Marrow Transplant October 18-20 Meetings Group Treviso, Italy February 21-25 “Psychosocial Aspects of HSCT” webinar Salt Lake City, Utah September 20 ISCT-ASBMT Cell Therapy Training Course •MARCH ASBMT October 23-27 European School of Haematology Corporate Council Meeting Seattle, Washington Clinical Updates on CLL and Indolent September 24-25 Lymphoma Fernandina Beach, Florida ASBMT March 2-4 4th Annual BMT Clinical Education Paris, France •OCTOBER Conference for NPs, PAs and Fellows Histiocyte Society October 26-28 European School of Haematology 33rd Annual Meeting Seattle, Washington 4th International Conference on Hematologic October 3-4 Malignancies at Older Age: Biology and Singapore •NOVEMBER Therapy Society for Immunotherapy of Cancer March 9-11 American Association of Tissue Banks Annual Meeting Mandelieu, France 41st Annual Meeting November 8-12 October 3-6 National Harbor, Maryland Association of Community Cancer Orlando, Florida Centers National Donor Marrow Program/Be The 44th Annual Meeting National Comprehensive Cancer Match March 14-16 Network Council Meeting Washington, D.C. 12th Annual Congress: Hematologic November 10-11 Malignancies Minneapolis, Minnesota Regenerative Medicine Workshop October 6-7 March 21-24 San Francisco, California Canadian Blood and Marrow Transplant Isle of Palms, South Carolina Group AABB “Quality and Accreditation” webinar National Comprehensive Cancer Network Annual Meeting November 15 23rd Annual Conference October 7-10 March 22-24 San Diego, California Orlando, Florida

Copyright © 2017 American Society for Blood and Marrow Transplantation. All rights reserved. The editor for ASBMT eNews is Jean Sanders, M.D.; managing editor is Dan Kotheimer; ASBMT eNews services provided by Lori O’Keefe. Do you have news, responses or opinions to share with us? Please e-mail the association office at [email protected]. .