Feb 22, 2018 - Accreditation of Cellular Therapy, International. Society for Cellular Therapy, Be The Match,. American .
ASBMT eNews AMERICAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION
February 2018
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ASSOCIATION NEWS ASBMT and CBMTG Release BMT Recommendations ASBMT and the Canadian Blood and Marrow Transplant Group (CBMTG) have partnered with Choosing Wisely and Choosing Wisely Canada to develop the first Choosing Wisely BMT list of five transplantation practices that clinicians should consider. Choosing Wisely is an initiative of the American Board of Internal Medicine (ABIM) Foundation, developed to spark conversations between providers and patients to ensure the right care is delivered at the right time. Choosing Wisely centers around evidence-based recommendations of “Things Providers and Patients Should Question.” Through the efforts of the Choosing Wisely Task Force, led by Sita Bhella, M.D., M.Ed., FRCPC, and Matthew Seftel, M.D., M.B.Ch.B., M.P.H., M.R.C.P., the ASBMT and CBMTG have jointly released the following recommendations: 1. Don't routinely use peripheral blood stem cells for patients with aplastic anemia when a ASSOCIATION NEWS suitable bone marrow donor is available due to a higher risk of graft-versus-host disease.
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A Word From the President
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Legislation & Regulation
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BMT Tandem Meetings
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2. Don't use greater than 2 mg/kg/day of methylprednisolone (or equivalent) for the initial treatment of graft-versus-host disease. 3. Don't routinely use two cord blood units for standard umbilical cord blood transplantation when a single unit of adequate size is available, recognizing that higher cell doses are preferred when using units with greater HLA mismatch. 4. Don't routinely use peripheral blood stem cells for matched unrelated donor transplantation using myeloablative conditioning and standard graft-versus-host disease prevention regimens when a suitable bone marrow donor is available. Continues on page 4
Association News
FACT Update
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Clinical Research
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Translational Science Studies
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Calendar of Events
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A WORD FROM PRESIDENT KRISHNA KOMANDURI, M.D. My dear colleagues, Shakespeare, in All’s Well That Ends Well, counsels that we should “Love all, trust a few, do wrong to none.” [ Act I.i] As I write this, my last column as your president, I realize that there is no ending approaching (despite the portents and rumblings) but that this column is an appropriate occasion for reflection about the current and future of our Society. I have tried in the past year to live by the words of the above quote. Love. I can say after this past year that my love for this Society and all it represents is deeper, as is my appreciation for its diverse, talented and dedicated membership. Our members face some of the most challenging diseases and difficult clinical scenarios in modern medicine and perform small and large miracles daily. Trust. I have absolute trust in ASBMT volunteer leaders who so wonderfully represent all of you, from the leaders of our special interest groups (SIGs), task forces and committees to the Board of Directors, Executive Committee and other volunteer scientific and editorial leaders. Do wrong to none. As Hippocrates also advised, I am often mindful to do no wrong to this proud organization. While taking chances, I believe we have succeeded in leaving it more secure and better poised to face its future. What have we accomplished in the past year? While the best answer is “not enough” I am pleased that we did some measurable good. As we mark our silver anniversary in Salt Lake City, we will reflect on the progress of the past quarter century while trying to glimpse our future. In the past year, ASBMT members have contributed scientifically to new Food and Drug Administration approvals across the spectrum of hematopoietic transplantation and adoptive immunotherapy. One example of our impact is the remarkable achievement of seeing each of
last year’s Tandem17 Late Breaker abstracts yielding publications in the New England Journal of Medicine, with all three presentations reflecting practice-changing studies. Critically, our members led scientific and clinical studies that led to approvals of engineered T cell therapies for pediatric acute leukemia and adult lymphoma. We performed studies that resulted in the first human gene therapies, the first antiviral agent for cytomegalovirus (CMV) prophylaxis after hematopoietic cell transplantation (HCT), the first approval of a drug to treat graft-versus-host disease (GVHD) and a critical affirmation of autologous transplantation as a treatment for systemic sclerosis. I am happy to say that the Society has embraced not only the scientific foundations of these transforming advances, but also reacted to their seismic impacts. This has included taking a leading position in defining clinical standards for immune effector cell therapies and also helping to create the infrastructure for coding, regulatory safety and reimbursement. By developing a formal health policy program within the ASBMT, we have better advocated for our clinicians and our patients to increase their access to a broader range of cell therapies. Significant challenges still need to be addressed as these therapies are unprecedented in cost and as reimbursement frameworks have not yet evolved to create sustainable health systems. This means that patients who could be cured by these therapies are not yet guaranteed the access and coverage they deserve. But I am proud of our ongoing efforts with our partners (including the Center for International Blood and Marrow Transplant Research, Foundation for the Accreditation of Cellular Therapy, International Society for Cellular Therapy, Be The Match, American Society of Hematology, American Society of Clinical Oncology and others) to address the many challenges that remain. Continues on page 3
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PRESIDENT’S MESSAGE (CONTINUED FROM PAGE 2) In addition to our nascent health policy efforts, I am happy to report that our ASBMT leadership has been working hard behind the scenes to create a new management framework that should prepare us for the next 25 years. Following careful self-reflection, we decided to transition to new association management and recently finalized an arrangement with SmithBucklin, a leading association management company who will provide new resources and directions to support our membership and our broad efforts. We are grateful to our staff at Executive Administration, Inc (EAI) for their dedicated service in the past year, including their gracious support for this critical transition. This past fall, our Board also began a critical strategic planning process that will be completed later this year once critical members of our new management team are in place. This will result in a redefinition of our vision and goals and will help define the structure, tactics and financial commitments needed to ensure our continued growth and impact. I am very pleased that John DiPersio, an accomplished and thoughtful scientist and clinician, will lead the ASBMT as your next president during this critical process. The input of members with diverse interests and needs will be critical, and we look forward to hearing from you as we redefine our scientific, clinical and policy priorities. While we have made progress, I recognize how much work remains to be done. Despite the approvals I mentioned above, we are still far from reliable cures, either through HCT or T cell therapies, for most patients we see. Even the approvals for CMV prevention and GVHD therapy have been meaningful but incomplete advances leaving need for further progress. Profound uncertainty remains about basic questions of conditioning intensity and graft choice and how to selectively suppress alloreactivity while limiting relapse. While we have made great scientific progress, we must
also admit too few of these advances have translated to daily practice. While we have established SIGs focused on palliative and supportive care, health economics and infectious diseases, these and other areas of research remain underdeveloped despite their importance. We have also done little to strengthen a collapsing funding environment for basic science. And while there are some encouraging signs (e.g., a record number of ASBMT New Investigator Award applications) I remain concerned about the numbers of clinicians and especially scientists who will replace our aging membership and carry our torches forward. With these caveats, I am sincerely optimistic about the future of hematopoietic transplantation and cellular therapy, and the critical role of the ASBMT in advancing science and patient care in these disciplines. It has been the greatest privilege of my career to serve as your president, and I am profoundly thankful for your support and for the lasting friendships I’ve made in your service. I look forward to continuing to support our talented Executive Committee and Board in the coming year, including our new board members VicePresident Pavan Reddy and new Board Members Stephanie Sarantopoulos, Bipin Savani and John Koreth. As the Bard also reminded us in Macbeth [Act V.v], “Life’s but a walking shadow.” I’m confident that all of us in the ASBMT will use our relatively brief hour upon the stage to make the world of cellular therapies a better place, for our members, our communities and most of all our patients. With appreciation, Krishna
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ASSOCIATION NEWS (CONTINUED FROM PAGE 1) Choosing Wisely 5. Don't routinely give immunoglobulin replacement to adult hematopoietic cell transplantation recipients in the absence of recurrent infections regardless of the IgG level. Suggestions for the list were solicited from ASBMT’s Quality Outcomes, Practice Guidelines and Education Committees, CBMTG program directors, and chairs of Center for International Blood and Marrow Transplant Research scientific working committees. The 119 unique suggestions received were ranked, and the final short-listed recommendations were subjected to systematic reviews to establish the final five. “These recommendations were uniformly based on Choosing Wisely’s guiding principles of focusing on tests, treatments or procedures, that may be harmful, wasteful, or for which there is no clinical benefit” said Dr. Bhella.
According to Dr. Seftel, “They were written with the intention of facilitating wise decisions about the most appropriate care based on a patient’s individual situation, and will foster discussion among clinicians and between clinicians and patients about these practices.” The list, with the rationale and key references for each recommendation, is available here. A manuscript detailing the methodology and summary of evidence for each recommendation is online now at ASBMT’s journal – Biology for Blood and Marrow Transplantation. Other members of the Choosing Wisely BMT Task Force included: Jeffrey Betcher, Christopher Bredeson, Luciano Costa, Andrew Daly, Christopher Dandoy, Zachariah De Filipp, Vi Doan, Alison Gulbis, Lisa Hicks, Mark Juckett, Nandita Khera, Amrita Krishnan, Navneet Majhail, George Selby, Nirav Shah, Melisa Stricherz, and Auro Viswabandya.
Celebrating 25 Proud Years of ASBMT This year, the ASBMT will celebrate its 25th anniversary! To celebrate, each monthly issue of ASBMT’s journal, Biology for Blood and Marrow Transplantation, will contain something special about ASBMT’s anniversary, including histories, photos, and personal memories from those who were there when it all started. Be sure to follow ASBMT on Facebook and Twitter for videos from ASBMT leadership – past and present – who will share stories about ASBMT’s early years, and they will tell you why they are proud to be a member of ASBMT. You are also encouraged to share your own video wishing ASBMT a happy anniversary or tell others what ASBMT means to you!
And of course, this February, drop by the ASBMT booth (407 and 409) at the 2018 BMT Tandem Meetings in Salt Lake City to walk down memory lane and revisit some of the accomplishments ASBMT has helped to bring about since 1993. We will have a photographer at the booth who will provide you with a FREE professional headshot. You can also schedule a time to create your own "What Does ASBMT Mean to You" video. There will also be a plenary celebration which will include presentations about the Society’s beginnings, and critical advances in clinical research and transplantation immunology. Join us Friday, Feb. 23, 8:30 a.m.-10 a.m.
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ASSOCIATION NEWS (CONTINUED FROM PAGE 4) ASBMT Election of 2018-2019 Officers and Board Members After a close race, we are excited to announce the following people have been elected to serve as our newest offices and board members: • Vice President: Pavan Reddy, M.D. • Director, Laboratory Science: Stefanie Sarantopoulos, M.D., Ph.D. • Treasurer: Corey Cutler, M.D. • Director, Community or Clinical Practice: Bipin Savani, M.D. • Director, At Large: John Koreth, M.B.B.S., D.Phil.
Many thanks to all of you who ran for your willingness to serve the Society. Also, thanks to those board members rotating off the board, including Shakila P. Khan, M.D., Colleen Delaney, M.D., and Patrick J. Stiff, M.D. A special thanks also to Chris Bredeson, M.D., who leaves the board as our immediate past president.
Fundamentals of HCT Training Course
Join us at the 2018 Fundamentals of Hematopoietic Cell Transplantation (HCT) Training course. This course provides practitioners with the skills required to manage patients undergoing HCT, also referred to as blood or marrow transplant (BMT), with a focus on pharmacotherapeutic management throughout the transplant process. The course also incorporates case-based learning to emphasize application of concepts taught through didactic lecture. When: March 24-25, 2018; held immediately following the Hematology/Oncology Pharmacy Association (HOPA) 14th Annual Conference Where: Colorado Convention Center in Denver, Colorado
Cost: $175 if registered by Feb. 14; $200 if registered after Feb. 14 This activity is eligible for ACPE credit; see final CPE activity announcement for specific details. Click here for additional details and to register for the course! Introduction to HCT Free Webinar – March 6 Register here today. Back by popular demand, the ASBMT Pharmacy Special Interest Group will provide a free Introduction to HCT webinar, hosted by Susannah Koontz, Pharm.D., BCOP, on March 6, 4 p.m.-5:15 p.m. (EST). All are welcome to attend. Click here for more details. Please note this webinar is not eligible for ACPE credit.
February ASBMT Self-Directed Learning Quiz On behalf of the ASBMT Committee on Education, we are pleased to continue our monthly self-directed learning quiz "Basic Principles and Practices of Hematopoietic Cell Transplantation and Cell Therapy – Question and Answer Approach” for fellows in training, highlighting hematopoietic cell transplantation and cell therapy topics. Answers to each question
appear with evidence-based “non-exhaustive” peer-review commentary. Our aim is to provide you with a credible and freely available educational resource. Questions generated and reviewed by Syed Abutalib, M.D., Mark R. Litzow, M.D., and William A. Wood, M.D. To take this month’s quiz, click here.
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ASSOCIATION NEWS (CONTINUED FROM PAGE 5) 2018 ASBMT Clinical Research Training Course (CRTC) – Applications Now Being Accepted ASBMT’s 2018 ASBMT Clinical Research Training Course (CRTC) will be held July 11-16 in Park City, Utah. Tuition, travel, housing and meal expenses will be paid by the Society and corporate sponsors for up to 12 scholars to attend the course. Participants will be competitively selected. Preference will be given to fellows and faculty with no more than two years of blood or marrow transplantation experience following training or a faculty appointment.
The ASBMT Board of Directors created the course in 2006 because of concerns that clinical fellowship programs do not adequately cover the principles of research and how to take findings from the laboratory to the clinic. The course helps close the gap by addressing those deficiencies. Please send your application and all required materials no later than March 12. For course and application details, click here.
ASBMT Pharmacy SIG Recent Publications The ASBMT Pharmacy Special Interest Group (SIG) has recently published two articles and produced a brochure. Current workforce shortages within the hematopoietic stem cell transplant field necessitate capitalizing on oncology-trained pharmacists. Working within an agreed-upon protocol, pharmacists are able to expand patient care delivery through optimal medication therapy management. To read “Characterization of Collaborative Practice Agreements Held by Hematopoietic Stem Cell Transplant
Pharmacists,” click here. The second publication, “The Hematopoietic Cell Transplant Pharmacist: Roles, Responsibilities, and Recommendations From the ASBMT Pharmacy SIG,” recently appeared in ASBMT’s journal, Biology of Blood and Marrow Transplantation (BBMT). The article can be accessed online here. The ASBMT Pharmacy SIG brochure will be available at the ASBMT booth (407) at the 2018 BMT Tandem Meetings.
1st International Symposium on HCT-Related Toxicities More than 10,000 hematopoietic cell transplantations (HCT) are performed annually in the United States as a potentially curative treatment for over 70 life-threatening illnesses including hematologic cancers, genetic disorders, and other diseases. Despite significant advances in supportive care and improvements in HCT outcomes, one in every three patients may succumb to HCT-related toxicities, half of which are not related to graftversus-host disease (GVHD). Following the successful examples of the international symposia focusing on GVHD and relapse after HCT, ASBMT leaders Sergio Giralt, M.D., and Miguel-Angel Perales, M.D., are holding the First International Symposium on Hematopoietic Cell Transplantation-Related Toxicities.
This symposium will bring together experts and thought leaders from around the world in the fields of HCT and related medical subspecialties. Speakers will discuss mechanisms of toxicities and symptom burden after HCT in order to identify best practices to both prevent and treat HCT-related toxicities. Over the two-day symposium, speakers and attendees will also convene in dedicated sessions that will facilitate collaborative efforts in planning future research aimed at mitigating serious toxicities, reducing symptom burden, and improving patients’ outcomes. The symposium will be held April 27-28, 2018 at Memorial Sloan Kettering Cancer Center in New York, New York. Additional information and registration can be found online at: www.mskcc.org/Toxicities.
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LEGISLATION & REGULATION All Things Tandem By Stephanie Farnia, ASBMT Director of Health Policy and Strategic Relations But first, ICER The Institute for Clinical and Economic Review (ICER) has been moving through a process of evaluating the cost-effectiveness of chimeric antigen receptor T cell therapy (CAR-T). The ASBMT has submitted a comment letter on the draft evidence report as an identified stakeholder, and Dr. Komanduri will be participating as part of an expert panel when the documents are up for a vote on March 2. The session will be live-streamed for free for those who are registered – information on the ICER CAR-T page. A few selected Tandem highlights Tandem can be overwhelming. There are always 18 things happening at once, and your calendar is packed from 6 a.m. – 9 p.m. All of the sessions will be valuable, but I try to scour the various tracks to find those most key to what I need to know in my position. I’m sharing some of that with you below in case it is of interest. (And I know I missed some great things on this list – apologies in advance!) Sessions – open to any attendee type, even if listed in various tracks Wednesday: • 8:15 a.m.-10 a.m. – Gene Therapy (and opening ceremonies) (Hall C): The official kick-off to the BMT Tandem meetings, the Gene Therapy plenary session, will be chaired by John Tisdale of the National Institutes of Health. Thursday: • 10:30 a.m.-noon – CMS Super Session (251 A-F): Susan Leppke (National Marrow Donor Program), Jugna Shah (Nimitt) and myself are teaming up for a fast-paced, extended session on all things hematopoietic cell transplantation (HCT) and Medicare.
• 1 p.m.-4 p.m. – Informatics for Cell Therapy and HCT (355 F): All things Center for International Blood and Marrow Transplant Research (CIBMTR) and electronic health records/electronic medical records. • 1:15 p.m.-4:15 p.m. – Value and Health Economics Special Interest Group Sessions (251 A-F): Three great sessions on valuing CAR-T, PROs in HCT and health policy hot topics. • 1:15 p.m.-2 p.m. – Ethical Considerations of Precision Medicine (250 ABC): The title says it all! • 1:30 p.m.-3 p.m. – Hidden Costs of Transplant (255 EF): This Pediatric Special Interest Group (SIG) session includes a discussion of the effects of poverty on pediatric transplant. • 3:15 p.m.-4:15 p.m. – Exporting Biotechnologies Across Borders (255 perspectives on moving research EF): Representatives from the Food and Drug Administration and Novartis will be sharing breakthroughs into the market. Friday: • 8:30 a.m.-10 a.m. – ASBMT Presidential Symposium: 25 Years of Progress (Hall C): A must-see session showcasing the history of the ASBMT, the science that moved the field forward and the partnership between the ASBMT and CIBMTR over that time. • 9:15 a.m.-10 a.m. – CAR-T Money Matters (251 A-F): I will be presenting the latest on CAR-T reimbursement, with a focus on resources and solutions for commonly asked questions. Continues on page 8
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LEGISLATION & REGULATION (CONTINUED FROM PAGE 7) All Things Tandem (continued from page 7) •
11:15 a.m. -noon - Financial Counseling in the New Era of BMT and Cellular Therapies (251 A-F) • 12:15 p.m.-1:15 p.m. – Quality Outcomes Forum: Making Data Your Friend (255 EF): This is a late addition session that is focused on how to use the new tools in the CIBMTR’s Stem Cell Therapeutic Outcomes Database for useful analysis of your program’s data. A powerhouse group of speakers will share how they use the tools in their own programs. • 1:15 p.m.-2:15 p.m. – New and Emerging Drugs (255 BC): It is always useful to hear the Pharmacy SIG talk through the pharmaceutical landscape. • 2:45 p.m.-3:30 p.m. – Management of CRS/Neurotoxicity at the Bedside (250 A-F): An important topic as CAR-T rolls out to more centers • 2:45 p.m.-6:45 p.m. – Awards, Best Abstracts, the Mortimer M. Bortin and E. Donnall Thomas lectures (Hall C): I will be camped out in Hall C, watching all of the awesomeness and frantically googling things to try to follow along with the science. Saturday: • 8:30 a.m.-9:30 a.m. – Pharmacy Advocacy (255 BC): HCT pharmacists are fierce advocates for their field and the patients they treat. This session will talk through those efforts in the last year and what’s to come in 2018.
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8:30 a.m.-10 a.m. – Building a Safer and Faster CAR (Hall C): Clustered regularly interspaced short palindromic repeats and I are still getting to know each other. I’m hoping this will help. • 10:45 a.m.-11:30 a.m. – What we do right…What can we do better? Survivors share their thoughts (250 A-F): Sessions that involve those we serve are always opportunities to learn and grow. Sunday: • 12:30 p.m.-1:30 p.m. - Late-breaking Abstracts (Hall C): Because they save (some of) the best for last. A note on posters I’m absolutely positive that there are dozens (hundreds?) of compelling posters that will be shown at this year’s meeting; I just haven’t had time to make my checklist yet. Let me know @HCT_policy if you spy a good one. 25th Anniversary Videos If you haven’t seen the 25th Anniversary videos that are popping up on the @ASBMT Twitter and Facebook pages, take some time to watch – they give a real sense of the history and progress the field has made in the last 25 years. And don’t forget the hashtag #BMTTandem18!
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BMT TANDEM MEETINGS Combined Meetings and Awards Once again, the ASBMT Business Meeting and Awards will be held in conjunction with the Center for International Blood and Marrow Transplant Research General Assembly, followed by the Mortimer M. Bortin and E. Donnall Thomas lectureships. Join us on Friday evening, Feb. 23, to celebrate the 2018 ASBMT Lifetime Achievement Award recipient, A. John Barrett, M.D. We are also honored to present the 2018 ASBMT Public Service Awards Jeffrey W. Chell, M.D., and Michael Boo, J.D. James Young, M.D., Memorial Sloan Kettering Cancer Center, ASBMT New Investigator Awards Review Committee chair, will present the 2018-2019 New Investigator Awards to the following recipients: ASBMT/Gabrielle’s Angel New Investigator Award Hanna Kraus, M.D., Johns Hopkins University Reinvigorating T Cell Function in Patients with Post-Transplant Relapsed AML ASBMT New Investigator Award Challice L. Bonifant, M.D., Ph.D., University of Michigan Chimeric Antigen Receptor NK Cell Therapy for AML ASBMT/Merck New Investigator Award Jonathan L. Golob, M.D., Ph.D., Fred Hutchinson Cancer Research Center Hematopoietic Cell Transplant Outcomes and Microbial Metabolism ASBMT New Investigator Award Daniel C. Peltier, M.D., Ph.D., Mott Children’s Hospital Non-Coding RNA-Mediated Mechanisms of T-Cell Autoimmunity ASBMT New Investigator Award Federico Simonetta, M.D., Ph.D., Stanford University Chimeric Antigen Receptor-Invariant Natural Killer T Cells in Allogeneic HCT ASBMT/Sanofi Genzyme New Investigator Award Christoph K. Stein-Thoeringer, M.D., Memorial Sloan Kettering Cancer Center Studying the Role of the Intestinal Enterococcus spp. In the Development of GVHD ASBMT New Investigator Award Amy N. Suthers, B.S., Ph.D., Duke University Increased TLR7 Signaling of BCR-Activated Patient B Cells in Chronic GHVD The evening will end with Robert S. Negrin, M.D., who will present the 2017 E. Donnall Thomas Lecture, and Eliane Gluckman, M.D., FRCP, who will present the Mortimer M. Bortin Lecture.
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BMT TANDEM MEETINGS (CONTINUED FROM PAGE 9) ASBMT Nursing SIG Awards Two individuals will be honored with ASBMT Nursing Special Interest Group (SIG) Awards at the BMT Tandem Meetings in Salt Lake City on Thursday, Feb. 22, at 8:30 a.m. Margaret Bellerjeau RN, MSN, OCN, BMTCN, CHTC, of Temple University Hospital in Philadelphia, will receive the ASBMT Nursing SIG Lifetime Achievement Award. Margaret is committed and dedicated to her work in the field of blood and marrow transplantation after 25 years. She is always seeking more information and sharing her knowledge with all.
Theresa M. Latchford RN, MS, CNS, BMTCN, AOCNS, of Stanford University Medical Center in Stanford, California, will be honored with the ASBMT Nursing SIG Clinical Excellence Award. Theresa is an expert clinician and her practice as a clinical nurse specialist is characterized by her tireless efforts to improve the care of blood and marrow transplant recipients through educating other nurses. The awards will be presented in Ballroom JHF of the Salt Palace Convention Center.
ASBMT Survivorship SIG to Hold Second Annual Meeting Feb. 21 Sharhukh Hashmi and Linda Burns, cochairs of the Survivorship Special Interest Group (SIG), invite you to attend the SIG’s second annual meeting to be held on Wednesday, Feb. 21, 7 a.m.-8:30 a.m. in Room 151 G during the upcoming BMT Tandem Meetings. You do not need to be a current SIG member to attend. All with an interest in survivorship are welcome! Breakfast will be available for all attendees at the start of the SIG meeting. We have a packed agenda, so please try to come a bit early to get breakfast so we can begin on time. The agenda includes updates on other
programs and SIGs with related interests, as well astime to brief attendees on the development of a Survivorship Program Directory and plans for a SIG position paper on delivery of care models. Based on feedback from the inaugural SIG meeting in 2017, the majority of the time will be spent presenting, discussing and providing feedback on research proposals submitted to the SIG this year. This is a great opportunity to meet colleagues in the field, hear what others are interested in and develop collaborations. If you have any questions, contact Linda at
[email protected]. See you in Salt Lake City!
FACT UPDATE Webinar to Outline Changes in 7th Edition Hematopoietic Cellular Therapy Standards The webinar titled, “Effectively Transition to the 7th Edition Hematopoietic Cellular Therapy Standards” is scheduled for March 14. Paul Eldridge, Ph.D., director of Cell GMP Facility at UNC Bone Marrow Transplant and Cellular Therapy Program and chair of the FACT
Committee, will describe changes to the 7th edition Standards and provide examples for effective implementation. The Standards will be published on March 1 and programs must comply with them by May 30. View meeting details and register here.
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FACT UPDATE (CONTINUED FROM PAGE 10) NEW FACT Inspector Handbook Mobile Application The fourth edition of the FACT Inspector Handbook is now available to download as an application on your mobile phone! The mobile application provides inspectors tips in an easyto-read format and shares the guidelines and wisdom that inspectors and accredited organizations have developed since the inception of FACT. If you have an iPhone, follow these steps: • Go to your web browser on your phone and type: inspectorhandbook.factwebsite.org • At the bottom of your screen, click the upload box with arrow • Click the box “Add to Home Screen” • Click “Add”
For Android users, follow these steps: • Go to your web browser on your phone and type: inspectorhandbook.factwebsite.org • Select three dots in the upper right-hand corner Select “Add to Home Screen” • Select “Add” • Select “Add Automatically” • For a step-by-step tutorial with graphics, please use this link.
FACT Events at the 2018 BMT Tandem Meetings FACT will host several popular events at the 2018 BMT Tandem Meetings in Salt Lake City, Utah. Join us for this educational programming to gain well-rounded knowledge about the FACT Standards and accreditation process. Cellular Therapy Inspection and Accreditation Workshop – Feb. 20 The blood and marrow transplant field has been a leader in voluntarily improving quality, and accredited clinical programs are currently adapting to several new FACT Standards and procedural changes to the accreditation process. This workshop will provide background on these changes. The morning workshop sessions include major topics such as how to effectively transition to the 7th Edition Hematopoietic Cellular Therapy Standards, Center for International Blood and Marrow Transplant Research data audits, clinical outcomes, including center reported causes of low survival, and the accreditation of immune effector cellular therapy programs. The afternoon session includes two different tracks: New Inspector Training Orientation and
Common Citations. The Inspector Training track includes sessions on the FACT accreditation process, what documents to review before an inspection, the ins and outs of performing an onsite inspection, how to conduct an exit interview, and finally, how to make your case via the inspection report. The Common Citations track will review recent deficiencies and corrections related to commonly cited Standards in the areas of quality management, personnel, and donor information and consent to donate. Sessions are accompanied by exercises and group discussions to practically apply lecture concepts to real-world experiences. Note: Inspector trainees are required to attend the New Inspector Training Orientation Track. View meeting details and register here. Cellular Therapy Leadership Course 101 – Feb. 20 Do you want to improve your leadership skills? Everyone wins when leaders get better, and this Continues on page 12
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FACT UPDATE (CONTINUED FROM PAGE 11) FACT Events (continued from page 11) half-day course is designed for that outcome. The course is open to anyone who has (or aspires to) a leadership position in cell therapy – whether you direct a transplant center or laboratory, lead a cell collection service or cord blood bank, head a staff of nurses or transplant coordinators, hold an office or board position in a volunteer organization, chair a committee, or have any position in which you are expected to motivate and lead a team. View meeting details and register here. Cellular Therapy Advanced Leadership Course 201 – Feb. 20 If you completed FACT’s Cell Therapy Leadership 101 course previously and want more, the 201 course is for you. This advanced workshop drills deeper into organizational development and leadership skills. Topics include: • Characteristics of a healthy organization • Adapting an organization to a changing environment • Multiplying and diminishing the effectiveness of a team • Leading from where you are • Servant leadership • Effective governing boards • Strategic planning with a focus on outcomes Participants in the prerequisite Cell Therapy Leadership 101 course in the morning also are eligible to register for the 201 course in the afternoon. View meeting details and register here.
FACT-ASBMT Quality Boot Camp – Feb. 21 This year’s boot camp will focus on topics identified to be challenging by transplant programs complying with FACT requirements. The boot camp will strengthen your quality assurance activities through an in-person workshop. Members of the FACT Quality Committee and the ASBMT Administrative Directors SIG Quality Working Group encourage you in the months leading up to the BMT Tandem Meetings to review your quality program and identify strengths and weaknesses. Experts, including Karen Collum, DNP, OCN and Ann Wilson, MT(ASCP)SBB, MHA, will present quality concepts and lead roundtables. Their session will examine the overall quality management program from the perspective of a large and small (autologous only) program. Presenters will share their insight on the advantages and challenges of implementing quality management principles in programs of all sizes. View meeting details and register online. View agenda FACT Accreditation Portal Open House – Feb. 22 The new Accreditation Portal is coming! FACT invites you to attend an Accreditation Portal Open House at the BMT Tandem Meetings in Salt Lake City. FACT’s IT Business Analyst will be performing a demo of the new portal’s functionality in an open forum. You are invited to ask questions and see the new portal launching this year. BMT Tandem Meetings Salt Lake City, Utah Salt Palace Convention Center, RM 260A Feb. 22, 2018 Applicant Session: 1 p.m. – 2:30 p.m. Inspector Session: 3:30 p.m. – 5 p.m.
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CLINICAL RESEARCH Autologous HCT Superior to Cyclophosphamide for Severe Scleroderma Severe scleroderma patients who receive an autologous hematopoietic cell transplantation have better event-free and overall survival outcomes than patients treated with cyclophosphamide, reports a study from The New England Journal of Medicine. For the study, researchers randomly assigned 36 adults to undergo myeloablative autologous transplantation and 39 adults to receive immunosuppression with 12 monthly infusions of cyclophosphamide. At 54 months follow up, the rate of event-free survival was 79% in the transplantation group compared to 50% in the cyclophosphamide group. Estimates of eventfree survival and overall survival at 72 months
also were superior with transplantation: 74% vs. 47% and 86% vs. 51%, respectively. In addition, 9% of the transplant recipients started disease-modifying antirheumatic drugs (DMARDS) by 54 months vs. 44% of the cyclophosphamide recipients. Finally, none of the cyclophosphamide patients died from treatment, but 3% of the transplant recipients experienced treatment-related mortality at 54 months and 6% had died at 72 months. However, rates of treatment-related death and use of DMARDs were lower than those in previous reports of nonmyeloablative transplantation. More...
Letermovir Lowers Risk of Cytomegalovirus A study published in The New England Journal of Medicine reports that letermovir prophylaxis for cytomegalovirus effectively reduces the risk of the complication after allogeneic hematopoietic cell transplantation. According to the study, 565 patients were randomly assigned to receive either 480 mg of letermovir or a placebo every day beginning approximately nine days after transplant. Patients taking cyclosporine received only 240 mg of letermovir per day. At week 24, 37.5% of
letermovir patients had developed cytomegalovirus vs. 60.6% of the placebo recipients. However, adverse events, such as vomiting, edema and atrial fibrillation or flutter, were similar between the two groups, as were the rates for myelotoxic and nephrotoxic events. Mortality from all causes also was similar between the two groups at week 48: 20.9% for the letermovir group and 25.5% for the placebo group. More...
Active SCID at HCT Increases Risk of Death in Children Severe combined immunodeficiency at the time of allogeneic hematopoietic cell transplantation adversely impacts survival in children, according to the Primary Immune Deficiency Treatment Consortium’s study appearing in a recent issue of Blood. The study included 68 children with typical SCID and 32 with leaky SCID, Omenn syndrome or reticular dysgenesis. Newborn screening or family history identified 59% of the patients. The twoyear overall survival for patients who were infection free at the time of transplant was 95% vs. 81% for patients with active infection. Preparative chemotherapy improved one-year
post-transplant median CD4 counts and freedom from IV immunoglobulin, while other immunologic reconstitution parameters were not impacted. Approximately three to four months after a T-cell-replete graft, patients were at a twofold risk of a second transplant or death if CD3 was less than 300 cells/µL, CD8 was less than 50 cells/µL, CD45RA was less than 10% or there was a restricted Vβ T-cell receptor repertoire. The researchers concluded that newborn screening is effective at identifying patients with SCID, but approaches to ensure that patients are infection free at the time of transplant are needed. More...
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TRANSLATIONAL SCIENCE STUDIES New Immunotherapy Prevents or Manages Leukemia Relapse Researchers have developed a T-cell immunotherapy to prevent or manage leukemia relapse after hematopoietic cell transplantation, according to a study published in Blood. The researchers report that T-cell receptors (TCRs) isolated from minor H antigen-specific T cells represent an unused resource for developing targeted T-cell immunotherapy to manage leukemia relapse after transplantation. Recognizing that several elements may be crucial to the efficacy and safety of engineered T-cell immunotherapy, researchers developed a therapeutic transgene with four components: 1) a TCR specific for the hematopoietic-restricted, leukemia-associated minor H antigen, HA-1;
2) a CD8 coreceptor to promote function of the class I-restricted TCR in CD4+ T cells; 3) an inducible caspase 9 safety switch to enable elimination of the HA-1 TCR T cells in case of toxicity; and 4) a CD34-CD20 epitope to facilitate selection of the engineered cell product and tracking of transferred HA-1 TCR T cells. The T-cell product includes HA-1 TCR CD4+ T cells to enhance the persistence and function of the HA-1 TCR CD8+ T cells and includes only memory T cells. However, naïve T cells were excluded to limit the potential for alloreactivity mediated by native TCR coexpressed by HA-1 TCR T cells. More...
Identification of Marrow Cell that Regulates Engraftment and Influences Endosteal Osteoblasts A study from Blood Advances reports that novel CD45-F4/80lo marrow cells come from hematopoietic progenitors and reside among endosteal osteoblasts. In addition, in situ depletion of CD45 cells prior to marrow radioablation results in flattened osteoblasts and absent engraftment. To evaluate the effect of hematopoieitic cells on marrow remodeling, researchers used a mouse model to selectively deplete hematopoietic cells in situ. Cell depletion just prior to radioablation and hematopoietic cell transplantation abrogated donor engraftment. It also was associated with
flattened endosteal osteoblasts with preserved osteoblast proliferation and megakaryocyte migration. Monocyte, macrophage or megakaryocyte deplection did not alter osteoblast morphology. This suggests that a hematopoietic-derived cell outside these lineages regulates osteoblast morphologic adaptation after irradiation. Researchers concluded that this newly identified marrow cell may be an important regulator of hematopoietic stem cell engraftment and may influence the shape and function of endosteal osteoblasts. More...
Blocking Donor T Cell-Mediated TNFα Signaling Improves Engraftment Donor immune cell-derived inflammatory signals directly influence hematopoietic stem cell fate, according to a study appearing in Science Translational Medicine. To simulate hematopoietic cell transplantation, researchers injected human umbilical cord blood cells into immunodeficient mice. They discovered that higher cell doses inversely correlated with stem and progenitor cell engraftment, which they indicated was attributed to increased donor cellderived inflammatory signals. Specifically, donor T cell-derived tumor necrosis factor-α
(TNFα) was found to impair survival and division of transplanted hematopoietic stem cells and progenitor cells. When donor T cellderived TNFα was neutralized in vivo, it improved short-term stem and progenitor cell engraftment and hematopoietic recovery. In addition, it altered donor immune cell compositions. The effect of TNFα on transplanted cells could be decoupled from the indirect effect of alleviating graft-versus-host disease by interleukin-6 blockade. More...
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CALENDAR OF EVENTS C•FALENDAR OF EVENTS EBRUARY BMT Tandem Meetings Combined ASBMT and CIBMTR Annual Meetings February 21-25 Salt Lake City, Utah •MARCH European School of Haematology Clinical Updates on CLL and Indolent Lymphoma March 2-4 Paris, France European School of Haematology 4th International Conference on Hematologic Malignancies at Older Age: Biology and Therapy March 9-11 Mandelieu, France
•APRIL American Association for Cancer Research Annual Meeting April 14-18 Chicago, Illinois
•MAY International Society for Biological and Environmental Repositories Annual Meeting May 20-24 Dallas, Texas
European School of Haematology 6th International Conference on Myelodysplastic Syndromes April 26-28 Mandelieu, France
•JUNE American Society of Clinical Oncology Annual Meeting June 1-5 Chicago, Illinois
Memorial Sloan Kettering Cancer Center 1st International Symposium on Hematopoietic Cell TransplantationRelated Toxicities April 27-28 New York, New York
American Society of Transplant Surgeons American Transplant Congress June 2-6 Seattle, Washington
Association of Community Cancer Centers 44th Annual Meeting March 14-16 Washington, D.C.
•MAY The American Society of Pediatric Hematology/Oncology Annual Conference May 2-5 Pittsburgh, Pennsylvania
European Society for Blood and Marrow Transplantation 44th Annual Meeting March 18-21 Lisbon, Portugal
International Society for Cellular Therapy Annual Meeting May 2-5 Montreal, Canada
Regenerative Medicine Workshop March 21-24 Isle of Palms, South Carolina
European School of Haematology Clinical Updates on Acute Leukemias May 4-6 Budapest, Hungary
National Comprehensive Cancer Network 23rd Annual Conference March 22-24 Orlando, Florida ASBMT/NMDP Fundamentals of HCT Training Course March 24-25 Denver, Colorado
American Association of Immunologists Annual Meeting May 4-8 Austin, Texas American Society of Gene and Cell Therapy Annual Meeting May 16-19 Chicago, Illinois
Canadian Blood and Marrow Transplant Group Annual Conference June 7-9 Ottawa, Canada Federation of Clinical Immunology Societies Annual Meeting June 14-17 Chicago, Illinois European Hematology Association 23rd Congress June 14-17 Stockholm, Sweden International Society for Stem Cell Research Annual Meeting June 20-23 Melbourne, Australia •JULY Society for Crybiology CRYO 2018 July 10-13 Madrid, Spain
Oncology Nursing Society 43rd Annual Congress May 17-20 Washington, D.C.
Copyright © 2018 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
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