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November 2019 Newsletter
RCI BMT to Oversee CD33 CAR-T IND ProtocolBMT CTN Enrolls >11,000 Patients Health Services Research Update Four New Summaries for PatientsCIBMTR 2019 Facts and FiguresCIBMTR TriviaCIBMTR on Facebook and TwitterOur SupportersAbbreviations
We are transplanters. We take care of patients with life-threatening illnesses. We apply a therapy that can save many patients but can also cut short the lives of some. We confront death on a routine basis. Every time we lose a patient, whether it be to malignancy or a treatment-related complication, we are professionally humbled, recognizing that as a field, although we have made remarkable progress, we still have a long way to go. We are also personally shaken, witnessing the pain and loss suffered by family members and friends of patients we do not save. Now, of course, death is inevitable for us all. That is something many of us conveniently forget. We are supposed to be the healers, but that does not mean we are invincible. We, too, will ultimately succumb and that will lead to personal grief for our loved ones and a profound void in our professional community.
That void has been quite evident since John Hansen, MD, passed away on July 31 from pancreatic cancer. John devoted his professional life to unraveling the immunogenetics of allogeneic transplantation. His impact on the field has been nearly unparalleled. Working at the Fred Hutchinson Cancer Research Center since 1977, he played a central role in making "the Hutch" and Seattle synonymous with BMT. A prolific researcher, his academic contributions are legendary. But John's legacy extends far beyond his entries in PubMed. He was a remarkable colleague and mentor, not only to all the trainees and scientists with whom he interacted with in Seattle but to national and international colleagues as well. He was always willing to give of his time and expertise to develop new collaborations and provide sage advice for academic and professional development.
John was front and center in establishing the National Marrow Donor Program (NMDP), now Be The Match, in 1986. His vision and courage forged the foundation of an organization that has facilitated more than 100,000 transplants worldwide. It is hard to fathom that when I started in transplantation, the donor pool was essentially limited to HLA matched siblings. His insight, hard work, and perseverance paved the way for thousands and thousands of patients to be cured of their illnesses, returning to their families and to their lives. We all collectively owe John a debt of gratitude that is impossible to repay.
While John's passing does leave a void, he would probably have been the first to say that there are hundreds (if not thousands) of dedicated clinicians, scientists, and other professionals ready to fill that chasm, to take up his mantle and continue this fight to make sure that everyone who needs a transplant can receive one and that the procedure can be performed safely and effectively. John's leadership and devotion to the field guaranteed that his work would live on and his dreams would be fulfilled. Onward.
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Krishna Komanduri, MD, University of Miami, Miami, FLMiguel-Angel Perales, MD, Memorial Sloan Kettering Cancer Center, New York, NYRoy Chemaly, MD, MPH, M.D. Cancer Center, Houston, TX
Marcie Riches, MD, MS, University of North Carolina Hospitals, Chapel Hill, NC
Soyoung Kim, PhDMin Chen, MS
Based on the 2018 CIBMTR Summary Slides, infection is the reported primary cause of death in 19-21% of allogeneic HCT recipients in the first 100 days and 11-13% beyond 100 days. It accounts for 7% of deaths in autologous HCT recipients. However, transplant clinicians recognize that infection accounts for significantly greater morbidity in our patients. This burden of infections and its correlation with post-transplant immune reconstitution is the focus of our committee's efforts.
The Infection and Immune Reconstitution Working Committee recognizes that data collection for infections and immune reconstitution is complex and particularly demanding. In part, the significant effort required to document infection endpoints has contributed to under-reporting biases evident in prior analyses. Given this complexity, we greatly appreciate the time spent by data managers to provide us with the high-quality data needed to understand and improve infection-related transplant outcomes. Several revisions to forms in the past 2 years allow for additional detail on infections of interest, thus improving our understanding of this highly vulnerable population.
Our committee faces unique challenges due to the complex interactions of multiple time-dependent co-variates as well as the complexities associated with reporting multiple (and often recurrent) infections caused by a diverse range of pathogens. The rates of post-transplant infections are also associated with many other factors, including graft type, donor-recipient mismatch, recipient age, and GVHD incidence. Given the complexity of these factors, our analyses rely heavily on novel statistical techniques provided by our excellent Master's-level Statistician, Min Chen, MS, and our Statistical Director, Soyoung Kim, PhD. Dr. Kim presented information on the use of dynamic landmark analyses at our working committee meeting in February 2019 during the TCT Meetings. This technique allows for improved examination of the interactions of infections on other time-dependent outcomes, such as GVHD. Watch for her upcoming manuscript as well as a presentation in the Statistical Concurrent: Novel Advancements in Design and Statistical Analysis of TCT Studies at the 2020 TCT Meetings, better detailing this method and its application to our infection studies!
Despite the aforementioned challenges, our committee has been quite productive. In the past year, investigators have published 5 manuscripts of study results, including:
IN07-01/11-01 A: Bacterial blood stream infections (BSIs), particularly post-engraftment BSIs, are associated with increased mortality after allogeneic hematopoietic cell transplantation.
IN07-01/11-01 B: Bloodstream infection (BSI) due to Vancomycin-Resistant Enterococcus (VRE) is associated with increased mortality after hematopoietic cell transplantation for acute leukemia and myelodysplastic syndrome: A multicenter, retrospective cohort study.
IN13-01: Increased overall and bacterial infections following myeloablative allogeneic HCT for patients with AML in CR1.
IN14-01: Survival outcomes of allogeneic hematopoietic cell transplants with EBV-positive or EBV-negative post-transplant lymphoproliferative disorder, A CIBMTR study.
IN16-01: Virus detection in the cerebrospinal fluid of hematopoietic stem cell transplant recipients is associated with poor patient outcomes: a CIBMTR contemporary longitudinal study.
We have several exciting studies in process as well. The increased use of haploidentical transplants has led to multiple analyses examining differences in infectious complications. The committee has two ongoing studies probing for differences in CMV, non-CMV herpes viral infections, community respiratory viral infections, fungal infections, and bacterial infections between patients receiving haploidentical transplant with post-transplant cyclophosphamide, identical sibling transplant with post-transplant cyclophosphamide, and identical sibling transplants with conventional tacrolimus-based GVHD prophylaxis. The analyses for the viral infections are complete, and abstracts were submitted for the 2020 TCT Meetings. Studies examining the impact of infection prophylaxis and immune reconstitution have been limited in our committee by prior data collection methods and underreporting. Fortunately, this is improving, and 2 proposals examining immune recovery on post-transplant outcomes (IN19-01) and the impact of antibacterial prophylaxis on transplant outcomes (IN19-02) are in development.
We welcome the input and participation of our working committee members. Meeting attendance and the number of proposals submitted to the committee both continue to increase. We encourage continued participation and look forward to a robust and lively meeting at the 2020 TCT Meetings.
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Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, Boston, MAPeiman Hematti, MD, University of Wisconsin Hospital and Clinics, Madison, WI Newly Appointed Chair: Cameron Turtle, MBBS, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA (term starts March 2020)
Marcelo Pasquini, MD, MS, Medical College of Wisconsin, Milwaukee, WI
Ruta Brazauskas, PhD
Hilary Hall, Dana Farber Cancer Institute, Boston, MA
Kenny Xu, MPHDaniel Klaver, MPH
The Cellular Immunotherapy for Cancer Working Committee (CICWC) is the newest CIBMTR working committee, and it is part of the Cellular Immunotherapy Data Resource (CIDR). The NCI-funded CIDR focuses on all cellular therapies for the treatment of cancer and requires the development of a working committee to provide statistical resources and streamlined access to cellular therapy data for the community at large.
The CIBMTR had an existing committee with a combined focus on hematopoietic cell transplantation for autoimmune diseases and cellular therapies in general (ACWC), which was modified to accommodate the CIDR requirements. The CIBMTR Advisory Committee oversees the activities of all working committees and in 2019 recommended that studies focused on HCT for autoimmune diseases be redirected to the Non-Malignant Diseases Working Committee and that the focus of this newly developed working committee should align with CIDR directives. The portfolio of approved studies in the ACWC (with focus on cellular therapy) will remain in the CICWC until their completion and leadership has been extended to add a new chair. The CICWC chair selection followed the standard process of nomination that is in place for all working committees; a new chair was appointed by the CIBMTR Executive Committee and will start his term in March 2020. The CICWC's first in person meeting will be at the 2020 TCT Meetings in Orlando, FL.
The CIBMTR Advisory committee will oversee the performance and activities of CICWC, and all established performance metrics will apply. Additionally, as part of the CIDR governance structure, the activities of the CICWC and study portfolio will also be overseen by the CIDR Executive Committee.
The current CICWC portfolio includes:
Utility of donor lymphocyte infusions for the
treatment of drug-resistant viral or fungal infections in allogeneic HCT
recipients led by Gorgun Akpek, MD, MHS, and Omer Bilal, MD, in analysis.
Pattern of use and outcomes with donor
lymphocyte infusion after HLA-haploidentical allogeneic hematopoietic stem cell
transplant, led by Vivek Roy, MD, James Foran, MD, and Eva Gupta, MD, in data file
Chimeric antigen receptor T Cells with or
without hematopoietic cell transplantation for treatment of refractory Acute
Lymphocytic Leukemia led by Jae Park, MD, Miguel-Angel Perales, MD, and Sarah Nikiforow MD, PhD, in
Effect of stem cell boost and donor lymphocyte
infusion on the incidence of GVHD, led by James Yoon, MD, and Edmund Waller, MD, PhD, FACP, in protocol
Allogeneic hematopoietic cell transplantation vs
chimeric antigen receptor T-cell therapy for diffuse large B-cell lymphoma
patients with prior autologous transplant failure or refractory disease, led by
Mehdi Hamadani, MD, in protocol development.
Prolonged cytopenia following CD-19 targeted
CAR-T therapy for diffuse large B-cell lymphoma led by Mazyar Shadman, MD, in
The cellular therapy registry was launched in 2016, and data is still maturing. There is an opportunity now to move the portfolio of studies forward and accept new proposals to develop high-impact studies. The CICWC is accepting new study proposals which will be processed similarly to all proposals received annually by the CIBMTR. Study proposals will be triaged to the CICWC according to the study question and scope, and they will be reviewed by CICWC leadership. Selected proposals will be presented at the 2020 TCT Meetings for the committee to vote on and prioritize.
The CIBMTR is ecstatic with this newly developed working committee. The CICWC will continue to work towards improving the community's access to cellular therapy, while promoting the CIBMTR's mission of providing access to and improving outcomes of cellular therapies. Stay tuned for a standing column about cellular therapy news and updates coming soon!
The TCT | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (TCT Meetings) are the combined annual meetings of the ASTCT and the CIBMTR. This has been North America's largest international gathering of administrators, clinicians, data manager / clinical research professionals, fellows-in-training, investigators, laboratory technicians, MD / PhDs, nurses, nurse practitioners, pharmacists, physician assistants, and other allied health professionals in HCT and cellular therapy since 1999.
2020 Scientific Organizing Chairs, Mary Flowers, MD, and Katy Rezvani, MD, PhD, along with the scientific organizing committee and session chairs have assembled an excellent program. Over five days, leading international experts in the field of transplantation and cellular therapy will present the latest developments during plenary, concurrent, oral abstract, and poster sessions.
We hope to see you at the World Center Marriott in Orlando, Florida, February 19-23, 2020.
Go to the 2020 TCT Meetings Home Page to register and view additional details. The last day to receive general registration rates is January 25, 2020. After registering, take advantage of conference-specific guest room rates offered at several hotels in the TCT Meetings housing block. All hotels within this block are located within 4 miles of the TCT Meeting venues.
Grab your feathers, fedoras, and roaring 20s attire! Join your colleagues and friends to end a memorable week with the TCT Meetings Reception on Saturday, February 22, beginning at 7:45 PM at the World Center Marriott. Tickets are available online (and during the meetings at the registration desk) until Thursday, February 20, at 5:00 PM.
Attend several networking opportunities during the TCT Meetings, including poster sessions, the networking reception, the TCT Meetings Reception, and more.
The year 2020 marks 25 years of combined ASTCT and CIBMTR meetings. If you have meeting photos from previous years that you would like displayed and shared, please email them to TCTMeetings@mcw.edu or share them on Facebook or Twitter using the hashtags #TCTM20 and #CelebrateTCTM25
Questions regarding support opportunities at the 2020 TCT Meetings of ASTCT & CIBMTR may be directed to the TCT Meetings Conference Office: TCTMeetings@mcw.edu.
We look forward to seeing you in Orlando, Florida!
The fourth meeting of Data Managers in Bone Marrow Transplantation was held at the end of July – prior to the XXIII Congress of the Brazilian Society of Bone Marrow Transplantation (SBTMO). A core group of Brazilian data managers - Anderson Simione, Cinthya Correa da Silva, and Heliz Regina Alves das Neves - along with Marcelo Pasquini, MD, MS, were instrumental in getting this meeting off the ground, and it continues to grow! This year there were more than 50 participants, the largest number to date.
Presenters included CIBMTR Scientific Directors Marcelo Pasquini, MD, MS, and Bronwen Shaw, MD, PhD, as well as CIBMTR staff members Janet Brunner-Grady, PA-C, and Monique Ammi. Monique presented her topic "Step by Step for New Centers" in Portuguese. Other topics included:
AML, including a case study
Multiple myeloma, including a case study
Complications and late effects after HCT
NMDP/Be The Match and the Pediatric Bone Marrow Transplant Consortium (PBMTC) are excited to announce that the FDA has issued notification to proceed with a new CD33 CAR-T IND protocol. The study is overseen by the Data Safety Monitoring Committee of the PBMTC, with IND sponsorship held by Be The Match. The RCI BMT is overseeing all operational aspects of clinical study start-up, execution, and closure. Study co-chairs are Richard Aplenc, MD, PhD, of the Children's Hospital of Philadelphia and Nirali Shah, MD, MHSc, of the NCI.
This Phase I / II trial aims to determine the safety and feasibility of anti-CD33 chimeric antigen receptor (CAR) expressing T cells (CD33CART) in children and adolescents / young adults with relapsed / refractory AML. Phase I will determine the maximum tolerated dose of CD33CART cells using a 3+3 trial design. Phase II is an expansion phase designed to evaluate the rate of response to CD33CART. This trial will be conducted with the intent to use the CD33CART as a bridge for patients to receive a consolidative allogeneic HCT following CAR-T cell therapy.
The CD33CART product will be manufactured at NCI's Biopharmaceutical Development Program Frederick National Laboratory for Cancer Research with logistics, supply chain, and case management support provided by Be The Match. The study is expected to enroll 34 subjects over 36 months, with subjects followed for one year post-CAR-T infusion and for a total of 15 years for long-term follow-up.
The BMT CTN, with its 38 Core / Consortia Centers and approximately 75 Affiliate Centers, has now enrolled more than 11,000 patients! The Network was established in 2001 and is currently in its fourth grant cycle funded by the NHLBI and NCI.
The BMT CTN encourages widespread transplant community participation in all of its clinical trials. There are 11 trials open and 2 released to centers:
If your center is interested in participating, please visit the BMT CTN website.
There are 109 BMT CTN published articles, including 28 primary analyses. The following manuscripts were recently published:
Waller et al. Kinetics of immune cell reconstitution predict survival in allogeneic bone marrow and G-CSF-mobilized stem cell transplantation. Blood Advances. 2019 Aug 13;3(15):2250-2263. Epub 2019 Aug 13. ncbi.nlm.nih.gov/pubmed/31345792
Rashidi et al. The association of CMV with NK-cell reconstitution depends on grafts source: Results from BMT CTN-0201 samples. Blood Advances. 2019 Aug 27;3(16):2465-2469. Epub 2019 Aug 27. ncbi.nlm.nih.gov/pubmed/31427278
Pidala et al. Factors associated with successful discontinuation of immune suppression after allogeneic hematopoietic cell transplantation. JAMA Oncology. 2019 Sep 26. doi: 10.1001/jamaoncol.2019.2974. [Epub ahead of print] ncbi.nlm.nih.gov/pubmed/31556923
The CIBMTR shares administration of the BMT CTN Data and Coordinating Center with NMDP/Be The Match and The Emmes Company. Together, these three organizations support all BMT CTN activities. The BMT CTN Steering Committee is currently under the leadership of Chair Rick Jones, MD (Johns Hopkins). Helen Heslop, MD (Baylor College of Medicine), is Chair-Elect, and Ed Stadtmauer, MD (University of Pennsylvania), is Vice-Chair.
To receive up-to-date information about BMT CTN studies, meetings, and news:
Like us on Facebook: facebook.com/BMTCTN Follow us on Twitter: twitter.com/BMTCTN (@BMTCTN)
The Health Services Research Program collaborates with stakeholders, including patients, caregivers, researchers, and clinicians, to conduct and disseminate research that contributes knowledge to the cellular therapy field, and informs policy, clinical practice and survivorship care. The current research portfolio includes a focus on three areas: 1) Access to HCT / value and health economics / health disparities, 2) Survivorship / late effects / patient-reported outcomes, and 3) Treatment decision-making. Select studies from each portfolio are highlighted below.
Financial costs can be a barrier for patients accessing HCT. Medicare claims data and CIBMTR registry data were merged to create a unique dataset that contained data on reimbursement, service utilization, and overall survival. This merged dataset allowed for adjustment of more comprehensive patient- and HCT-related characteristics than administrative claims data or registry data alone. The aim of a recently published study was to assess reimbursement, utilization, and overall survival up to one year post-allogeneic HCT for Medicare beneficiaries aged 65 years or older with AML. Mean total reimbursement was $230,815 (95% CI: $214,381-$247,249) one year after alloHCT, with pharmacy being the costliest inpatient service category. Mortality increased with age, poorer Karnofsky Performance Score, and receipt of myeloablative conditioning. More information about this study can be found in the manuscript published in the Journal of the National Cancer Institute, Cancer Spectrum.
The Health Services Research Program recently acquired the most currently available Medicare (2010-2016) and Medicaid (2010-2014) data. These data will be linked to the CIBMTR registry data for the conduct of future studies focusing on access and health economics.
Patient-reported outcomes provide information from the patient perspective and can be used to inform care decisions. The Health Services Research Program and the Survey Research Group are collaborating on an upcoming study, "INSPIRE: A Multicenter Randomized Controlled Trial Integrating Health Informatics in a Scalable Stepped Care Self-Management Program for Survivors After HCT," funded by the NIH (PI: K. Syrjala, Fred Hutchinson Cancer Research Center). This multi-center clinical trial aims to determine the efficacy of a tailored, online, self-managed stepped care program and survivorship care plan intervention in adults 2-5 years post-HCT.
Donor selection practices are important for improving patient access to transplant and outcomes, as well as treatment decision-making. In collaboration with the NMDP/Be The Match Histocompatibility Advisory Group, the Health Services Research Program conducted a national survey of transplant physicians and search coordinators to better understand donor search and selection practices. Survey results showed that "urgent time to transplant" was most commonly defined as "transplant within 4 to 6 weeks of search initiation." Higher HCT urgency was associated with a higher disease risk index. For urgent cases with low probability of an 8 / 8 matched unrelated donor, 75% and 80% of physicians and coordinators endorsed a short (1 to 2 weeks) unrelated donor search before proceeding to an alternative donor source. Survey respondents strongly endorsed NMDP/Be The Match-provided solutions to expedite donor identification. Further details on this study are provided in the manuscript published in the Biology of Blood and Marrow Transplantation.
For more information about the program, please visit the Health Services Research webpage.
Share the 4 new patient-friendly summaries of CIMBTR publications with your patients. Watch for new summaries on the Study Summaries for Patients webpage:
BMT can treat blood cancers in people with HIV / AIDS
An early study shows that blood or marrow transplant (BMT) is as safe for people with HIV as for those without it.
Blood or marrow transplant helps treat leukemia after age 60
People older than 60 lived longer after getting BMT than chemo alone.
Haplo transplant helps people with leukemia
News may help people of all ethnicities get BMT sooner.
For some people older than 50, transplant from young, matched donors is better than half-matched donors
Study looked at ages and types of donors to treat people with acute leukemia.
Summaries are created through a collaborative process. Each quarter, members of the CIBMTR's Consumer Advocacy Committee (CAC) select studies. A team from the CIBMTR and NMDP/Be The Match writes, reviews, designs, posts, and promotes the summaries; team members include a Medical Writer, a Senior Patient Education Specialist, Scientific Directors, and a Communications Specialist.
The CAC was created in 2005 to communicate CIBMTR research to the public and to provide patient and donor perspectives during the development of the CIBMTR research agenda. Many CAC members have personal experience as a donor, recipient, or family member.
The CIBMTR fiscal year 2019 Facts and Figures document is now available to view on the CIBMTR Administrative and Progress Reports webpage.
This document provides an annual summary of the CIBMTR's accomplishments in each research program, key publications, and high priority initiatives.
Please email firstname.lastname@example.org if you would like a printed copy or copies mailed to you.
At any given time, the CIBMTR has >200 retrospective research studies and >20 prospective research studies ongoing. How many major areas of research activity does the CIBMTR have?
A. 7 B. 6 C. 5 D. 4
Enter your answer online. If you answer correctly, you will be entered into a drawing to win a CIBMTR prize.
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The CIBMTR is supported primarily by Public Health Service grant/cooperative agreement U24CA076518 with the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID); grant/cooperative agreement U24HL138660 with NHLBI and NCI; grant U24CA233032 from the NCI; grants OT3HL147741, R21HL140314 and U01HL128568 from the NHLBI; contract HHSH250201700006C with Health Resources and Services Administration (HRSA); grants N00014-18-1-2888 and N00014-17-1-2850 from the Office of Naval Research; subaward from prime contract award SC1MC31881-01-00 with HRSA; subawards from prime grant awards R01HL131731 and R01HL126589 from NHLBI; subawards from prime grant awards 5P01CA111412, 5R01HL129472, R01CA152108, 1R01HL131731, 1U01AI126612 and 1R01CA231141 from the NIH; and commercial funds from our corporate and private contributors.
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