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November 2024 Newsletter

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In Memory of Kristin Page

1974-2024

Kristin Page, MD, MHS, MEdCIBMTR mourns the recent loss of our colleague and friend, Kristin Page Chartrand, MD, MHS, MEd, after fighting a months-long illness.

Kristin was born in Cedar Rapids, IA, on June 7, 1974. Kristin, along with her brother, Joel Page were raised in Falmouth, MA, by their wonderful parents, Allan and Janice Page. In 2002, Kristin married Doug Chartrand and became the mother to three amazing children: Nate, Anna, and Mea.

She began her professional career as a high school science teacher and moved on to become a pediatric physician and researcher – finding joy in pursuing advancements in cancer research and even more comfort in taking care of families during their difficult journey.

In February 2021, Kristin joined CIBMTR as the Senior Scientific Director of Data Operations and Scientific Director of the Acute Leukemia Working Committee. She also served as Associate Professor of Pediatrics at Children’s Wisconsin. Before joining CIBMTR, she developed significant expertise in various aspects of transplant and cellular therapy, including focusing on data, quality, and informatics, at Duke University.

Kristin was warm, affectionate, funny, endlessly helpful, and a complete pleasure to work with. She loved meeting new people, heading into new situations, and helping however she could. She quickly became known throughout the CIBMTR community and formed relationships with many in the US and internationally. She grew our partnerships with several international groups, including EBMT and SBTMO. 

Kristin had a major impact across the organization – working closely with all groups to improve our data and drive innovation. She made important contributions to practice-changing research, such as in MRD. One of her most significant achievements was, with Larisa Broglie, MD, MS (CIBMTR Scientific Director), developing and pioneering the Working Committee Training and Leadership program. She had a passion for teaching, mentorship, and elevating the next generation of leaders in our field. From building a curriculum to arranging team building and making the best graphics with Canva (another thing she was good at!), she threw her energy and “spare time” into ensuring success for young investigators. She also played an integral role in mentoring medical students, residents, and fellows through work at Duke, the Medical College of Wisconsin, and ASH-a-Palooza. In honor of Kristin’s work and impact, the CIBMTR Working Committee Training and Leadership program will now be known as CIBMTR’s Page Scholars.

Outside of work, Kristin loved gardening, baking, and most importantly spending time with family and friends. Kristin had numerous roles in her amazing life; however, the role she cherished the most was that of “mom.” Spending time with her three children Nate, Anna, and Mea was her life's passion.

Kristin made numerous important contributions to CIBMTR – many of which will have a long-lasting impact. We all miss her greatly.

A Go Fund Me fundraiser has been created to help with Kristin's medical bills, the family's cost of living, and the children's educational futures. If you would like to offer support, please visit the fundraiser page.

Click here to donate.

Many colleagues and friends also offered words about Kristin; here are a few of them:

  • “She was one of the most impressive people I have met. She was such a wonderful mentor and advocate for all of us. She will be missed.”
    -Brian Ball, WCTL participant Non-Malignant Diseases Working Committee
     
  • “She was a fearless leader and will be missed!”
    -Jennifer Saultz, WCTL participant Immunobiology Working Committee
     
  • “I am completely speechless. What a loss for all of us.”
    -Mariam Nawas, WCTL participant Acute Leukemia Working Committee
     
  • “She has left an everlasting mark in this world personally and professionally and will be sorely missed. Her legacy will live on.”
    -Najla El Jurdi, WCTL participant GVHD Working Committee
     
  • “Kristin was such a wonderful person. I'm saddened at losing her as a mentor and advocate, and I'm sure I only was exposed to a sliver of all the wonderful things she has done. She went above and beyond and will truly be missed.”  
    -Megan Herr, WCTL participant Donor and Recipient Health Services Working Committee
     
  • “As one of Kristin's mentees, she was instrumental in my professional growth and always provided unwavering support. Kristin had a unique ability to inspire and uplift those around her.”
    -Firas El Chaer, Acute Leukemia Working Committee PI and mentee
     
  • “My heart is hurting from the loss of such a beautiful soul. I had so much love and respect for her.”
    -Nelli Bejanyan, Acute Leukemia Working Committee Chair
     
  • “It is hard to process. To me, Kristin has always been more than just a director—she was like a teacher, consistently kind and supportive. Having her as my Scientific Director during my first year at CIBMTR was truly fantastic. I still cherish the memory of our wonderful ‘yacht’ trip in San Antonio at the 2024 Tandem (Meetings). This moment, along with many others, will always be missed.”
    -Dylan Liu, Acute Leukemia Working Committee Statistician
     
  • “You said it perfectly, she was ’warm, affectionate, funny, endlessly helpful, and a complete pleasure to work with.’ She lived the ’work hard and be nice to people’ ethic. Kristin was an amazing advocate for early career faculty even as she grew her own career.”
    -Christopher Hourigan, Acute Leukemia Working Committee Chair
     
  • “I also echo what others have written and remember Kristin’s great dedication to the field of BMT and her tireless efforts in this regard. She stepped into the role of Scientific Director for the Acute Leukemia Working Committee effortlessly and carried the mission of our committee along without us missing a beat.”
    -Mark Litzow, Acute Leukemia Working Committee Chair
     
  • “I can only echo everyone's words and share your profound sense of loss. I met her 5 years ago as I was just starting in this field, and she became one of my best mentors and advocates. It is a true loss.”
    -Alexandra Gomez-Arteaga, Acute Leukemia Working Committee PI and mentee

 

Perspectives: 
Navigating the Heterogeneity in Blood and Marrow Transplantation and Cellular Therapy

By Michael Verneris, MD

Michael Verneris, MD

The BMT and cellular therapy field has witnessed remarkable advancements over the past few decades. From the early introduction of calcineurin inhibitors and the recognition of HLA-C matching to recent innovations in GVHD prophylaxis and CAR-T therapy, our field has made numerous advances that have led to standardized approaches and significantly improved patient outcomes. Despite these advancements, significant variability in clinical practice remains across individual physicians and centers. While some of this is necessary due to patient needs and practical considerations, there is room to re-examine our care model and identify areas that could further improve patient outcomes. Here, I aim to explore the dichotomy between established treatment approaches and the heterogeneous nature of care delivery within BMT and cellular therapy and to discuss the potential for unifying approaches to improve patient care.

Standardization: Achievements and Gaps

In many respects, the BMT and cellular therapy fields have made significant strides towards standardization. Approaches for patient selection, stem cell collection, conditioning regimens, and GVHD prophylaxis are well-established and widely adopted. Research studies from CIBMTR have led to guidelines put forth by individual researcher teams or organizations like the ASTCT and serve as valuable resources, providing evidence-based recommendations that have been instrumental in guiding practice.

Yet, it is also important to acknowledge that not all aspects of our care are uniform. Certain treatment approaches can differ considerably. This variation often stems from patient-specific needs, such as underlying disease characteristics, comorbidities, and individual responses to therapy. Moreover, the decision to use a different stem cell source or preparative regimen—often reflects the nuanced needs of a patient rather than a clear superiority of one source over another. 

Areas of Significant Variation

While some aspects of BMT and cellular therapy have coalesced around a few predominant approaches, other areas exhibit considerable heterogeneity, which seem to not be evidence-based. A few that come to mind include the following:

  1. Nutritional Support: The details around nutritional management seem to vary considerably before, during, and after transplantation. For instance, some physicians are quick to introduce enteral feeding while others start parental nutrition, and still others take a watchful waiting approach to patients with anorexia after transplantation. Given the growing recognition of the importance of the microbiome, these variations may have considerable impact.  
  2. Monitoring Immune Reconstitution: The assessment of immune reconstitution post-transplant is critical for evaluating immune recovery and managing prophylactic medications and other decisions including return to school or work. However, the timing, methodology, and response to immune reconstitution data seem to vary widely.  
  3. Management of Mixed Donor-Recipient Chimerism: The approach to mixed chimerism post-transplant also exemplifies the diversity of practice patterns. Some advocate for a strategy of tapering immunosuppression while others augment it, and still others deliver donor lymphocyte infusions or simply observe the patient. While some of this is context-dependent (i.e., malignant vs. non-malignant disease), this lack of consensus highlights the need for more standardized approaches.  
  4. Vaccination Strategies: Revaccination approaches after transplantation also demonstrate considerable variability. Some centers base their vaccination schedules on time post-transplant while others utilize immune reconstitution metrics to inform decisions, and the ones that use the latter approach (immune-based metric) vary in what immune parameters trigger vaccination. 

While some of these practices can be captured in databases such as CIBMTR, the complexity of these data can lead to challenges in reporting and analyzing these data effectively. The heterogeneity in clinical practices often translates into data that are difficult to standardize and interpret, limiting the applicability of findings to broader populations.

The Need for Unification

Given the significant variability in practice, it is time for the BMT and cellular therapy community to consider unifying some of the above clinical management strategies. Establishing and implementing consensus guidelines for areas that lack standardized practices may improve patient outcomes by fostering a more cohesive approach to care. This is particularly important in the pediatric space, where smaller and heterogeneous patient populations make achieving statistical power and drawing robust conclusions from isolated practices extremely challenging. 

Unifying practices could also facilitate more effective data collection and analysis, allowing for better understanding of the efficacy of various interventions. Standardized reporting metrics would also enhance the ability to assess the utility of different approaches, enabling the field to move forward based on evidence rather than anecdote.

Challenges to Implementation

Implementing a more unified approach is not without its challenges. The diversity of practice patterns often reflects local expertise, resources, patient demographics, and treatment philosophy. Additionally, distinct institutional policies and logistical constraints may also undermine reaching consensus. To be sure, the need for flexibility must be considered. And, while some degree of standardization is beneficial, it is equally important to allow for adaptations based on individual patient needs. Thus, a “one-size-fits-all” approach will not be feasible or appropriate in every case. 

Moving Forward

Nevertheless, the pursuit of unifying these practices is worth undertaking. One important approach would be to engage stakeholders with specific expertise in the above areas during workshops and / or consensus conferences and to publish guidelines that outline best practices. We have achieved similar success in developing consensus guidelines in other aspects of our field, such as chronic GVHD. Pursuing an analogous consensus-building framework in the BMT context could yield immense benefits.

Despite challenges, data-driven unification of these clinical practices is worthwhile. A key step in this process is engaging a wide range of stakeholders with varying expertise to foster collaboration and consensus-building. One effective approach to achieving consensus is through the organization of focused workshops or consensus conferences, where experts come together to share their perspectives and experiences, review existing data, and collectively decide on best practices. These events could be structured to tackle specific areas of variability, promoting in-depth discussion and analysis of current practices and emerging evidence. The publication of evidence-based guidelines that outline best practices is another crucial step. This approach has proven successful in other areas of the field, such as the management of chronic GVHD, in which gaps have been identified and standardized protocols proposed. A similar strategy could be applied to the areas of variability discussed earlier, providing clinicians with clear recommendations that reduce heterogeneity and ensure a higher level of consistency in patient care across centers.

In summary, while the task of unifying clinical practices in BMT and cellular therapy is complex, the potential benefits for patients and the field make it a pursuit well worth undertaking. Through collaborative efforts, focused workshops, and the development of robust, adaptable guidelines, we can work toward greater standardization, improved patient outcomes, and more efficient use of resources. 

 

Kwang Woo Ahn Promoted to CIBMTR Statistical Director

Kwang Woo Ahn, PhDWe are pleased to announce that Kwang Woo Ahn, PhD, has been appointed Chief Statistical Director of the CIBMTR, effective September 1, 2024. Dr. Ahn is Professor in the Division of Biostatistics at MCW. He has served as Statistical Director for various committees of the CIBMTR since joining MCW in 2008, including the Lymphoma, Pediatric Cancer, Regimen-Related Toxicity and Supportive Care, Chronic Leukemia, and Infection and Immune Reconstitution Working Committees. For the past 2 years, he has served as CIBMTR’s Associate Chief Statistical Director.  
 
Dr. Ahn has published more than 150 peer reviewed manuscripts, including 25 methodologic publications in leading statistical journals focusing on survival and competing risks data, variable selection, and high-dimensional data analysis; most of these publications are directly applicable to the analysis of transplant and cell therapy data. He also successfully obtained multiple research grants, including a contract as PI with the FDA on “Commensurate Prior Models Accommodating Historical Controls for Clinical Trials with Matched and/or Interval-Censored Data” as well as past grants with the National Science Foundation, the Advancing a Healthier Wisconsin endowment, and the MCW Cancer Center. Dr. Ahn takes over this role from Dr. Mei-Jie Zhang, who has served since 2015.   

Dr. Ahn said, “I am truly honored to step into the role of Statistical Director. CIBMTR has been instrumental in shaping my journey as a statistician. I am always grateful to CIBMTR for this and view this opportunity as a chance to give back. Together with the talented team and leadership, I look forward to advancing our shared mission of improving patient outcomes through collaborative research and innovative statistical approaches.”

Please join us in congratulating Dr. Ahn on his promotion to this role, and thanking Dr. Zhang for his wonderful service to CIBMTR as Chief Statistical Director these past 9 years.  

 

Chronic Leukemia Working Committee

Committee Leadership
Betul Oran Mark Juckett Michael Grunwald  

Betul Oran, Co-Chair
M.D. Anderson Cancer Center, Houston, TX

 

Mark Juckett, Co-Chair
University of Minnesota, Minneapolis, MN

 

Michael Grunwald, Co-Chair
Levine Cancer Institute, Charlotte, NC

 

 
Hany Elmariah Wael Saber Soyoung Kim Charimar Santiago Parrilla

Hany Elmariah, CIBMTR Page Scholar
Moffitt Cancer Center, Tampa, FL

Wael Saber, Scientific Director
CIBMTR MCW

Soyoung Kim, Statistical Director
CIBMTR MCW

Charimar Santiago Parrilla, Statistician
CIBMTR MCW

The main goals of the Chronic Leukemia Working Committee are to help establish the optimal timing of HCT for patients with MDS, CML, CLL, MPN, and MDS/MPN overlap syndromes and to improve HCT outcomes for these patients. While the role of HCT for many of these diseases is increasing, relatively low disease prevalence limits the feasibility of prospective clinical trials. Of critical importance, many high-impact HCT trials exclude patients with chronic leukemias, such as myelofibrosis and chronic myelomonocytic leukemia. High-quality, retrospective data are often the most rigorous option to optimize HCT strategies for chronic leukemias. 

Leveraging the resources of CIBMTR’s database, the Chronic Leukemia Working Committee published practice-changing studies that answer key questions regarding HCT for chronic hematologic malignancies. During the last few years, the committee's efforts resulted in several published manuscripts and oral and poster presentations. The committee also maintains the MDS and MPN data collection forms to capture disease status and treatment responses more accurately.   

To help foster new study proposals, our committee provides members with disease-specific lists of accepted studies and prior proposals that were not accepted, with the rationale for the decision. This information allows investigators to focus new study proposals on feasible and not redundant concepts. 

A few of the recently published studies are described below:

  • CK21-01 Haploidentical donor transplantation versus matched or mismatched-unrelated donor allogeneic BMT outcomes in patients with myelofibrosis
    Citation: Jain T, Estrada-Merly N, Queralt Salas M, et al. Donor types and outcomes of transplantation in myelofibrosis: A CIBMTR study. Blood Advances. 2024 Aug 27; 8(16):4281-4293. doi:10.1182/bloodadvances.2024013451. Epub 2024 Jun 25. PMC11372592.Despite the increasing use of haploidentical donor HCT with post-transplant cyclophosphamide (PTCy), data supporting this platform in myelofibrosis has been limited. Tania Jain, MBBS, and the Chronic Leukemia Working Committee compared matched sibling donors (MSD), matched unrelated donors (MUD), MMUD, and haploidentical HCT with PTCy in 1,032 patients with myelofibrosis. HCTs using MSDs yielded superior overall survival, while MUDs, MMUDs, and haploidentical HCTs had comparable outcomes. This study has provided the strongest published evidence to support the usage of haploidentical donor HCT for myelofibrosis.    
     
  • CK20-01 Outcomes of allogeneic HCT for myelofibrosis based on the conditioning regimen
    Citation: Murthy GSG, Kim S, Estrada-Merly N, et al. Association between the choice of the conditioning regimen and outcomes of allogeneic hematopoietic cell transplantation for myelofibrosis. Haematologica. 2023 Jul 1; 108(7):1900-1908. doi:10.3324/haematol.2022.281958. Epub 2023 Feb 14. PMC10316233.While prospective studies have compared conditioning regimens in AML and MDS, Hemant Murthy, MD, and the committee recognized the need for clearer guidance for HCT conditioning for myelofibrosis. We compared specific conditioning regimens in 872 patients with myelofibrosis, stratified by MAC and RIC. Among 379 patients receiving MAC regimens, fludarabine / busulfan yielded optimal GVHD-free relapse-free survival and lower rates of GVHD by multivariate analysis. Similarly, multivariate analysis of 493 patients who received RIC regimens demonstrated superior overall survival, non-relapse mortality, and acute GVHD with fludarabine / busulfan. Since a prospective study is unlikely, this publication justifies the adoption of busulfan containing conditioning regimens for patients with myelofibrosis. 
     
  • CK18-02 The impact of somatic mutations on allogeneic transplant in chronic myelomonocytic leukemia
    Citation: Mei M, Pillai R, Kim S, et al. The mutational landscape in chronic myelomonocytic leukemia and its impact on allogeneic hematopoietic cell transplantation outcomes: A Center for Blood and Marrow Transplantation Research (CIBMTR) analysis. Haematologica. 2023 Jan 1; 108(1):150-160. doi:10.3324/haematol.2021.280203. Epub 2022 Apr 21. PMC9827167.The mutational landscape has proven a critical prognostic tool across myeloid malignancies in both the pre-transplant and post-transplant settings. Led by Matthew Mei, MD, our committee studied the prognostic significance of somatic mutations in chronic myelomonocytic leukemia (CMML) patients receiving an allogeneic HCT. Clinical data was collected from CIBMTR’s database while mutational profiling was performed ad hoc for this study in 313 patients with an available pre-conditioning peripheral blood sample. Mutations in DNMT3A, JAK2, and TP53 were associated with worse disease-free survival while mutations in DNMT3A and TP53 were associated with decreased overall survival. While refining prognostic predictive tools for CMML patients undergoing allogeneic HCT, this study also demonstrated CIBMTR’s unique ability to combine existing data with well-designed lab-based science to perform a meaningful study. 
     
  • CK17-01 Development of a prognostic scoring system predictive of outcomes in patients with myelofibrosis after allogeneic hematopoietic cell transplantation
    Citation: Tamari R, McLornan DP, Ahn KW, et al. A simple prognostic system in patients with myelofibrosis undergoing allogeneic stem cell transplantation: A CIBMTR/EBMT analysis. Blood Advances. 2023 Aug 8; 7(15):3993-4002. doi:10.1182/bloodadvances.2023009886. Epub 2023 May 3. PMC10410129. Multiple prognostic models have been developed for myelofibrosis in the pre-transplant setting. To develop a prognostic model for myelofibrosis in the post-transplant setting, Roni Tamari, MD, studied a cohort of myelofibrosis patients in CIBMTR’s database and supplemented it with a cohort from the EBMT registry. We were able to identify significant predictors of survival, including patient age, anemia, and donor source, leading to the development of a prognostic score validated in both cohorts.

In addition to these recent publications, the Chronic Leukemia Working Committee keeps the momentum going with many exciting studies currently in progress, including: 

  • CK16-01B Identification of germline predisposition mutations in young MDS patients 
  • CK22-01 Impact of somatic mutations on outcomes after allogeneic HCT in patients with MDS with ring sideroblasts (MDS-RS) and MDS / MPN with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T)
  • CK22-02 Toxicity and survival of AML / MDS patients receiving allogeneic HCT using RIC: A propensity score analysis
  • CK23-01 Identifying the optimal GVHD regimen in allogeneic HCT for myelofibrosis
  • CK24-01 Identifying the optimal stem cell dosing for PBSC HCT with post-transplant cyclophosphamide
  • CK24-02 Outcomes of allogeneic HCT in patients with DDX41-mutated MDS and AML
  • CK24-03 Comparison of RIC regimens for haploidentical donor HCT with post-transplant cyclophosphamide in patients with AML or MDS
  • CK24-04 Comparison of post-transplant cyclophosphamide-based RIC regimens for older patients with AML and MDS
  • CK23-02 The mutational landscape in MDS arising from aplastic anemia and its impact on allogeneic HCT outcomes

The Chronic Leukemia Working Committee welcomes new participants and new proposals. We also encourage collaboration with other committees and the use of outside data sets, which can better define the role and timing of transplantation as new non-transplant strategies emerge. We encourage young investigators to take part in the committee, which provides them with an excellent opportunity to become familiar with novel study designs for observational research and the statistical methodologies utilized. The next in-person meeting of the Chronic Leukemia Working Committee will be at the Tandem Meetings in Honolulu, Hawaii, in February 2025. The working committee is proud to have advanced the field with the great work done over the past years. We look forward to reviewing new proposals to continue improving patient outcomes. 

View planned, in-progress, and completed studies and publications on the Chronic Leukemia Working Committee webpage.

 

Morbidity, Recovery and Survivorship Working Committee

Committee Leadership
Hélène Schoemans Mohamed Sorror Sairah Ahmed Seth Rotz
Hélène Schoemans, Co-Chair
University Hospital Gasthuisberg, Leuven, BE
Mohamed Sorror, Co-Chair
Fred Hutchinson Cancer Center, Seattle, WA
Sairah Ahmed, Co-Chair
M.D. Anderson Cancer Center, Houston, TX
Seth Rotz, Co-Chair
Cleveland Clinic Foundation, Cleveland, OH
Michelle Schoettler Amy Moskop Rachel Phelan Ruta Brazauskas
Michelle Schoettler, CIBMTR Page Scholar
Emory/Children’s Healthcare of Atlanta, Atlanta, GA
Amy Moskop, Scientific Director
CIBMTR MCW
Rachel Phelan, Scientific Director
CIBMTR MCW
Ruta Brazauskas, Statistical Director
CIBMTR MCW
Andrew Peterson Brandon Nuechterlein Rebecca Higgins  
Andrew Peterson, Statistician
CIBMTR NMDP
Brandon Nuechterlein, Consumer Advocacy Committee Member
Denver, CO
Rebecca Higgins, Consumer Advocacy Committee Member
Milwaukee, WI
 

The Morbidity, Recovery and Survivorship Working Committee provides scientific oversight for studies related to the post-HCT / cellular therapy trajectory, except for GVHD toxicities. This ranges from consequences of using particular preparative regimens, prevention strategies, or supportive care to issues related to long-term survivorship, such as organ toxicity and psychosocial effects on health-related quality of life. We take advantage of CIBMTR’s large and representative clinical database to study these early complications and late effects of treatment, which include characterizing specific toxicities as well as understanding which survivors are at greatest risk for developing these complications. A better understanding of HCT / cellular therapy-related early toxicities and late effects is important not only for improving supportive care and surveillance in clinical practice but also for the development of new strategies associated with low rates of undesirable complications of transplantation, resulting in improvements in recovery and subsequent survivorship. Such studies also allow us to give our patients and their loved ones realistic prospects regarding their recovery after undergoing HCT / cellular therapy. 

Because of the increased number of CAR-T-focused proposals submitted in recent years, CIBMTR decided that CAR-T studies focusing on toxicities will now be part of the Morbidity, Recovery and Survivorship Working Committee. These studies were previously part of the phased-out Cellular Immunotherapy for the Cancer Working Committee. That committee’s research portfolio made great progress in pushing their studies forward with several manuscripts submitted this fall. 

The Morbidity, Recovery and Survivorship Working Committee meets annually in person at the Tandem Meetings. The Co-chairs, CIBMTR Scientific Director, Consumer Advocacy Committee members, and statisticians meet monthly by teleconference to ensure the timely completion of projects, reassess priority areas, and promote and develop the scientific agenda. The 2024 committee meeting in San Antonio, TX, enjoyed continued strong participation with many in-person and virtual attendees. 

A list of the committee’s studies, including recent publications, is provided on the committee’s webpage. Our recent work highlights several complications based on data collected in the pediatric intensive care setting; the importance of late effects in non-malignant diseases; and psychosocial issues, such as return to work after HCT and how social wellbeing is associated with conserved transcriptional response to adversity transcription profiles. The committee also recently provided international guidance on male-specific late effects and general screening and preventative practices in long-term survivors. The enthusiasm and engagement of the participants in the activities of the Morbidity, Recovery and Survivorship Working Committee have been and will continue to be key to the accomplishments achieved, making us very grateful for all that has been achieved in the past few years.

Our extensive portfolio also comprises multiple protocols in development, analysis, and manuscripts in preparation. These cover a wide range of topics germane to the care of HCT and cellular therapy recipients, including long-term survivors. The Morbidity, Recovery and Survivorship Working Committee has also engaged in collaborative efforts between CIBMTR and other organizations, such as the United Network for Organ Sharing (UNOS), Virtual Pediatric Systems (VPS), and the Childhood Cancer Survivor Study (CCSS).

Additionally, the committee remains involved in other activities, including projects that lead to published reviews targeting areas of post-transplant late effects. These projects have created additional opportunities for junior investigators to become actively involved in our committee. Building on our strong history of international collaboration, we have worked diligently with our EBMT colleagues to develop a process calling for proposals, choosing a pertinent late effects topic, and then conducting a formal systematic review on the chosen topic. Currently, a systematic review focused on female-specific late effects is being initiated, and we are excited to continue this effort moving forward.

The Morbidity, Recovery and Survivorship Committee’s success depends on new ideas, testable hypotheses, and the participation of individuals with different perspectives and scientific backgrounds. We seek novel ideas and encourage the involvement of junior investigators interested in outcomes research. We believe early involvement leads to the long-term participation of junior investigators in committee activities.

Before you propose a study, please review the data forms available online to determine if the data are available to answer your question. Since some of the proposed studies may require data across many decades, it is possible the type and way the data was collected also changed with time. Please reach out early if there are any questions regarding data availability.

To learn more about the committee or to discuss your research ideas and proposals, we encourage you to contact one of the members of the committee leadership team (listed above). We look forward to your participation in upcoming studies and at future Tandem Meetings!

 

Register for the 2025 Tandem Meetings Today

Tandem Meetings

By Alicia Halfmann and Maira Brey

ASTCT® and CIBMTR® are pleased to announce that registration is open for the 2025 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR. We hope you will join us February 12-15, 2025, in Honolulu, HI, for opportunities to learn, collaborate, and advance your expertise.

The 2025 Tandem Meetings will feature In-Person Attendee and Digital Access Attendee options, so everyone can experience this premier event in the evolving HCT and cellular therapy field. Once registration is completed, please be sure to book your hotel within the Tandem Meetings Housing Block. For more housing details, click here.

To access the online agenda, please visit the Tandem Meetings Website.

Follow ASTCT and CIBMTR on social media, as well as the official hashtag, #Tandem25, for updates.

We look forward to seeing you at the 2025 Tandem Meetings in Honolulu, HI!

 

HLA-DQ Heterodimers Tool to Enhance Donor Selection by Lowering Relapse Risk

By Ray W. Sajulga Jr.

Transplant centers considering HLA-DQB1 for matching can further optimize their matches by considering HLA-DQA1 to lower relapse risk. Previous research by Petersdorf et al. (2022) identified the importance of HLA-DQ heterodimers; researchers found that the presence of or mismatching against group 2 (G2) HLA-DQ heterodimers was associated with a higher relapse risk relative to group 1 (G1) HLA-DQ heterodimers. To enhance donor selection, we collaborated with Effie Petersdorf, MD, from the Fred Hutchinson Cancer Center to design a tool called QLASSy (HLA-DQA1 and HLA-DQB1 Heterodimers Assessment Tool) for classifying these HLA-DQ heterodimers. To validate the tool, we leveraged 352,148 high-confidence, statistically phased haplotypes, 1,052 pedigree-phased haplotypes, and 13,663 historical transplants with HLA-DQ data.

For data quality, we:

  1. Provided in-silico validations for 99.7% of our observed trans-heterodimers (which serve as the basis for individuals containing as many as four unique HLA-DQ heterodimers)
  2. Developed high-confidence imputation of HLA-DQ information via HLA-DRB1 with >99% correct predictions
  3. Corrected a handful of HLA genotypes in the database that had unexpected combinations that did not adhere to G1 / G2 assignments

Additionally, we embellished our knowledge of HLA-DQ heterodimers by:

  1. Providing a synopsis of HLA-DQ frequencies across various self-identified race and ethnicity categories 
  2. Visualizing the effect of HLA-DQA1 and HLA-DQB1 mismatching on HLA-DQ annotations for x/8 and x/10 models
  3. Analyzing historical search lists that would have benefited from a tool like this

Our tool supports the findings from these analyses by flagging any unexpected data from either cis-heterodimers or trans-heterodimers, imputing missing HLA-DQA1, and annotating HLA-DQ information in a highly interactive, visual, and comprehensive tool. By automating this complex model, we can stay up to date with the latest science to select the best possible source for our patients.

Access the tool: https://dq-dimers.nmdp.org/

Study Contributors: Ray Sajulga, Yung-Tsi Bolon, Martin J. Maiers, Effie W. Petersdorf. Click here to view the study published in the Transplantation and Cellular Therapy journal on August 15, 2024.

HLA-DQ Heterodimers Tool to Enhance Donor Selection by Lowering Relapse Risk

 

Data Science Competition

By Tushar Deshpande

The Bioinformatics Research team has partnered with Kaggle (Google) to organize a hackathon that will bring together data scientists, machine learning practitioners, industry and academic partners, and healthcare enthusiasts for a collaborative and competitive event. The competition has two main objectives:

  • 1) To enhance the accuracy of predictions for transplant survival rates for patients undergoing allogeneic HCT by developing fair predictive models that can accelerate progress
  • 2) To raise awareness about our organization's mission and its dedication to expanding access to healthcare.

This initiative aims to drive scientific innovation and establish our organization as a leading partner in data science and machine learning, encouraging stakeholders to join us in our journey toward a more equitable and impactful future in healthcare.

NOW AVAILABLE: https://www.kaggle.com/competitions/equity-post-HCT-survival-predictions

 

CIBMTR CRO Services: PRESERVE 1 First Patient Enrolled

CIBMTR is thrilled to announce the first enrollment in the PRESERVE I trial (NCT05589896) “A first-in-human study of HLA-partially to fully matched allogeneic cryopreserved deceased donor BMT for patients with hematologic malignancies.” The first patient to receive Ossium HPC, Marrow was infused at the University of Utah – Huntsman Cancer Institute. 

The clinical trial sponsor, Ossium Health, has developed a proprietary process to recover bone marrow from deceased organ donor vertebral bodies and to cryopreserve the product. Ossium’s HPC, Marrow product aims to provide an on-demand, bone marrow stem cell graft of adequate cell dose from a screened deceased donor that would enable patients without an available, suitable living donor to receive allogeneic HCT.

The PRESERVE I study will continue to enroll 11 more subjects over the next year at 10-12 transplant centers across the US. Subjects will be followed for 1 year post-transplant to evaluate the safety and efficacy of the Ossium HPC, Marrow product.

For more information about the clinical trial, please contact the PRESERVE I study team at PRESERVE@NMDP.ORG.

 

BMT CTN Grant Renewed

By Mykala Heuer, BSN, RN

BMT CTN LogoThe BMT CTN has now enrolled more than 17,000 patients. The BMT CTN was established in 2001 and is funded by the NHLBI and NCI. We are excited to announce that BMT CTN was renewed for a fifth grant cycle, which started this summer! 19 Core Centers were awarded with several being consortia, which are comprised of two or more centers. 

Clinical Trials: Open Enrollment

The BMT CTN encourages widespread transplant community participation in clinical trials. If your center is interested in participating, please visit the BMT CTN website.

There are 9 trials open to accrual. Of the BMT CTN-led trials, one has been released to centers, and three are in development. The following BMT CTN protocol was recently released to centers: 

  • BMT CTN 2207 - A Phase II trial of non-myeloablative conditioning and transplantation of haploidentical related, partially HLA-mismatched, or matched unrelated bone marrow for newly diagnosed patients with severe aplastic anemia.

If your center is participating in BMT CTN 2203 or BMT CTN 2207, please consider participating in BMT CTN 2302 - Facilitating Activation of Study Trials (FAST), which is a time-and-motion study to understand the infrastructure, processes, barriers, and effective and ineffective center practices related to activation of a cooperative group trial.

BMT CTN Publications

There are 182 BMT CTN published articles, including 44 primary analyses. In 2024, we published 2 primary results articles:

  • BMT CTN 1506: Levis et al. Gilteritinib as post-transplant maintenance for AML with internal tandem duplication mutation of FLT3. Journal of Clinical Oncology. 2024 May 20; 42(15):1766-1775. Epub 2024 Mar 12. https://pubmed.ncbi.nlm.nih.gov/38801746/ 
  • BMT CTN 2101: Hill et al. SARS-CoV-2 vaccination in the first year after HCT or CAR T-cell therapy: A prospective, multicenter, observational study. Clinical Infectious Diseases: An official publication of the Infectious Diseases Society of America. 2024 Aug 16;79(2):542-554. Epub 2024 May 27. https://pubmed.ncbi.nlm.nih.gov/38801746/
About the BMT CTN

CIBMTR shares administration of the BMT CTN Data and Coordinating Center with NMDP and The Emmes Company. Together, these three organizations support all BMT CTN activities. The BMT CTN Steering Committee is currently under the leadership of John Levine, MD, MS (Mount Sinai), as Steering Committee Chair; Stephanie Lee, MD, MPH (Fred Hutchinson Cancer Center), as Steering Committee Vice-Chair; and Edward Stadtmauer, MD (University of Pennsylvania), as Steering Committee Past-Chair.

To receive up-to-date information about BMT CTN studies, meetings, and news, be sure to follow us on X (previously known as Twitter): @BMTCTN

 

Stem Cell Therapeutic Outcomes Database: Center-Specific Survival Analysis

By Carol Doleysh

The SCTOD is part of the US Health Resources and Services Administration (HRSA)-funded C.W. Bill Young Cell Transplantation Program that collects data on all allogeneic hematopoietic cell transplants performed in the US and on transplants done elsewhere using cellular products that originated in the US. 

During the 2023 Center Outcomes Forum, stakeholders recommended CIBMTR hold an informational session during the Tandem Meetings to provide key updates about the Center-Specific Survival Analysis, including updated risk adjustment, analysis findings, and future plans. This session will offer important stakeholder groups, especially center medical and administrative directors, and data professionals the opportunity to learn more about the Center-Specific Survival Analysis firsthand. Planning for this meeting is underway.

CIBMTR is also implementing other stakeholder recommendations from the Center Outcomes Forum, including: 

  • Providing a simplified one-page summary of the Center-Specific Survival Analysis report that highlights changes in the risk adjustment model and provides overall center results as a cover page to the full report.
  • Maintaining educational tools that explain the Center-Specific Survival Analysis risk-adjustment model and address common misunderstandings about the analysis. This information can be found on the dedicated Center-Specific Survival Analysis page on the CIBMTR website.
  • Reminding centers about quality improvement tools available on the CIBMTR Portal, where the Center-Specific Survival Analysis report, one-year survival calculator, individual center datasets, and additional tools are accessible.
  • Continuing to collect essential information about recipients’ participation in clinical trials, including trial sponsor, study number (for national / cooperative group studies), and ClinicalTrials.gov identification number (NCT #) to facilitate data linking as appropriate and to support future research proposals about the impact of trial participation on outcomes.
  • Revising data collection tools to include new or updated information and remove information that is obsolete or no longer used. Specific recommendations included:
    • Accommodating the World Health Organization 2022 disease classification.
    • Updating cytogenetic, fluorescence in situ hybridization, and molecular options for several diseases.
    • Updating disease response criteria, particularly MRD status assessment for AML, ALL, and MDS.
    • Other revisions to disease- and treatment- related characteristics.

 

Risk Assessment for T-Cell Malignancies After CAR-T Therapy

In November 2023, the FDA released a communication about the risks of T-cell malignancies after CAR-T therapy. Read more about CIBMTR’s response to this communication in our May 2024 newsletter.

CIBMTR remains focused on assessing the risk of subsequent primary malignancies, timing, subtypes of malignancies, and risk factors. CIBMTR’s ACT Council created a Subsequent Primary Malignancies Task Force that includes insight and expertise of the ACT Council members, physicians, patient support groups, and professional societies to address the magnitude of the risk of these complications and to create an integrated approach to recognize and capture these events. The Task Force’s recommendations will be presented to the CIBMTR Advisory Committee in February 2025.

 

Gene Therapy Post-Authorization Safety Studies (PASS)

CIBMTR continues to work with two pharmaceutical companies to establish long-term follow-up studies for patients receiving gene therapy for the treatment of cerebral adrenoleukodystrophy, transfusion-dependent beta thalassemia, and sickle cell disease.

Several of these studies have launched, with others planned to follow late 2024 / early 2025. Each study will have a 15-year follow-up period as part of PASS. CIBMTR recently began site training for these PASS at multiple centers and also updated the disease-specific forms to capture key data elements. The sickle cell form updates will be released in Fall 2024.

 

Publicly Available Datasets

Public Datasets Webpage ScreenshotView a summary of the Publicly Available Datasets and the data dictionary containing the most commonly used variables.

In accordance with the NIH Data Sharing Policy and NCI Cancer Moonshot Public Access and Data Sharing Policy, CIBMTR makes the final datasets from published studies publicly available on CIBMTR’s Research Datasets for Secondary Analysis webpage. These publication analysis datasets are freely available to the public for secondary analysis.

While providing these data, CIBMTR is committed to safeguarding the privacy of participants and protecting confidential and proprietary data. Upon accessing the datasets page on CIBMTR’s public website, the viewer is notified that the dataset was collected by CIBMTR, and CIBMTR’s supporters are listed. The webpage also clearly notes the terms and conditions of dataset usage.

NEW datasets are now available online.

 

Share Your Research in Plain Language

By Jennifer Motl

These new plain-language summaries of CIBMTR research may help your patients:

Medicine given to donors of blood stem cells is safe: 7-year study of filgrastim shows it does not cause blood disorders

Medicine given to donors of blood stem cells is safe: 7-year study of filgrastim shows it does not cause blood disorders; read more: 

Umbilical cord blood transplants help people of all races

Umbilical cord blood transplants help people of all races; read more: 

Overweight not linked to relapse or quality of life after transplant: For adults with multiple myeloma, being overweight is not worrisome

Overweight not linked to relapse or quality of life after transplant: For adults with multiple myeloma, being overweight is not worrisome; read more:

Find more summaries on the Study Summaries for Patients webpage.

 

Our Supporters

CIBMTR is supported primarily by Public Health Service U24CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); U24HL138660 from NHLBI and NCI; 75R60222C00008, 75R60222C00009, and 75R60222C00011 from the Health Resources and Services Administration (HRSA); and N00014-23-1-2057 and N00014-24-1-2057 from the Office of Naval Research. 

Additional federal support is provided by OT3HL147741, P01CA111412, R01CA100019, R01CA218285, R01CA231838, R01CA262899, R01AI128775, R01AI150999, R01AI158861, R01HL155741, R01HL171117, R21AG077024, U01AI069197, U01AI184132, U24HL157560, and UG1HL174426. 

Support is also provided by Boston Children’s Hospital; Fred Hutchinson Cancer Center; Gateway for Cancer Research, Inc.; Jeff Gordon Children’s Foundation; Medical College of Wisconsin; NMDP; Patient Center Outcomes Research Institute; PBMTF; St. Baldricks’s Foundation; Stanford University; Stichting European Myeloma Network (EMN); and from the following commercial entities: AbbVie; Actinium Pharmaceuticals, Inc.; Adaptive Biotechnologies Corporation; ADC Therapeutics; Adienne SA; Alexion; AlloVir, Inc.; Amgen, Inc.; Astellas Pharma US; AstraZeneca; Atara Biotherapeutics; Autolus Limited; BeiGene; BioLineRX; Blue Spark Technologies; bluebird bio, inc.; Blueprint Medicines; Bristol Myers Squibb Co.; CareDx Inc.; CSL Behring; CytoSen Therapeutics, Inc.; DKMS; Editas Medicine; Elevance Health; Eurofins Viracor, DBA Eurofins Transplant Diagnostics; Gamida-Cell, Ltd.; Gift of Life Biologics; Gift of Life Marrow Registry; HistoGenetics; Incyte Corporation; Iovance; Janssen Research & Development, LLC; Janssen/Johnson & Johnson; Jasper Therapeutics; Jazz Pharmaceuticals, Inc.; Karius; Kashi Clinical Laboratories; Kiadis Pharma; Kite, a Gilead Company; Kyowa Kirin; Labcorp; Legend Biotech; Mallinckrodt Pharmaceuticals; Med Learning Group; Medac GmbH; Merck & Co.; Millennium, the Takeda Oncology Co.; Miller Pharmacal Group, Inc.; Miltenyi Biomedicine; Miltenyi Biotec, Inc.; MorphoSys; MSA-EDITLife; Neovii Pharmaceuticals AG; Novartis Pharmaceuticals Corporation; Omeros Corporation; Orca Biosystems, Inc.; OriGen BioMedical; Ossium Health, Inc.; Pfizer, Inc.; Pharmacyclics, LLC, An AbbVie Company; PPD Development, LP; Registry Partners; Rigel Pharmaceuticals; Sanofi; Sarah Cannon; Seagen Inc.; Sobi, Inc.; Stemcell Technologies; Stemline Technologies; STEMSOFT; Takeda Pharmaceuticals; Talaris Therapeutics; Vertex Pharmaceuticals; Vor Biopharma Inc.; Xenikos BV. 

 

Abbreviations

Need an acronym defined? Review our list of common abbreviations.