Perspectives:
What a Christmas Tree on The Beach Teaches Us About Data Sharing
By Michael Verneris, MD
For many years now, every December, my family has traveled to Seabrook Island, South Carolina, where my father-in-law has a home. One of our cherished traditions is taking long walks on the beach, where my wife and I find moments of reflection amid the sound of waves and the salty breeze. These walks often spark meaningful conversations—some solving problems, others creating them—but always fostering connection and clarity. This year, however, something on the beach captured my attention and spurred a deeper contemplation.
When we arrived in mid-December, a lone Christmas tree stood planted in the sand near the beach entrance. It was bare, unadorned, and its purpose was unclear. Yet, as we walked by it multiple times per day, we noticed something extraordinary. Slowly but surely, the tree began to transform. People passing by started decorating it—seaweed became tinsel, a horseshoe crab carcass and shells were repurposed as ornaments, old Christmas lights and bulbs appeared, and an eclectic assortment of decorations accumulated. It became a collective creation, a symbol of community in which each contribution, no matter how small, added to something larger.
This tree reminded me of my two years as chair of the CIBMTR Advisory Committee. Much like that tree on the beach, CIBMTR’s database is a collaborative effort in which medical centers around the world contribute small amounts of data that, together, form an invaluable resource for advancing our field of transplantation. My experience in this role has been profoundly humbling, as I’ve come to appreciate the extraordinary effort required to maintain and grow such a robust database. It’s not just the physician leaders but also the countless staff members behind the scenes whose dedication ensures its success.
As I reflect on this role, I find myself contemplating the future of CIBMTR in the context of rapidly evolving technologies, particularly the rise of large language models (LLMs). Models like OpenAI’s ChatGPT, Google’s Gemini, and Microsoft’s Co-pilot are just a few examples of the offerings that are significantly reshaping how we process and analyze information. For instance, in healthcare, these LLMs are already being tested and/or used to assist with tasks such as clinic visit documentation and response to patient queries, but also for summarizing patient records, predicting disease outcomes, and identifying potential treatment options based on vast datasets. Their capabilities raise intriguing questions about the future of data collection, analysis, and utilization in clinical research. On those walks on the beach, I found myself wondering whether CIBMTR’s database will eventually be supplanted by these technologies, or will they be integrated into its framework to enhance its functionality?
Traditionally, the work of analyzing clinical data has been painstakingly manual. Medical students, residents, fellows, and researchers have historically spent countless hours combing through hundreds of charts to identify risk factors for cellular therapy complications or relapse risks. This process, while rigorous, is also labor-intensive and time-consuming. The advent of LLMs promises a paradigm shift. These models can process vast amounts of data, identify patterns and associations that might elude humans, and do so with remarkable speed. Envision a future where LLMs are seamlessly integrated into EMRs, funneling data directly to CIBMTR’s database. The potential for real-time data analysis and insight is immense.
However, this vision is not without its challenges. One of the most immediate concerns is data privacy and compliance. The Health Insurance Portability and Accountability Act (HIPAA) sets stringent standards for protecting patient information, and the sophisticated capabilities of LLMs may necessitate rethinking these standards. For instance, the models’ ability to infer associations or reconstruct identities from de-identified data could inadvertently compromise patient privacy. Ensuring HIPAA compliance in the age of LLMs will require innovative solutions and possibly new regulatory frameworks.
Another critical issue is the inherent biases within these models. LLMs are trained on vast datasets, which often reflect existing societal, cultural, and systemic biases. When applied to clinical research and decision-making, these biases could lead to skewed analyses or recommendations, particularly for underrepresented populations. Addressing algorithmic bias is essential to ensure equitable and accurate outcomes.
Perhaps the most challenging aspect of integrating LLMs into clinical research is their lack of transparency. These models are often described as “black boxes” because they do not easily explain how they arrive at their conclusions. While they may identify correlations or generate hypotheses, the absence of clear (human) reasoning can complicate the application of their findings. Furthermore, the phenomenon of “hallucinations”—in which models generate inaccurate or entirely fabricated data—poses a significant risk in the high-stakes context of medical research and patient care.
Despite these challenges, the potential benefits of LLMs are too significant to ignore. They could revolutionize our understanding of complex datasets, uncovering insights that drive innovation in patient care. For example, the integration of daily patient data—blood work, vitals, imaging, and even patient-reported outcomes—into these models could provide a more dynamic and holistic view of a patient’s health status during transplantation or cellular therapy, enabling a more personalized and accurate treatment strategy than current protocol-driven patient care. Moreover, these models could enhance the efficiency of data collection and analysis, freeing up research staff to focus on interpretation and application rather than data collection.
CIBMTR is well-positioned to be at the forefront of this transformation. The organization has already begun testing data transfer mechanisms from the EMRs of individual transplant centers to its database, paving the way for more seamless and comprehensive data collection and integration. As LLMs become more sophisticated and accessible, it’s imperative that CIBMTR anticipate their incorporation into clinical research workflows. This will involve not only technological adaptation but also thoughtful consideration of ethical, legal, and practical implications.
Returning to the Christmas tree on the beach, I find its evolution to be a fitting metaphor for CIBMTR’s journey. Each beachgoer’s contribution, no matter how small, mirrored the collaborative spirit of transplant centers contributing data to build something extraordinary. Just as beachgoers contributed to the tree, each transplant center’s data contributions build the foundation of CIBMTR’s database. The tree’s final form was not dictated by any single individual but emerged organically through collective effort and creativity. Similarly, CIBMTR’s future will be shaped by the contributions of its many stakeholders, informed by emerging technologies, and guided by a shared commitment to advancing the science of transplantation and cellular therapy.
As I reflect on my tenure as chair of CIBMTR’s Advisory Committee, I am filled with gratitude for the opportunity to be part of such a meaningful endeavor. The dedication of this community—from the physicians and researchers to the administrative and technical staff—is truly inspiring. I am confident that, like the tree on the beach, CIBMTR will continue to grow and evolve, incorporating new tools and perspectives to address the challenges and opportunities ahead. Together, we will ensure that this collaborative effort remains a beacon of innovation and hope in the field of transplantation and cellular therapy.
Graft-versus-Host Disease Working Committee
Pictured left to right: Tao Wang, Stephen Spellman, Jakob Devos, Nosha Farhadfar, Zachariah DeFilipp, Stephanie Lee, and Najla El Jurdi.
Committee Leadership
Co-Chairs:
- Carrie Kitko, Vanderbilt University Medical Center, Nashville, TN
- Nosha Farhadfar, Methodist Hospital, San Antonio, TX
- Zachariah DeFilipp, Massachusetts General Hospital, Boston, MA
Scientific Directors:
- Stephanie Lee, Fred Hutchinson Cancer Center, Seattle, WA
- Stephen Spellman, CIBMTR NMDP, Minneapolis, MN
Statistical Director:
- Tao Wang, CIBMTR MCW, Milwaukee, WI
Statistician:
- Jakob Devos, CIBMTR MCW, Milwaukee, WI
CIBMTR Page Scholar:
- Najla El Jurdi, National Cancer Institute, Bethesda, MD
GVHD remains a major complication of allogeneic HCT. Ongoing efforts to understand, prevent, and treat both acute and chronic GVHD are critical to improving transplant safety. The GVHD Working Committee is committed to leveraging CIBMTR’s robust data resources and statistical expertise to address impactful research questions in the field.
As an update to the committee leadership structure, the GVHD Working Committee announces Pooja Khandelwal, MD, from Cincinnati Children’s Hospital as the incoming Co-Chair and Najla El Jurdi, MD, from NIH/NCI as our Page Scholar. Thank you to our outgoing Chair, Carrie Kitko, MD, and our outgoing Scientific Director, Stephen Spellman, MBS, for all their contributions.
The committee remains very active, with several recent presentations and publications arising from the GVHD Working Committee study portfolio. Some recent notable examples include studies comparing GVHD incidence and risk factors among pediatric patients; evaluating the risk for bloodstream infections after acute GVHD; and determining the impact of racial, ethnic, and socioeconomic health disparities on the outcome of acute and chronic GVHD.
The committee currently has nine studies in progress that will explore new and exciting research avenues. One study examines the probability and determinants of durable discontinuation of immune suppression after allogeneic HCT. Another study is assessing the incidence of chronic GVHD in cryopreserved compared to fresh peripheral blood HCT grafts. Other studies will implement new analytic approaches to large data sets, including machine learning in one study and multi-state modeling in another. These studies demonstrate the collaborative nature of the GVHD Working Committee to address complex research questions. Additionally, the committee is currently working on a large dataset to support four studies in the post-transplant cyclophosphamide (PTCy) context, aligning with the high level of interest within the field:
- Comparing PTCy to calcineurin inhibitor (CNI)-based GVHD prophylaxis in the HLA-matched setting
- Comparing PTCy with sirolimus versus PTCy with CNI
- Assessing the effect of acute GVHD on HCT outcomes after PTCy
- Characterizing acute and chronic GVHD after PTCy versus CNI-based prophylaxis
Overall, the current study portfolio has great potential to address priority research questions in the field.
The GVHD Working Committee encourages the submission of new proposals and specifically welcomes the involvement of junior investigators. Four new proposals will be presented for consideration at the committee's meeting during the 2025 Tandem Meetings of ASTCT & CIBMTR. Committee leaders are also available to discuss new concepts and give input on proposal development. Please note that data sets from previously published studies are now publicly available for secondary analyses on CIBMTR’s website. Additional information about the committee can also be found on the GVHD Working Committee’s webpage.
BMT CTN: Open Trials and Recent Publications
By Mykala Heuer, BSN, RN
The BMT CTN is in its fifth grant cycle and has now enrolled more than 17,000 patients. Two BMT CTN-led protocols, 2203 and 2207, were recently released to centers, and researchers published 3 primary manuscripts in 2024.
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 active BMT CTN trials. Of the BMT CTN-led trials, 2 protocols were recently released to centers, and 3 are in development. The following BMT CTN protocols were recently released to centers:
- BMT CTN 2203 – A randomized, multicenter, Phase III trial of tacrolimus / methotrexate / ruxolitinib versus post-transplant cyclophosphamide / tacrolimus / mycophenolate mofetil in non-myeloablative / reduced intensity conditioning allogeneic peripheral blood stem cell transplantation
- 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
- This trial also offers a Proactive Financial Navigation service as part of a BMT CTN Access to Clinical Trials initiative.
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 186 BMT CTN published articles, including 45 primary analyses. There were 3 primary manuscripts published in 2024:
- BMT CTN 1506: Levis MJ, Hamadani M, Logan D 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. doi: 10.1200/JCO.23.02474. Epub 2024 Mar 12. PMC11095884. https://pubmed.ncbi.nlm.nih.gov/38471061/
- BMT CTN 2101 (Full Cohort): Hill JA, Martens MJ, Young JH, et al. SARS-CoV-2 vaccination in the first year after hematopoietic cell transplant or chimeric antigen receptor 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. doi: 10.1093/cid/ciae291. Epub 2024 May 27. PMC11327798. https://pubmed.ncbi.nlm.nih.gov/38801746/
- BMT CTN 1503: Walters MC, Eapen M, Liu Y, et al. Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease. Blood Advances. 2024 Oct 29; bloodadvances.2024013926. doi: 10.1182/bloodadvances.2024013926. [Epub ahead of print.] https://pubmed.ncbi.nlm.nih.gov/39471440/
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 (Mount Sinai), as Steering Committee Chair; Stephanie Lee, MD (Fred Hutchinson Cancer Center), as Steering Committee Chair-Elect; and Miguel-Angel Perales, MD (Memorial Sloan Kettering Cancer Center), as Steering Committee Vice-Chair.
To get up-to-date information about BMT CTN studies, meetings, and news be sure to follow us on X (previously known as Twitter): @BMTCTN
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.; Adaptimmune LLC; 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.; Caribou Biosciences, Inc.; 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; In8bio, Inc.; 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.; Sociedade Brasileira de Terapia Celular e Transplante de Medula Óssea (SBTMO); Stemcell Technologies; Stemline Technologies; STEMSOFT; Takeda Pharmaceuticals; Talaris Therapeutics; Vertex Pharmaceuticals; Vor Biopharma Inc.; Xenikos BV.