A study by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) found that a donor search prognosis-based strategy helps physicians move patients with blood cancers and other blood diseases to hematopoietic cell transplant (HCT) faster, resulting in similar transplant success regardless of likelihood of finding a matched unrelated donor (MUD).
Investigators from the consortium of U.S. transplant centers, CIBMTR® (Center for International Blood and Marrow Transplant Research®), the Emmes Company and NMDPSM, which collectively make up the BMT CTN, used an NMDP-developed donor search prognosis strategy based on patients’ human leukocyte antigen (HLA) type and self-identified race/ethnicity to determine patients’ likelihood of identifying a MUD. The findings, published in the peer-reviewed Journal of Clinical Oncology showed that by rapidly pivoting to alternative donors when the chance of finding an HLA-matched (8/8) MUD was low, time to transplant and survival rates did not differ—overturning previously-established research.
The first national, multi-center study to evaluate the full donor search process using a score-based strategy,1 researchers tested whether the approach could help transplant centers avoid lengthy, unsuccessful searches for fully matched donors for patients very unlikely to find one. Physicians could instead focus their efforts on alternative donors who are readily available—including mismatched unrelated donors (MMUD), haploidentical related donors and/or umbilical cord blood—to improve transplant rates and patient outcomes. The study found no difference in overall survival at two years post-evaluability. Investigators also observed no differences in any other transplant clinical outcomes, such as relapse, treatment-related mortality, disease-free survival, and chronic or acute (grade II-IV) graft-versus-host disease.
1 Wadsworth K, Albrecht M, Fonstad R, Spellman S, Maiers M, Dehn J. Unrelated donor search prognostic score to support early HLA consultation and clinical decisions. Bone Marrow Transplant. 2016;51(11):1476-1481. doi: 10.1038/bmt.2016.162. Epub 2016 Jun 6. PMID: 27272451.
“As a physician, a primary concern following a blood cancer diagnosis is getting my patient to transplant quickly, as the benefits of transplant diminish as the disease progresses. Patients can face complications, greater resistance to treatment and worse outcomes if a prolonged search for a fully matched unrelated donor delays transplant,” said Stephanie Lee, MD, MPH, principal study investigator. Dr. Lee is professor and section head of hematologic malignancies in the Clinical Research Division at Fred Hutch Cancer Center and holds the David and Patricia Giuliani/Oliver Press Endowed Chair in Cancer Research.
Dr. Lee added, “The transplant team needs every advanced method to save their patients’ lives and a search prognosis score-based strategy can help them make rapid, evidence-based decisions about which type of donor to look for.”
Researchers enrolled 1,751 patients with malignant and non-malignant blood diseases, including lymphoma, leukemia and sickle cell disease, at 47 transplant centers. Designed as a biological assignment study, the search prognosis scoring system classified patients with blood cancers and disorders requiring transplant into three groups—Very Likely, Less Likely and Very Unlikely—based on their chances of finding a MUD. Patients assigned to the Very Likely group proceeded to a MUD transplant of their physician’s choosing. Patients in the Very Unlikely group proceeded to their physician’s preferred alternative donor source (haploidentical relative, umbilical cord blood or MMUD). Researchers used a pre-specified statistical model to compare overall survival between the Very Likely and Very Unlikely groups.
“Historically, the established approach was to delay transplant while physicians searched for a matched unrelated donor, even when chances were statistically slim. This new evidence gives clinicians unequivocal evidence to immediately proceed to transplant with the best available alternative donor—whether fully matched or not—so patients can have the greatest chances for survival,” said Steven Devine, MD, chief medical officer, NMDP, and senior scientific director, CIBMTR. “NMDP is redefining how research advances transplant care and cell therapy available for every patient in need, closing the access gap for those who historically have had lower chances of finding a matched donor.”
As a result of these findings and other scientific evidence influencing donor selection practices, NMDP and CIBMTR recently published updated donor selection guidelines in the peer-reviewed Journal of Transplantation and Cellular Therapy, recommending broader use of the BMT CTN 1702 findings. Work is underway at NMDP to incorporate guidance from the search prognosis strategy into donor selection operational systems.
The BMT CTN conducted the study and resulting analysis—and initially presented these data at the American Society of Hematology (ASH) Annual Meeting in December 2024.
About the Blood and Marrow Transplant Clinical Trials Network
The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) conducts rigorous multi-institutional clinical trials of high scientific merit, focused on improving survival for patients undergoing hematopoietic cell transplantation and/or receiving cellular therapies. The BMT CTN has completed accrual to 52 Phase II and III trials at more than 100 transplant centers and enrolled over 16,600 study participants. BMT CTN is funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute, both parts of the National Institutes of Health (NIH), and is a collaborative effort of 19 Core Transplant Centers/Consortia, CIBMTR (Center for International Blood and Marrow Transplant Research), NMDP and the Emmes Company, LLC, a clinical research organization. CIBMTR is a research collaboration between the Medical College of Wisconsin and NMDP. More information about the BMT CTN can be found at bmtctn.net.
About CIBMTR®
CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a nonprofit research collaboration between NMDPSM, in Minneapolis, and the Medical College of Wisconsin (MCW), in Milwaukee. CIBMTR collaborates with the global scientific community to increase survival and enrich quality of life for patients. CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of centers, and a unique database of long-term clinical data for more than 680,000 people who have received hematopoietic cell transplantation and other cellular therapies. Learn more at cibmtr.org. It is funded by the National Cancer Institute, the National Heart, Lung and Blood Institute and the National Institute for Allergy and Infectious Disease, the Health Resources and Services Administration, the Office of Naval Research, industry sponsors, MCW, and NMDP.
About the Medical College of Wisconsin
With a history dating back to 1893, The Medical College of Wisconsin is dedicated to leadership and excellence in education, patient care, research, and community engagement. More than 1,500 students are enrolled in MCW’s medical school and graduate school programs in Milwaukee, Green Bay, and Central Wisconsin. MCW’s School of Pharmacy opened in 2017. A major national research center, MCW is the largest research institution in the Milwaukee metro area and second largest in Wisconsin. In the last 10 years, faculty received more than $1.5 billion in external support for research, teaching, training, and related purposes. This total includes highly competitive research and training awards from the National Institutes of Health (NIH). Annually, MCW faculty direct or collaborate on more than 3,100 research studies, including clinical trials. Additionally, more than 1,650 physicians provide care in virtually every specialty of medicine for more than 2.8 million patients annually. Learn more at mcw.edu.
About Emmes
Founded more than 45 years ago, Emmes is a global, full-service Clinical Research Organization dedicated to excellence in supporting the advancement of public health and biopharmaceutical innovation. The company’s clients include numerous agencies and institutes of the U.S. federal government and a wide range of biotechnology, pharmaceutical and medical device companies throughout the world. To learn more about how our research is making a positive impact on human health, go to the Emmes website at emmes.com.
About NMDPSM
At NMDPSM, we believe each of us holds the key to curing blood cancers and disorders. As a global nonprofit leader in cell therapy, NMDP creates essential connections between researchers and supporters to inspire action and accelerate innovation to find life-saving cures. With the help of blood stem cell donors from the world’s most diverse registry and our extensive network of transplant partners, physicians and caregivers, we’re expanding access to treatment so that every patient can receive their life-saving cell therapy. NMDP. Find cures. Save lives. Learn more at nmdp.org.