Data from oral presentation show similar transplant success regardless of likelihood of finding a matched unrelated donor

First national, multicenter study using donor search prognosis score enables providers to more quickly move patients with blood cancers and diseases to transplant using alternative donors by estimating likelihood of finding a fully matched blood stem cell donor

The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) will present new data showing a donor search prognosis score helps physicians move patients with blood cancers and diseases to hematopoietic cell transplant (HCT) more quickly with alternative donors, resulting in no difference in any outcomes like overall survival (OS) at two years post-evaluability. Investigators from the consortium of U.S. transplant centers, CIBMTR^® (Center for International Blood and Marrow Transplant Research^®), the Emmes Company and NMDP^SM that make up the BMT CTN, used an NMDP-developed donor search prognosis score based on patients’ human leukocyte antigen (HLA) type and self-identified race/ethnicity to determine patients’ likelihood of identifying an HLA-matched (8/8) unrelated donor (MUD). By turning to alternative donors when the chance of finding a MUD is low, patients were able to achieve similar survival rates, results which will be presented orally today at the 66th American Society of Hematology (ASH) Annual Meeting in San Diego.

In the first national study of the entire donor search process, researchers tested whether a defined, score-based search strategy¹ could help transplant centers prevent futile searches for a fully matched donor for patients unlikely to find them, instead focusing efforts on alternative donors who are readily available—including mismatched unrelated donors (MMUD), haploidentical related donors and/or umbilical cord blood—and improve transplant rates and patient outcomes. Investigators also observed no differences in any other clinical outcomes of transplant, such as relapse, treatment-related mortality, disease-free survival, and chronic or acute (grade II-IV) graft-versus-host disease.

“Getting to transplant sooner is often the best option for patients, since the benefits of transplant diminish as a patient’s disease progresses. A prolonged search for a fully matched unrelated donor can delay transplant and be futile for some patients, leading to complications, greater disease resistance and worse outcomes,” said Stephanie Lee, MD, MPH, principal study investigator who will present the findings at ASH. She is professor and section head of hematologic malignancies in the Clinical Research Division and holds the David and Patricia Giuliani/Oliver Press Endowed Chair in Cancer Research at Fred Hutch Cancer Center.

Lee added, “This search prognosis score can help the transplant team make rapid, evidence-based decisions about pursuing alternative donor options, when needed.”

Researchers enrolled 1,751 patients with malignant and non-malignant blood diseases—including lymphoma, leukemia and sickle cell disease—at 47 national 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 mismatched unrelated donor). Researchers used a pre-specified statistical model to compare overall survival between the Very Likely and Very Unlikely groups.

“While traditional practice has been to postpone transplant in favor of finding a MUD, these results give physicians the confidence to proceed with identifying the best available alternative donor for their patients—improving outcomes for those who historically have had lower chances of finding donor matches,” said Steven Devine, MD, chief medical officer, NMDP, and senior scientific director, CIBMTR. “NMDP is changing what’s possible for patients who need a cure by investing in practice-changing research, tools and clinical methodologies that will help all patients receive their life-saving cell therapy.”

As a result of these findings, NMDP is implementing a search prognosis-based strategy at six U.S. centers. The organization aims to study optimal approaches for identifying donors for patients traditionally underserved in transplant, using this research to improve transplant practice.

The BMT CTN, which conducts multi-institutional clinical trials to evaluate promising cell therapies for patients facing life-threatening blood cancers and disorders, conducted the study and resulting analysis. Additional analyses are in progress.

ASH Annual Meeting Oral Presentation:

• Title: Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Finding the Ideal Donor and Graft: Going Beyond HLA
• Presenting author: Stephanie J. Lee, MD, MPH, Professor and Associate Director, Clinical Research Division, Fred Hutch Cancer Center
• Session type and title: Oral; Likelihood of finding an 8/8 HLA-matched unrelated donor (Donor Search Prognosis) is not associated with survival: Primary results from BMT CTN 1702
• Abstract number: 695
• Date & time: Sunday, Dec. 8; 4:30–6 p.m. PST
• Location: San Diego Convention Center, Room 6A

Additional Relevant Sessions:

• Title: OPTIMIZE: A Phase II Study of Reduced Dose Post Transplantation Cyclophosphamide as GVHD Prophylaxis in Adult Patients with Hematologic Malignancies Receiving HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation
• Presenting author: Jeffery J. Auletta, MD
• Session type and title: Poster; Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Toxicities: Poster II
• Abstract number: 3514.1
• Date & time: Sunday, Dec. 8; 6-8 p.m. PST
• Location: San Diego Convention Center, Halls G-H
  
  
• Title: Addressing Patient Representation in Hematopoietic Cell Transplant Clinical Trials: Insights Gained Through the ACCESS Trial
• Presenting author: Jeffery J. Auletta, MD
• Session type and title: Poster; Health Services and Quality Improvement: Myeloid Malignancies: Poster II
• Abstract number: 3672
• Date & time: Sunday, Dec. 8; 6-8 p.m. PST
• Location: San Diego Convention Center, Halls G-H

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 20 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 NMDP^SM, 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 NMDP^SM

At NMDP^SM, 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.

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.

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