Phase III Clinical Trial Published in New England Journal Of Medicine Identifies New Standards for Prevention of Graft Versus Host Disease Following Transplant

 // News

June 22  

Practice-changing novel approach to preventing graft vs host disease may become the new standard of care in adults who receive transplant.

MINNEAPOLIS, MINN., June 22, 2023 –  Results of a Phase III randomized clinical trial published in the New England Journal of Medicine indicates that a new strategy for prevention of graft versus host disease (GVHD) for blood cancer patients receiving allogeneic hematopoietic cell transplantation (HCT) from well-matched donors significantly improves GVHD-free/relapse -free survival (GRFS).

This study offers hope for the thousands of people fighting blood cancers in the United States alone – and their families – who face the uncertainty of a GVHD diagnoses after HCT,” said Amy Ronneberg, CEO of National Marrow Donor Program (NMDP)/Be The Match. “It may change standard treatment practices and offer a higher quality of life for HCT patients. We are proud to continue advancing cell therapy through the Blood and Marrow Transplant Clinical Trials Network® (BMT CTN).”

GVHD is a condition that can develop after HCT. Donor cells (the graft) that a person (the host) has received can attack that person’s organs and/or tissues. The donor cells do this because they mistake the host’s organs and/or tissues as a threat. GVHD symptoms can impact people’s ability to live normal lives – even daily routines can be affected. In severe cases, GVHD can be life-threatening.

The study, completed by the Blood and Marrow Transplant Clinical Trials Network,  compared the outcomes of HCT patients with blood cancers receiving standard GVHD prevention therapy to patients who received a three-drug combination of PTCy/Tac/MMF (post-transplant cyclophosphamide, tacrolimus and mycophenolate mofetil) with reduced-intensity conditioning regimens – lower doses or different types of chemotherapy or radiation.

The trial enrolled 431 patients aged 18 years and older with blood cancers who were undergoing reduced-intensity conditioning allogeneic (cells from a donor) HCT. Patients were randomized 1:1 to receive either PTCy/Tac/MMF (n = 214; median age, 66.1 years; 62.6% men) or the current standard of care consisting of Tac plus methotrexate (Tac/MTX) (n = 217; median age, 66.3 years; 58.1% men). The primary end point was GRFS at one-year post-HCT defined as the absence of severe (grade III-IV) acute GVHD, chronic GVHD requiring systemic immunosuppression, relapse/progression, or death. Secondary endpoints of this study included engraftment, incidence and severity of acute and chronic GVHD, infections, relapse/progression, transplant-related mortality and survival.

At one-year post-HCT, GRFS was significantly improved in patients receiving PTCy/Tac/MMF vs Tac/MTX (61.9% vs 44.9%, p = .0004).  The improvement in GRFS was attributed to a significantly reduced risk of severe acute GVHD at 100 days (6.3% vs. 14.7%, p = .001) and chronic GVHD requiring systemic immune suppression at one year (12.5% vs 25%, p=.001) in the PTCy/Tac/MMF arm. Overall survival, relapse/progression and transplant-related mortality did not significantly differ between the treatment arms (p>.1). Median time to neutrophil engraftment did not differ between arms, but platelet engraftment was significantly delayed in the PTCy/Tac/MMF arm (median 24 vs 18 days, p<.001).  Grade 2 infections were higher with PTCy/Tac/MMF (33.7% vs 20.5%, p=.002), however Grade 3 infection rates did not differ (12.2% vs 13.3%, p=.817).

Based on these positive results the study team concluded that PTCy/Tac/MMF should be the new standard for GVHD prevention for patients receiving reduced intensity conditioning HCT with well-matched donors for treatment of blood cancers.

“We agree with the study team that the results set a new standard for GVHD prophylaxis using PTCy/Tac/MMF for older patients receiving peripheral blood grafts from matched related and unrelated donors following reduced intensity conditioning,” said Dr. Steven Devine, Chief Medical Officer, NMDP/Be The Match.

National Marrow Donor Program® (NMDP)/Be The Match®

The National Marrow Donor Program® (NMDP)/Be The Match® is the leading global partner working to save lives through cellular therapy. With 35 years of experience managing the most diverse registry of potential unrelated blood stem cell donors and cord blood units in the world, NMDP/Be The Match is a proven partner in providing cures to patients with life-threatening blood and marrow cancers and diseases. Through their global network, they connect centers and patients to their best cell therapy option—from blood stem cell transplant to a next-generation therapy—and collaborate with cell and gene therapy companies to support therapy development and delivery through Be The Match BioTherapies®. NMDP/Be The Match is a tireless advocate for the cell therapy community, working with hematologists/oncologists to remove barriers to consultation and treatment, and supporting patients through no-cost programs to eliminate non-medical obstacles to cell therapy. In addition, they are a global leader in research through the CIBMTR® (Center for International Blood and Marrow Transplant Research®)—a collaboration with Medical College of Wisconsin, investing in and managing research studies that improve patient outcomes and advance the future of care.

CIBMTR® (Center for International Blood and Marrow Transplant Research®)

Center for International Blood and Marrow Transplant Research is a nonprofit research collaboration between the National Marrow Donor Program (NMDP)/ Be The Match, in Minneapolis, and the Medical College of Wisconsin, in Milwaukee. The 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 630,000 people who have received hematopoietic cell transplantation and other cellular therapies. Learn more at

The BMT CTN (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, The Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Donor Program (NMDP)/Be The Match and the Emmes Company, LLC, a clinical research organization. CIBMTR is a research collaboration between the NMDP/Be The Match and the Medical College of Wisconsin.

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