MINNEAPOLIS, June 14, 2024 — NMDPSM, a global nonprofit leader in cell therapy, and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) announced interim results from the ACCESS trial as an oral abstract during the European Hematology Association (EHA) Annual Meeting in Madrid, Spain. The study demonstrated that adults with hematologic malignancies who received peripheral blood stem cell (PBSC) transplant from HLA-mismatched unrelated donors (MMUD) followed by post-transplant cyclophosphamide (PTCy) graft-versus-host-disease (GvHD) prophylaxis exhibited a 79% overall survival (OS), the primary endpoint of the study, with an encouraging 51% GvHD-free, relapse-free survival (GRFS) probability at one-year post transplant.
“Our goal is to discover how we can improve outcomes for all patients – extending their life and improving their quality of life,” said Antonio Jimenez Jimenez, M.D., Hematology & Oncology Specialist, Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, presenting author. “We are encouraged by these early findings, which strongly advocate for the inclusion of this innovative treatment approach for patients receiving PBSC from partially matched, unrelated donors.”
In addition to achieving very good OS and GRFS clinical endpoints, adult participants also exhibited low rates of severe acute and moderate to severe chronic GvHD, both at 9%. The NMDP-sponsored ACCESS trial, conducted through the CIBMTR — a research collaboration between the Medical College of Wisconsin and NMDP — enrolled 70 adult patients with blood cancers and disorders, including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and acute lymphoblastic leukemia (ALL), from 13 participating transplant centers including Sylvester Comprehensive Cancer Center. All patients in this stratum received reduced intensity conditioning, most often fludarabine/melphalan (63%) or fludarabine/busulfan (20%), and PBSCs from donors matched at 5-7 of 8 HLA loci, predominantly at the 7/8 match level (67%).
Expanding Access to Transplant: NMDP-led Donor for All Research
NMDP’s network of transplant center partners, many of which participate in CIBMTR trials, are bringing new research to light that is challenging previously established stem cell transplantation science. NMDP’s Donor For All initiative includes several research efforts that aim to establish a new safe and effective approach for using MMUD transplants worldwide.
“Low likelihood of matched sibling donors and the substantial variance of matched, unrelated donor availability on international registries have historically hindered the likelihood of finding a suitable donor,” said ACCESS study co-author Steven M. Devine, M.D., Chief Medical Officer, NMDP; Senior Scientific Director, CIBMTR. “Our Donor For All research is expanding the definition of a suitable donor for patients without a fully matched donor. Through CIBMTR, we are developing innovative protocols to prevent GvHD and reduce risk for post-transplant complications in patients with hematologic malignancies.”
ACCESS builds upon findings from the first Donor for All trial, the groundbreaking NMDP-sponsored 15-MMUD study, which showed that PTCy was effective in decreasing risk for GvHD in adults with hematologic malignancies receiving bone marrow (BM) transplants from MMUD. Similar to ACCESS, 15-MMUD patients showed a one-year OS rate of 76% and exhibited a low rate of moderate / severe GvHD.
Enrolling now, the OPTIMIZE trial is evaluating whether a reduced dose of PTCy will safely and effectively prevent GvHD while reducing infection risk in patients with hematologic malignancies receiving PBSC HCT from MMUDs. Finally, a recent observational study by the CIBMTR presented at the 2024 Tandem Meetings, reported no discernable differences in OS or GRFS for adult patients with hematologic malignancies using MMUD HCT at an 8/8 or 7/8 HLA match level using PTCy GvHD prophylaxis.
2024 EHA Presentation Details
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Oral Presentation (Abstract #S260) Friday, June 14; 2:45-3:00 p.m. CEST; Room Hall Dali 2 Post-transplant cyclophosphamide-based graft-versus-host disease prophylaxis following mismatched unrelated donor peripheral blood stem cell (PBSC) transplantation Antonio Jimenez Jimenez, M.D. |
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, 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 675,000 people who have received hematopoietic cell transplantation and other cellular therapies. Learn more at cibmtr.org.
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.
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CONTACT:
Erica Sevilla
NMDP