Jeff Auletta, MD, discusses the background, methods, design and findings from the ACCESS trial.
Jeff Auletta, MD, senior vice president of health equity at the National Marrow Donor Program and chief scientific officer of the Center for International Blood and Marrow Transplant Research, discusses the background, methods, design, and findings from the ACCESS trial of posttransplant cyclophosphamide as graft-vs-host disease (GVHD) prophylaxis in mismatched unrelated donor (MMUD) peripheral blood stem cell transplants.
“The ACCESS trial is a multicenter, phase 2 trial designed to compare overall survival in patients receiving mismatched unrelated donor peripheral blood stem cells as an allograft source for hematologic malignancies. We are comparing this overall survival data to our previous trial, the 15 MMUD trial, which used mismatched, unrelated donor bone marrow,” explains Auletta.
In the phase 2 trial, investigators compared outcomes using peripheral blood stem cells to the previously conducted 15 MMUD trial, which used bone marrow. Findings from the study were recently presented at the 2025 Transplantation & Cellular Therapy Meetings.
“Historically, mismatched, unrelated donor transplants with calcineurin inhibitor-based GVHD prophylaxis, such as cyclosporine or tacrolimus, have been associated with inferior overall survival. However, the use of posttransplant cyclophosphamide as GVHD prophylaxis has demonstrated favorable results in both the 15 MMUD trial and now the ACCESS trial,” says Auletta.
According to Auletta, the 1-year overall survival rate in myeloablative adult patients was 84% (95% CI, 73%-90%) and PTCy was safe and effective, reducing barriers for diverse patients.
“These results reinforce the effectiveness of this approach in providing life-saving treatment, particularly for ethnically diverse patients, who comprised approximately 51% of the enrolled participants in the trial,” adds Auletta.