Comparing Fludarabine and Melphalan With or Without Venetoclax for Blood Stem Cell Transplant in AML and MDS Patients Over 50 Years

What this Trial Is About

Researchers are studying conditioning regimens for elderly patients over 50 years old with acute myeloid leukemia AML or myelodysplastic syndrome MDS who are undergoing allogeneic hematopoietic cell transplantation Allo-HCT. The study evaluates whether adding Venetoclax, a targeted drug, to the standard fludarabine plus melphalan FluMel regimen improves long-term outcomes. This trial is a multicenter, randomized phase 3 study addressing the challenge that elderly patients have higher relapse rates and need safer, more effective transplant conditioning. Participants are randomly assigned to one of two groups. The control group receives the standard conditioning regimen of fludarabine plus melphalan, with melphalan doses adjusted by age. The experimental group receives Venetoclax along with fludarabine and melphalan, with Venetoclax doses adjusted if certain antifungal medications are used and melphalan dose adjusted by age. Venetoclax is given daily from days -8 to -2, fludarabine from days -7 to -3, and melphalan on day -2 before transplant. During the study, participants will be monitored for graft-versus-host disease GVHD and relapse-free survival over two years as the main outcome. Other outcomes include overall survival, progression-free survival, non-relapse mortality, and immune recovery. Researchers will assess safety and effectiveness through regular follow-ups. The trial aims to determine if adding Venetoclax can improve survival and reduce complications after transplant in this patient group.

Fludarabine Plus Melphalan Versus Addition of Venetoclax to Fludarabine/Melphalan Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in AML/MDS Patients Aged > 50 Years: a Multicenter, Randomized, Phase 3 Trial