Comparing Immediate Stem Cell Transplantation to Bridging Therapy Before Transplant in Adults with High-Risk Myelodysplastic Syndrome

What this Trial Is About

Researchers are evaluating the effectiveness of immediate allogeneic hematopoietic stem cell transplantation HSCT compared to HSCT performed after bridging therapy in patients with higher-risk myelodysplastic syndrome HR-MDS. This phase 2, multicenter, randomized controlled trial seeks to determine if starting HSCT right away is not worse than delaying it until after initial treatment, helping to guide optimal timing for transplantation in this patient group. Participants will be randomly assigned to one of two groups the immediate HSCT group, where patients receive direct allogeneic HSCT or the bridging therapy group, where patients first undergo one to two cycles of bridging therapy before HSCT. Bridging therapy may include hypomethylating agents alone or combined with chemotherapy, such as azacitidine and venetoclax, and targeted therapies like IDH1 inhibitors for eligible patients. The study will continue until at least 124 events occur, ensuring sufficient data to assess outcomes. During the study, researchers will monitor participants for disease-free survival over two years after HSCT as the primary outcome. Secondary measures include the proportion of patients undergoing HSCT at various time points, complete remission rates, overall survival, leukemia-free survival, quality of life assessments, and molecular clearance rates over two years. Participants will be closely followed with clinical evaluations and laboratory tests throughout the study duration, which extends through at least two years post-treatment to fully assess outcomes and safety.

Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients