Study of Venetoclax and Azacitidine Before Stem Cell Transplant in Adults with High-Risk Myelodysplastic Neoplasms with Increased Blasts

What this Trial Is About

Researchers are evaluating a treatment approach for adults aged 18 and older with higher-risk Myelodysplastic Neoplasms with Increased Blasts 2 MDS-IB2. This single-arm, prospective study investigates the use of Venetoclax combined with Azacitidine as a bridging therapy before allogeneic hematopoietic stem cell transplantation allo-HSCT. The goal is to assess treatment response and survival outcomes in this patient population. Participants will receive Venetoclax and Azacitidine in 28-day cycles, with dosing starting at 100 mg of Venetoclax on Day 1 and escalating to 400 mg by Days 3-14, alongside Azacitidine at 75 mgm on Days 1-7. They may receive one or two cycles depending on their treatment response. Those achieving a modified Composite Complete Remission after the first cycle proceed directly to transplantation, while others receive a second cycle before undergoing allo-HSCT within three months. Patients unable to proceed to transplantation will receive standard care and monitoring. During the study, participants will be closely monitored for treatment response using established criteria and followed for outcomes such as relapse-free survival over two years. Additional measures include overall survival, relapse rates, and complete response rates assessed up to four years. Safety and organ function will be evaluated, and follow-up care will continue after transplantation or standard treatment as appropriate.

Clinical Study of Venetoclax Combined With Azacitidine as Bridging Therapy Prior to Hematopoietic Stem Cell Transplantation in Patients With Higher-Risk Myelodysplastic Syndromes