Study of a Modified Stem Cell Transplant for Severe Aplastic Anemia to Improve Safety and Success

What this Trial Is About

Researchers are evaluating a modified allogeneic hematopoietic stem cell transplantation allo-HSCT regimen for patients with severe aplastic anemia, a condition where the bone marrow fails to produce enough blood cells. This study aims to improve the success rate and patient survival by optimizing the treatment and reducing complications such as graft-versus-host disease GVHD. It is a phase 2 clinical trial led by the Hematology department of the 920th hospital, enrolling 30 participants aged 3 to 65 years. The treatment involves a modified conditioning regimen including fludarabine, cyclophosphamide, and melphalan before transplantation. To prevent acute GVHD, the regimen includes PTCY, ATG, MMF, ruxolitinib, and cyclosporine CSA administered over a specified timeline before and after transplantation. This single-arm study evaluates this improved transplant system to enhance safety and efficacy in severe aplastic anemia patients. Participants will be monitored for two years after transplantation to assess overall survival and the incidence of acute and chronic GVHD. Additional evaluations include transplantation-related mortality, infections such as fungal diseases, and viral reactivations like EB virus and CMV. The study involves regular clinical assessments, laboratory tests, and safety monitoring to track outcomes and adverse events during this period.

Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia