Phase II Study of Bone Marrow Transplant from Haploidentical or Unrelated Donors for Patients Aged 3 to 75 Years with Newly Diagnosed Severe Aplastic Anemia

What this Trial Is About

Researchers are evaluating bone marrow transplantation as a first treatment for patients aged 3 to 75 years with severe aplastic anemia SAA who have not received prior therapy. SAA is a rare condition where the body stops producing enough blood cells, leading to low white blood cells, red blood cells, and platelets. This study aims to determine if transplantation from half-matched related donors or matched unrelated donors can restore normal blood cell production safely and effectively in untreated patients. Participants will receive a reduced-intensity conditioning regimen including fludarabine, cyclophosphamide, low-dose total body irradiation, and Thymoglobulin before receiving fresh bone marrow cells from either a haploidentical related donor or an unrelated donor. Graft-versus-host disease GVHD prevention will include post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil. The study includes two parallel groups based on donor type, each aiming to enroll 30 participants. Throughout the study, participants will be closely monitored for one year after transplant to assess survival without GVHD or treatment failure, recovery of blood cells, graft success, and incidence of acute and chronic GVHD. Assessments will include blood counts at multiple time points, evaluation of graft failure, and overall and failure-free survival. Participants will be followed regularly to track these outcomes and their health status during the year after transplantation.

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia