Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

What this Trial Is About

Researchers are evaluating allogeneic hematopoietic stem cell transplantation HSCT regimens for patients with high-risk hemoglobinopathies and other red cell transfusion-dependent disorders in this phase II trial. The study aims to assess different transplant approaches and their effects on outcomes such as graft versus host disease GvHD and survival. It is an open-label trial conducted at a single center, focusing on conditions like sickle cell disease, thalassemia, and Diamond Blackfan anemia. Participants will receive one of several transplantation regimens depending on donor type and age. These include matched sibling donor regimens using Alemtuzumab Campath and total body irradiation TBI, or regimens with Thymoglobulin ATG, Fludarabine, Busulfan, Cyclophosphamide, and other drugs tailored to donor match type. Cells will be infused on day 0 following conditioning treatments. Immunosuppressive drugs such as sirolimus or tacrolimus and mycophenolate mofetil are given to prevent rejection and GvHD, with dosing schedules varying by treatment arm. During the study, participants will be closely monitored for graft success and complications, including the incidence of GvHD over one year and survival outcomes at one and two years. Assessments include clinical evaluations, laboratory tests, and drug monitoring. The trial will also track failure-free survival and chronic GvHD status up to two years. Participants may be followed for up to eight years, with safety and efficacy evaluations guiding ongoing care.

Allo HSCT for High Risk Hemoglobinopathies