Immune Suppression Treatment Before Haploidentical Hematopoietic Cell Transplantation for Children and Adults With Severe Sickle Cell Disease or Beta-Thalassemia

What this Trial Is About

Researchers are evaluating a treatment approach for people with severe sickle cell disease or transfusion-dependent beta-thalassemia who will receive an allogeneic hematopoietic cell transplantation HCT. The study focuses on whether giving immune-suppressing drugs like fludarabine and dexamethasone before the standard transplant conditioning can reduce serious side effects such as graft failure and graft-versus-host disease GvHD. Depending on participants responses, additional drugs such as cyclophosphamide, bortezomib, and rituximab may be used to help control the immune system. Participants will receive personalized doses of fludarabine guided by drug monitoring, with dexamethasone given on specific days before transplant. If needed, cyclophosphamide will be given after transplant. Other drugs like tacrolimus and mycophenolate mofetil will start from day 5 after transplant, and rabbit antithymocyte globulin ATG will be dosed according to a set plan. Bortezomib and rituximab are also given on scheduled days before transplant to support immune suppression. During the study, participants will be closely followed for one year to monitor outcomes such as treatment-related mortality and primary graft failure. Researchers will evaluate blood tests, organ function, and immune responses throughout the process. Participants will have regular visits for assessments, including heart and lung function tests, and will be monitored for side effects and overall health during and after the transplant procedure.

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)