Study of Immune Suppression Treatment for Sickle Cell Disease or β-Thalassemia
In this study, researchers are looking at how to make blood stem cell transplants safer for people with Sickle Cell Disease or β-Thalassemia. This type of transplant, called Hematopoietic Cell Transplantation (HCT), replaces unhealthy blood cells with healthy ones from a donor. Before the transplant, patients will receive medicines called fludarabine and dexamethasone, which help lower the immune system's activity. This might reduce side effects like graft failure, where the new cells don't work properly, and GvHD, a condition where new cells attack the body. Depending on how patients respond, other medicines like cyclophosphamide, bortezomib, or rituximab could be used.
Key Points:
- Eligibility: Age 2-50, with suitable donor and good organ function.
- Treatment: Involves immune-suppressing drugs before and during the transplant.
- Exclusions: Recent stroke, active infections, or other cancers.
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