About this study
This study is being done to test a transplant method that may have fewer side effects or be less toxic than standard transplants that use high-dose chemotherapy for children and young adults with sickle cell disease.
Sickle cell disease symptoms are usually treated by giving the medicine hydroxyurea and/or regular red blood cell transfusions. These treatments have improved survival in patients with sickle cell disease. Performing a transplant of blood-forming cells from a donor may provide a cure for this disease. However, getting a transplant can have many bad side effects.
Normally, patients undergoing this type of blood transplant for sickle cell disease receive high-dose chemotherapy to prepare the body for the transplant. Researchers leading this study want to see if a different conditioning treatment works just as well with fewer side effects. This new method uses fewer drugs to suppress your immune system before transplant.
Eligibility overview
- 25 years old or younger
- Diagnosed with severe sickle cell disease
- Has a suitable HLA-matched sibling donor or single haplotype matched family member donor