Abstract:Allogeneic hematopoietic stem cell transplantation (HSCT) is the only widely available curative treatment for sickle cell disease (SCD). Myeloablative conditioning regimens are associated with excellent outcomes in children with HLA-identical sibling donors but are limited by organ toxicity in adults. Here we report 48 children and adults who underwent HLA-identical sibling HSCT for SCD using a reduced toxicity conditioning (RTC) regimen (fludarabine, busulfan, and anti-thymocyte globulin), followed by cyclosp… Show more
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