Outcomes of Hematopoietic Stem Cell Transplantation in Children with Sickle Cell Disease: Does Donor Sickle Cell Trait Status Matter?

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) remains the gold standard curative therapy for sickle cell disease (SCD). A matched related donor (MRD) with sickle cell trait (SCT) is a viable option, with no evidence of adversely affecting transplant outcomes. We conducted a retrospective chart review of children (≤ 14 years) who underwent myeloablative HSCT from MRDs for SCD between 2013 and 2023, comparing outcomes for MRD with SCT to those with normal hemoglobin (Hgb) electrophoresis. Overall, 73 children underwent HSCT at a median age of 11.3 years. The main indication for HSCT was central nervous system insult in 43 (60%) patients. Among the donors, 48 (66%) had SCT, whereas 25 (34%) had normal Hgb electrophoresis. The mean time to neutrophil and platelet engraftment was 21.3 (10–84) and 22.61 (14–78) days, respectively. None of the patients developed graft rejection. The incidence of both acute and chronic graft-versus-host disease (GVHD) was similar across the donor groups. With a remarkable 5-year overall survival and disease-free survival rate of 97.3%, our findings demonstrate that there is no significant difference between donor groups. Our results align with international HSCT outcomes for SCD, reinforcing the efficacy of allogeneic HSCT from MRDs, regardless of SCT status.