Longer-Term HCT Outcomes for Severe Sickle Cell Disease in Childhood
Allogeneic HCT for sickle cell disease (SCD) can lead to survival rates exceeding 90%, according to results of a single-center study of 50 children with severe SCD. Median age at HCT was 8.3 years, and stem cell sources were bone marrow (n=39), cord blood (n=3), bone marrow and cord blood (n=7) and peripheral blood stem cells (n=1). All patients had one or more severe manifestations of SCD, including recurrent vaso-occlusive crises/acute chest syndrome (n=37), cerebral vasculopathy (n=27), and nephropathy (n=1). At a median follow up of 7.7 years, eight-year survival and event-free survival was 94.1% and 85.6%, respectively.