Hematopoietic cell transplantation (HCT) to treat sickle cell disease (SCD) can result in up to 96% survival at two years, according to research results presented at the ASH Annual Meeting. In this registry study, researchers analyzed outcomes data reported to CIBMTR (Center for International Blood and Marrow Transplant Research) on 161 HCT recipients ≤21 years old with SCD. Two-year overall survival (OS) for all 161 recipients was 90%, and OS was highest in umbilical cord blood transplant recipients, at 96%. Risk of death was significantly higher for children ≥10 years old (HR 2.1, p=0.003). The researchers concluded that for children with an available matched sibling donor, allogeneic HCT is the preferred therapy, especially in children before age 10 years, and preferably prior to onset of SCD complications.
Early HCT for SCD in Children is Preferred Therapy Prior to Onset of SCD Complications