Results presented at ASH from a study of 1,000 matched sibling donor transplants in patients with severe sickle cell disease (SCD) have demonstrated a three-year overall survival of 94% and an event-free survival of 90%. The researchers characterized these results as “excellent” and noted that hematopoietic cell transplant (HCT) recipients experienced low transplant-related toxicity despite the use of myeloablative conditioning regimens in 87% of the transplants.
Patients in the study were transplanted between 1991 and 2013, with outcomes reported to CIBMTR (Center for International Blood and Marrow Transplant Research; n=439) and EBMT (European Society for Blood and Marrow Transplantation; n=561). Median patient age was 9 years (range, 1-54), with 85% of patients age <16 years. The most common indication for HCT was stroke.
A multivariate analysis showed that every advancing year in age increment resulted in higher mortality (HR 1.1, 95% CI 1.07-1.14, p<0.001). The researchers concluded that pediatric hematologists should discuss HCT as a curative therapeutic option with parents of young patients with severe SCD early in their disease course.