HSCT Shows Curative Potential in Adults With Sickle Cell Disease

Hematopoietic stem cell transplantation (HSCT) is both possible and effective in adult patients with sickle cell disease (SCD), according to a retrospective single-center study published in Annals of Hematology.

The retrospective case series enrolled 14 adult patients who underwent allogeneic HSCT at a single center in India between 2018 and 2024. Nine patients received matched sibling donor transplants, and 5 underwent haploidentical transplants.

All patients received myeloablative conditioning and had a baseline symptom burden of at least 2 vaso-occlusive pain crises per year that required hospitalization.

At a median follow-up of 22.4 months, event-free survival was 71.4% and overall survival was 78.6%. The authors reported that 84.6% of patients achieved full or mixed donor chimerism, and all patients reaching this threshold remained free of sickle-related events post-transplant.