Allogenic hematopoietic stem cell transplantation in sickle cell disease

Discussing the currently available HSCT options for Hb SS patients highlighting advantages and disadvantages of each modality in the light of recently published data.

When MSD is available, myeloablative regimen is the preferred approach for otherwise healthy children whereas the nonmyeloablative (NMA) regimen is of choice for adults as well as children with SCD-associated morbidities. Mixed chimerism is common especially with NMA conditioning and is usually enough for cure. Alternative donor HSCT outcomes are progressively improving especially with posttransplant cyclophosphamide for GVHD prophylaxis.

Recent studies comparing HSCT and chronic transfusion in Hb SS patients increasingly come in favor of HSCT arm. Advances in HSCT field led to donor pool expansion and better tolerated regimens. It is easier now to tailor a personalized transplantation plan for almost every patient. A successful management plan should be sufficiently comprehensive addressing patients' and families' social and psychological concerns to ensure compliance and improve outcome.