Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease.

Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative therapy for sickle cell disease (SCD).41 Several barriers prevent its widespread application, including the lack of a suitable donor, risk of early and late onset of regimen-related toxicities, rejection and mortality. Despite these limitations, the number of transplants for hemoglobinopathies has been increasing in the last decade. The overall probability of survival (OS) for patients with SCD transplanted with a human lymphocyte antigen (HLA)- identical sibling graft ranges between 91 and 100% with an event-free survival (EFS) of 73-100%.3 A controversial issue is the ideal age to perform HSCT in SCD patients. In fact, whilst early age HSCT could prevent SCD-related organ damage, resulting in better patient outcomes, the emergence of new available SCD supportive care, promising curative therapies could justify not proceeding with HSCT in certain cases [...