Haematopoietic cell transplantation in the treatment of sickle cell disease

Allogeneic haematopoietic cell transplantation (HCT) is presently the only treatment which offers the possibility of a cure for patients with sickle cell disease (SCD). While ~ 84% of patients survive disease-free after human leukocyte antigen (HLA)-identical sibling donor HCT, this therapy has traditionally been reserved for patients who have suffered serious complications due to the risk of transplant-related morbidity and mortality. Typically, these sickle-related complications have included recurrent episodes of acute chest syndrome, recurrent vaso-occlusive episodes and stroke. The future of HCT for haemoglobinopathies undoubtedly will evolve as transplant-related complications are reduced and as the process of selecting patients for HCT is refined.