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 approximately 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.
Atkins
Robert C
RC
Children's Hospital and Research Center at Oakland, Blood and Marrow Transplantation Program, 747 52nd Street, Oakland, CA 94609, USA.
Walters
Mark C
MC
D000755:Anemia, Sickle Cell / Q000401:mortality / Q000628:therapy*; D018572:Disease-Free Survival; D006680:HLA Antigens / Q000737:chemistry; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006453:Hemoglobinopathies / Q000628:therapy; D006650:Histocompatibility Testing; D006801:Humans; D013997:Time Factors; D014019:Tissue Donors; D016896:Treatment Outcome
D006680:HLA Antigens