title: Haematopoietic stem cell transplantation for sickle cell disease - current practice and new approaches.
abstract: Sickle cell disease is an inherited disorder that affects over 5 million people worldwide. Current maintenance therapy has been successful in reducing complications and enhancing life expectancy; yet subclinical complications persist. To date, allogeneic haematopoietic stem cell transplant (HSCT) remains the only available curative therapy for sickle cell disease. With declining incidences of rejection and transplant- related mortality, disease-free survival after human leucocyte antigen-identical sibling transplant exceeds 90%. However, the majority of individuals with sickle cell disease do not have an human leucocyte antigen (HLA)-identical sibling; therefore, research is expanding to focus on new approaches to alternative donor transplant. Advances in supportive care and conditioning regimens have led to expansion of the pool of donors to unrelated donors and haploidentical donors. Challenges remain in improving the safety and efficacy of HSCT from alternate donors. Early results from gene therapy may provide another curative option in patients with sickle cell disease. These approaches show early promise, but larger, longitudinal studies are needed to better determine the optimal clinical circumstances for transplant in sickle cell disease.
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Department of Pediatrics, Atlanta, GA, USA.
authors.forename: Staci D
authors.initials: SD
authors.lastname: Arnold
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, NY, USA.
authors.forename: Monica
authors.initials: M
authors.lastname: Bhatia
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Department of Pediatrics, Atlanta, GA, USA.
authors.forename: John
authors.initials: J
authors.lastname: Horan
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Department of Pediatrics, Atlanta, GA, USA.
authors.forename: Lakshmanan
authors.initials: L
authors.lastname: Krishnamurti
mesh_terms: D000755:Anemia, Sickle Cell / Q000628:therapy*; D018572:Disease-Free Survival; D015316:Genetic Therapy; D006680:HLA Antigens / Q000276:immunology; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000639:trends; D006801:Humans; D061349:Unrelated Donors
chemical_list: D006680:HLA Antigens
keywords: SCT; Transplantation; sickle cell disease