title: Hematopoietic stem cell transplantation for patients with sickle cell disease: progress and future directions.
abstract: Research has solidified matched sibling marrow, cord blood, or mobilized peripheral blood as the best source for allogeneic hematopoietic stem cell transplantation for patients with sickle cell disease, with low graft rejection and graft-versus-host disease (GVHD) and high disease-free survival rates. Fully allelic matched unrelated donor is an option for transplant-eligible patients without HLA-matched sibling donors. Unrelated cord transplant studies reported high GVHD and low engraftment rates. Haploidentical transplants have less GVHD, but improvements are needed to increase the low engraftment rate. The decision to use unrelated cord blood units or haploidentical donors depends on institutional expertise.
authors.affiliation: 9000 Rockville Pike, Building 10/9N112, Bethesda, MD 20892, USA.
authors.forename: Courtney D
authors.initials: CD
authors.lastname: Fitzhugh
authors.affiliation: Division of Blood and Marrow Transplantation, Children's National Health System, George Washington University School of Medicine and Health Sciences, 111 Michigan Avenue, North West, Washington, DC 20010, USA.
authors.forename: Allistair A
authors.initials: AA
authors.lastname: Abraham
authors.affiliation: 9000 Rockville Pike, Building 10/9N112, Bethesda, MD 20892, USA.
authors.forename: John F
authors.initials: JF
authors.lastname: Tisdale
authors.affiliation: 9000 Rockville Pike, Building 10/9N112, Bethesda, MD 20892, USA. Electronic address: matthewhs@mail.nih.gov.
authors.forename: Matthew M
authors.initials: MM
authors.lastname: Hsieh
mesh_terms: D000755:Anemia, Sickle Cell / Q000628:therapy*; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods; D005544:Forecasting; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000639:trends; D006650:Histocompatibility Testing; D006801:Humans; D016019:Survival Analysis; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D061349:Unrelated Donors
keywords: Cord blood (CB) donor; Haploidentical donor; Matched sibling donor; Matched unrelated donor; Sickle cell disease