title: Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research.
abstract: We report outcomes after myeloablative haematopoietic cell transplantation (HCT) from human leucocyte antigen (HLA)-matched sibling donors in 67 patients with sickle cell disease transplanted between 1989 and 2002. The most common indications for transplantation were stroke and recurrent vaso-occlusive crisis in 38% and 37% of patients respectively. The median age at transplantation was 10 years and 67% of patients had received >10 red blood cell transfusions before HCT. Twenty-seven percent of patients had a poor performance score at transplantation. Ninety-four percent received busulfan and cyclophosphamide-containing conditioning regimens and bone marrow was the predominant source of donor cells. Most patients achieved haematopoietic recovery and no deaths occurred during the early post-transplant period. Rates of acute and chronic graft-versus-host disease were 10% and 22% respectively. Sixty-four of 67 patients are alive with 5-year probabilities of disease-free and overall survival of 85% and 97% respectively. Nine patients had graft failure with recovery of sickle erythropoiesis, eight of who had recurrent sickle-related events. This report confirms and extends earlier reports that HCT from HLA-matched related donors offers a very high survival rate, with few transplant-related complications and the elimination of sickle-related complications in the majority of patients who undergo this therapy.
authors: Department of Hematology/Oncology/Bone Marrow Transplant, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI 53226, USA. jpanepin@mcw.edu
authors: Julie A
authors: JA
authors: Panepinto
authors: Mark C
authors: MC
authors: Walters
authors: Jeanette
authors: J
authors: Carreras
authors: Judith
authors: J
authors: Marsh
authors: Christopher N
authors: CN
authors: Bredeson
authors: Robert Peter
authors: RP
authors: Gale
authors: Gregory A
authors: GA
authors: Hale
authors: John
authors: J
authors: Horan
authors: Jill M
authors: JM
authors: Hows
authors: John P
authors: JP
authors: Klein
authors: Ricardo
authors: R
authors: Pasquini
authors: Irene
authors: I
authors: Roberts
authors: Keith
authors: K
authors: Sullivan
authors: Mary
authors: M
authors: Eapen
authors: Alina
authors: A
authors: Ferster
mesh_terms: D000755:Anemia, Sickle Cell / Q000276:immunology / Q000401:mortality / Q000601:surgery*; D018572:Disease-Free Survival; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D007958:Leukocyte Count; D009504:Neutrophils / Q000276:immunology; D011336:Probability; D012042:Registries; D015996:Survival Rate; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous