Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research.
(Journal Article;Multicenter Study)
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.
Panepinto, JA; Walters, MC; Carreras, J; Marsh, J; Bredeson, CN; Gale, RP; Hale, GA; Horan, J; Hows, JM; Klein, JP; Pasquini, R; Roberts, I; Sullivan, K; Eapen, M; Ferster, A; Non-Malignant Marrow Disorders Working Committee, Center for International Blood and Marrow Transplant Research,
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