Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research.

Published

Journal Article

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.

Full Text

Duke Authors

Cited Authors

  • 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,

Published Date

  • June 2007

Published In

Volume / Issue

  • 137 / 5

Start / End Page

  • 479 - 485

PubMed ID

  • 17459050

Pubmed Central ID

  • 17459050

International Standard Serial Number (ISSN)

  • 0007-1048

Digital Object Identifier (DOI)

  • 10.1111/j.1365-2141.2007.06592.x

Language

  • eng

Conference Location

  • England