Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol 2007 Jun;137(5):479-85
Date
04/27/2007Pubmed ID
17459050DOI
10.1111/j.1365-2141.2007.06592.xScopus ID
2-s2.0-34247858963 (requires institutional sign-in at Scopus site) 148 CitationsAbstract
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.
Author List
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 ResearchAuthor
Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
Anemia, Sickle CellDisease-Free Survival
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation
Histocompatibility Testing
Humans
Leukocyte Count
Neutrophils
Probability
Registries
Survival Rate
Transplantation Conditioning
Transplantation, Homologous