Matched‐related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research
- 1 May 2007
- journal article
- research article
- Published by Wiley in British Journal of Haematology
- Vol. 137 (5), 479-485
- https://doi.org/10.1111/j.1365-2141.2007.06592.x
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.Journal ArticleMulticenter StudyResearch Support, N.I.H. ExtramuralResearch Support, Non-U.S. Gov'tResearch Support, U.S. Gov't, Non-P.H.S.SCOPUS: ar.jFLWINinfo:eu-repo/semantics/publisheKeywords
This publication has 28 references indexed in Scilit:
- Right ventricular abnormalities in sickle cell anemia: Evidence of a progressive increase in pulmonary vascular resistanceThe Journal of Pediatrics, 2006
- Discontinuing Prophylactic Transfusions Used to Prevent Stroke in Sickle Cell DiseaseNew England Journal of Medicine, 2005
- Bone marrow transplantation for sickle cell anemia: Progress and prospectsPediatric Blood & Cancer, 2004
- Sickle cell anemia and hematopoietic cell transplantation: When is a pound of cure worth more than an ounce of prevention?Pediatric Transplantation, 2004
- Pulmonary Hypertension as a Risk Factor for Death in Patients with Sickle Cell DiseaseNew England Journal of Medicine, 2004
- Brain injury in children with sickle cell disease: Prevalence and etiologyAnnals of Neurology, 2003
- Silent Cerebral Infarcts in Sickle Cell Anemia: A Risk Factor AnalysisPediatrics, 1999
- Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in BelgiumBone Marrow Transplantation, 1998
- Neurocognitive Aspects of Pediatric Sickle Cell DiseaseJournal of Learning Disabilities, 1993
- Pain in Sickle Cell DiseaseNew England Journal of Medicine, 1991