Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation
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Accepted version
Author(s)
Type
Journal Article
Abstract
Despite advances in supportive therapy to prevent complications of sickle cell disease (SCD), access to care is not universal. Hematopoietic cell transplantation is, to date, the only curative therapy for SCD, but its application is limited by availability of a suitable HLA-matched donor and lack of awareness of the benefits of transplant. Included in this study are 1000 recipients of HLA-identical sibling transplants performed between 1986 and 2013 and reported to the European Society for Blood and Marrow Transplantation, Eurocord, and the Center for International Blood and Marrow Transplant Research. The primary endpoint was event-free survival, defined as being alive without graft failure; risk factors were studied using a Cox regression models. The median age at transplantation was 9 years, and the median follow-up was longer than 5 years. Most patients received a myeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bone marrow was the predominant stem cell source (n = 839; 84%); peripheral blood and cord blood progenitors were used in 73 (7%) and 88 (9%) patients, respectively. The 5-year event-free survival and overall survival were 91.4% (95% confidence interval, 89.6%-93.3%) and 92.9% (95% confidence interval, 91.1%-94.6%), respectively. Event-free survival was lower with increasing age at transplantation (hazard ratio [HR], 1.09; P < .001) and higher for transplantations performed after 2006 (HR, 0.95; P = .013). Twenty-three patients experienced graft failure, and 70 patients (7%) died, with the most common cause of death being infection. The excellent outcome of a cohort transplanted over the course of 3 decades confirms the role of HLA-identical sibling transplantation for children and adults with SCD.
Date Issued
2017-03-16
Date Acceptance
2016-12-01
Citation
BLOOD, 2017, 129 (11), pp.1548-1556
ISSN
0006-4971
Publisher
American Society of Hematology
Start Page
1548
End Page
1556
Journal / Book Title
BLOOD
Volume
129
Issue
11
Copyright Statement
© 2017 The American Society of Hematology. This research was originally published in Blood 2017 129:1548-1556; doi: https://doi.org/10.1182/blood-2016-10-745711
Sponsor
National Institute for Health Research
Identifier
http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000397294700022&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=1ba7043ffcc86c417c072aa74d649202
Grant Number
NF-SI-0611-10275
Subjects
Science & Technology
Life Sciences & Biomedicine
Hematology
BONE-MARROW TRANSPLANT
EXPERT PANEL
FOLLOW-UP
CHILDREN
ANEMIA
LIFE
ADULTS
HYDROXYUREA
THALASSEMIA
MANAGEMENT
Immunology
1102 Cardiovascular Medicine And Haematology
1103 Clinical Sciences
1114 Paediatrics And Reproductive Medicine
Publication Status
Published