1. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.
- Author
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Gluckman, Eliane, Cappelli, Barbara, Bernaudin, Francoise, Labopin, Myriam, Volt, Fernanda, Carreras, Jeanette, Pinto Simões, Belinda, Ferster, Alina, Dupont, Sophie, de la Fuente, Josu, Dalle, Jean-Hugues, Zecca, Marco, Walters, Mark C, Krishnamurti, Lakshmanan, Bhatia, Monica, Leung, Kathryn, Yanik, Gregory, Kurtzberg, Joanne, Dhedin, Nathalie, Kuentz, Mathieu, Michel, Gerard, Apperley, Jane, Lutz, Patrick, Neven, Bénédicte, Bertrand, Yves, Vannier, Jean Pierre, Ayas, Mouhab, Cavazzana, Marina, Matthes-Martin, Susanne, Rocha, Vanderson, Elayoubi, Hanadi, Kenzey, Chantal, Bader, Peter, Locatelli, Franco, Ruggeri, Annalisa, and Eapen, Mary
- Subjects
Biomedical and Clinical Sciences ,Cardiovascular Medicine and Haematology ,Immunology ,Hematology ,Prevention ,Sickle Cell Disease ,Stem Cell Research ,Rare Diseases ,Clinical Research ,Stem Cell Research - Nonembryonic - Human ,Regenerative Medicine ,Transplantation ,Adolescent ,Anemia ,Sickle Cell ,Child ,Child ,Preschool ,Disease-Free Survival ,Female ,Graft Survival ,HLA Antigens ,Hematopoietic Stem Cell Transplantation ,Histocompatibility ,Humans ,Infant ,Male ,Siblings ,Surveys and Questionnaires ,Survival Rate ,Transplantation Conditioning ,Treatment Outcome ,Eurocord ,the Pediatric Working Party of the European Society for Blood and Marrow Transplantation ,and the Center for International Blood and Marrow Transplant Research ,Cardiorespiratory Medicine and Haematology ,Clinical Sciences ,Paediatrics and Reproductive Medicine ,Biochemistry and cell biology ,Cardiovascular medicine and haematology ,Paediatrics - 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.
- Published
- 2017