Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

Eliane Gluckman, Barbara Cappelli, Francoise Bernaudin, Myriam Labopin, Fernanda Volt, Jeanette Carreras, Belinda Pinto Simões, Alina Ferster, Sophie Dupont, Josu de la Fuente, Jean Hugues Dalle, Marco Zecca, Mark C. Walters, Lakshmanan Krishnamurti, Monica Bhatia, Kathryn Leung, Gregory Yanik, Joanne Kurtzberg, Nathalie Dhedin, Mathieu KuentzGerard Michel, Jane F. Apperley, Patrick Lutz, Bénédicte Neven, Yves Bertrand, Jean Pierre Vannier, Mouhab Ayas, Marina Cavazzana, Susanne Matthes-Martin, Vanderson Rocha, Hanadi Elayoubi, Chantal Kenzey, Peter Bader, Franco Locatelli, Annalisa Ruggeri, Mary Eapen, Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)1548-1556
Number of pages9
JournalBlood
Volume129
Issue number11
DOIs
Publication statusPublished - Dec 13 2016

Keywords

  • Journal Article

Fingerprint Dive into the research topics of 'Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation'. Together they form a unique fingerprint.

  • Cite this

    Gluckman, E., Cappelli, B., Bernaudin, F., Labopin, M., Volt, F., Carreras, J., Pinto Simões, B., Ferster, A., Dupont, S., de la Fuente, J., Dalle, J. H., Zecca, M., Walters, M. C., Krishnamurti, L., Bhatia, M., Leung, K., Yanik, G., Kurtzberg, J., Dhedin, N., ... Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research (2016). Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood, 129(11), 1548-1556. https://doi.org/10.1182/blood-2016-10-745711