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Multicenter Study
. 2017 Mar 16;129(11):1548-1556.
doi: 10.1182/blood-2016-10-745711. Epub 2016 Dec 13.

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

Eliane Gluckman  1   2 , Barbara Cappelli  2 , Francoise Bernaudin  3 , Myriam Labopin  4 , Fernanda Volt  1   2 , Jeanette Carreras  5 , Belinda Pinto Simões  6 , Alina Ferster  7 , Sophie Dupont  8 , Josu de la Fuente  9 , Jean-Hugues Dalle  10 , Marco Zecca  11 , Mark C Walters  12 , Lakshmanan Krishnamurti  13 , Monica Bhatia  14 , Kathryn Leung  15 , Gregory Yanik  16 , Joanne Kurtzberg  17 , Nathalie Dhedin  18 , Mathieu Kuentz  3 , Gerard Michel  19 , Jane Apperley  20 , Patrick Lutz  21 , Bénédicte Neven  22 , Yves Bertrand  23 , Jean Pierre Vannier  24 , Mouhab Ayas  25 , Marina Cavazzana  26   27   28 , Susanne Matthes-Martin  29 , Vanderson Rocha  1   30   31 , Hanadi Elayoubi  1   2 , Chantal Kenzey  1   2 , Peter Bader  32 , Franco Locatelli  33   34 , Annalisa Ruggeri  1   2   35 , Mary Eapen  5 ; Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research
Affiliations
Multicenter Study

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

Eliane Gluckman et al. Blood. .

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.

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Figures

Figure 1.
Figure 1.
Outcomes after HSCT for SCD. (A) Unadjusted chronic GVHD, according to age; and (B) OS, according to stem cell source.
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