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Blood. 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.

Author information

1
Eurocord, Paris-Diderot University Equipe d'Accueil 3518, Hospital Saint Louis, Paris, France.
2
Monacord, International Observatory on Sickle Cell Disease, Centre Scientifique de Monaco, Monaco.
3
Department of Pediatrics, Referral Center for Sickle Cell Disease, Centre Hospitalier Intercommunal Créteil, Paris XII University, Créteil, France.
4
European Society for Blood and Marrow Transplantation Statistical Unit, Hospital Saint Antoine, Assistance Publique-Hôpitaux de Paris, Paris, France.
5
Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI.
6
Ribeirão Preto Medical School, University of São Paulo, São Paulo, Brazil.
7
Hemato-Oncology Unit, Hospital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels, Belgium.
8
Cliniques Universitaires Saint Luc, Hemato-Oncology Unit, Brussels, Belgium.
9
Centre for Haematology, Imperial College London, London, United Kingdom.
10
Hemato-immunology, Hospital Robert Debré and Paris-Diderot University, Paris, France.
11
Pediatric Hematology-Oncology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
12
University of California San Francisco Benioff Children's Hospital, Oakland, CA.
13
Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA.
14
Morgan Stanley Children's Hospital of New York, New York, NY.
15
Baylor College of Medicine, Houston, TX.
16
Children's Hospital of Michigan, Detroit, MI.
17
Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, NC.
18
Adolescent and Young Adults Hematology Department, Hospital Saint-Louis, Paris, France.
19
Department of Pediatric Hematology and Oncology and Research Unit Equipe d'Accueil 3279, Aix-Marseille University and Timone Children's Hospital Marseille, Marseille, France.
20
Imperial College London, Hammersmith Hospital, London, United Kingdom.
21
Service D'hémato Oncologie Pédiatrique, Centre Hospitalier Universitaire de Strasbourg, Strasbourg, France.
22
Pediatric Hematology-Immunology Department, Hospital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.
23
Department of Pediatric Hematology and Oncology, University Hospital of Lyon, Lyon, France.
24
Equipe d'Accueil 3829, Institut de Recherche et d'Innovation Biomédicale, Faculté de Médecine-Pharmacie, Rouen, France.
25
Paediatric Haematology/Oncology, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia.
26
Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
27
Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM, Paris, France.
28
Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France.
29
Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, Austria.
30
Hospital Sirio-Libanes, and Serviço de Hematologia, Hemoterapia e Terapia Celular, São Paulo University, São Paulo, Brazil.
31
Churchill Hospital, Oxford, United Kingdom.
32
Department for Stem Cell Transplantation and Immunology, Clinic for Children and Adolescents, University Hospital Frankfurt, Goethe University, Frankfurt, Germany.
33
Dipartimento di Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico, Ospedale Bambino Gesù, Rome, Italy.
34
Dipartimento di Scienze Pediatriche, Università di Pavia, Pavia, Italy; and.
35
Department of Hematology and Cell Therapy, Hospital Saint Antoine, Paris, France.

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.

PMID:
27965196
PMCID:
PMC5356458
DOI:
10.1182/blood-2016-10-745711
[Indexed for MEDLINE]
Free PMC Article

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