title: Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.
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.
authors.affiliation: Eurocord, Paris-Diderot University Equipe d'Accueil 3518, Hospital Saint Louis, Paris, France.|Monacord, International Observatory on Sickle Cell Disease, Centre Scientifique de Monaco, Monaco.
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
authors.affiliation: Monacord, International Observatory on Sickle Cell Disease, Centre Scientifique de Monaco, Monaco.
authors.forename: Barbara
authors.initials: B
authors.lastname: Cappelli
authors.affiliation: Department of Pediatrics, Referral Center for Sickle Cell Disease, Centre Hospitalier Intercommunal Créteil, Paris XII University, Créteil, France.
authors.forename: Francoise
authors.initials: F
authors.lastname: Bernaudin
authors.affiliation: European Society for Blood and Marrow Transplantation Statistical Unit, Hospital Saint Antoine, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors.forename: Myriam
authors.initials: M
authors.lastname: Labopin
authors.affiliation: Eurocord, Paris-Diderot University Equipe d'Accueil 3518, Hospital Saint Louis, Paris, France.|Monacord, International Observatory on Sickle Cell Disease, Centre Scientifique de Monaco, Monaco.
authors.forename: Fernanda
authors.initials: F
authors.lastname: Volt
authors.affiliation: Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI.
authors.forename: Jeanette
authors.initials: J
authors.lastname: Carreras
authors.affiliation: Ribeirão Preto Medical School, University of São Paulo, São Paulo, Brazil.
authors.forename: Belinda
authors.initials: B
authors.lastname: Pinto Simões
authors.affiliation: Hemato-Oncology Unit, Hospital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels, Belgium.
authors.forename: Alina
authors.initials: A
authors.lastname: Ferster
authors.affiliation: Cliniques Universitaires Saint Luc, Hemato-Oncology Unit, Brussels, Belgium.
authors.forename: Sophie
authors.initials: S
authors.lastname: Dupont
authors.affiliation: Centre for Haematology, Imperial College London, London, United Kingdom.
authors.forename: Josu
authors.initials: J
authors.lastname: de la Fuente
authors.affiliation: Hemato-immunology, Hospital Robert Debré and Paris-Diderot University, Paris, France.
authors.forename: Jean-Hugues
authors.initials: JH
authors.lastname: Dalle
authors.affiliation: Pediatric Hematology-Oncology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.affiliation: University of California San Francisco Benioff Children's Hospital, Oakland, CA.
authors.forename: Mark C
authors.initials: MC
authors.lastname: Walters
authors.affiliation: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA.
authors.forename: Lakshmanan
authors.initials: L
authors.lastname: Krishnamurti
authors.affiliation: Morgan Stanley Children's Hospital of New York, New York, NY.
authors.forename: Monica
authors.initials: M
authors.lastname: Bhatia
authors.affiliation: Baylor College of Medicine, Houston, TX.
authors.forename: Kathryn
authors.initials: K
authors.lastname: Leung
authors.affiliation: Children's Hospital of Michigan, Detroit, MI.
authors.forename: Gregory
authors.initials: G
authors.lastname: Yanik
authors.affiliation: Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, NC.
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.affiliation: Adolescent and Young Adults Hematology Department, Hospital Saint-Louis, Paris, France.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Dhedin
authors.affiliation: Department of Pediatrics, Referral Center for Sickle Cell Disease, Centre Hospitalier Intercommunal Créteil, Paris XII University, Créteil, France.
authors.forename: Mathieu
authors.initials: M
authors.lastname: Kuentz
authors.affiliation: Department of Pediatric Hematology and Oncology and Research Unit Equipe d'Accueil 3279, Aix-Marseille University and Timone Children's Hospital Marseille, Marseille, France.
authors.forename: Gerard
authors.initials: G
authors.lastname: Michel
authors.affiliation: Imperial College London, Hammersmith Hospital, London, United Kingdom.
authors.forename: Jane
authors.initials: J
authors.lastname: Apperley
authors.affiliation: Service D'hémato Oncologie Pédiatrique, Centre Hospitalier Universitaire de Strasbourg, Strasbourg, France.
authors.forename: Patrick
authors.initials: P
authors.lastname: Lutz
authors.affiliation: Pediatric Hematology-Immunology Department, Hospital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors.forename: Bénédicte
authors.initials: B
authors.lastname: Neven
authors.affiliation: Department of Pediatric Hematology and Oncology, University Hospital of Lyon, Lyon, France.
authors.forename: Yves
authors.initials: Y
authors.lastname: Bertrand
authors.affiliation: Equipe d'Accueil 3829, Institut de Recherche et d'Innovation Biomédicale, Faculté de Médecine-Pharmacie, Rouen, France.
authors.forename: Jean Pierre
authors.initials: JP
authors.lastname: Vannier
authors.affiliation: Paediatric Haematology/Oncology, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia.
authors.forename: Mouhab
authors.initials: M
authors.lastname: Ayas
authors.affiliation: Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.|Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM, Paris, France.|Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France.
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana
authors.affiliation: Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, Austria.
authors.forename: Susanne
authors.initials: S
authors.lastname: Matthes-Martin
authors.affiliation: Eurocord, Paris-Diderot University Equipe d'Accueil 3518, Hospital Saint Louis, Paris, France.|Hospital Sirio-Libanes, and Serviço de Hematologia, Hemoterapia e Terapia Celular, São Paulo University, São Paulo, Brazil.|Churchill Hospital, Oxford, United Kingdom.
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
authors.affiliation: Eurocord, Paris-Diderot University Equipe d'Accueil 3518, Hospital Saint Louis, Paris, France.|Monacord, International Observatory on Sickle Cell Disease, Centre Scientifique de Monaco, Monaco.
authors.forename: Hanadi
authors.initials: H
authors.lastname: Elayoubi
authors.affiliation: Eurocord, Paris-Diderot University Equipe d'Accueil 3518, Hospital Saint Louis, Paris, France.|Monacord, International Observatory on Sickle Cell Disease, Centre Scientifique de Monaco, Monaco.
authors.forename: Chantal
authors.initials: C
authors.lastname: Kenzey
authors.affiliation: Department for Stem Cell Transplantation and Immunology, Clinic for Children and Adolescents, University Hospital Frankfurt, Goethe University, Frankfurt, Germany.
authors.forename: Peter
authors.initials: P
authors.lastname: Bader
authors.affiliation: Dipartimento di Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico, Ospedale Bambino Gesù, Rome, Italy.|Dipartimento di Scienze Pediatriche, Università di Pavia, Pavia, Italy; and.
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.affiliation: Eurocord, Paris-Diderot University Equipe d'Accueil 3518, Hospital Saint Louis, Paris, France.|Monacord, International Observatory on Sickle Cell Disease, Centre Scientifique de Monaco, Monaco.|Department of Hematology and Cell Therapy, Hospital Saint Antoine, Paris, France.
authors.forename: Annalisa
authors.initials: A
authors.lastname: Ruggeri
authors.affiliation: Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI.
authors.forename: Mary
authors.initials: M
authors.lastname: Eapen
mesh_terms: D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000453:epidemiology / Q000401:mortality / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006085:Graft Survival; D006680:HLA Antigens; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006648:Histocompatibility; D006801:Humans; D007223:Infant; D008297:Male; D035781:Siblings; D011795:Surveys and Questionnaires; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens