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

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

Eapen, Mary

Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research

2022-03-23T16:03:39Z

2022-03-23T16:03:39Z

2017-03

2022-03-23T16:03:38Z

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.

S0006-4971(20)33599-0

0006-4971

1528-0020

https://hdl.handle.net/10161/24616

eng

American Society of Hematology

Blood

10.1182/blood-2016-10-745711

Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research

Humans

Anemia, Sickle Cell

HLA Antigens

Disease-Free Survival

Treatment Outcome

Transplantation Conditioning

Hematopoietic Stem Cell Transplantation

Survival Rate

Siblings

Histocompatibility

Graft Survival

Adolescent

Child

Child, Preschool

Infant

Female

Male

Surveys and Questionnaires

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

Journal article

Kurtzberg, Joanne|0000-0002-3370-0703

1548

1556

11

Duke

School of Medicine

Clinical Science Departments

Institutes and Centers

Pathology

Pediatrics

Duke Cancer Institute

Institutes and Provost's Academic Units

Initiatives

Duke Innovation & Entrepreneurship

Pediatrics, Transplant and Cellular Therapy

Published

129