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

on behalf of 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 asuitable 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. Themedian age at transplantation was 9 years, and themedian follow-up was longer than 5 years. Most patients received amyeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bonemarrow 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. Eventfree 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 (US)
Pages (from-to)1548-1556
Number of pages9
JournalBlood
Volume129
Issue number11
DOIs
StatePublished - Mar 16 2017

Fingerprint

Hematopoietic Stem Cell Transplantation
Sickle Cell Anemia
Stem cells
Transplants
Siblings
Blood
Transplantation
Grafts
Hazards
Disease-Free Survival
Bone Marrow
Confidence Intervals
Survival
Cell Transplantation
Fetal Blood
Proportional Hazards Models
Availability
Cause of Death
Stem Cells
Surveys and Questionnaires

ASJC Scopus subject areas

  • Immunology
  • Biochemistry
  • Hematology
  • Cell Biology

Cite this

on behalf of Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research (2017). 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

Sickle cell disease : An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. / on behalf of Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research.

In: Blood, Vol. 129, No. 11, 16.03.2017, p. 1548-1556.

Research output: Contribution to journalArticle

on behalf of Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research 2017, 'Sickle cell disease: An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation', Blood, vol. 129, no. 11, pp. 1548-1556. https://doi.org/10.1182/blood-2016-10-745711
on behalf of Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research. Sickle cell disease: An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood. 2017 Mar 16;129(11):1548-1556. https://doi.org/10.1182/blood-2016-10-745711
on behalf of Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research. / Sickle cell disease : An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. In: Blood. 2017 ; Vol. 129, No. 11. pp. 1548-1556.
@article{ae4be7520492402fa55f027f8a486be3,
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 asuitable 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. Themedian age at transplantation was 9 years, and themedian follow-up was longer than 5 years. Most patients received amyeloablative conditioning regimen (n = 873; 87{\%}); the remainder received reduced-intensity conditioning regimens (n = 125; 13{\%}). Bonemarrow 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. Eventfree 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.",
author = "{on behalf of Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research} and Eliane Gluckman and Barbara Cappelli and Francoise Bernaudin and Myriam Labopin and Fernanda Volt and Jeanette Carreras and Sim{\~o}es, {Belinda Pinto} and Alina Ferster and Sophie Dupont and {De La Fuente}, Josu and Dalle, {Jean Hugues} and Marco Zecca and Walters, {Mark C.} and Lakshmanan Krishnamurti and Monica Bhatia and Kathryn Leung and Gregory Yanik and Joanne Kurtzberg and Nathalie Dhedin and Mathieu Kuentz and Gerard Michel and Jane Apperley and Patrick Lutz and B{\'e}n{\'e}dicte Neven and Yves Bertrand and Vannier, {Jean Pierre} and Mouhab Ayas and Marina Cavazzana and Susanne Matthes-Martin and Vanderson Rocha and Hanadi Elayoubi and Chantal Kenzey and Peter Bader and Franco Locatelli and Annalisa Ruggeri and Mary Eapen and Victoria Bordon and Veerle Labarque and Maguy Pereira and Henrique Bittencourt and Heidi Petersen and Eric Deconninck and Charlotte Jubert and Jean Perrin and Cahn, {Jean Yves} and B{\'e}n{\'e}dicte Bruno and Pierre Bordigoni and Francoise Mechinaud and Vernant, {Jean Paul} and {Bolanos Meade}, {F Javier}",
year = "2017",
month = "3",
day = "16",
doi = "10.1182/blood-2016-10-745711",
language = "English (US)",
volume = "129",
pages = "1548--1556",
journal = "Blood",
issn = "0006-4971",
publisher = "American Society of Hematology",
number = "11",

}

TY - JOUR

T1 - Sickle cell disease

T2 - An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

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

AU - Gluckman, Eliane

AU - Cappelli, Barbara

AU - Bernaudin, Francoise

AU - Labopin, Myriam

AU - Volt, Fernanda

AU - Carreras, Jeanette

AU - Simões, Belinda Pinto

AU - Ferster, Alina

AU - Dupont, Sophie

AU - De La Fuente, Josu

AU - Dalle, Jean Hugues

AU - Zecca, Marco

AU - Walters, Mark C.

AU - Krishnamurti, Lakshmanan

AU - Bhatia, Monica

AU - Leung, Kathryn

AU - Yanik, Gregory

AU - Kurtzberg, Joanne

AU - Dhedin, Nathalie

AU - Kuentz, Mathieu

AU - Michel, Gerard

AU - Apperley, Jane

AU - Lutz, Patrick

AU - Neven, Bénédicte

AU - Bertrand, Yves

AU - Vannier, Jean Pierre

AU - Ayas, Mouhab

AU - Cavazzana, Marina

AU - Matthes-Martin, Susanne

AU - Rocha, Vanderson

AU - Elayoubi, Hanadi

AU - Kenzey, Chantal

AU - Bader, Peter

AU - Locatelli, Franco

AU - Ruggeri, Annalisa

AU - Eapen, Mary

AU - Bordon, Victoria

AU - Labarque, Veerle

AU - Pereira, Maguy

AU - Bittencourt, Henrique

AU - Petersen, Heidi

AU - Deconninck, Eric

AU - Jubert, Charlotte

AU - Perrin, Jean

AU - Cahn, Jean Yves

AU - Bruno, Bénédicte

AU - Bordigoni, Pierre

AU - Mechinaud, Francoise

AU - Vernant, Jean Paul

AU - Bolanos Meade, F Javier

PY - 2017/3/16

Y1 - 2017/3/16

N2 - 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 asuitable 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. Themedian age at transplantation was 9 years, and themedian follow-up was longer than 5 years. Most patients received amyeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bonemarrow 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. Eventfree 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.

AB - 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 asuitable 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. Themedian age at transplantation was 9 years, and themedian follow-up was longer than 5 years. Most patients received amyeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bonemarrow 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. Eventfree 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.

UR - http://www.scopus.com/inward/record.url?scp=85015934863&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85015934863&partnerID=8YFLogxK

U2 - 10.1182/blood-2016-10-745711

DO - 10.1182/blood-2016-10-745711

M3 - Article

C2 - 27965196

AN - SCOPUS:85015934863

VL - 129

SP - 1548

EP - 1556

JO - Blood

JF - Blood

SN - 0006-4971

IS - 11

ER -