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Hematopoietic Stem Cell Transplantation from HLA Identical Sibling Forsickle Cell Disease an International Survey on Behalf of Eurocord-Monacord, EBMT Paediatric Disease Working Party and CIBMTR

Authors :
Agnès Devergie
Marie Robin
Yves Bertrand
Mark C. Walters
Bénédicte Neven
Sophie Dupont
Josu de la Fuente
Françoise Bernaudin
Fernanda Volt
Christina Peters
Gérard Michel
Annalisa Ruggeri
Marco Zecca
Eliane Gluckman
Chantal Kenzey
J P Vannier
Susanne Matthes
Franco Locatelli
Patrick Lutz
Alina Ferster
Hanadi Rafii El-Ayoubi
Mary Eapen
Belinda Pinto Simões
Nathalie Dhedin
Myriam Labopin
Mouhab Ayas
Vanderson Rocha
Barbara Cappelli
Jean-Hugues Dalle
Source :
Blood. 126:541-541
Publication Year :
2015
Publisher :
American Society of Hematology, 2015.

Abstract

Introduction: Hematopoietic stem cell transplantation (HSCT) is, to date, the only curative therapy for sickle cell disease (SCD). However, HSCT is offered to relatively few patients with SCD for a number of reasons including lack of a suitable HLA-matched donor, lack of consensus on indications for HSCT, the potential for trading one chronic condition (i.e., SCD) for another, such as chronic graft-versus-host disease (GVHD), and the mortality associated with the procedure. To-date, most HSCTs for SCD have utilized matched siblings as donors and are performed in children and adolescents. We report outcomes after HLA-matched sibling HSCT of patients reported to the Eurocord-Monacord/European Group for Blood and Marrow Transplantation (EBMT) and Center for International Blood and Marrow Transplant Research (CIBMTR). Material and methods: One thousandpatients with SCD received HLA identical sibling HSCT between 1991 and 2013; n=439 from CIBMTR and n=561 from EBMT centers. HSCTs were performed in 90 centers in 23 countries. Results: Median age at HSCT was 9 years (range 1-54y); 85% of patients were aged The cumulative incidence (CI) of neutrophil engraftment at day+60 was 98% (96.6% for CB, 98.3% for BM and 95.2% for PB) with a median time to recovery of 19 days, while that for platelet engraftment was 98 % (96±2% for CB, 99±1% for BM and 98±9% for PBSC) with a median time to recovery of 25 days. Twenty-six patients experienced primary and 47 patients secondary graft failure; 67 patients died mainly due to GVH or infection. The 3-year probabilities of overall (OS) and event-free survival (EFS, alive with engraftment) were 94% (95% CI 92-95) and 90% (95% CI 68-82), respectively. According to stem cell source, 3-year OS was 99% after CB, 94% after BM and 80% after PBS (p Conclusion: This large registry based international study shows that HLA identical sibling transplant is successful more than 90% of the patients with severe SCD with limited transplant related complications (rejection, GVHD). Strategies aimed at lowering graft failure and GVHD are desirable to further optimize the observed 3-year event-free survival. Importantly, these data should increase awareness to early referral to HSCT of patients with severe SCD. Disclosures Walters: ViaCord and AllCells, Inc: Other: Medical director. Bertrand:ERYTECH Pharma: Consultancy. Peters:Medac: Research Funding; Fresenius: Research Funding; Amgen: Research Funding; Jazz: Research Funding; Novartis: Research Funding; Pfizer: Research Funding; Sanovi: Research Funding; Pierre-Fabre: Research Funding.

Details

ISSN :
15280020 and 00064971
Volume :
126
Database :
OpenAIRE
Journal :
Blood
Accession number :
edsair.doi...........2aee52600ca4baa3afceecab5d8eb7f9
Full Text :
https://doi.org/10.1182/blood.v126.23.541.541