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 y

SUBMITTER: Gluckman E 

PROVIDER: S-EPMC5356458 | biostudies-literature | 2017 Mar

REPOSITORIES: biostudies-literature