Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry …

D Baronciani, E Angelucci, U Potschger… - Bone marrow …, 2016 - nature.com
D Baronciani, E Angelucci, U Potschger, J Gaziev, A Yesilipek, M Zecca, MG Orofino…
Bone marrow transplantation, 2016nature.com
Allogeneic hemopoietic stem cell transplantation (HSCT) is the only method currently
available to cure transfusion-dependent thalassemia major that has been widely used
worldwide. To verify transplantation distribution, demography, activity, policies and
outcomes inside the European Group for Blood and Marrow Transplantation (EBMT), we
performed a retrospective non-interventional study, extracting data from the EBMT
hemoglobinopathy prospective registry database. We included 1493 consecutive patients …
Abstract
Allogeneic hemopoietic stem cell transplantation (HSCT) is the only method currently available to cure transfusion-dependent thalassemia major that has been widely used worldwide. To verify transplantation distribution, demography, activity, policies and outcomes inside the European Group for Blood and Marrow Transplantation (EBMT), we performed a retrospective non-interventional study, extracting data from the EBMT hemoglobinopathy prospective registry database. We included 1493 consecutive patients with thalassemia major transplanted between 1 January 2000 and 31 December 2010. In total, 1359 (91%) transplants were performed on patients< 18 years old, 1061 were from a human leukocyte Ag-identical sibling donor. After a median observation time of 2 years, the 2-year overall survival (OS) and event-free survival (EFS; that is, thalassemia-free survival) were 88±1% and 81±1%, respectively. Transplantation from a human leukocyte Ag-identical sibling offered the best results, with OS and EFS of 91±1% and 83±1%, respectively. No significant differences in survival were reported between countries. The threshold age for optimal transplant outcomes was around 14 years, with an OS of 90–96% and an EFS of 83–93% when transplants were performed before this age. Allogeneic HSCT for thalassemia is a curative approach that is employed internationally and produces excellent results.
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