TY - JOUR
T1 - Hemopoietic stem cell transplantation in thalassemia: A report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010
AU - Baronciani, D.
AU - Angelucci, E.
AU - Potschger, U.
AU - Gaziev, J.
AU - Yesilipek, A.
AU - Zecca, M.
AU - Orofino, M. G.
AU - Giardini, C.
AU - Al-Ahmari, A.
AU - Marktel, S.
AU - De La Fuente, J.
AU - Ghavamzadeh, A.
AU - Hussein, A. A.
AU - Targhetta, C.
AU - Pilo, F.
AU - Locatelli, Franco
AU - Dini, G.
AU - Bader, P.
AU - Peters, C.
PY - 2016
Y1 - 2016
N2 - 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.
AB - 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.
KW - HSCT
KW - HSCT
UR - http://hdl.handle.net/10807/229887
U2 - 10.1038/bmt.2015.293
DO - 10.1038/bmt.2015.293
M3 - Article
SN - 0268-3369
VL - 51
SP - 536
EP - 541
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
ER -