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.

Baronciani, D., Angelucci, E., Potschger, U., Gaziev, J., Yesilipek, A., Zecca, M., Orofino, M. G., Giardini, C., Al-Ahmari, A., Marktel, S., De La Fuente, J., Ghavamzadeh, A., Hussein, A. A., Targhetta, C., Pilo, F., Locatelli, F., Dini, G., Bader, P., Peters, C., Hemopoietic stem cell transplantation in thalassemia: A report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010, <<BONE MARROW TRANSPLANTATION>>, 2016; 51 (4): 536-541. [doi:10.1038/bmt.2015.293] [https://hdl.handle.net/10807/229887]

Hemopoietic stem cell transplantation in thalassemia: A report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010

Locatelli, Franco
Writing – Review & Editing
;
2016

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.
2016
Inglese
Baronciani, D., Angelucci, E., Potschger, U., Gaziev, J., Yesilipek, A., Zecca, M., Orofino, M. G., Giardini, C., Al-Ahmari, A., Marktel, S., De La Fuente, J., Ghavamzadeh, A., Hussein, A. A., Targhetta, C., Pilo, F., Locatelli, F., Dini, G., Bader, P., Peters, C., Hemopoietic stem cell transplantation in thalassemia: A report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010, <<BONE MARROW TRANSPLANTATION>>, 2016; 51 (4): 536-541. [doi:10.1038/bmt.2015.293] [https://hdl.handle.net/10807/229887]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/229887
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