Journal Article

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

Angelucci E., Giardini C., Al-Ahmari A., Peters C., Zecca M., Pilo F., Targhetta C., Hussein A. A., Yesilipek A., Ghavamzadeh A., Baronciani D., Marktel S., Dini G., Locatelli F., De La Fuente J., Potschger U., Gaziev J., Orofino M. G., Bader P.

2016

Overview

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.

• Publisher: Springer Science and Business Media LLC,Nature Publishing Group UK,Nature Publishing Group
• Subject: 692/308/2171 / 692/699/1541 / Adolescent / Adult / Blood / Blood diseases / Bone marrow / Bone marrow transplantation / Care and treatment / Cell Biology / Demography / Disease-Free Survival / Europe / Female / Health aspects / Hematology / Hematopoietic Stem Cell Transplantation / Hemoglobinopathy / hemopoietic stem cell transplantation; thalassemia; young patients / HSCT / Humans / Internal Medicine / Leukocytes / Male / Medicine / Medicine & Public Health / Middle Aged / original-article / Public Health / Registries / Societies, Medical / Stem cell transplantation / Stem Cells / Survival / Survival Rate / Thalassemia / Thalassemia - mortality / Thalassemia - therapy / Transfusion / Transplantation / Transplants