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Bone Marrow Transplant. 2016 Apr;51(4):536-41. doi: 10.1038/bmt.2015.293. Epub 2016 Jan 11.

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

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Ematologia e Centro Trapianti, Ospedale Oncologico di Riferimento Regionale 'Armando Businco', Azienda Ospedaliera 'Brotzu', Cagliari, Italy.
St Anna Children's Hospital, Department of Pediatrics, Medical University, Vienna, Austria.
International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Rome, Italy.
Pediatric Stem Cell Transplantation Unit, Akdeniz University School of Medicine, Antalya, Turkiye.
Department of Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico 'San Matteo', Pavia, Italy.
Department of Biomedical Science and Biotechnology, Second Paediatric Clinic, Bone Marrow Transplant Unit, University of Cagliari, Cagliari, Italy.
Ematologia e Centro Trapianti, Ospedali Riuniti Marche Nord, Pesaro, Italy.
King Faisal Specialist Hospital and Research Centre, Department of PHO and Stem Cell Transplantation, Riyadh, Saudi Arabia.
Stem Cells Programme, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Division of Paediatrics, St Mary Hospital, London, UK.
Hematology, Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
Pediatric Bone Marrow and Stem Cell Transplant, King Hussein Cancer Center, Al-Jubeiha Amman, Jordan.
Department of Pediatric Hematology and Oncology IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.
EBMT member.
Division for Stem Cell Transplantation and Immunology Hospital for Children and Adolescents, Frankfurt a. Main, Germany.


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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