title: Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010.
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.
authors.affiliation: Ematologia e Centro Trapianti, Ospedale Oncologico di Riferimento Regionale 'Armando Businco', Azienda Ospedaliera 'Brotzu', Cagliari, Italy.
authors.forename: D
authors.initials: D
authors.lastname: Baronciani
authors.affiliation: Ematologia e Centro Trapianti, Ospedale Oncologico di Riferimento Regionale 'Armando Businco', Azienda Ospedaliera 'Brotzu', Cagliari, Italy.
authors.forename: E
authors.initials: E
authors.lastname: Angelucci
authors.affiliation: St Anna Children's Hospital, Department of Pediatrics, Medical University, Vienna, Austria.
authors.forename: U
authors.initials: U
authors.lastname: Potschger
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Rome, Italy.
authors.forename: J
authors.initials: J
authors.lastname: Gaziev
authors.affiliation: Pediatric Stem Cell Transplantation Unit, Akdeniz University School of Medicine, Antalya, Turkiye.
authors.forename: A
authors.initials: A
authors.lastname: Yesilipek
authors.affiliation: Department of Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico 'San Matteo', Pavia, Italy.
authors.forename: M
authors.initials: M
authors.lastname: Zecca
authors.affiliation: Department of Biomedical Science and Biotechnology, Second Paediatric Clinic, Bone Marrow Transplant Unit, University of Cagliari, Cagliari, Italy.
authors.forename: M G
authors.initials: MG
authors.lastname: Orofino
authors.affiliation: Ematologia e Centro Trapianti, Ospedali Riuniti Marche Nord, Pesaro, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Giardini
authors.affiliation: King Faisal Specialist Hospital and Research Centre, Department of PHO and Stem Cell Transplantation, Riyadh, Saudi Arabia.
authors.forename: A
authors.initials: A
authors.lastname: Al-Ahmari
authors.affiliation: Stem Cells Programme, IRCCS San Raffaele Scientific Institute, Milan, Italy.
authors.forename: S
authors.initials: S
authors.lastname: Marktel
authors.affiliation: Division of Paediatrics, St Mary Hospital, London, UK.
authors.forename: J
authors.initials: J
authors.lastname: de la Fuente
authors.affiliation: Hematology, Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: A
authors.initials: A
authors.lastname: Ghavamzadeh
authors.affiliation: Pediatric Bone Marrow and Stem Cell Transplant, King Hussein Cancer Center, Al-Jubeiha Amman, Jordan.
authors.forename: A A
authors.initials: AA
authors.lastname: Hussein
authors.affiliation: Ematologia e Centro Trapianti, Ospedale Oncologico di Riferimento Regionale 'Armando Businco', Azienda Ospedaliera 'Brotzu', Cagliari, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Targhetta
authors.affiliation: Ematologia e Centro Trapianti, Ospedale Oncologico di Riferimento Regionale 'Armando Businco', Azienda Ospedaliera 'Brotzu', Cagliari, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Pilo
authors.affiliation: Department of Pediatric Hematology and Oncology IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.affiliation: EBMT member.
authors.forename: G
authors.initials: G
authors.lastname: Dini
authors.affiliation: Division for Stem Cell Transplantation and Immunology Hospital for Children and Adolescents, Frankfurt a. Main, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Bader
authors.affiliation: St Anna Children's Hospital, Department of Pediatrics, Medical University, Vienna, Austria.
authors.forename: C
authors.initials: C
authors.lastname: Peters
mesh_terms: D000293:Adolescent; D000328:Adult; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D008875:Middle Aged; D012042:Registries; D012955:Societies, Medical; D015996:Survival Rate; D013789:Thalassemia / Q000401:mortality / Q000628:therapy*