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

Baronciani D, Angelucci E, Potschger U, Gaziev J, Yesilipek A, Zecca M, Orofino MG, Giardini C, Al-Ahmari A, Marktel S, de la Fuente J, Ghavamzadeh A, Hussein AA, 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 Transplant. 2016 Jan 11. doi: 10.1038/bmt.2015.293. [Epub ahead of print]

In this manuscript, EBMT provides the retrospective transplant data of 1493 consecutive patients with thalassemia major from the data extracted from the EBMT prospective registry database.  Of these patients,1359 (91%)  were  <18 years old at time of transplantation, 1061 were transplanted from a human leukocyte Ag-identical sibling donor. The 2-year overall survival (OS) and thalassemia-free survival) were reported to be as 88±1% and 81±1%, respectively. Transplantation from a  HLA 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.  Hematopoietic stem cell transplantation has been reported to offer curative treatment option with successful outcomes across the countries. (Summarized by Şule Ünal, M.D.)

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