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

被引：163

作者：

Baronciani, D. ^{[1
,2
]}

Angelucci, E. ^{[1
,2
]}

Potschger, U. ^{[3
]}

Gaziev, J. ^{[4
]}

Yesilipek, A. ^{[5
]}

Zecca, M. ^{[6
]}

Orofino, M. G. ^{[7
]}

Giardini, C. ^{[8
,9
]}

Al-Ahmari, A. ^{[10
]}

Marktel, S. ^{[11
]}

de la Fuente, J. ^{[12
]}

Ghavamzadeh, A. ^{[13
]}

Hussein, A. A. ^{[14
]}

Targhetta, C. ^{[1
,2
]}

Pilo, F. ^{[1
,2
]}

Locatelli, F. ^{[15
]}

Dini, G.

Bader, P. ^{[16
]}

Peters, C. ^{[3
]}

机构：

[1] Azienda Osped Brotzu, Osped Oncol Riferimento Reg Armando Businco, Ematol, Via Edward Jenner, I-09121 Cagliari, Italy

[2] Azienda Osped Brotzu, Osped Oncol Riferimento Reg Armando Businco, Ctr Trapianti, Via Edward Jenner, I-09121 Cagliari, Italy

[3] Med Univ, Dept Pediat, St Anna Childrens Hosp, Vienna, Austria

[4] Int Ctr Transplantat Thalassemia & Sickle Cell An, Rome, Italy

[5] Akdeniz Univ, Sch Med, Pediat Stem Cell Transplantat Unit, TR-07058 Antalya, Turkey

[6] Fdn IRCCS Policlin San Matteo, Dept Pediat Hematol Oncol, Pavia, Italy

[7] Univ Cagliari, Bone Marrow Transplant Unit, Paediat Clin 2, Dept Biomed Sci & Biotechnol, Cagliari, Italy

[8] Osped Riuniti Marche Nord, Ematol, Pesaro, Italy

[9] Osped Riuniti Marche Nord, Ctr Trapianti, Pesaro, Italy

[10] King Faisal Specialist Hosp & Res Ctr, Dept PHO & Stem Cell Transplantat, Riyadh 11211, Saudi Arabia

[11] IRCCS San Raffaele Sci Inst, Stem Cells Programme, Milan, Italy

[12] St Mary Hosp, Div Paediat, London, England

[13] Univ Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Tehran, Iran

[14] King Hussein Canc Ctr, Pediat Bone Marrow & Stem Cell Transplant, Al Jubeiha Amman, Jordan

[15] IRCCS Osped Pediat Bambino Gesu, Dept Pediat Hematol & Oncol, Rome, Italy

[16] Hosp Children & Adolescents, Div Stem Cell Transplantat & Immunol, Frankfurt, Germany

来源：

BONE MARROW TRANSPLANTATION | 2016年 / 51卷 / 04期

关键词：

BETA-THALASSEMIA; CONDITIONING REGIMEN; MANAGEMENT; THERAPY;

D O I：

10.1038/bmt.2015.293

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

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.

引用

页码：536 / 541

页数：6

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