Treatment for beta-thalassemia with hematopoietic stem cell transplantation.

doi:10.7504/ek2014110605

Abstract

Abstract:

Abstract：Allogeneic hematopoietic stem cell transplantation （HSCT） is the only curative therapy for severe beta-thalassemia major. Patients with an available human leukocyte antigen （HLA） identical sibling donor should be offered HSCT as soon as possible before development of iron overload and transfusion associated complications， and the cure rate was up to 80%～90%. High-resolution HLA typing matched unrelated donor transplants also achieved good results. HSCT from HLA-mismatched relatives or HLA-phenotypically-identical donor is an option to be performed in expert centers. Optimization of conditioning regimen and techniques to control transplant-related complications will further improve outcomes.

Key words: hematopoietic stem cell transplantation（HSCT）, beta-thalassemia, bone marrow transplatation（BMT）

摘要：

异基因造血干细胞移植（allo-HSCT）是目前临床根治重型β地中海贫血的惟一方法。对有人类白细胞抗原（HLA）全相合同胞供者的患者，应尽早进行HSCT，治愈率可达80%～90%。高分辨HLA配型相合的无关供体移植亦取得良好结果。血缘相关HLA 不全相合和单倍型HSCT扩大了移植物来源，移植效果尚不肯定。移植后需密切检测嵌合体变化，优化预处理方案，提高移植相关并发症防治技术，将进一步改善移植效果。

关键词: 造血干细胞移植；&beta, 地中海贫血；骨髓移植

CLC Number:

Ｒ72

XU Hong-gui，FANG Jian-pei.. Treatment for beta-thalassemia with hematopoietic stem cell transplantation.[J]. Acta Metallurgica Sinica, DOI: 10.7504/ek2014110605.

徐宏贵，方建培. 造血干细胞移植治疗β地中海贫血[J]. 中国实用儿科杂志, DOI: 10.7504/ek2014110605.

References

[1］ Angelucci E， Matthes-Martin S， Baronciani D， et al. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease： indications and management recommendations from an international expert panel［J］. Haematologica，2014，99：811-819.

[2］ Angelucci E. Hematopoietic stem cell transplantation in thalassemia［J］. Am Soc Hematol，2010，2010：456-462.

[3］ Gaziev J， Lucarelli G. Hematopoietic stem cell transplantation for thalassemia［J］. Curr Stem Cell Res Ther，2011，6：162-169.

[4］ Sabloff M， Chandy M， Wang Z， et al. HLA-matched sibling bone marrow transplantation for β-thalassemia major［J］.Blood，2011，117： 1745-1750.

[5］ Hubail Z，Morsy MF. Splenectomized versus nonsplenectomized thalassemia patients with thalassemia major［J］. Saudi Med J， 2009， 30：450-454.

[6］ Sodani P， Gaziev J， Polchi P， et al. New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years［J］. Blood，2004，104：1201-1203.

[7］ Mathews M， George M， Viswabandya A， et al. Improved clinical outcomes of high risk β-thalassemia major patients undergoing a HLA matched related allogeneic stem cell transplant with a treosulfan based conditioning regimen and peripheral blood stem cell grafts［J］. Plos One，2013，8：1-8.

[8］ Ghavamzadeh A，Iravani M，Ashouri A，et al. Peripheral blood versus bone marrow as a source of hematopoietic stem cells for allogeneic transplantation in children with classⅠandⅡβ-thalassemia major［J］. Biol Blood Marrow Transplant，2008，14（3）：301-308.

[9］ Li C， Wu X， Feng X， et al. A novel conditioning regimen improves outcomes in beta-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation［J］. Blood，2012，120： 3875-3881.

[10］ Jaing TH，Hung IJ，Yang CP，et al. Unrelated cord blood transplantation for thalassaemia： a single-institution experience of 35 patients［J］. Bone Marrow Transplant，2012，47：33-39.

[11］ Baronciani D，Pilo F，Lyon-Caen S，et al. Hematopoietic stem cell transplantation in thalassemia major：report from the EBMT hemoglobinopathy registry［C/CD］. 53rd ASH annual meeting and exposition. 2011 American Society of Hematology.San Diego，2011.

[12］ Kabbara N， Locatelli F， Rocha V， et al. A multicentric comparative analysis of outcomes of HLA identical related cord blood and bone marrow transplantation in patients with betathalassemia or sickle cell disease［J］. Biol Blood Marrow Transplant，2008，14：3-4.

[13］ Locatelli F， Kabbara N， Ruggeri A， et al. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling［J］. Blood，2013： 1072-1078.

[14］ Gaziev D， Galimberti M， Lucarelli G， et al. Bone marrow transplantation from alternative donors for thalassemia： HLA phenotypically identical relative and HLA-nonidentical sibling or parent transplants［J］. Bone Marrow Transplant， 2000; 25： 815-821.

[15］ Gaziev J， Marziali M， Isgro A， et al. Bone marrow transplantation for thalassemia from alternative related donors： improved outcomes with a new approach［J］. Blood， 2013， 122： 2751-2756.

[16］ Sodani P， Isgro A， Gaziev J， et al. Purified T-depleted， CD34＋peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia［J］. Blood， 2010， 115： 1296-1302.

[17］ La Nasa G， Argiolu F， Giardini C， et al. Unrelated bone marrow transplantation for beta-thalassemia patients： the experience of the Italian Bone Marrow Transplant Group［J］. Ann NY Acad Sci， 2005， 1054： 186-195.

[18］ Locatelli F， Littera R， Pagliard D， et al. Outcome of unrelated donor bone marrow transplantation for thalassemia major patients［C/CD］. 53rd ASH annual meeting and exposition. 2011 American Society of Hematology.San Diego，2011.

[19］ Ruggeri A，Eapen M，Scaravadou A，et al. Survey of outcomes of unrelated cord blood transplant in patients with haemoglobinopathies： a retrospective study on behalf of CIBMTR， NYCB and EUROCORD［J］. Bone Marrow Transplant， 2010， 45： S73.

[20］ Littera R， Orru N， Vacca A， et al. The role of killer immunoglobulin-like receptor haplotypes on the outcome of unrelated donor haematopoietic SCT for thalassaemia［J］. Bone Marrow Transplant， 2010， 45： 1618-1624.

[21］ Fleischhauer K， Locatelli F， Zecca M， et al. rejection after unrelated donor hematopoietic stem cell transplantation for thalassemia is associated with nonpermissive HLA-DPB1 disparity in host-versus-graft direction［J］. Blood， 2006， 107： 2984-2992.

[22］ Gaziev J，SodaniP，Lucarelli G，et al. Second stem cell transplantation for thalassemia recurrence following graft rejection of the first grafe［J］.Bone Marrow Transplant，2008，42： 397-404.

[23］ Karasu GT， Yesilipek MA， Karauzum SB， et al. The value of donor lymphocyte infusions in thalassemia patients at imminent risk of graft rejection following stem cell transplantation［J］. Pediatr Blood Cancer， 2012， 58： 453-458.

[24］ Tavil B， Kazik M， Kuskonmaz B， et al. A prompt graft-versus-thalassemia effect upon withdrawal of cyclosporine A in a child who received allogeneic peripheral blood stem cell transplantation［J］. Bone Marrow Transplant， 2006， 38： 315-316.

[25］ Andreani M， Testi M， Gaziev J， et al. Quantitatively different red cell/nucleated cell chimerism in patients with long-term， persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease［J］. Haematologica， 2011， 96（1）： 128-133.