造血干细胞移植治疗β地中海贫血

doi:10.7504/ek2014110605

摘要/Abstract

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

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）

中图分类号:

Ｒ72

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.

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

参考文献

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