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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。
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is currently the only method of clinical treatment of severe β-thalassemia. HSCT should be done as early as possible for patients with HLA-identical sibling donors, with a cure rate of 80% -90%. High-resolution HLA matching coincide with donor-free donor also achieved good results. Hemophilia-related incomplete HLA haplotype and haploidentical HSCT expand the source of the graft, the effect of transplantation is not yet confirmed. After transplantation, the changes of chimerism should be closely monitored, the pretreatment program should be optimized, and the prevention and treatment of transplantation-related complications should be improved, which will further improve the transplantation effect.