重型地中海贫血的移植进展

Research advances in transplantation for thalassemia major

摘要:

地中海贫血是因调节血红蛋白的基因突变导致珠蛋白链形成障碍的遗传性血液疾病。目前异基因造血干细胞移植是公认的唯一治愈手段，其经历了预处理方案的演进，供体及移植物来源的对比选择等阶段，现生存情况已得到提高。该文回顾了地中海贫血移植的发展过程及研究进展，以期给临床提供更合适的治疗选择决策。

Abstract:

Thalassemia is an inherited blood disorder caused by disordered globin chain synthesis due to mutations in the regulatory genes for hemoglobin. At present, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is recognized as the only curative method for treatment. Through the revolution of pretransplantation regimens and selection of donor and source of stem cells, patients' survival has been greatly improved. This article reviews the development of transplantation for thalassemia and related research advances, in order to provide suitable treatment options for clinical application.