骨髓增生异常综合征去甲基化治疗的研究新进展*

Research progress on treatment of myelodysplastic syndromes with demethylating drugs

摘要: 表观遗传学异常是骨髓增生异常综合征（myelodysplastic syndromes，MDS ）发病的重要机制之一，DNA 甲基化异常是MDS 最常见的表观遗传学改变。抑制DNA 异常甲基化可以改善部分MDS 患者的病情，延长其生存期。目前被批准用于MDS 临床治疗的去甲基化药物主要有阿扎胞苷和地西他滨，这两种药物在MDS 患者中均显示出一定的疗效。本文对该药的应用及进展研究进行综述。

Abstract: Abnormal epigenetics play important roles in the pathogenesis of myelodysplastic syndromes (MDS). DNA hypermeth -ylation is the most common epigenetic abnormality in MDS. Demethylating DNA hypermethylation may improve the quality of life of MDS patients and prolong their overall survival. Azacitidine and decitabine are the demethylating drugs approved for MDS treatment. These drugs showed clinical effects on all subgroups of MDS patients.