骨髓增生异常综合征是一组影响骨髓造血干细胞、祖细胞的克隆性疾病。近年来，随着对骨髓增生异常综合征发病机制的深入研究产生了许多新型药物。目前对骨髓增生异常综合征治疗的药物包括缺氧诱导因子脯氨酰羟化酶抑制剂、端粒酶抑制剂、剪切体抑制剂、异柠檬酸脱氢酶抑制剂、新型去甲基化剂、免疫检测点抑制剂、针对TP53突变药物、酪氨酸激酶抑制剂、抗CD47的单克隆抗体。对这些药物的作用机制、临床使用情况进行了总结，为患者治疗药物的选择提供参考。

Myelodysplastic syndromes are a group of clonal diseases that affect bone marrow hematopoietic stem cells and progenitor cells. In recent years, with the in-depth research on the pathogenesis of myelodysplastic syndrome, many new drugs have been produced. At present, drugs for treatment of myelodysplastic syndromes include hypoxia-inducible factor prolyl hydroxylase inhibitors, telomerase inhibitors, spliceosome inhibitors, isocitrate dehydrogenase inhibitors, new demethylation drugs, immune checkpoint inhibitors, targeting TP53 mutant drugs, tyrosine kinase inhibitors, and anti-CD47 monoclonal antibodies. This article summarizes the mechanism of action and clinical use of these drugs, in order to provide a reference for the selection of therapeutic drugs for patients.

R977