去甲基化药物及免疫检查点抑制剂在MDS中的研究进展

Research Progress of Hypomethylating Agents and Immune Checkpoint Inhibitors in MDS

摘要: 骨髓增生异常综合征(Myelodysplastic syndrome, MDS)是一种以无效造血为特征的高度异质性疾病，表现范围广泛，从惰性和无症状的细胞减少到急性髓系白血病(AML)。目前MDS的发病机制还尚未完全清楚，其唯一的治疗方法是异基因造血干细胞移植(allo-HSCT)，然而，由于allo-HSCT具有很高的毒性风险，人们常常考虑替代治疗。去甲基化药物及免疫检查点抑制剂的研究及临床应用，为MDS发病机制的探索和临床治疗带来了新方向。故本文主要就这两类药物在MDS中的作用机制、分类及临床疗效等方面进行综述，加深对MDS发病机制及新时代药物的了解，为MDS的治疗提供临床参考价值。

Abstract: Myelodysplastic syndrome (MDS) is a highly heterogeneous disease characterized by ineffective hematopoiesis, with a wide range of manifestations ranging from indolent and asymptomatic cell depletion to acute myeloid leukemia (AML). At present, the pathogenesis of MDS is not fully under-stood. The only treatment for MDS is allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, because of the high toxicity risk of allo-HSCT, alternative treatment is often considered. The research and clinical application of hypomethylating agents and immune checkpoint inhibitors have brought a new direction for the exploration of pathogenesis and clinical treatment of MDS. Therefore, this article mainly reviews the mechanism of action, classification and clinical efficacy of these two types of drugs in MDS, deepening the understanding of the pathogenesis of MDS and the new era of drugs, and providing reference value for the treatment of MDS.