组蛋白去乙酰化酶抑制剂治疗骨髓增生异常综合征

目前,常规药物治疗骨髓增生异常综合征(MDS)的效果并不显著,异基因造血干细胞移植也有一定的局限性。鉴于表观遗传学在MDS的发生和发展中具有重要作用,且表观遗传调控药组蛋白去乙酰化酶抑制剂已用于多种实体瘤的治疗,因而该抑制剂在MDS中的治疗作用也愈来愈受到关注。本文主要综述组蛋白去乙酰化酶抑制剂的作用机制,治疗MDS的基本原理和临床治疗的现状及进展。

Histone deacetylase inhibitors for the treatment of myelodysplastic syndrome

The effects of conventional treatment for myelodysplastic syndrome (MDS) are not remarkable to date, while only a minority of patients was eligible for allogeneic stem cell transplantation. As epigenetics plays a significant role during the occurrence and development of MDS, and histone deacetylase inhibitors (HDACI), a class of gene expression modulating drugs, are currently being developed for therapy of several types of solid tumor, more attention is paying to HDACI as potential therapy of MDS. This review summarizes briefly the rationale for HDACI use in MDS, the common mechanism of HDACI, the present state of the clinical efficiency, and future development in this field.