去甲基化药物治疗骨髓增生异常综合征的临床现状与前景

Clinical application status and prospect of hypomethylating agents in treatment of myelodysplastic syndromes

骨髓增生异常综合征(MDS)是一组起源于造血干细胞的异质性髓系克隆性疾病,具有高风险向急性髓细胞白血病(AML)转化的特点。MDS患者的自然病程及预后异质性较大,个体化治疗策略对其尤为重要。自2004年去甲基化药物(HAM)获得美国食品药品监督管理局(FDA)批准上市,用于治疗MDS以来,迄今该药仍为治疗较高危MDS患者的一线药物。笔者拟就HMA单药治疗较高危MDS患者、以HMA为基础的联合治疗MDS方案、HMA用于治疗接受造血干细胞移植(HSCT)MDS患者的研究进展及临床治疗现状与应用前景进行阐述。

Myelodysplastic syndromes(MDS)are a group of heterogeneous myeloid clonal diseases originated from hematopoietic stem cells,which have a high risk of transformation to acute myeloid leukemia(AML).The natural course and prognosis of MDS patients are very heterogeneous,so a individualized treatment measure should be taken.Hypomethylating agents(HMA)have been approved for treatment of MDS since 2004 by Food and Drug Administration(FDA)of American,and they are recommended as the first line drug therapy for higher-risk MDS patients.This article summarizes the current situations and research progresses of monotherapy with HMA in patient with higher-risk MDS,combination protocols based on HMA treatment of MDS patients,and HMA treatment of MDS patients after hematopoietic stem cell transplantation(HSCT),especially on the prospect of combined therapy based on HMA.