骨髓增生异常综合征的去甲基化治疗

骨髓增生异常综合征(MDS)是一组异质性后天性克隆性恶性疾病,其基本病变是克隆性造血干、祖细胞发育异常,导致无效造血以及恶性转化危险性增高。MDS作为恶性克隆性疾病,只有异基因造血干细胞移植(allo-HSCT)才可以治愈。而MDS主要发生于老年人群,多数患者由于年龄、体能状况及严重并发症无法接受异基因造血干细胞移植治疗。在去甲基化药物(HMAs)出现之前,大多数患者只能采取支持治疗作为主要甚至唯一的治疗手段。

Demethylation therapy in myelodysplastic syndrome

Myelodysplastic syndrome（MDS） is a group of clonal heterogeneous malignancies, which is characterized by dysplasia of clonal hematopoietic/progenitor that lead to ineffective hematopoiesis and higher risk of progressing to acute myeloid leukemia. The only curable method to MDS is allogeneic hematopoietic stem cell transplantation（allo-HSCT）. However, most of MDS patients who are aged can＇t accept the treatment because of age, physical condition and severe complication. The main or even the only treatment to MDS is supportive treatment for most of MDS patients before hypomethylating agents（HMAs） were invented.