地西他滨治疗环形铁粒幼细胞性难治性贫血的研究现状

Current status of decitabine in refractory anemia with ringed sideroblasts

环形铁粒幼细胞性难治性贫血(RAS)是骨髓增生异常综合征(MDS)的法国-美国-英国协作组(FAB)分型之一,通常属于相对低危组MDS。去甲基化药物的使用对于RAS患者的主要治疗目标是改善血细胞减少,使患者脱离输血依赖,提高其生存质量和延长生存期。地西他滨(DAC)是重要的去甲基化药物,在RAS治疗中发挥重要作用。目前,DAC对RAS的最佳治疗剂量为20 mg/(m2·d)×5 d。由于较大剂量的DAC可能引起更严重的骨髓抑制,因此近年来,DAC对包括RAS在内的较低危MDS的治疗剂量有减低趋势。笔者通过对RAS发病机制,DAC治疗RAS的作用机制、最佳用药剂量和不良反应等方面的研究现状进行综述,旨在为RAS的临床治疗提供理论依据。

Refractory anemia with ringed sideroblasts (RAS) is one of myelodysplastic syndrome (MDS) in the French-American-British cooperative group (FAB) classifications, and it often belongs to a relatively low-risk group of MDS. The main therapeutic goals for the usage of demethylated drugs for RAS patients are improving cytopenia, detaching from blood transfusion, and improving quality of life and prolonging survival. Decitabine (DAC) is an important demethylating drug that plays an important role in the treatment of RAS. Currently, the optimal therapeutic dose for DAC treatment of RAS is 20 mg/(m2·d). Since larger dose of DAC may cause more severe levels of myelosuppression, recently DAC has shown a decreasing trend in therapeutic dose for lower-risk MDS, which including RAS. This article summarizes the current status in pathogenesis of RAS, and the mechanisms, optimal dosage and adverse reactions of DAC treatment in RAS, in order to provide theoretical basis for clinical treatment of RAS.