【摘要】 目的观察低剂量DNA甲基化抑制剂地西他滨(decitabine,DAC)用于骨髓增生异常综合征治疗的临床疗效,药物安全性和针对毒副作用所采取的相应措施及反应。方法应用小剂量地西他滨治疗10例骨髓增生异常综合征(MDS),剂量为25 mg/次,持续静脉滴注1 h以上,每天1次,共5 d;4周1个周期。结果完成2个疗程治疗后,患者均有程度不同的治疗反应,其中2例患者达到完全缓解(CR)20%。完成4个疗程治疗后,4例患者达到完全缓解(CR)40%,2例达到部分缓解(PR)20%,1例患者达到骨髓缓解(mCR 10%),总有效率60%。治疗期间均出现不同程度的骨髓抑制,但不良反应轻微,经对症支持治疗后患者均可耐受。结论地西他滨作为DNA甲基化的有效抑制剂用于MDS,尤其是对于不能耐受强化疗的高危老年患者,能够使患者病情稳定,减轻输血依赖,是具有一定疗效的治疗手段之一。更多还原

【Abstract】 Objective To observe the effect of low dose DNA methylation inhibitor decitabine( DAC) for clinical treatment of myelodysplastic syndrome,the drug’s safety,corresponding measures for toxicity and response. Methods 10 patients with myelodysplastic syndrome( MDS) were treated with low-dose decitabine therapy,25mg daily,continuous infusion over 1 hour for 5 days,4 weeks as a cycle. Results After 2 courses of treatment,all patients manifested response to treatment in various degrees,among whom 3 patients completely remitted( CR20%). By the completion of 4 courses,4 patients were in complete remission( CR40%),and 2 in partial remission( PR20%),1 achieved remission of marrow( mCR10%),with a total efficiency rate of 60%. In the process of treatment,bone marrow suppression of various degrees occurred,but adverse reactions were mild. After symptomatic treatment,the reactions became endurable. Conclusion Decitabine as a potent inhibitor of DNA methylation for MDS,especially for those elderly patients in high-risk who cannot tolerate intensive chemotherapy,can make the disease stabilized and reduce blood transfusion dependence. It is one of the effective means of treatment.更多还原