二氯醋酸二异丙胺治疗非酒精性脂肪性肝病的随机双盲临床研究

Diisopropylamine dichloroacetate in the treatment of nonalcoholic fatty liver disease: a multicenter random double-blind controlled trial

目的 研究二氯醋酸二异丙胺治疗非酒精性脂肪性肝病的临床疗效和安全性。 方法 用随机双盲多中心剂量平行对照的临床试验设计选择非酒精性脂肪性肝病患者,分别用二氧醋酸二异丙胺高剂量(120mg／d)和低剂量(60 mg／d)治疗8周,观察其疗效和安全性。 结果 本试验共有127例患者入选,高剂量组63例,低剂量组64例。未见脱落病例,剔除4例,剔除率3.1％,实际完成病例数123例,高剂量组61例,低剂量组62例。治疗后8周高剂量组和低剂量组临床疗效总有效率分别为87.8％和79.6％(P=0.2536),丙氨酸氨基转移酶复常总有效率分别为55.7％和69.4％(P=0.0807),血脂总有效率分别为67.2％和67.7％(P=O.9320),B超脂肪肝影像学检查总有效率分别为51.7％和43.5％(P=0.2879),两组比较,差异均无统计学意义。两组患者中各有1例发生不良反应,高和低剂量组的发生率分别为1.6％和1,6％,表现为口干,未见严重的不良反应。 结论 二氯醋酸二异丙胺可安全有效地用于治疗非酒精性脂肪性肝病。

Objective To investigate the efficacy and safety of diisopropylamine dicbloroacetate in the treatment of nonalcohlic fatty liver diseases (NAFLD). Methods A randomized, double-blind, dose-paralleled control trial was carried out with NAFLD patients. The patients were randomly assigned to 2 groups treated with either a high dosage (120 mg/d) or a low dosage (60 mg/d) of diisopropylamine dichloroacetate for 8 weeks and the efficacy and safety of the drug were examined. Results 127 cases were recruited for the trial, 63 in the high dosage group, and 64 in the low dosage group. No case dropped out in the trial but four cases were eliminated (4/127, 3.1%). The final number in this trial was 123, with 61 in the high dosage group and 62 in the low dosage group. After 8 weeks of treatment, the overall improvement of clinical symptoms in the high dosage and in the low dosage group was 87.8% and 79.6%, respectively (P= 0.2536). ALT normalization was found in 55.7% and 69.4% (P = 0.0807) of the cases in the two groups, serum lipids were lowered in 67.2% and 67.7% (P = 0.9320) and ultrasound grading of the liver alteration severity was lowered in 51.7% and 43.5% (P = 0.2879) in the two groups. The differences found between the two groups were of no statistical significance. One case from each group was found having an adverse drug reaction of dryness of the mouth (1.6%). No severe adverse drug reactions were found. Conclusion Diisopropylamine dichloroacetate could be used as a safe and effective drug in the treatment of nonalcohlic fatty liver diseases.