熄风止动片与安慰剂对照治疗小儿抽动障碍肝风内动挟痰证的临床研究

Xifeng Zhidong Tablet and the Placebo Control Treatment of Tic Disorder Children Patients of Internal Disturbance of Gan-wind with Phlegm Syndrome:a Clinical Study

目的评价熄风止动片治疗小儿抽动障碍肝风内动挟痰证的有效性和安全性。方法采用分层区组随机、双盲、安慰剂平行对照、多中心、优效性检验的方法。160例受试者随机分为试验组与对照组,每组80例。试验组应用熄风止动片治疗,对照组采用安慰剂治疗。疗程4周。有效性指标为:主要指标:耶鲁综合抽动严重程度量表(Yale global tic severity scale,YGTSS)抽动积分;次要指标:社会功能受损改善情况、疾病疗效、中医证候单项指标及中医证候疗效;安全性指标包括生命体征,血、尿、大便常规,肝、肾功能,心电图以及临床不良事件。结果主要指标:YGTSS抽动积分:试验组治疗前后分别为(22.10±6.38)分和(11.34±6.58)分;对照组治疗前后分别为(22.65±6.70)分和(16.82±6.53)分,与本组治疗前比较,治疗后两组YGTSS抽动积分均下降,差异有统计学意义(P<0.01),且治疗后试验组较对照组下降更为显著(P<0.05)。次要指标:疾病疗效:试验组与对照组总有效率分别为83.54%、34.18%,组间比较差异有统计学意义(P<0.05);社会功能受损改善情况:治疗后试验组无损害、极轻度、轻度、中度、明显的等级分别有20、38、16、3、1例,对照组为1、24、45、7、0例,试验组对社会功能受损改善情况优于对照组(P<0.05);中医证候疗效:试验组与对照组总有效率分别为87.34%、64.56%,试验组优于对照组(P<0.05);单项证候改善情况:运动性抽动、烦躁易怒、多梦、异常舌质、舌苔及脉象的消失率,试验组分别为78.67%、34.72%、62.26%、34.62%、58.97%、39.74%,对照组分别为34.67%、13.11%、21.82%、15.58%、25.97%、19.48,试验组高于对照组,差异具有统计学意义(P<0.05)。试验中,共发现5例不良事件,试验组和对照组的发生率分别为3.75%和2.53%。结论熄风止动片治疗4周,能够明显减少YGTSS抽动积分,改善社会功能受损程度和中医证候,且未发现不良反应。

Objective To assess the efficacy and safety of Xifeng Zhidong Tablet （XZT） in treating tic disorder children patients of internal disturbance of Gan-wind with phlegm syndrome （IDGWPS）.Methods A stratified randomized, double-blinded, parallel control of placebo, multi-center trial was conducted in 160 subjects from 5 hospitals in China. They were randomly assigned to 2 groups, the test group and the control group, 80 in each group. Those in the test group were treated with xz＇r, while those in the control group were treated with placebos. The therapeutic course was 4 weeks for all. The effectiveness in- dicators covered main indicators and secondary indicators. Yale global tic severity scale （YGTSS） was taken as the main indicators. The amelioration of social function impairment, efficacy, single index of Chi- nese medical syndromes, Chinese medical syndrome efficacy as well as disappearance rate of single Chi- nese medical symptoms were evaluated as secondary indicators. The safety indicators included clinical ad- verse events, vital signs, blood/urine/stool routines, renal and liver functions, and electrocardiogram （ECG）. Results As for main indicators, the score of YGTSS decreased from 22.10 ＋6.38 to 11.34 ＋6.58 in the test group, while it decreased from 22.65 ＋6.70 to 16.82 ＋6.53 in the control group, showing statisti- cal difference when compared with the same group before treatment （P 〈0.01 ）. Besides, the decrement was more significant in the test group after treatment （P 〈0.05）. As for secondary indicators, the total ef- fective rate was 83.54% in the test group and 34. 18% in the controlled group, showing statistical difference between the two groups （P 〈0.05）. As for social function impairment, 20, 38, 16, 3, 1 case（s） in the test group were ranked as normal, minimal, mild, moderate, obvious degree, while 1, 24, 45, 7, and 0 case（s） in the control group were ranked as normal, minimal, mild, moderate, obvious degree. Better effect was obtained in the test group （P 〈0.05）. As for Chinese medical syndrome efficacy, it was 87.34% in the test group and 64.56% in the control group （P 〈0.05）. As for single index of Chinese medical syn- dromes, the disappearance rate of motor tics, irritability, dreaminess, abnormal tongue proper ,abnormal tongue fur, and abnormal tongue pulse condition was 78.67%, 34.72%, 62.26%, 34.62%, 58.97%, and 39.74%, respectively in the test group, while they were 34.67%, 13.11%, 21.82%, 15.58%, 25.97%, and 19.48%, respectively in the control group. Better results were shown in the test group （P 〈0.05）. Totally 5 adverse events occurred. The incidence of adverse events was 3.75% in the test group and 2.53% in the control group. Conclusions After 4 weeks of XZT treatment, the integral of YGTSS could be obviously reduced, the degree of social function impairment ameliorated, and Chinese medical syndromes improved. In addition, no adverse reaction occurred in this study.