【作者】 万斌；

【导师】 罗才贵；

【作者基本信息】 成都中医药大学 ， 中西医结合临床， 2009， 博士

【摘要】 目的：探讨非劣效／等效性界值在中药临床再评价临床试验中的作用。方法：(1)通过理论研究和临床试验,探讨目前中药临床再评价的目的、意义及方法。(2)对非劣效／等效性界值在中药临床再评价临床试验中的作用、确定原则及应用方法进行分析研究。结果：(1)目前中药临床再评价存在的不足之处：试验样本量较小,试验缺乏随机对照,统计学检验存在误差,评价标准不规范,临床定位不清,新的方法技术在中药临床再评价中的应用太少,中药数据库不完善,中药重点剂型再评价未引起足够重视。(2)目前从文献检索的结果来看,几乎所有的非劣效／等效性临床试验都没有规定非劣效／等效性界值的取值,造成了其结论只具有统计学意义,未考虑临床意义。(3)为了临床试验结果更为可信,具有实际临床意义,必须在试验设计的时候,考虑到非劣效／等效性界值的取值。(4)根据设计方案所确定的非劣效性界值为0.1,按证候疗效进行非劣效性检验,FAS(u=5.597,P=0.000)和PP(u=6.388,P=0.000),试验组证候疗效非劣效于对照组；对贫血有效率进行非劣效性检验,FAS(u=4.993,P=0.000)和PP(u=5.317,P=0.000),试验组贫血有效率非劣效于对照组：对月经不调有效率进行非劣效性检验,FAS(u=3.979,P=0.000)和PP(u=4.888,P=0.000),试验组月经不调有效率非劣效于对照组。结论：(1)中药临床再评价的方法中最重要的是前瞻性多中心随机对照临床试验。(2)基于显著性检验得到的的药物疗效统计学结果未考虑临床意义。(3)非劣效／等效性界值的应用是统计学结果是否具有临床实际意义的关键。(4)非劣效／等效性界值的具体取值,需要慎重的确定,必须在制定试验方案时予以提前确定。(5)非劣效／等效性界值的最终确定,必须紧密结合试验病证的临床特征,由相关学科临床专家和统计学家共同认可。更多还原

【Abstract】 PurposeTo explore the role of non-inferiority/equivalence value in re-evaluation clinical trials for traditional Chinese medicine.Methods(1) Through theoretical research and clinical trials to explore the purpose, the clinical significance and evaluation of the current re-evaluation of traditional Chinese medicine.(2) To analyze the function, the determining principles and the application of non-inferiority/equivalence value in re-evaluation clinical trials for traditional Chinese medicine.Results(1)The current clinical re-evaluation of traditional Chinese medicine had some deficiencies:the smaller volume of test samples, the lack of randomized controlled trial, the existence of statistical error, non-standard evaluation criteria, unclear clinical orientation, limited application of the new methods and techniques in clinical re-evaluation of traditional Chinese medicine, little attention to clinical re-evaluation of important Chinese herbal pharmaceutics.(2)At present, literature research released that almost all of the non-inferiority /equivalence of clinical trials had not applied non-inferiority/equivalence cutoff values, which lead to the conclusions only have statistical significance. But the clinical significances did not take into account.(3)It is necessary to consider the non-inferiority/equivalence value in trials’ designs in order to get credible results with more clinical significances.(4)According to the program’s design, the non-inferiority of the sector value was 0.1. Based on the syndrome effects of non-inferiority test, FAS (u=5.597, P=0.000) and PP (u=6.388, P=0.000), the experimental group had non-inferiority efficacy compared with the control group. For anemia efficient non-inferiority test, FAS (u=4.993, P=0.000) and PP (u=5.317, P=0.000), the effective rate in experimental group had non-inferiority to that of the control group. For irregular menstruation efficient non-inferiority test, FAS (u=3979, P=0.000) and PP (u=4.888, P=0.000), the effective rate in experimental group had non-inferiority to that of the control group.Conclusion(1)The most important method of clinical re-evaluation of traditional Chinese medicine is forward-looking, multi-center randomized and controlled clinical trials.(2)The clinical significances had not been take into account in statistical results of herbals efficacy based on the significance test.(3)The application of Non-inferiority/equivalence was a vital factor to decide whether the statistical results have clinical meaning.(4)Circumspect consideration should be needed to determine the specific cutoff value of non-inferiority/equivalence. The values must be decided before trials design.(5)To determine the ultimate value of non-inferiority/equivalence cutoff, clinical experts of relevant disciplines must draw a common recognition with statisticians according to the clinical features of diseases.更多还原