中医药临床疗效评价中结局指标的选择与应用

Choice and Application of the Outcome Indexes for Clinical Effectiveness Assessment of Traditional Chinese Medicine

论述中医药临床疗效评价中结局评价指标的选择和应用。总结了我国临床试验中结局指标选择的现状及与国外的差距,阐述了主要结局指标与次要结局指标的联系与区别,介绍了临床试验中结局指标的选择方法。指出只有选择对患者有直接影响的主要结局指标才能够证实干预措施的真正疗效,以实验室检验为主的生物学指标只是一种次要结局指标,只有在主要结局指标不可行、且次要结局指标被证实与主要结局指标具有因果关系的情况下才能够作为代替主要结局指标的替代指标使用;并提出了临床研究中亟需注意的三点事项:①应尽快引进与完善功能与生存质量评价;②寻找能发扬中医药优势的证候相关指标;③分清主要结局指标与次要结局指标的临床意义,严格进行结论的推导。

The choice of effectiveness indexes (outcome assessment indexes) is one of the key points in clinical trials. Only the primary outcomes which affects the patients directly can prove the real effectiveness of the interventions. Biological indexes from laboratory are called secondary outcomes. Only when the primary outcome indexes are not feasible and the secondary ones are proved to have causal relations with them, can the latter be the substitute for the former. In this paper, the status of choosing outcome indexes in clinical trials and the gap between China and other countries were analysed. Relations and the differences between the primary outcomes and the secondary ones were introduced and how to choose the outcome indexes in clinical trials was also discussed. At present, in the clinical trials of the traditional Chinese medicine (TCM) , special attention should be paid to the following: (1) introducing the functional assessment and quality of life assessment, (2) searching for syndrome - related indexes advantageous to the development of TCM, (3)dis-tinguishing the clinical meanings of the primary outcome indexes and the secondary ones and deducing results strictly.