以COPD临床试验为示范的辨证论治疗效结局测量与评价关键技术研究

中医疗效评价目的是验证干预措施与结局间因果关系，结局测量与评价是临床研究的关键环节。目前，缺乏对中医证候动态转归及量化测评、缺乏基于生存质量与生存时间联合评价预后研究、缺乏结局的最小临床差异值（MCID）评价、缺乏中医疾病负担科学评估有效方法技术，影响中医临床研究的水平和质量及可信度。本研究以COPD循证临床研究为示范，拟开展以下研究：一是研制证候评价量表客观精确评价证候疗效；二是运用生存质量和生存时间联合模型评价中医干预的远后效应；三是采用MCID方法评估急性加重次数等结局指标客观变化；四是运用伤残调整生命年和质量调整生命年评估COPD疾病负担和中医卫生经济学优势。通过循证研究，客观阐释中医证候变化特色优势及远后效应优势，客观评价结局指标变化的临床意义和疾病负担。建立适宜中医药自身特点、科学规范、可行的结局测量与评价关键技术，为中医临床疗效评价方法学研究提供可借鉴的思路与方法。

The purpose of curative effect evaluation for traditional Chinese medicine (TCM) is to verify the cause and effect between intervention and outcome. The outcome measurement and evaluation are key links of curative effect evaluation of TCM. At present, accurate evaluation for TCM ZHENG, prognosis evaluation researches based on the combination of quality of life and survival time, evaluation methods of minimal clinical important difference (MCID), as well as effective methods and techniques of scientific assessment for burden of disease in TCM are all deficient. These deficiencies will affect the level, quality and credibility of TCM clinical researches. This study based on evidence-based clinical trials of chronic obstructive pulmonary disease (COPD) will include several major sections. First of all, ZHENG scale of COPD will be developed to measure dynamic ZHENG changes objectively and accurately. Secondly, combined model of quality of life and survival time will be used to evaluate long-term effects of TCM. Thirdly, MCID methods will adopted to analyze the objective changes of frequency of acute exacerbation of COPD, etc. Finally, disability adjusted life year and quality adjusted life year will be used to evaluate burden of disease and health economics advantages of TCM for COPD. Through the study, we will objectively interpret TCM ZHENG changes and long-term effects, and evaluate the clinical significance of outcome changes and burden of disease. Key techniques of outcome measure and evaluation suitable for TCM will be established to provide ideas and methods for methodology research on clinical efficacy evaluation of TCM.