Establishing an evaluation mode with multiple primary outcomes based on combination of diseases and symptoms in TCM clinical trials

摘要

The development of a scientific evaluation tool is a prerequisite to reflect the efficacy of traditional Chinese medicine (TCM). At present, separate analysis of each outcome is the most commonly used method in randomized controlled trials (RCTs) of TCM, while this method has been reported to have some limitations. Combination evaluation of multiple primary outcomes including diseases and symptoms can reflect the efficacy of TCM comprehensively and objectively. Now, several multivariate statistical methods have been proposed to analyze RCTs with multiple clinical outcomes. In this article, we highlighted the two commonly used methods, global statistical test (GST) methodology and alpha-adjustment procedures, and introduced an evaluation mode. First, we used one stage adaptive adjustment strategy to estimate the correlation among multiple primary outcomes in blindness, which can resolve the inappropriate correlation estimation among multiple primary outcomes, then we introduced an adaptation of the Bonferroni procedure which can account for correlated data to calculate the alpha of individual outcome. GST using O’Brien ranking procedure and the corresponding global treatment effect (GTE) measure were used to assess the treatment’s global impact. We will conduct a randomized, double-blind, controlled trial with compound Danshen dripping pills for stable angina to explore establishing the evaluation mode. We hope that the introduction of this approach can provide methodological aid for the assessment of therapeutic effect of TCM.