Analyzing multiple endpoints in clinical trials of pain treatments: IMMPACT recommendations

作者：Turk Dennis C^*; Dworkin Robert H; McDermott Michael P; Bellamy Nicholas; Burke Laurie B; Chandler Julie M; Cleeland Charles S; Cowan Penney; Dimitrova Rozalina; Farrar John T; Hertz Sharon; Heyse Joseph F; Iyengar Smriti; Jadad Alejandro R; Jay Gary W; Jermano John A; Katz Nathaniel P; Manning Donald C; Martin Susan; Max Mitchell B; McGrath Patrick; McQuay Henry J; Quessy Steve; Rappaport Bob A; Revicki Dennis A; Rothman Margaret; Stauffer Joseph W

来源：Pain, 2008, 139(3): 485-493.

DOI：10.1016/j.pain.2008.06.025

• Multiple endpoints
• Multiplicity
• Clinical trials
• Treatment outcomes
• Chronic pain
• Acute pain
• Sampling error
• Type I error

The increasing complexity of randomized clinical trials and the practice of obtaining a wide variety of measurements from Study participants have made the consideration Of Multiple endpoints a critically important issue in the design, analysis, and interpretation of clinical trials. Failure to consider important outcomes call limit the validity and utility of clinical trials; specifying multiple endpoints for the evaluation of treatment efficacy, however, call increase the rate of false positive conclusions about the efficacy of a treatment. We describe the use of multiple endpoints in the design, analysis, and interpretation of pain clinical trials, and review available strategies and methods for addressing multiplicity. To decrease the probability of a Type I error (i.e., the likelihood of obtaining statistically significant results by chance) in pain clinical trials.. the use of gatekeeping procedures and other methods that correct for multiple analyses is recommended when a single primary endpoint does not adequately reflect the overall benefits of treatment. We emphasize the importance of specifying in advance the Outcomes and clinical decision rule that will serve as the basis for determining that a treatment is efficacious and the methods that will be used to control the overall Type I error rate.

• 出版日期2008-10-31