Pocock S J
Medical Statistics Unit, London School of Hygiene & Tropical Medicine, United Kingdom.
Control Clin Trials. 1997 Dec;18(6):530-45; discussion 546-9. doi: 10.1016/s0197-2456(97)00008-1.
This article tackles both practical and statistical issues in the handling of multiple outcomes in clinical trials, with relevance to trial design, analysis, and reporting. Specific topics illustrated by examples include: the advantage of prespecifying priorities amongst outcomes and analyses, corrections for multiple significance testing and their limited value, problems with adverse event data, the use of a single global test of significance for clinically related outcomes, the use of a combined outcome for clinical event data, and the value of exploring interrelationships amongst outcomes. The problems in handling multiple outcomes are enhanced by trials being too small, dichotomous attitudes (is the trial "positive" or not?), obsession with p-values, and the manipulative instincts of human nature. While predeclarations of priorities in analysis and reporting of multiple outcomes are important in suppressing distortive claims, it would be unfortunate if too inflexible an approach suppressed unpredictable findings from being seriously considered.
本文探讨了临床试验中处理多个结果时的实际问题和统计学问题，涉及试验设计、分析和报告。文中通过实例说明的具体主题包括：预先确定结果和分析的优先级的优势、多重显著性检验的校正及其有限价值、不良事件数据的问题、对临床相关结果使用单一全局显著性检验、对临床事件数据使用综合结果以及探索结果之间相互关系的价值。试验规模过小、二分法态度（试验是否“阳性”？）、对p值的痴迷以及人性的操纵本能加剧了处理多个结果时的问题。虽然在分析和报告多个结果时预先声明优先级对于抑制歪曲性主张很重要，但如果过于僵化的方法抑制了对不可预测发现的认真考虑，那将是不幸的。
