随机对照试验中多个主要结局是否得到恰当分析？一项综述。

Are multiple primary outcomes analysed appropriately in randomised controlled trials? A review.

作者信息

Vickerstaff Victoria, Ambler Gareth, King Michael, Nazareth Irwin, Omar Rumana Z

机构信息

Division of Psychiatry, University College London, 6th Floor, Maple House, 149 Tottenham Court Road, London W1T 7NF, UK; Department of Statistical Science, University College London, Gower Street, London WC1E 6BT, UK; The Research Department of Primary Care and Population Health, University College London, Rowland Hill Street, London NW3 2PF, UK.

Department of Statistical Science, University College London, Gower Street, London WC1E 6BT, UK.

出版信息

Contemp Clin Trials. 2015 Nov;45(Pt A):8-12. doi: 10.1016/j.cct.2015.07.016. Epub 2015 Jul 26.

DOI:10.1016/j.cct.2015.07.016

PMID:26215934

Abstract

OBJECTIVES

To review how multiple primary outcomes are currently considered in the analysis of randomised controlled trials. We briefly describe the methods available to safeguard the inferences and to raise awareness of the potential problems caused by multiple outcomes.

METHODS/DESIGN: We reviewed randomised controlled trials (RCTs) in neurology and psychiatry disease areas, as these frequently analyse multiple outcomes. We reviewed all published RCTs from July 2011 to June 2014 inclusive in the following high impact journals: The New England Journal of Medicine, The Lancet, The American Journal of Psychiatry, JAMA Psychiatry, The Lancet Neurology and Neurology. We examined the information presented in the abstract and the methods used for sample size calculation and statistical analysis. We recorded the number of primary outcomes, the methods used to account for multiple primary outcomes, the number of outcomes discussed in the abstract and the number of outcomes used in the sample size calculation.

RESULTS

Of the 209 RCTs that we identified, 60 (29%) analysed multiple primary outcomes. Of these, 45 (75%) did not adjust for multiplicity in their analyses. Had multiplicity been addressed, some of the trial conclusions would have changed. Of the 15 (25%) trials which accounted for multiplicity, Bonferroni's correction was the most commonly used method.

CONCLUSIONS

Our review shows that trials with multiple primary outcomes are common. However, appropriate steps are not usually taken in most of the analyses to safeguard the inferences against multiplicity. Authors should state their chosen primary outcomes clearly and justify their methods of analysis.

摘要

目的

回顾目前在随机对照试验分析中对多个主要结局的考量方式。我们简要描述可用于保障推断以及提高对多个结局所引发潜在问题认识的方法。

方法/设计：我们回顾了神经病学和精神病学疾病领域的随机对照试验（RCT），因为这些领域经常分析多个结局。我们回顾了2011年7月至2014年6月期间（含）在以下高影响力期刊上发表的所有RCT：《新英格兰医学杂志》《柳叶刀》《美国精神病学杂志》《美国医学会杂志·精神病学》《柳叶刀·神经病学》和《神经病学》。我们检查了摘要中呈现的信息以及样本量计算和统计分析所使用的方法。我们记录了主要结局的数量、用于处理多个主要结局的方法、摘要中讨论的结局数量以及样本量计算中使用的结局数量。