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Common Misconceptions in Randomized Controlled Trials
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摘要: 随机对照试验是评估不同干预措施疗效和常见不良反应的标准设计方案，但我国随机对照试验在研究设计、实施、效应指标选择、数据分析和结果解释等方面仍有提升空间。本文从随机对照试验实施的关键技术、结果展示和结果解读3方面分析了6点常见误区，期望帮助临床研究者明确随机化概念，正确实施随机分配方法和分配方案隐藏、正确理解随机对照试验中的基线平衡问题、合理选择效应指标、正确解读研究结果的统计学意义和临床意义，以生产更多高质量的随机对照试验证据，同时避免被误导。Abstract: A well-conducted randomized controlled trial(RCT) is considered the gold standard for comparing the efficacy and common adverse events of different interventions. However, for China's RCTs, there is still room for the improvement in research design, implementation, selection of effective measures, data analysis, and interpretation of results. In this article, we present six misunderstandings or errors to help clinicians in clarifying the concept of randomization, correctly implementing the process of randomization and concealment of allocation, understanding the baseline imbalance in RCTs, reasonably selecting effective measuers, and correctly interpreting the statistical significance and clinical significance in an effort to produce more RCTs of high-quality and protect clinicians from potentially misleading presentations and interpretations of research findings.
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Key words:
- randomized controlled trial /
- allocation concealment /
- odds ratio /
- risk ratio /
- subgroup analysis
利益冲突 无 -
表 1 抗凝剂A和B治疗房颤患者胃肠出血结果比较
治疗 出血(n) 未出血(n) 比值 风险 高风险人群 抗凝剂A 50 50 50÷50=1 50÷100=0.5 抗凝剂B 20 80 20÷80=0.25 20÷100=0.2 关联强度 OR=1÷0.25=4 RR=0.5÷0.2=2.5 低风险人群 抗凝剂A 5 95 5÷95≈0.05 5÷100=0.05 抗凝剂B 2 98 2÷98≈0.02 2÷100=0.02 关联强度 OR=0.05÷0.02=2.5 RR=0.05÷0.02=2.5 OR:比值比; RR:风险比/相对危险度 
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表 2 RITA-3试验随访1年及亚组分析结果[n(%)]
结局 介入治疗(n=895) 保守治疗(n=915) RR/OR(95% CI) P值 随访1年主要结果 死亡 41(4.6) 36(3.9) 1.16(0.75~1.80) 0.50 死亡和心肌梗死 68(7.6) 76(8.3) 0.91(0.67~1.25) 0.58 亚组分析结果* 死亡 男性 23(4.2) 28(4.8) 0.78(0.44~1.41) 0.031 女性 18(5.1) 8(2.4) 2.43(1.01~5.84) 死亡或心肌梗死 男性 38(7.0) 59(10.1) 0.63(0.41~0.98) 0.007 女性 30(8.6) 17(5.1) 1.79(0.95~3.35) 死亡、心肌梗死或顽固性心绞痛 男性 67(12.3) 125(21.4) 0.48(0.34~0.67) 0.002 女性 55(15.7) 46(13.9) 1.14(0.74~1.76) *亚组分析结果为校正OR和交互P值；RR、OR：同表 1
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