在主要心血管试验中事件速率和效果大小估计的准确性:系统性审查
Christoph B Olivier1, Lasse Struß1, Nathalie Sünnen1
1Department of Cardiology and Angiology, Cardiovascular Clinical Research Center, University Heart Center Freiburg-Bad Krozingen, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
心血管随机临床试验经常高估事件发生率和效果大小. 这种过度估计可能导致研究不足,并阻碍临床研究中的假设测试.
科学领域:
- 临床试验 临床试验
- 心血管医学 心血管医学
- 生物统计学 生物统计学
背景情况:
- 准确估计事件发生率和效应大小对于设计不足的随机临床试验 (RCT) 至关重要.
- 过度估计这些参数可能会导致试验不足,影响检测显著干预效应的能力.
研究的目的:
- 评估当代心血管RCT中事件率和效果大小的公布估计的准确性.
- 确定过高估计的程度及其对试验结果的潜在影响.
主要方法:
- 从2010年至2019年,在MEDLINE系统搜索在领先期刊 (NEJM,JAMA,Lancet) 上发表的多中心心血管RCT.
- 从研究方案或出版物中提取数据,然后使用随机效应模型进行聚合.
- 通过比较观察到与假设的事件率和效果大小来分析准确性,使用线性回归来评估与试验特征的关联.
主要成果:
- 对344个RCT的分析显示,事件发生率的中位数高估了12.3%,影响大小高估了23.1%.
- 超过61%的试验高估了预期事件率,82.1%高估了效应大小.
- 准确的事件率估计与更高的反驳零假设的可能性有关.
结论:
- 当代心血管RCT经常高估主终点事件率和干预效应大小.
- 这种系统的高估可能会损害充分测试试验假设的能力,并可能导致研究不足.
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