平台试验中的特征,设计和统计方法:系统性审查
Clément R Massonnaud1, Christof Manuel Schönenberger2, Malena Chiaborelli2
1Université Paris Cité, Inserm, IAME, F-75018 Paris, France; AP-HP, Hôpital Bichat, Département d'Épidémiologie, Biostatistique et Recherche Clinique, F-75018 Paris, France.
Journal of clinical epidemiology
|May 11, 2025
概括
平台试验 (PT) 在临床研究中越来越受欢迎,采用各种方法. 提高复杂的贝叶斯 PTs 的透明度对于可靠性和接受性至关重要.
科学领域:
- 临床研究 临床研究
- 生物统计学 生物统计学
- 流行病学 流行病学
背景情况:
- 平台试验 (PTs) 提供创新和灵活的方法,在临床研究中获得了引力.
- 这项研究旨在描述 PT 的不断变化的景观.
研究的目的:
- 确定平台试验中使用的特征,方法方法和统计实践.
- 分析PT启动和设计的趋势.
主要方法:
- 通过对试验登记册和文献数据库的系统搜索,确定了189个符合条件的平台试验.
- 数据提取的重点是试验状态,设计特征,统计方法和报告质量.
- 符合条件的PT是随机对照试验,在单一人群中进行多次干预,允许手臂灵活性.
主要成果:
- 传染病和瘤学是PT的主要重点领域,其中大量与COVID-19相关.
- 贝叶斯设计很普遍,有23%的试验缺乏预先确定的样本大小,通常被指定为永久试验.
- 频率主义PT更常见地预先确定了临时分析,而贝叶斯主义PT广泛使用模拟,尽管代码共享是最小的.
结论:
- 平台试验仍然是一个受欢迎和多样化的研究模式.
- 提高透明度和报告,特别是对于复杂的贝叶斯设计,对于确保平台试验的可靠性和接受性至关重要.
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