在个性化随机对照 (PRACTical) 试验中确定样本大小
Rebecca M Turner1, Kim May Lee2, A Sarah Walker1
1MRC Clinical Trials Unit at University College London, London, UK.
Statistics in medicine
|July 9, 2024
概括
这项研究引入了个性化随机对照试验 (PRACTical) 中样本大小确定的新方法. 这些方法有助于确定个别患者最有效的治疗方法,减少复杂的临床环境中的不良结果.
科学领域:
- 临床试验 临床试验
- 生物统计学 生物统计学
- 个性化医疗是个性化的医疗.
背景情况:
- 在复杂的临床环境中,通常缺乏标准护理,因此需要个性化治疗方法.
- 个性化随机对照试验 (PRACTical) 只在适当的治疗中随机选择患者.
- 现有的样本大小计算方法不适用于PRACTical设计.
研究的目的:
- 提出用于PRACTICAL设计中确定样本大小的新方法.
- 量化使用试验结果用于个性化治疗选择的好处.
- 在具有多种治疗选择的环境中,为临床决策和政策制定提供信息.
主要方法:
- 通过评估从不同规模的试验中获得的信息来推导样本大小.
- 通过选择排名最高的治疗方法来量化减少不良结果的数量.
- 使用模拟来评估性能指标,包括治疗有效性和患者结果.
主要成果:
- 提出的方法为实践试验中的样本大小计算提供了一个框架.
- 模拟证明了通过使用个性化治疗排名来减少不良结果的潜力.
- 这种方法应用于新生儿败血症抗生素治疗方案试验 (NeoSep1).
结论:
- 开发的方法为个性化临床试验中样本大小的确定提供了相关的方法.
- 这有助于明智的临床决策,通过根据个体患者的适宜性对治疗进行排名.
- 这些发现支持在非标准化护理的复杂治疗领域使用PRACTical设计.
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