基于模拟的样本大小计算,对具有竞争风险的反复事件进行边际比例平均模型的计算
Julie Funch Furberg1,2, Per Kragh Andersen1, Thomas Scheike1
1Section of Biostatistics, University of Copenhagen, Copenhagen, Denmark.
Pharmaceutical statistics
|March 20, 2024
概括
本研究介绍了一种模拟方法,用于在临床试验中进行功率计算,这些临床试验具有反复事件和竞争风险. 该方法有助于确定未来试验的样本大小,确保可靠的统计分析.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 流行病学 流行病学
背景情况:
- 复发性事件 (例如,心力衰竭住院) 是临床试验中常见的结果.
- 当死亡率显著时,复发性和竞争性终端事件都需要仔细的统计考虑.
- 准确的样本大小确定对于设计具有重复事件终点和竞争风险的未来试验至关重要.
研究的目的:
- 引入基于模拟的方法,用于在随机对照试验 (RCT) 中进行功率估计,这些试验具有重复事件和竞争风险.
- 为设计未来RCT提供一个样本大小计算方法,这对于设计未来RCT至关重要.
- 为了促进可靠的统计分析,当经常性和终端事件都感兴趣时.
主要方法:
- 建议采用基于模拟的方法来估计功率.
- 该方法使用比例平均模型用于反复发生的事件和比例危险模型用于终端事件.
- 该程序是灵活的,依赖于易于指定的边际数量,并包括对强度的敏感性分析.
主要成果:
- 模拟程序是详细的,概述了功率估计的用户规格.
- 灵敏度分析表明,尽管缺乏某种依赖性,但拟议方法的功率是强大的.
- 来自LEADER试验的数据为模拟目的的数据生成提供了信息.
结论:
- 开发的模拟方法提供了一种灵活和强大的方法,用于在具有反复事件和竞争风险的试验中进行功率计算.
- 这些发现支持使用反复事件方法而不是第一次事件方法,以获得潜在更大的功率.
- 这种方法对于设计解决复杂事件结构的未来临床试验至关重要.
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