将样本大小再计算规则的条件性能得分扩展到对二进制终点的设置
Björn Bokelmann1, Geraldine Rauch2,3, Jan Meis4
1Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Charitéplatz 1, Berlin, 10117, Germany. bjoern.bokelmann@charite.de.
BMC medical research methodology
|January 19, 2024
概括
这项研究将适应性临床试验的条件性能得分扩展到二元终点. 新方法准确评估试验性能,有助于调整样本大小,以更好地设计试验.
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
- 统计建模 统计建模
背景情况:
- 临床试验中的样本大小计算依赖于参数假设,引入不确定性和不足或超大尺寸的风险.
- 适应性设计提供了一个解决方案,允许在中间分析期间调整样本大小.
- 适应性设计的现有方法,如条件性能得分,主要是为正常分布的终点开发的.
研究的目的:
- 将条件性绩效评分方法扩展到具有二进制终点的临床试验.
- 评估这个扩展分数对二元结果的性能和适用性.
主要方法:
- 开发了针对二进制终点量身定制的条件性能得分的一维得分参数化.
- 进行模拟研究,以评估拟议方法的操作特性.
- 演示了扩展分数的实际应用.
主要成果:
- 条件性能得分理论可以直接扩展到二进制终点,而无需修改.
- 评分结果以单个分布参数表示.
- 得到一个统一的效果指标,包括比例差异和对照组比例.
结论:
- 条件性能得分成功地扩展到二进制终点,扩大其在临床试验设计中的适用性.
- 这项研究提供了一种实用工具,用于指导选择适应性设计,并对二进制结果进行样本大小重新计算.
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