对于二进制终点的最佳徒劳性停止边界
Michaela Maria Freitag1, Xieran Li2,3, Geraldine Rauch2,4,5
1Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Charitéplatz 1, 10117, Berlin, Germany. michaela-maria.freitag@charite.de.
BMC medical research methodology
|March 28, 2024
概括
在II期临床试验中优化无用性边界通过减少错误的无用性停止来改善决策. 这种新方法为西蒙的设计提供了灵活的替代方案,提高了研究效率.
科学领域:
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
- 药学研究 药学研究
背景情况:
- 在临床试验中节约资源. 组序列设计与徒劳停止节约资源.
- 西蒙的设计在单臂II期研究中对于二进制终点是常见的,但在虚假无用声明和样本大小效率方面存在局限性.
- 优化徒劳界限对于学习表现至关重要,影响停止决策的权力和准确性.
研究的目的:
- 在单臂II期研究中,将徒劳边界的最佳性标准扩展到二元终点.
- 介绍一个算法来导出优化的无用性边界.
- 将这些优化边界的性能与已建立的西蒙设计及其修改进行比较.
主要方法:
- 开发了一种算法,以根据Schueler等的标准得出优化的徒劳边界.
- 扩展了对单臂II期研究的二进制终点的方法.
- 与Simon的最佳和最小设计以及Kim等人的最佳和最小设计比较操作特性 (例如,样本大小,功率,徒劳性错误率). 的修改设计.
主要成果:
- 优化的徒劳边界最大化正确的徒劳停止,同时限制功耗损失和错误的徒劳停止.
- 操作特性,包括最大和预期的样本大小,与西蒙的设计相比或优于它.
- 拟议的边界没有约束力,与西蒙的约束性规则相比,提供了灵活性.
结论:
- 徒劳性边界选择和中间分析时间显著影响研究设计性能.
- 拟议的方法提供了一个灵活的,不具约束力的替代方案,西蒙的设计的第二阶段研究.
- 优化的徒劳界限将错误地停止徒劳的可能性降至最低,并避免西蒙设计的常见缺点.
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