用GEE对集群随机试验中计数结果的比例进行样本大小估计
Jijia Wang1, Song Zhang2, Chul Ahn2
1Department of Applied Clinical Research, UT Southwestern Medical Center, Dallas, TX.
概括
本研究提出了一个新的样本大小公式,用于集群随机试验,集群大小各不相同. 该方法使用通用估计方程,通过发病率比率来提高评估干预有效性的能力.
科学领域:
- 流行病学 流行病学
- 生物统计学 生物统计学
- 临床试验 临床试验
背景情况:
- 计数结果在集群随机试验 (CRT) 中很常见,特别是在流行病学中.
- 发病率的比率是干预有效性的关键指标,但不同集群大小可能导致研究不足.
- 现有的样本大小方法通常假设恒定的集群大小,这是不现实的.
研究的目的:
- 为CRT开发一个灵活的样本大小方法,以考虑不同的集群大小.
- 为测试两个发病率的比率提供一个封闭形式的样本大小公式.
- 提高CRT中样本大小估计的准确性和功率,特别是在疫苗疗效试验中.
主要方法:
- 提出了一种基于通用估计方程 (GEE) 方法的样本大小方法.
- 开发了一个封闭形式的样本大小公式,容纳不平衡的随机化和可变的集群大小.
- 进行模拟以评估拟议方法的性能.
主要成果:
- 提出的基于GEE的样本大小公式有效地处理不同集群大小和不平衡的随机化.
- 模拟表明,与恒定集群大小假设相比,该方法能够保持研究功率.
- 该公式为设计更强大的CRT提供了一个实用的工具.
结论:
- 开发的样本大小方法为具有可变集群大小的CRT提供了显著的改进.
- 这种方法提高了流行病学和疫苗疗效研究中的统计功率计算的可靠性.
- 准确的样本大小确定对于CRT的成功执行和解释至关重要.
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