模糊集群随机试验和观察性研究:用于亚抽样,缺失和少数独立单元的两阶段TMLE
Joshua R Nugent1, Carina Marquez2, Edwin D Charlebois3
1Division of Research, Kaiser Permanente Northern California, 2000 Broadway, Oakland, CA 94612, USA.
Biostatistics (Oxford, England)
|August 2, 2023
概括
集群随机试验 (CRT) 面临参与者采样和缺失数据的挑战. 新的方法准确估计了干预效应,显示结核病感染率下降了27%.
科学领域:
- 生物统计学 生物统计学
- 流行病学 流行病学
- 临床试验 临床试验
背景情况:
- 集群随机试验 (CRT) 通常涉及大量参与者,但由于资源限制,结果评估有限.
- 亚样本和CRT中缺少的数据可能会导致对干预效应的偏见估计,如果样本中的个人与一般集群人群不同.
- 在CRT中很少有集群减少了统计能力,并引起了对有限样本性能的担忧.
研究的目的:
- 开发和应用统计方法来应对CRT的挑战,特别是亚抽样,缺失的结果数据和集群内部依赖.
- 准确估计SEARCH-TB试验中的干预效应,该试验旨在减少发生的结核病感染.
主要方法:
- 扩展的两阶段针对性最低损失的估计,以处理三种缺失来源:亚抽样,基线状态测量和最终状态测量.
- 对集群中的子单元的条件独立性进行了批判性评估,以提高精度和功率.
- 应用方法用于SEARCH-TB集群随机试验数据.
主要成果:
- 不切实际的假设导致结核病感染增加了约18% (RR=1.18,95% CI:0.85-1.63).
- 考虑到抽样,失踪和社区内依赖的方法显示结核病发病率下降了27% (RR=0.73,95% CI:0.57-0.92).
结论:
- 准确的统计方法对于在复杂采样和缺少数据的CRT中对干预效应的公正估计至关重要.
- 在SEARCH-TB试验中的干预表明,当考虑试验复杂性时,结核病感染发生率显著降低.
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