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Efficient study design to estimate population means with multiple measurement instruments
Statistics in Medicine ( IF 2 ) Pub Date : 2021-05-20 , DOI: 10.1002/sim.9032
Michal Bitan 1 , Malka Gorfine 1 , Laura Rosen 2 , David M Steinberg 1
Affiliation  

Outcomes from studies assessing exposure often use multiple measurements. In previous work, using a model first proposed by Buonoccorsi (1991), we showed that combining direct (eg, biomarkers) and indirect (eg, self-report) measurements provides a more accurate picture of true exposure than estimates obtained when using a single type of measurement. In this article, we propose a tool for efficient design of studies that include both direct and indirect measurements of a relevant outcome. Based on data from a pilot or preliminary study, the tool, which is available online as a shiny app at https://michalbitan.shinyapps.io/shinyApp/, can be used to compute: (1) the sample size required for a statistical power analysis, while optimizing the percent of participants who should provide direct measures of exposure (biomarkers) in addition to the indirect (self-report) measures provided by all participants; (2) the ideal number of replicates; and (3) the allocation of resources to intervention and control arms. In addition we show how to examine the sensitivity of results to underlying assumptions. We illustrate our analysis using studies of tobacco smoke exposure and nutrition. In these examples, a near-optimal allocation of the resources can be found even if the assumptions are not precise.

中文翻译:

使用多种测量仪器估计总体均值的有效研究设计

评估暴露的研究结果通常使用多次测量。在之前的工作中,使用 Buonoccorsi (1991) 首次提出的模型,我们表明,结合直接(例如,生物标志物)和间接(例如,自我报告)测量提供了比使用单一方法获得的估计值更准确的真实暴露情况。测量类型。在本文中,我们提出了一种有效设计研究的工具,其中包括对相关结果的直接和间接测量。根据试点或初步研究的数据,该工具可作为闪亮的应用程序在线获取,网址为 https://michalbitan.shinyapps.io/shinyApp/,可用于计算:(1)统计功效分析,同时优化除了所有参与者提供的间接(自我报告)措施外,还应提供直接暴露措施(生物标志物)的参与者百分比;(2) 理想的重复次数;(3) 为干预和控制武器分配资源。此外,我们还展示了如何检查结果对基本假设的敏感性。我们使用烟草烟雾暴露和营养研究来说明我们的分析。在这些示例中,即使假设不准确,也可以找到接近最佳的资源分配。我们使用烟草烟雾暴露和营养研究来说明我们的分析。在这些示例中,即使假设不准确,也可以找到接近最佳的资源分配。我们使用烟草烟雾暴露和营养研究来说明我们的分析。在这些示例中,即使假设不准确,也可以找到接近最佳的资源分配。
更新日期:2021-07-19
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