Identifying predictors of resilience to stressors in single-arm studies of pre-post change.

IF 1.8 3区 数学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY Biostatistics Pub Date : 2024-10-01 DOI:10.1093/biostatistics/kxad018
Ravi Varadhan, Jiafeng Zhu, Karen Bandeen-Roche
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Abstract

Many older adults experience a major stressor at some point in their lives. The ability to recover well after a major stressor is known as resilience. An important goal of geriatric research is to identify factors that influence resilience to stressors. Studies of resilience in older adults are typically conducted with a single-arm where everyone experiences the stressor. The simplistic approach of regressing change versus baseline yields biased estimates due to mathematical coupling and regression to the mean (RTM). We develop a method to correct the bias. We extend the method to include covariates. Our approach considers a counterfactual control group and involves sensitivity analyses to evaluate different settings of control group parameters. Only minimal distributional assumptions are required. Simulation studies demonstrate the validity of the method. We illustrate the method using a large, registry of older adults (N  =7239) who underwent total knee replacement (TKR). We demonstrate how external data can be utilized to constrain the sensitivity analysis. Naive analyses implicated several treatment effect modifiers including baseline function, age, body-mass index (BMI), gender, number of comorbidities, income, and race. Corrected analysis revealed that baseline (pre-stressor) function was not strongly linked to recovery after TKR and among the covariates, only age and number of comorbidities were consistently and negatively associated with post-stressor recovery in all functional domains. Correction of mathematical coupling and RTM is necessary for drawing valid inferences regarding the effect of covariates and baseline status on pre-post change. Our method provides a simple estimator to this end.

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在前后变化的单臂研究中确定对压力的恢复能力的预测因素。
许多老年人在一生中都会遇到重大压力。在经历重大压力后能够很好地恢复的能力被称为恢复力。老年医学研究的一个重要目标就是找出影响压力恢复能力的因素。对老年人复原力的研究通常采用单臂法,即每个人都经历压力源。由于数学耦合和均值回归(RTM)的原因,将变化与基线进行回归的简单方法会产生有偏差的估计值。我们开发了一种方法来纠正这种偏差。我们将该方法扩展到包括协变量。我们的方法考虑了反事实对照组,并进行了敏感性分析,以评估对照组参数的不同设置。只需要最低限度的分布假设。模拟研究证明了该方法的有效性。我们使用一个接受全膝关节置换术(TKR)的大型老年人登记册(N = 7239)来说明该方法。我们展示了如何利用外部数据来限制敏感性分析。原始分析揭示了多个治疗效果调节因素,包括基线功能、年龄、体重指数 (BMI)、性别、合并症数量、收入和种族。校正分析表明,基线(应激前)功能与 TKR 术后恢复的关系不大,在协变量中,只有年龄和合并症数量与应激后所有功能领域的恢复持续负相关。为了有效推断协变量和基线状态对前后变化的影响,有必要对数学耦合和 RTM 进行校正。我们的方法为此提供了一个简单的估算器。
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来源期刊
Biostatistics
Biostatistics 生物-数学与计算生物学
CiteScore
5.10
自引率
4.80%
发文量
45
审稿时长
6-12 weeks
期刊介绍: Among the important scientific developments of the 20th century is the explosive growth in statistical reasoning and methods for application to studies of human health. Examples include developments in likelihood methods for inference, epidemiologic statistics, clinical trials, survival analysis, and statistical genetics. Substantive problems in public health and biomedical research have fueled the development of statistical methods, which in turn have improved our ability to draw valid inferences from data. The objective of Biostatistics is to advance statistical science and its application to problems of human health and disease, with the ultimate goal of advancing the public''s health.
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