Propensity score weighting analysis with complex survey data for estimating population-level treatment effects on survival: a simulation study.

IF 2.2 Q3 HEALTH CARE SCIENCES & SERVICES
Lihua Li, Chen Yang, Wei Zhang, Yulei He, John R Pleis, Lauren M Rossen, Bian Liu, Morgan Earp, Madhu Mazumdar
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Abstract

Propensity score weighting (PSW) is a valuable tool for estimating treatment effects on survival outcomes in observational studies. However, there is no clear best practice for applying PSW to complex survey data with survival outcomes. This paper addresses this gap by exploring how to integrate PSW into complex survey with design features (strata, clusters, sampling weights) for unbiased population-level estimates. We evaluate three PSW methods where: Method I: neither the propensity score (PS) model nor the outcome model accounts for the survey design; Method II: the PS model does not account for the survey design, but the outcome model does; Method III: both the PS model and outcome model account for the survey design. Through extensive simulations, we compare performance in estimating absolute treatment effects measured by population survival quantile effects and relative treatment effects measured by population marginal hazard ratios. Mean relative bias, mean absolute bias and coverage probability are estimated for model evaluations under various scenarios, including varying treatment effect magnitude, censoring type and rate, level of PS overlap, presence of outliers and nonresponse. Findings reveal that both survey-weighted Methods II and III outperform the unweighted Method I under most scenarios for both measures of treatment effects, especially when there is a true treatment effect. Both weighted methods II and III are found to perform closely, including when there exists informative censoring, influential outliers, or non-response. We recommend that when considering PSW with complex survey data for estimating population-level treatment effects on survival outcomes, both modelling stages should incorporate survey designs, but it is most critical for the outcome modelling. For illustration, all methods are applied to the public-use 2000-2018 National Health Interview Survey (NHIS) Linked to Mortality Files with mortality information through 2019 to estimate the effect of smoking cessation after a cancer diagnosis on subsequent overall survival.

用复杂调查数据进行倾向评分加权分析,估计人群水平治疗对生存的影响:一项模拟研究。
在观察性研究中,倾向评分加权(PSW)是估计治疗对生存结果影响的一种有价值的工具。然而,对于将PSW应用于具有生存结果的复杂调查数据尚无明确的最佳实践。本文通过探索如何将PSW整合到具有设计特征(地层、聚类、抽样权重)的复杂调查中,以实现无偏总体水平估计,从而解决了这一差距。我们评估了三种PSW方法,其中:方法一:倾向得分(PS)模型和结果模型都不能解释调查设计;方法二:PS模型不考虑调查设计,但结果模型考虑了;方法三:采用PS模型和结果模型进行调查设计。通过广泛的模拟,我们比较了用种群生存分位数效应衡量绝对治疗效果和用种群边际风险比衡量相对治疗效果的表现。估计了不同情景下模型评估的平均相对偏倚、平均绝对偏倚和覆盖概率,包括不同的处理效果大小、审查类型和比率、PS重叠水平、异常值的存在和无反应。研究结果表明,在大多数情况下,对于两种治疗效果的测量,调查加权方法II和III都优于未加权方法I,特别是当存在真实的治疗效果时。发现加权方法II和III的表现都很接近,包括当存在信息审查、有影响力的异常值或无响应时。我们建议在考虑具有复杂调查数据的PSW以估计人群水平治疗对生存结果的影响时,两个建模阶段都应纳入调查设计,但这是结果建模最关键的。例如,所有方法都应用于公共使用的2000-2018年全国健康访谈调查(NHIS)与死亡率文件相关,其中包含到2019年的死亡率信息,以估计癌症诊断后戒烟对随后总体生存的影响。
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来源期刊
Health Services and Outcomes Research Methodology
Health Services and Outcomes Research Methodology HEALTH CARE SCIENCES & SERVICES-
CiteScore
3.40
自引率
6.70%
发文量
28
期刊介绍: The journal reflects the multidisciplinary nature of the field of health services and outcomes research. It addresses the needs of multiple, interlocking communities, including methodologists in statistics, econometrics, social and behavioral sciences; designers and analysts of health policy and health services research projects; and health care providers and policy makers who need to properly understand and evaluate the results of published research. The journal strives to enhance the level of methodologic rigor in health services and outcomes research and contributes to the development of methodologic standards in the field. In pursuing its main objective, the journal also provides a meeting ground for researchers from a number of traditional disciplines and fosters the development of new quantitative, qualitative, and mixed methods by statisticians, econometricians, health services researchers, and methodologists in other fields. Health Services and Outcomes Research Methodology publishes: Research papers on quantitative, qualitative, and mixed methods; Case Studies describing applications of quantitative and qualitative methodology in health services and outcomes research; Review Articles synthesizing and popularizing methodologic developments; Tutorials; Articles on computational issues and software reviews; Book reviews; and Notices. Special issues will be devoted to papers presented at important workshops and conferences.
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