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arXiv 2202.09164stat.ME
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观测资料比较有效性研究中的三角化工具变量、混杂因素调整和差中差方法

Triangulating Instrumental Variable, confounder adjustment and Difference-in-Difference methods for comparative effectiveness research in observational data

Laura Güdemann, John M. Dennis, Andrew P. McGovern, Lauren R. Rodgers, Beverley M. Shields, William Henley, Jack Bowden

摘要:

观察性研究可以在评估竞争性治疗的相对有效性方面发挥有用的作用。在临床试验中,将受试者随机分配到治疗组和对照组通常会导致在可能的混杂因素方面平衡良好的组,这使得分析简单明了。然而,在分析观察性数据时,潜在的未测量混杂因素使得比较处理效应更具挑战性。因果推断方法,如工具变量和先验偶率比方法,可以避免调整数据中未测量或测量错误的混杂因素。通过多变量回归和倾向得分匹配进行的直接混杂因素调整也具有相当大的实用性。每种方法都依赖于一组不同的假设,并利用数据的不同方面。在本文中,我们描述了每种方法的假设,并评估违反这些假设的影响,在模拟研究。我们提出了先前结果增强的工具变量方法,该方法利用了治疗开始前后的数据,并且对违反关键假设具有鲁棒性。最后,我们建议使用异质性统计量来决定两个或多个估计是否在统计上相似,并考虑它们的相关性。我们使用临床实践研究数据链的观察数据,说明我们的因果框架,以评估将钠-葡萄糖协同转运蛋白-2抑制剂与二肽基肽酶-4抑制剂作为2型糖尿病二线治疗的患者生殖器感染的风险。

英文摘要:

Observational studies can play a useful role in assessing the comparative effectiveness of competing treatments. In a clinical trial the randomization of participants to treatment and control groups generally results in well-balanced groups with respect to possible confounders, which makes the analysis straightforward. However, when analysing observational data, the potential for unmeasured confounding makes comparing treatment effects much more challenging. Causal inference methods such as Instrumental Variable and Prior Even Rate Ratio approaches make it possible to circumvent the need to adjust for confounding factors that have not been measured in the data or measured with error. Direct confounder adjustment via multivariable regression and Propensity score matching also have considerable utility. Each method relies on a different set of assumptions and leverages different aspects of the data. In this paper, we describe the assumptions of each method and assess the impact of violating these assumptions in a simulation study. We propose the prior outcome augmented Instrumental Variable method that leverages data from before and after treatment initiation, and is robust to the violation of key assumptions. Finally, we propose the use of a heterogeneity statistic to decide if two or more estimates are statistically similar, taking into account their correlation. We illustrate our causal framework to assess the risk of genital infection in patients prescribed Sodium-glucose co-transporter-2 inhibitors versus Dipeptidyl peptidase-4 inhibitors as second-line treatment for Type 2 Diabets using observational data from the Clinical Practice Research Datalink.

机构、总结、中文摘要均由AI分析而来,可能存在错误,请以论文正文为准。

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