Comparisons of Propensity Score Methods for Time to Event Outcomes: Evaluation through Simulations and Oral Squamous Cell Carcinoma Case Study

Sophie Ma, B. I. Said, A. Hosni, Wei Xu, S. Keshavarzi
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Abstract

Introduction & Objective: In observational studies, it is recommended to use propensity score (PS) methods or covariate adjustment for confounding effect adjustment. However, few guidelines are available regarding the choice of PS approaches or covariate adjustment for the best performance in a particular data. In this study, we compared different PS methods and conventional covariate adjustment to investigate the treatment effect for the overall population on time-to-event outcomes. Methods: In the Monte Carlo simulations, we compared the hazard ratio (HR) and precision estimated using covariate adjustment and eight different PS approaches, including matching, stratification, and inverse probability of treatment weighting (IPTW). In the Oral Squamous-Cell Carcinoma Cancer case study, we applied the aforementioned PS approaches to compare the effect of receiving post-operative radiation therapy (PORT) and having engraftable tumors on different time-to-event clinical outcomes. Results: In the simulations, both IPTW and covariate adjustment produced unbiased HR estimates with small uncertainty. In the case study, covariate adjustment showed that patients with engraftable tumors were twice as likely to have local/regional recurrence (HR 1.98 [1.23, 3.18], p-value<0.005) and any recurrence or death (HR 2.02 [1.38, 2.96], p-value<0.001); patients received PORT were twice as likely to develop either local, regional, or distance recurrence (HR 2.12 [1.32, 3.41], p-value<0.005). Results produced by IPTW were consistent with covariate adjustment method (within ± 0.1 differences). Conclusion: Covariate adjustment and the IPTW method performed well across simulations and the case study. In practice, care should be taken to select the most suitable method when estimating the treatment, exposure or intervention effect on time-to-event outcomes.
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事件发生时间的倾向评分方法的比较:通过模拟评估和口腔鳞状细胞癌病例研究
前言与目的:在观察性研究中,建议使用倾向评分(PS)方法或协变量校正来校正混杂效应。然而,关于在特定数据中选择PS方法或协变量调整以获得最佳性能的指导方针很少。在这项研究中,我们比较了不同的PS方法和传统的协变量调整,以调查治疗对总体人群的时间到事件结局的影响。方法:在蒙特卡罗模拟中,我们比较了使用协变量调整和8种不同PS方法(包括匹配、分层和处理加权逆概率(IPTW))估计的风险比(HR)和精度。在口腔鳞状细胞癌病例研究中,我们应用上述PS方法来比较接受术后放射治疗(PORT)和植入肿瘤对不同时间-事件临床结果的影响。结果:在模拟中,IPTW和协变量调整都产生了不确定性较小的无偏HR估计。在病例研究中,协变量调整显示,植瘤患者发生局部/区域复发(HR 1.98 [1.23, 3.18], p值<0.005)、复发或死亡(HR 2.02 [1.38, 2.96], p值<0.001)的可能性是植瘤患者的两倍;接受PORT治疗的患者发生局部、区域或远处复发的可能性是前者的两倍(HR 2.12 [1.32, 3.41], p值<0.005)。IPTW结果与协变量调整方法一致(误差在±0.1以内)。结论:协变量调整和IPTW方法在模拟和案例研究中都具有良好的效果。在实践中,在评估治疗、暴露或干预对事件发生时间结果的影响时,应注意选择最合适的方法。
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