Biostatistics Research Group, Department of Health Sciences, University of Leicester, Leicester, UK.
Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.
Stat Med. 2021 Nov 30;40(27):6069-6092. doi: 10.1002/sim.9171. Epub 2021 Sep 15.
A commonly reported measure when interested in the survival of cancer patients is relative survival. Relative survival circumvents issues with inaccurate cause of death information by incorporating the expected mortality rates of cancer individuals from population lifetables of the general population. A summary of the cancer population prognosis can be obtained using the marginal relative survival. To explore differences between exposure groups, such as socioeconomic groups, the difference in marginal relative survival between exposed and unexposed can be obtained and under assumptions is interpreted as the average causal effect of exposure to survival. In a modeling context, this is usually estimated by applying regression standardization as the average of the individual-specific estimates after fitting a relative survival model. Regression standardization yields an estimator that consistently estimates the causal effect under standard causal inference assumptions and if the relative survival model is correctly specified. We extend inverse probability weighting (IPW) and doubly robust standardization methods in the relative survival framework as additional valuable tools for obtaining average causal effects when correct model specification might not hold for the relative survival model. IPW yields an unbiased estimate of the average causal effect if a correctly specified model has been fitted for the exposure (propensity score) whereas doubly robust standardization requires that at least one of the propensity score model or the relative survival model is correctly specified. An example using data on melanoma is provided and a simulation study is conducted to investigate how sensitive are the methods to model misspecification, including different ways for obtaining standard errors.
当关注癌症患者的生存率时,相对生存率是一种常用的衡量指标。相对生存率通过纳入癌症患者的预期死亡率,从人群生命表中规避了死因信息不准确的问题。通过边际相对生存率可以获得癌症人群预后的总结。为了探索暴露组(如社会经济组)之间的差异,可以获得暴露组和非暴露组之间的边际相对生存率差异,并在假设条件下将其解释为暴露对生存率的平均因果效应。在建模背景下,通常通过应用回归标准化来估计,即在拟合相对生存率模型后,将个体特定估计值的平均值作为回归标准化的估计值。回归标准化产生的估计值在标准因果推理假设下一致估计因果效应,并且如果相对生存率模型正确指定。我们在相对生存率框架中扩展逆概率加权 (IPW) 和双重稳健标准化方法,作为在相对生存率模型可能不正确指定的情况下获得平均因果效应的额外有用工具。如果已经为暴露(倾向评分)拟合了正确指定的模型,IPW 会产生平均因果效应的无偏估计值,而双重稳健标准化要求至少正确指定倾向评分模型或相对生存率模型之一。提供了一个使用黑色素瘤数据的示例,并进行了模拟研究,以调查这些方法对模型误设的敏感性,包括获得标准误差的不同方法。