论文标题
当使用倾向分数方法将临床试验推广到目标人群时,多次插补的应用
Application of Multiple Imputation When Using Propensity Score Methods to Generalize Clinical Trials to Target Populations of Interest
论文作者
论文摘要
当试验样本和目标群体之间的治疗效应改性剂的分布不同时,可以应用逆概率加权(IPSW)来实现目标人群平均治疗效应的无偏估计。当目标人群中缺少数据时,IPSW的统计有效性受到威胁,包括试验样本中的潜在丢失。但是,目前的文献中尚未充分讨论缺少的数据方法。我们进行了一组仿真研究,以确定如何在IPSW的背景下应用多个插补(MI)。我们特别解决了诸如归纳模型中包括哪些变量的问题,以及它们是否来自目标人群的试验或非试验部分。根据我们的发现,我们建议在试验和非试验人群中包括所有潜在效应修饰符和试验指标,以及试用模型中试用样本中的治疗和结果变量作为主要影响。此外,我们通过将频繁的血液透析网络(FHN)每日试验的发现传输到美国肾脏阶段系统(USRDS)人群来说明思想。
When the distribution of treatment effect modifiers differs between the trial sample and target population, inverse probability weighting (IPSW) can be applied to achieve an unbiased estimate of the population average treatment effect in the target population. The statistical validity of IPSW is threatened when there are missing data in the target population, including potential missingness in trial sample. However, missing data methods have not been adequately discussed in the current literature. We conducted a set of simulation studies to determine how to apply multiple imputation (MI) in the context of IPSW. We specifically addressed questions such as which variables to include in the imputation model and whether they should come from trial or non-trial portion of the target population. Based on our findings, we recommend including all potential effect modifiers and trial indicator from both trial and non-trial populations, as well as treatment and outcome variables from trial sample in the imputation model as main effects. Additionally, we have illustrated ideas by transporting findings from the Frequent Hemodialysis Network (FHN) Daily Trial to the United States Renal Stage System (USRDS) population.