Clinical studies often encounter with truncation-by-death problems, which may render the outcomes undefined. Statistical analysis based only on observed survivors may lead to biased results because the characters of survivors may differ greatly between treatment groups. Under the principal stratification framework, a meaningful causal parameter, the survivor average causal effect, in the always-survivor group can be defined. This causal parameter may not be identifiable in observational studies where the treatment assignment and the survival or outcome process are confounded by unmeasured features. In this paper, we propose a new method to deal with unmeasured confounding when the outcome is truncated by death. First, a new method is proposed to identify the heterogeneous conditional survivor average causal effect based on a substitutional variable under monotonicity. Second, under additional assumptions, the survivor average causal effect on the overall population is also identified. Furthermore, we consider estimation and inference for the conditional survivor average causal effect based on parametric and nonparametric methods with good asymptotic properties. Good finite-sample properties are demonstrated by simulation and sensitivity analysis. The proposed method is applied to investigate the effect of allogeneic stem cell transplantation types on leukemia relapse.
翻译:临床研究经常遇到脱轨问题,这可能导致结果无法界定。仅根据观察到的幸存者进行统计分析,可能导致有偏差的结果,因为幸存者的个性在治疗群体之间差别很大。在主要分层框架下,可以确定一个有意义的因果参数,即幸存者在总幸存者群体中的平均因果效应。在治疗任务和生存或结果过程因不测特征而混杂的观察研究中,这一因果参数可能无法识别。在本文件中,我们提出一种新的方法,在结果因死亡而脱轨时,处理无法计量的混杂情况。首先,建议采用新的方法,根据单一性下的替代变量,确定混杂的有条件幸存者平均因果效应。第二,根据其他假设,还确定了幸存者对整个人口的平均因果效应。此外,我们考虑根据具有良好消化特性的对数和非对数方法,对有条件的幸存者平均因果效应进行估计和推论。模拟和敏感性分析表明良好的定数性细胞特征。拟议方法用于调查所有遗传性干细胞型移植的影响。