Clinical studies sometimes encounter truncation by death, rendering outcomes undefined. Statistical analysis based solely on observed survivors may give biased results because the characteristics of survivors differ between treatment groups. By principal stratification, the survivor average causal effect was proposed as a causal estimand defined in always-survivors. However, this estimand is not identifiable when there is unmeasured confounding between the treatment assignment and survival or outcome process. In this paper, we consider the comparison between an aggressive treatment and a conservative treatment with monotonicity on survival. First, we show that the survivor average causal effect on the conservative treatment is identifiable based on a substitutional variable under appropriate assumptions, even when the treatment assignment is not ignorable. Next, we propose an augmented inverse probability weighting (AIPW) type estimator for this estimand with double robustness. Finally, large sample properties of this estimator are established. The proposed method is applied to investigate the effect of allogeneic stem cell transplantation types on leukemia relapse.
翻译:临床研究有时会因死亡而缺勤,结果不确定。仅仅根据观察到的幸存者的统计分析可能得出偏差结果,因为幸存者的特征不同。根据主要分层,幸存者的平均因果效应被建议为总幸存者定义的因果估计值。然而,当治疗任务与生存或结果过程之间出现不测的混杂时,这一估计值是无法确定的。在本文件中,我们考虑了攻击性治疗和保守治疗与单一性治疗对生存的比较。首先,我们表明幸存者对保守治疗的平均因果效应是根据适当假设的替代变量确定的,即使治疗任务不可忽略。接下来,我们提议增加这一估计值的逆概率估计值类型,并同时具有双强性。最后,确定了这个估计值的大型样本特性。拟议方法用于调查所有遗传性干细胞移植类型对白血病复发的影响。