解决生存数据中删失问题的非参数断点回归方法,提升医疗决策评估的准确性。
Nonparametric Regression Discontinuity Designs with Survival Outcomes
- 采用双重稳健删失校正,适配现有断点回归估计器
- 在前列腺癌筛查试验中实现更高效率与抗模型误设能力
- 适用于多终点、长随访、协变量相关删失的生存分析场景
准实验评估是生成真实世界因果证据的核心方法,可补充随机试验的发现。断点回归设计(RDD)是一种准实验设计,用于估计基于连续变量是否越过阈值而分配的处理效应。这类阈值规则在医疗领域极为常见,常由预测性或预后性生物标志物指导治疗决策。然而,标准的RDD估计器依赖于完整的结局数据,这一假设在时间至事件分析中常因失访导致删失而被违反。为此,我们提出一种非参数方法,结合双重稳健删失校正,并可与现有RDD估计器配合使用。该方法能处理多个生存终点、长期随访及生存与删失的协变量相关变异。我们在多个应用领域讨论了其适用性,并通过模拟和前列腺、肺、结直肠及卵巢(PLCO)癌症筛查试验中的前列腺部分数据验证其有效性。新方法展现出更高效率和对模型误设的鲁棒性。我们还开发了开源R包\texttt{rdsurvival},支持实际应用。
原文摘要 · Abstract (English)
Quasi-experimental evaluations are central for generating real-world causal evidence and complementing insights from randomized trials. The regression discontinuity design (RDD) is a quasi-experimental design that can be used to estimate the causal effect of treatments that are assigned based on a running variable crossing a threshold. Such threshold-based rules are ubiquitous in healthcare, where predictive and prognostic biomarkers frequently guide treatment decisions. However, standard RD estimators rely on complete outcome data, an assumption often violated in time-to-event analyses where censoring arises from loss to follow-up. To address this issue, we propose a nonparametric approach that leverages doubly robust censoring corrections and can be paired with existing RD estimators. Our approach can handle multiple survival endpoints, long follow-up times, and covariate-dependent variation in survival and censoring. We discuss the relevance of our approach across multiple areas of applications and demonstrate its usefulness through simulations and the prostate component of the Prostate, Lung, Colorectal and Ovarian (PLCO) Cancer Screening Trial where our new approach offers several advantages, including higher efficiency and robustness to misspecification. We have also developed an open-source software package, $\texttt{rdsurvival}$, for the $\texttt{R}$ language.
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