arXiv:2502.01575stat.MLcs.LG2025-02ICML被引 1

解决生存数据中治疗效果异质性估计难题,适用于高删失场景。

Heterogeneous Treatment Effect in Time-to-Event Outcomes: Harnessing Censored Data with Recursively Imputed Trees

  • 用递归插补生存树处理删失数据,不依赖删失机制建模
  • 在重删失条件下优于已有方法,且在真实数据集上表现更优
  • 首次实现工具变量下非参数化的异质性治疗效应估计

个性化治疗是医学等领域的核心目标,关键在于估计异质性治疗效应(HTE),即治疗对不同亚群的影响差异。然而,在生存数据分析中,因右删失导致观测不完整,现有方法常因假设完全观测而产生偏差与效率损失。尽管Cui等人(2023)提出双重稳健方法,但在重删失场景下仍表现不佳,尤其在罕见结局如肌萎缩侧索硬化症(ALS)中。此外,多数方法无法处理工具变量,限制了其因果推断能力。本文提出多重插补生存治疗响应(MISTR),一种通用、非参数的生存数据HTE估计新方法。MISTR通过递归插补生存树处理删失,无需直接建模删失机制。在大规模模拟及两个真实数据集——艾滋病临床试验组协议175和伊利诺伊州失业数据集——上的实验表明,MISTR在无隐混杂因素下显著优于现有方法,且可扩展至工具变量设置。据我们所知,MISTR是首个通过工具变量实现未观测混杂因素下非参数HTE估计的方法。

原文摘要 · Abstract (English)

Tailoring treatments to individual needs is a central goal in fields such as medicine. A key step toward this goal is estimating Heterogeneous Treatment Effects (HTE) - the way treatments impact different subgroups. While crucial, HTE estimation is challenging with survival data, where time until an event (e.g., death) is key. Existing methods often assume complete observation, an assumption violated in survival data due to right-censoring, leading to bias and inefficiency. Cui et al. (2023) proposed a doubly-robust method for HTE estimation in survival data under no hidden confounders, combining a causal survival forest with an augmented inverse-censoring weighting estimator. However, we find it struggles under heavy censoring, which is common in rare-outcome problems such as Amyotrophic lateral sclerosis (ALS). Moreover, most current methods cannot handle instrumental variables, which are a crucial tool in the causal inference arsenal. We introduce Multiple Imputation for Survival Treatment Response (MISTR), a novel, general, and non-parametric method for estimating HTE in survival data. MISTR uses recursively imputed survival trees to handle censoring without directly modeling the censoring mechanism. Through extensive simulations and analysis of two real-world datasets-the AIDS Clinical Trials Group Protocol 175 and the Illinois unemployment dataset we show that MISTR outperforms prior methods under heavy censoring in the no-hidden-confounders setting, and extends to the instrumental variable setting. To our knowledge, MISTR is the first non-parametric approach for HTE estimation with unobserved confounders via instrumental variables.

生存分析异质性治疗删失数据工具变量

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