arXiv:2508.11060stat.MLcs.LG2025-08中稿 · JRSS C被引 2

提出一种新方法,用更稳健的方式从不完整随访数据中优化长期治疗方案。

Counterfactual Survival Q-learning via Buckley-James Boosting, with Applications to ACTG 175 and CALGB 8923

  • 结合加速失效时间模型与迭代提升,直接建模生存时间
  • 在ACTG175和CALGB8923试验中,决策准确率提升且反事实对比更稳定
  • 适合处理非线性协变量效应和多阶段治疗决策的临床研究

我们提出一种基于Buckley-James(BJ)提升的Q学习框架,用于从纵向随机临床试验中右删失生存结果估计最优动态治疗策略,应对随访不完整和协变量效应非线性时的个体化治疗决策需求。该方法将加速失效时间模型与灵活基学习器(如分量最小二乘、回归树)结合,在反事实Q学习框架内迭代提升。通过直接建模条件生存时间,避免比例风险假设,提供可临床解释的时间尺度对比,并在标准潜在结果假设下估计各阶段Q函数与个性化决策规则。相比依赖风险建模的Cox型Q学习,本方法对非比例风险和模型误设更鲁棒。模拟研究及对ACTG175艾滋病试验和CALGB 8923两阶段白血病试验的分析表明,该方法显著提升治疗决策准确性,增强个体内部反事实对比稳定性,尤其在多阶段场景中有效缓解跨阶段误差与偏倚累积问题。

原文摘要 · Abstract (English)

We propose a Buckley James (BJ) Boost Q learning framework for estimating optimal dynamic treatment regimes from right censored survival outcomes in longitudinal randomized clinical trials, motivated by the clinical need to support patient specific treatment decisions when follow up is incomplete and covariate effects may be nonlinear. The method combines accelerated failure time modeling with iterative boosting using flexible base learners, including componentwise least squares and regression trees, within a counterfactual Q learning framework. By modeling conditional survival time directly, BJ Boost Q learning avoids the proportional hazards assumption, yields clinically interpretable time scale contrasts, and enables estimation of stage specific Q functions and individualized decision rules under standard potential outcomes assumptions. In contrast to Cox based Q learning, which relies on hazard modeling and can be sensitive to nonproportional hazards and model misspecification, our approach provides a robust and flexible alternative for regime learning. Simulation studies and analyses of the ACTG175 HIV trial and the CALGB 8923 two stage leukemia trial show that BJ Boost Q learning improves treatment decision accuracy and produces more stable within participant counterfactual contrasts, particularly in multistage settings where estimation error and bias can compound across stages.

生存分析动态治疗因果推断

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