提出可解释的多臂治疗效应估计方法,提升医疗决策可信度。
Causal rule ensemble approach for multi-arm data
- 基于规则集成生成可解释的治疗效应预测模型
- 模拟与真实数据中偏差更低、估计更准确
- 适合需要透明决策依据的临床研究场景
异质性治疗效应(HTE)估计在医学研究中至关重要,可为精准医疗提供统计支持。现有方法多针对二元处理情形,而现实应用常涉及多个干预措施。当前的HTE方法主要面向二元比较,且依赖黑箱模型,限制了其在多臂场景下的适用性和可解释性。为此,我们提出一种用于多臂试验的可解释机器学习框架。该方法采用基于规则的集成策略,包括规则生成、规则集成和HTE估计三个步骤,兼顾预测精度与可解释性。通过大规模模拟研究及真实数据应用,我们的方法在性能上优于现有先进多臂HTE估计方法,表现出更低的偏差和更高的估计准确性。此外,该框架的可解释性有助于清晰揭示协变量如何影响治疗效应,促进临床决策。本研究填补了精度与可解释性之间的空白,为多臂HTE估计提供了有力工具,助力精准医疗发展。
原文摘要 · Abstract (English)
Heterogeneous treatment effect (HTE) estimation is critical in medical research. It provides insights into how treatment effects vary among individuals, which can provide statistical evidence for precision medicine. While most existing methods focus on binary treatment situations, real-world applications often involve multiple interventions. However, current HTE estimation methods are primarily designed for binary comparisons and often rely on black-box models, which limit their applicability and interpretability in multi-arm settings. To address these challenges, we propose an interpretable machine learning framework for HTE estimation in multi-arm trials. Our method employs a rule-based ensemble approach consisting of rule generation, rule ensemble, and HTE estimation, ensuring both predictive accuracy and interpretability. Through extensive simulation studies and real data applications, the performance of our method was evaluated against state-of-the-art multi-arm HTE estimation approaches. The results indicate that our approach achieved lower bias and higher estimation accuracy compared with those of existing methods. Furthermore, the interpretability of our framework allows clearer insights into how covariates influence treatment effects, facilitating clinical decision making. By bridging the gap between accuracy and interpretability, our study contributes a valuable tool for multi-arm HTE estimation, supporting precision medicine.
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