用优化方法提升分组分析精度,更好发现治疗效果差异人群
Efficient Subgroup Analysis via Optimal Trees with Global Parameter Fusion
- 基于混合整数优化构建全局最优分组树,避免传统方法的局部贪心问题
- 引入参数融合机制,在小样本下提升分组识别准确率和统计效率
- 在真实医疗数据中发现有意义的健康差异,适合临床研究与精准医疗
识别和推断差异性治疗效应(临床研究中的子群分析核心)是精准健康的关键。子群分析可定位对治疗特别受益或受保护的人群,推动靶向干预。基于树的递归划分方法因可解释性强而被广泛使用,但存在贪婪启发式导致的次优分组、局部拟合引发的过拟合等问题,尤其在样本量有限时更为显著。为此,我们提出一种融合最优因果树方法,利用混合整数优化(MIO)实现精确子群识别。该方法确保全局最优分组,并引入参数融合约束,促进相关子群间的信息共享,显著提升子群发现准确性和统计效率。我们通过严格推导给出了泛化风险界,并与经典树方法进行对比。实验表明,该方法在模拟中持续优于主流基线。最后,我们在健康与大脑老化研究健康差异(HABS-HD)数据集上验证了其实际价值,发现了具有临床意义的洞察。
原文摘要 · Abstract (English)
Identifying and making statistical inferences on differential treatment effects (commonly known as subgroup analysis in clinical research) is central to precision health. Subgroup analysis allows practitioners to pinpoint populations for whom a treatment is especially beneficial or protective, thereby advancing targeted interventions. Tree based recursive partitioning methods are widely used for subgroup analysis due to their interpretability. Nevertheless, these approaches encounter significant limitations, including suboptimal partitions induced by greedy heuristics and overfitting from locally estimated splits, especially under limited sample sizes. To address these limitations, we propose a fused optimal causal tree method that leverages mixed integer optimization (MIO) to facilitate precise subgroup identification. Our approach ensures globally optimal partitions and introduces a parameter fusion constraint to facilitate information sharing across related subgroups. This design substantially improves subgroup discovery accuracy and enhances statistical efficiency. We provide theoretical guarantees by rigorously establishing out of sample risk bounds and comparing them with those of classical tree based methods. Empirically, our method consistently outperforms popular baselines in simulations. Finally, we demonstrate its practical utility through a case study on the Health and Aging Brain Study Health Disparities (HABS-HD) dataset, where our approach yields clinically meaningful insights.
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