arXiv:2410.00509cs.LGcs.IT2024-10NeurIPS被引 2

破解临床数据中的治疗分配偏差,提升精准医疗决策的可靠性

Learning Personalized Treatment Decisions in Precision Medicine: Disentangling Treatment Assignment Bias in Counterfactual Outcome Prediction and Biomarker Identification

  • 用互信息建模多种治疗分配偏差,更贴近真实临床场景
  • 发现部分与结果无关的偏差对预测准确率影响小,关键需识别特定偏差
  • 适用于需处理真实世界医疗数据的精准医疗研究者

精准医疗利用机器学习和人工智能为个体患者定制治疗方案,但面临临床观察数据中复杂的偏差及生物数据高维性的挑战。本研究通过互信息建模多种治疗分配偏差,分析其对反事实预测和生物标志物识别模型的影响。不同于传统依赖固定治疗策略的基准,本文聚焦不同临床环境下观测治疗策略的特征差异。在模拟数据、半合成TCGA肿瘤基因组数据以及药物和CRISPR筛选的真实生物结果上验证方法。结合经验性生物学机制,构建更贴近现实的数据基准。分析表明,不同偏差导致模型表现各异,尤其与结果机制无关的偏差对预测精度影响较小。强调在反事实机器学习模型开发中必须考虑具体临床偏差,以提升精准医疗的个性化决策能力。

原文摘要 · Abstract (English)

Precision medicine has the potential to tailor treatment decisions to individual patients using machine learning (ML) and artificial intelligence (AI), but it faces significant challenges due to complex biases in clinical observational data and the high-dimensional nature of biological data. This study models various types of treatment assignment biases using mutual information and investigates their impact on ML models for counterfactual prediction and biomarker identification. Unlike traditional counterfactual benchmarks that rely on fixed treatment policies, our work focuses on modeling different characteristics of the underlying observational treatment policy in distinct clinical settings. We validate our approach through experiments on toy datasets, semi-synthetic tumor cancer genome atlas (TCGA) data, and real-world biological outcomes from drug and CRISPR screens. By incorporating empirical biological mechanisms, we create a more realistic benchmark that reflects the complexities of real-world data. Our analysis reveals that different biases lead to varying model performances, with some biases, especially those unrelated to outcome mechanisms, having minimal effect on prediction accuracy. This highlights the crucial need to account for specific biases in clinical observational data in counterfactual ML model development, ultimately enhancing the personalization of treatment decisions in precision medicine.

精准医疗反事实推理治疗偏差生物标志物

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