处理治疗导致结果不可观测的问题,用专家推测反事实结果提升神经病学预后模型评估可靠性。
Learning Under Treatment-Induced Label Indeterminacy with Expert Annotations of Counterfactual Outcomes: A Case Study in Neurological Prognostication

- 区分确定与不确定病例,利用专家对反事实结果的推测补充标签
- 模型在确定病例上表现相似时,对不确定病例的概率估计差异显著
- 揭示传统评估忽略的关键权衡:提升不确定病例预测会牺牲确定病例精度
临床预测模型通常假设所有患者的结果都能清晰观测,但当治疗决策使临床相关结果永久不可观测时,该假设失效。以2,497名心脏骤停患者为例,其中1,429名患者的结局因治疗决策变得不确定,这些患者由独立临床专家评估其反事实结局(即若未接受治疗会如何),称为不确定病例;其余患者结局可观察,称为确定病例。本文提出一种框架,将评估任务明确区分为确定与不确定病例。由于目标标签不同,难以统一评估。我们设计了一个简单预测模型,同时使用两类病例的标签进行训练,实现二者之间的权衡。在神经网络模型与多种表格基线模型中,尽管确定病例的AUROC相近,但其在确定病例上的布里尔分数及对不确定病例的概率估计仍存在显著差异。优化不确定病例的标签对齐通常以牺牲确定病例准确率为代价,揭示了标准评估所掩盖的显性权衡。结果表明,当治疗决策影响结果可观测性时,传统评估指标可能遗漏最需要预测支持的患者群体中的关键失败模式。
原文摘要 · Abstract (English)
Clinical prediction models are often developed as if the outcome of interest were cleanly observed for every patient. This assumption fails when treatment decisions make the clinically relevant outcome permanently unobservable. As a case study of this problem, we consider post-cardiac-arrest neurological prognostication using a cohort of 2,497 patients, including 1,429 patients whose outcomes were rendered indeterminate by treatment decisions. These patients with indeterminate outcomes were reviewed by independent clinical experts, who provided their guesses of counterfactual outcomes about what would have happened to the patients. We refer to these patients as uncertain cases. We also have patients for whom we observe their clinically relevant outcomes; we refer to these patients as certain cases. We propose a framework for evaluating prediction models that explicitly splits the evaluation between certain and uncertain cases. Here, we cannot easily evaluate both types of cases in a uniform manner as the available target labels differ. We then propose a simple prediction model that uses target labels from both certain and uncertain cases in a manner that allows us to trade off between them. Across the proposed neural model and a collection of tabular baselines, models with similar certain-case AUROC can nevertheless differ substantially in both certain-case Brier score and their probability estimates for uncertain cases. Improving alignment with target labels of uncertain cases for our proposed model generally comes at the cost of worse accuracy on certain cases, highlighting an explicit tradeoff that standard evaluation conceals. These results show that when treatment decisions determine whether clinically meaningful outcomes remain observable, conventional evaluation metrics can miss important failure modes in the very patients for whom prognostic support matters most.
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