arXiv:2605.26413stat.MEcs.AI2026-05

通过医生治疗意图识别隐藏混杂因素,提升真实世界医疗研究的因果推断可靠性。

Confounder Detection via Treatment Intent: A New Observational Study Design

论文配图:Confounder Detection via Treatment Intent: A New Observational Study Design
图 1 · 摘自论文原文
  • 让临床专家对比匹配患者对治疗决策差异,挖掘未观测到的混杂变量。
  • 在重症监护数据中发现电子病历存在显著未观测混杂,影响干预效果评估。
  • 结合临床文本与NLP技术,在模拟环境中验证方法有效性,适合医疗因果研究者。

理解干预效果是科学进步的核心,随机对照试验(RCT)被视为因果推断的金标准。然而,RCT成本高、耗时长,且常受伦理或实际限制,推动了从观察数据中进行因果推断的方法发展。尽管观察数据规模不断增长,但因部分影响治疗分配和结果的变量未被观测到,导致未观测混杂问题普遍存在。本文提出一种新研究设计——通过治疗意图检测混杂因子。该方法向做出治疗决策的临床专家询问,基于系统匹配策略提出的患者对,比较其治疗决策差异,以揭示解释差异的未观测变量。我们为该程序提供了理论基础,明确了可有效识别未观测混杂的条件。在此基础上,我们研究了重症监护室(ICU)中干预措施的效果。首先,实证证据强烈表明ICU电子健康记录(EHR)受到未观测混杂的影响。通过将临床文本笔记作为医生知识的代理,并利用自然语言处理技术,在具有已知真实情况的半合成环境中,为本方法提供了概念验证。

原文摘要 · Abstract (English)

Understanding the effects of interventions is central to scientific progress, with randomized controlled trials (RCTs) regarded as the gold standard for causal inference in many applied fields. However, RCTs are costly, time-consuming, and often constrained by ethical or practical limitations, motivating the need for causal methods able to draw conclusions from observational data. While such data is collected at ever larger scale, making its use for causal inference is often hindered by the fact that not all variables affecting treatment allocation and the outcome are observed: an issue known as unobserved confounding. In this paper, we introduce a new study design called confounder detection via treatment intent. The idea is to query a human expert who makes treatment decisions, and ask them to compare pairs of units proposed by a principled matching strategy, with the goal of eliciting unobserved variables that explain why treatment decisions differ. We provide a theoretical basis for such a procedure, ascertaining conditions under which such a study design may elicit unobserved confounders. Building on this newly established foundations, we study treatment effects of interventions in the intensive care unit (ICU). First, we show empirical evidence strongly indicating that electronic health records (EHRs) collected in ICUs are subject to unobserved confounding. By using clinical text notes as a proxy for physicians' knowledge and leveraging natural language processing, we provide a proof of concept for our methodology in a semi-synthetic environment with a known ground truth.

因果推断医疗数据混杂因素NLP应用

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