arXiv:2606.12623stat.APcs.LG2026-06

用因果模型预测中风患者个体化治疗效果,助力精准决策。

Estimating Individualized Treatment Effects in Acute Ischemic Stroke with Causal Transformation Models (TRAM-DAG): A Multi-Centre Observational Study with External RCT Validation

论文配图:Estimating Individualized Treatment Effects in Acute Ischemic Stroke with Causal Transformation Models (TRAM-DAG): A Multi-Centre Observational Study with External RCT Validation
图 1 · 摘自论文原文
  • 基于有向无环图的因果变换模型(TRAM-DAG)分析多中心观察数据
  • 在MR CLEAN试验人群上验证,平均治疗效应与真实结果一致
  • 能准确排序患者预后好坏,适合临床个性化治疗参考

急性缺血性中风的个体化治疗需超越平均治疗效应(ATE),转向个体治疗效应(ITE)估计以支持临床决策。尽管随机对照试验(如MR CLEAN)显示机械取栓优于溶栓,但无法确定哪些患者受益最大。本研究以3个月时改良秩次量表(mRS,0为无症状,6为死亡)为结局,利用多中心观察性MAGIC数据训练TRAM-DAG模型。为确保与MR CLEAN人群可比,仅使用入院时NIHSS≥6的子集进行训练。将该模型应用于MR CLEAN患者,估算其个体化治疗效应。虽无法实验验证,但估测的平均值与试验报告的ATE一致,且对良好预后(mRS≤2)发生频率排序准确。结果表明TRAM-DAG可有效实现观察证据与临床试验间的桥梁,支持中风治疗的个性化决策。

原文摘要 · Abstract (English)

Personalized medicine in acute ischemic stroke requires moving beyond average treatment effects (ATE) to individualized treatment effect (ITE) estimates to support treatment decisions. In acute ischemic stroke, mechanical thrombectomy has been shown to be more effective on average than lysis in randomized controlled trials (RCTs), such as the MR CLEAN study. We aim to identify which individual patients benefit most from mechanical thrombectomy compared to lysis. The outcome of interest is the modified Rankin Scale (mRS) at three months, an ordinal measure of functional disability (0: no symptoms, 6: death). We demonstrate that causal transformation models on directed acyclic graphs (TRAM-DAG) can be used for ITE estimation after being fitted on observational MAGIC multi-center stroke patient data. To ensure comparability with the MR CLEAN population, which we use for validation, we train the TRAM-DAG on a MAGIC sub-population with NIHSS at admission >= 6, corresponding to one inclusion criterion of MR CLEAN. The fitted model is then used to estimate ITEs for stroke patients in the MR CLEAN population. While these ITE estimates cannot be confirmed experimentally, we show that their average is consistent with the trial's reported ATE. Furthermore, the ITE estimates correctly rank trial patients by their observed frequency of a good outcome (mRS at three months <= 2). These findings support the use of causal models like TRAM-DAG for personalized decision-making in stroke care and highlight their ability to bridge the gap between observational evidence and clinical trials.

因果推断中风治疗个体化医疗

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