从治疗路径到患者原型:面向价值医疗基准化的潜在类别分析框架
From therapeutic pathways to patient archetypes: a latent class analysis framework for Value-Based Healthcare benchmarking
摘要
该研究针对价值医疗(VBHC)在跨机构基准比较中病例组合偏倚问题,提出以治疗路径而非单纯生物学特征划分患者原型。基于2018–2020年确诊的2485例乳腺癌患者队列,采集基线与6个月随访的人口学与治疗变量,在80%训练集中应用潜在类别分析(LCA)建模,以aBIC、熵值和后验概率评估拟合与分类质量,并在20%测试集中用随机森林验证分类稳健性,另在外部多中心队列(N=352)检验可推广性。结果选出六类原型方案,熵值0.932,与随机森林分类一致率达99.68%,外部队列平均后验概率0.965。作者认为LCA可透明、高效地识别并按治疗路径归类新患者,支持VBHC公平基准化,建议在前瞻性更广队列中验证。
原文摘要
BACKGROUND: Value-Based Healthcare (VBHC) focuses on Patient-Centred Outcomes (PCOs) to maximize healthcare value. Benchmarking across providers requires comparing outcomes among clinically and therapeutically comparable patients. However, traditional classification is based on biological features without accounting for the therapeutic trajectory. Establishing patient archetypes based on therapeutic pathways is therefore essential to prevent case-mix bias in VBHC benchmarking. The aim of this paper is to develop a model-based framework using Latent Class Analysis (LCA) to (i) identify patient archetypes in breast cancer and (ii) establish a probabilistic tool to classify new patients. METHODS: A cohort of 2485 patients diagnosed with breast cancer between 2018 and 2020 was followed-up. Demographic and treatment variables were collected at baseline and at 6-months follow-up. LCA was applied to derive archetypes in a training set (80%). Fit and classification quality were assessed using aBIC, entropy, and posterior probabilities. Tool classification robustness was evaluated using Random Forest (RF) in a test set (20%), and generalizability was tested in an external multicenter cohort (N = 352). RESULTS: A six-class solution (Arch-1-Arch-6) was selected, achieving optimal fit, parsimony, high entropy (0.932), and clinical interpretability. Evaluated against RF, the tool demonstrated high classification concordance (99.68% accuracy). In the external cohort, high individual classification certainty was maintained (mean posterior probability = 0.965), supporting consistent assignment across care settings. CONCLUSIONS: LCA provides a transparent, efficient and robust tool to identify patient archetypes and assign new patients based on therapeutic pathways, enabling fair benchmarking in VBHC. Prospective validation in broader cohorts is recommended to confirm transferability to other clinical conditions.
推荐理由:方法学(LCA划分治疗路径原型并做内外部验证)设计扎实、可迁移至ART结局基准化,但研究对象为乳腺癌,与生殖内分泌/辅助生殖无直接关联,仅对关注价值医疗与病例组合校正方法的研究者具方法学参考价值。
文献解读
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