Comparing Explainable Machine Learning Approaches With Traditional Statistical Methods for Evaluating Stroke Risk Models: Retrospective Cohort Study.

Background: Stroke has multiple modifiable and nonmodifiable risk factors and represents a leading cause of death globally. Understanding the complex interplay of stroke risk factors is thus not only a scientific necessity but a critical step toward improving global health outcomes.

Objective: We aim to assess the performance of explainable machine learning models in predicting stroke risk factors using real-world cohort data by comparing explainable machine learning models with conventional statistical methods.

Methods: This retrospective cohort included high-risk patients from Ramathibodi Hospital in Thailand between January 2010 and December 2020. We compared the performance and explainability of logistic regression (LR), Cox proportional hazard, Bayesian network (BN), tree-augmented Naïve Bayes (TAN), extreme gradient boosting (XGBoost), and explainable boosting machine (EBM) models. We used multiple imputation by chained equations for missing data and discretized continuous variables as needed. Models were evaluated using C-statistics and F₁-scores.

Results: Out of 275,247 high-risk patients, 9659 (3.5%) experienced a stroke. XGBoost demonstrated the highest performance with a C-statistic of 0.89 and an F₁-score of 0.80 followed by EBM and TAN with C-statistics of 0.87 and 0.83, respectively; LR and BN had similar C-statistics of 0.80. Significant factors associated with stroke included atrial fibrillation (AF), hypertension (HT), antiplatelets, HDL, and age. AF, HT, and antihypertensive medication were common significant factors across most models, with AF being the strongest factor in LR, XGBoost, BN, and TAN models.

Conclusions: Our study developed stroke prediction models to identify crucial predictive factors such as AF, HT, or systolic blood pressure or antihypertensive medication, anticoagulant medication, HDL, age, and statin use in high-risk patients. The explainable XGBoost was the best model in predicting stroke risk, followed by EBM.