Predicting clozapine-induced adverse drug reaction biomarkers using machine learning

Abstract Clozapine is an atypical antipsychotic used for patients with treatment-resistant schizophrenia. This drug has serious adverse drug reactions (ADRs), including the risk of severe neutropenia (agranulocytosis). Patients who could benefit from clozapine may not be administered it due to conce...

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Main Authors: John-Paul Cooper, Pierre Chue, Arno G. Siraki, Lusine Tonoyan
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-09472-3
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author John-Paul Cooper
Pierre Chue
Arno G. Siraki
Lusine Tonoyan
author_facet John-Paul Cooper
Pierre Chue
Arno G. Siraki
Lusine Tonoyan
author_sort John-Paul Cooper
collection DOAJ
description Abstract Clozapine is an atypical antipsychotic used for patients with treatment-resistant schizophrenia. This drug has serious adverse drug reactions (ADRs), including the risk of severe neutropenia (agranulocytosis). Patients who could benefit from clozapine may not be administered it due to concerns about monitoring ADRs. In addition, traditional toxicological assessments cannot predict clozapine-induced agranulocytosis. Predicting agranulocytosis could improve patient safety. Our study aimed to develop and validate machine learning (ML) models for predicting agranulocytosis in clozapine-prescribed patients using the Canada Vigilance Adverse Reaction Online Database (n = 9395 reports). We addressed the class imbalance (337 agranulocytosis-positive cases vs. 9058 agranulocytosis-negative cases) through systematically evaluating resampling techniques and selecting appropriate performance metrics for rare event prediction. Five ML algorithms were evaluated on a hold-out test set. The best-performing model was the Gradient Boosting with Synthetic Minority Over-sampling technique (GB-SMOTE), achieving recall (sensitivity) of 0.85, AUC-PR (area under the precision-recall (PR) curve) of 0.77, PPV (Positive Predictive Value) of 0.40 and a Matthews Correlation Coefficient of 0.56. SHAP feature analysis identified blood and lymphatic system disorders, leukocytosis, and neutropenia as the strongest predictors. Our results demonstrate the potential of ML for predicting clozapine-induced agranulocytosis and provide a framework for developing pharmacovigilance prediction models. This is clinically important and relevant to the management of schizophrenia, which remains a chronic disease with high morbidity and mortality.
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spelling doaj-art-1c33fc4012f04020ae5ceeed0ffbb7192025-08-20T03:05:27ZengNature PortfolioScientific Reports2045-23222025-07-0115111110.1038/s41598-025-09472-3Predicting clozapine-induced adverse drug reaction biomarkers using machine learningJohn-Paul Cooper0Pierre Chue1Arno G. Siraki2Lusine Tonoyan3Faculty of Pharmacy and Pharmaceutical Sciences, College of Health Sciences, University of AlbertaFaculty of Pharmacy and Pharmaceutical Sciences, College of Health Sciences, University of AlbertaFaculty of Pharmacy and Pharmaceutical Sciences, College of Health Sciences, University of AlbertaFaculty of Pharmacy and Pharmaceutical Sciences, College of Health Sciences, University of AlbertaAbstract Clozapine is an atypical antipsychotic used for patients with treatment-resistant schizophrenia. This drug has serious adverse drug reactions (ADRs), including the risk of severe neutropenia (agranulocytosis). Patients who could benefit from clozapine may not be administered it due to concerns about monitoring ADRs. In addition, traditional toxicological assessments cannot predict clozapine-induced agranulocytosis. Predicting agranulocytosis could improve patient safety. Our study aimed to develop and validate machine learning (ML) models for predicting agranulocytosis in clozapine-prescribed patients using the Canada Vigilance Adverse Reaction Online Database (n = 9395 reports). We addressed the class imbalance (337 agranulocytosis-positive cases vs. 9058 agranulocytosis-negative cases) through systematically evaluating resampling techniques and selecting appropriate performance metrics for rare event prediction. Five ML algorithms were evaluated on a hold-out test set. The best-performing model was the Gradient Boosting with Synthetic Minority Over-sampling technique (GB-SMOTE), achieving recall (sensitivity) of 0.85, AUC-PR (area under the precision-recall (PR) curve) of 0.77, PPV (Positive Predictive Value) of 0.40 and a Matthews Correlation Coefficient of 0.56. SHAP feature analysis identified blood and lymphatic system disorders, leukocytosis, and neutropenia as the strongest predictors. Our results demonstrate the potential of ML for predicting clozapine-induced agranulocytosis and provide a framework for developing pharmacovigilance prediction models. This is clinically important and relevant to the management of schizophrenia, which remains a chronic disease with high morbidity and mortality.https://doi.org/10.1038/s41598-025-09472-3ClozapineSchizophreniaAgranulocytosisMachine learningBiomarkers
spellingShingle John-Paul Cooper
Pierre Chue
Arno G. Siraki
Lusine Tonoyan
Predicting clozapine-induced adverse drug reaction biomarkers using machine learning
Scientific Reports
Clozapine
Schizophrenia
Agranulocytosis
Machine learning
Biomarkers
title Predicting clozapine-induced adverse drug reaction biomarkers using machine learning
title_full Predicting clozapine-induced adverse drug reaction biomarkers using machine learning
title_fullStr Predicting clozapine-induced adverse drug reaction biomarkers using machine learning
title_full_unstemmed Predicting clozapine-induced adverse drug reaction biomarkers using machine learning
title_short Predicting clozapine-induced adverse drug reaction biomarkers using machine learning
title_sort predicting clozapine induced adverse drug reaction biomarkers using machine learning
topic Clozapine
Schizophrenia
Agranulocytosis
Machine learning
Biomarkers
url https://doi.org/10.1038/s41598-025-09472-3
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AT arnogsiraki predictingclozapineinducedadversedrugreactionbiomarkersusingmachinelearning
AT lusinetonoyan predictingclozapineinducedadversedrugreactionbiomarkersusingmachinelearning