๐Ÿง‘๐Ÿผโ€๐Ÿ’ป Research - February 2, 2026

Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia.

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โšก Quick Summary

This study developed a machine learning mortality prediction model for children with aplastic anemia undergoing cyclosporine therapy, achieving an impressive AUC of 0.834 in training and 0.826 in validation. The model aims to enhance risk-stratified treatment strategies and improve patient outcomes.

๐Ÿ” Key Details

  • ๐Ÿ“Š Dataset: Children with acquired aplastic anemia receiving cyclosporine
  • ๐Ÿงฉ Features used: Reticulocyte count, platelet count, disease subtype, total bilirubin, bone marrow myeloid proportion
  • โš™๏ธ Technology: CatBoost machine learning model
  • ๐Ÿ† Performance: AUC 0.834 (training), AUC 0.826 (validation)

๐Ÿ”‘ Key Takeaways

  • ๐Ÿ“ˆ Machine learning can effectively predict mortality risk in pediatric aplastic anemia patients.
  • ๐Ÿ” Five key predictors were identified: reticulocyte count, platelet count, disease subtype, total bilirubin, and bone marrow myeloid proportion.
  • ๐Ÿ† The CatBoost model demonstrated the highest performance among ten tested models.
  • ๐Ÿ“Š Calibration scores were acceptable, indicating reliable predictions.
  • ๐Ÿ’ก SHAP analysis highlighted reticulocyte count as the most significant predictor of mortality risk.
  • ๐ŸŒŸ Adherence to TRIPODโ€‰+โ€‰AI guidelines ensures methodological rigor in the study.
  • ๐ŸŒ Potential for clinical application as a decision tool for stratifying patients into distinct mortality risk groups.

๐Ÿ“š Background

Aplastic anemia is a serious condition characterized by the failure of bone marrow to produce sufficient blood cells. In children, the variability in outcomes, particularly regarding mortality risk, necessitates the development of predictive models. With advancements in machine learning, there is an opportunity to enhance treatment strategies and improve patient care through more accurate risk assessments.

๐Ÿ—’๏ธ Study

This retrospective cohort study focused on children diagnosed with acquired aplastic anemia who were treated with cyclosporine-based immunosuppression. The cohort was stratified by disease severity and divided into training (70%) and validation (30%) groups. The researchers developed ten machine learning models, optimizing hyperparameters through grid search and 10-fold cross-validation to ensure robust performance.

๐Ÿ“ˆ Results

The CatBoost model emerged as the top performer, achieving an AUC of 0.834 in the training cohort and 0.826 in the validation cohort. The model’s calibration was deemed acceptable, with Brier scores of 0.206 and 0.207 for training and validation cohorts, respectively. The SHAP analysis confirmed that lower reticulocyte counts were associated with a higher predicted mortality risk, underscoring its importance as a predictive feature.

๐ŸŒ Impact and Implications

The findings from this study have significant implications for clinical practice. By utilizing machine learning to predict mortality risk in pediatric aplastic anemia patients, healthcare providers can implement more tailored treatment strategies. This approach not only optimizes patient care but also enhances the overall management of this challenging condition, potentially leading to improved survival rates and quality of life for affected children.

๐Ÿ”ฎ Conclusion

This study highlights the transformative potential of machine learning in predicting mortality risk for children undergoing cyclosporine therapy for aplastic anemia. The robust performance of the CatBoost model, combined with its transparency and adherence to methodological guidelines, positions it as a promising clinical decision tool. Continued research in this area is essential to further refine these models and enhance patient outcomes.

๐Ÿ’ฌ Your comments

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Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia.

Abstract

The outcomes of children with aplastic anemia receiving cyclosporine monotherapy vary significantly in terms of mortality risk; therefore, a prognostic model for predicting mortality risk was constructed to optimize risk-stratified treatment strategies. This retrospective cohort study included children with acquired AA receiving cyclosporine-based immunosuppression, stratified by disease severity (vSAA/SAA/NSAA) and randomly split into training (70%) and validation (30%) cohorts. Ten machine learning models were developed; hyperparameters were optimized via grid search with 10-fold cross-validation exclusively within the training cohort to prevent data leakage. Model performance was evaluated using area under the ROC curve (AUC), accuracy, recall, specificity, precision, F1 score, and Brier score. Decision curve analysis (DCA) quantified clinical net benefit. The calibration curve was used to evaluate the reliability of the predicted probabilities. The SHapley Additive exPlanations (SHAP) framework was used to interpret feature contributions and ensure model transparency. Least absolute shrinkage and selection operator (LASSO) regression on the training cohort identified 5 predictors: reticulocyte count (RC), platelet count (PLT), disease subtype (vSAA/SAA/NSAA), total bilirubin (TB), and bone marrow myeloid proportion. The CatBoost model achieved the highest performance: AUC 0.834 (95% CI: 0.774-0.895) in training and 0.826 (95% CI: 0.743-0.910) in validation, with acceptable calibration (Brier score: 0.206 in training cohort, 0.207 in validation cohort). SHAP analysis confirmed RC as the top contributor, with lower RC values associated with higher predicted mortality risk. The CatBoost model demonstrates robust performance and transparency for predicting mortality risk in children with AA after cyclosporine treatment. Adherence to TRIPODโ€‰+โ€‰AI guidelines ensures methodological rigor, supporting its potential as a clinical decision tool to stratify patients into distinct mortality risk groups and optimize individualized treatment strategies.

Author: [‘Wen X’, ‘Xiao L’, ‘Li D’, ‘Liao M’, ‘Liu Y’, ‘Liu Q’, ‘Guan X’, ‘Dou Y’, ‘Hua Z’]

Journal: Ann Hematol

Citation: Wen X, et al. Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia. Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia. 2026; 105:69. doi: 10.1007/s00277-026-06842-3

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