Machine learning in paediatric haematological malignancies: a systematic review of prognosis, toxicity and treatment response models

Abstract

Abstract Background Machine Learning (ML) has demonstrated potential in enhancing care in adult oncology. However, its application in paediatric haematological malignancies is still emerging, necessitating a comprehensive review of its capabilities and limitations in this area. Methods A literature search was conducted through Ovid. Studies included focused on ML models in paediatric patients with haematological malignancies. Studies were categorised into thematic groups for analysis. Results Twenty studies, primarily on leukaemia, were included in this review. Studies were organised into thematic categories such as prognoses, treatment responses and toxicity predictions. Prognostic studies showed AUC scores between 0.685 and 0.929, indicating moderate-high predictive accuracy. Treatment response studies demonstrated AUC scores between 0.840 and 0.875, reflecting moderate accuracy. Toxicity prediction studies reported high accuracy with AUC scores from 0.870 to 0.927. Only five studies (25%) performed external validation. Significant heterogeneity was noted in ML tasks, reporting formats, and effect measures across studies, highlighting a lack of standardised reporting and challenges in data comparability. Conclusion The clinical applicability of these ML models remains limited by the lack of external validation and methodological heterogeneity. Addressing these challenges through standardised reporting and rigorous external validation is needed to translate ML from a promising research tool into a reliable clinical practice component. Impact Key message: Machine Learning (ML) significantly enhances predictive models in paediatric haematological cancers, offering new avenues for personalised treatment strategies. Future research should focus on developing ML models that can integrate with real-time clinical workflows. Addition to literature: Provides a comprehensive overview of current ML applications and trends. It identifies limitations to its applicability, including the limited diversity in datasets, which may affect the generalisability of ML models across different populations. Impact: Encourages standardisation and external validation in ML studies, aiming to improve patient outcomes through precision medicine in paediatric haematological oncology.