Artificial Intelligence for Response Assessment in Pediatric Neuro-Oncology (AI-RAPNO), part 2: challenges,...

The Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria provide an important framework for evaluating treatment efficacy and tumour progression in clinical studies of paediatric brain tumours. As artificial intelligence (AI) rapidly transforms clinical practice, integrating AI into the RAPNO framework presents a unique opportunity to enhance quantitative, data-driven approaches for response assessment. However, successful clinical implementation faces challenges, including variability in imaging protocols, scarce annotated datasets, and regulatory and ethical considerations. To address these barriers, this Policy Review, led by the AI for Assessment in Pediatric Neuro-Oncology (AI-RAPNO) subcommittee, outlines key challenges and proposes recommendations to improve AI trustworthiness, generalisability, and implementation in paediatric neuro-oncology. We highlight the potential of AI for response assessment, multimodal integration, and synthetic control groups in clinical trials. Our recommendations emphasise the need for standardised imaging protocols, robust validation frameworks, and infrastructure to support AI readiness in clinical studies. By addressing these needs, AI-RAPNO aims to bridge the gap between AI research and clinical application, ensuring reliable and actionable AI-driven tools for paediatric neuro-oncology.