Benefit-risk balance of advanced therapies in pediatric inflammatory bowel disease: evidence and gaps versus adults.
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Although the incidence of pediatric Inflammatory Bowel Diseases (IBD) continues to rise globally, often presenting with more aggressive phenotypes than in adults, the therapeutic arsenal available for children lags significantly behind. Advanced therapies, pioneered by anti-TNF, have revolutionized adult IBD care. However, their application in pediatrics remains predominantly off-label, relying heavily on the extrapolation of adult evidence. This narrative review examines the critical discrepancy between adult and pediatric evidence regarding the benefit-risk balance of advanced therapies. Establishing the efficacy of treatment in children is hindered by multifactorial barriers to randomized controlled trials (RCTs), including ethical constraints, small sample sizes, and pharmacological challenges, inducing regulatory delays for marketing approval. For now, only infliximab, adalimumab, and ustekinumab are approved for the treatment of children with IBD. Regarding safety, while anti-TNF benefit-risk balance is documented, notably regarding the risk of serious infections, data remain scarce for other advanced therapies concerning major adverse events such as serious infections, malignancy, and venous thromboembolism. To fill the gap, real-world evidence (RWE), derived from large-scale population-based cohorts, can complement RCTs. The future of pediatric IBD management lies in shifting from empirical extrapolation of adult evidence to precision medicine based on evidence derived from pediatric IBD. This transition required integrating RWE with predictive modeling and shared decision-making frameworks to optimize therapeutic strategies and improve quality of life for this vulnerable population.