Promoting clinical trials in pediatric IgA nephropathy through extrapolation: an artificial intelligence-enhanced literature review of the disease in pediatrics sponsored by the Kidney Health Initiative.

BACKGROUND: There is an urgent need to evaluate the efficacy of novel therapeutics that have been approved for use in adults with IgA nephropathy (IgAN) in pediatric patients with this primary glomerular disease. METHODS: To explore the feasibility of deploying extrapolation to promote clinical trials in pediatric patients with IgA nephropathy (IgAN), an artificial intelligence (AI)-enhanced review of the published literature through 2023 was conducted to characterize the epidemiology and natural history of IgA nephropathy, summarize the clinical trial experience, and identify potential biomarkers for use in patient care and clinical trials. RESULTS: Using Distiller SR® software to identify relevant articles and Elicit®, an artificial intelligence tool to extract content, 83 articles were included in the review. The clinical trial reports (n = 9) and biomarker studies (n = 33) were limited in scope. The natural history and epidemiology articles (n = 41) suggested that the severity of kidney histopathology at the time of diagnosis indicated a higher risk of disease progression while proteinuria < 0.5-1 mg/mg creatinine was associated with a more favorable prognosis. CONCLUSIONS: In conjunction with the similar biological basis of the disease in pediatric and adult patients, these findings may enable the use of extrapolation in the design of clinical trials in pediatric patients with IgAN.