Safety and Efficacy of Upadacitinib for Pediatric Patients with Polyarticular Course Juvenile Idiopathic Arthritis: An Interim Analysis of an Open-Label, Phase-1 Trial

Join Ingrid Pan, PharmD, Pediatric Clinical Pharmacist at Children’s Hospital Colorado, as she reviews the latest findings from the Phase 1 trial of Upadacitinib (Rinvoq) in treating pediatric polyarticular course juvenile idiopathic arthritis (JIA). This interim analysis, published in Annals of the Rheumatic Diseases, explores the potential of JAK1-selective inhibition in pediatric rheumatology.

This open-label, three-part Phase 1 study evaluated pharmacokinetics, safety, and efficacy in patients aged 2-18 across North America, Europe, and Asia. Results demonstrated promising clinical responses, with an ACR 30 response rate of 92% at Week 12, ACR 50 response of 90%, and ACR 70 response of 69%. Safety data indicated mild-to-moderate adverse events, including upper respiratory tract infections, COVID-19 infections, gastroenteritis, neutropenia, and CPK elevations.

With FDA approval and orphan drug designation granted in June 2024, Upadacitinib represents a potential new treatment option for pediatric JIA, offering targeted JAK1 inhibition to improve disease outcomes.

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