Efficacy of Modified Upadacitinib Dosing in Pediatric Refractory IBD: A Case-Based Analysis.

Abstract

This case report describes four pediatric patients with severe, therapy-refractory ulcerative colitis (UC) and one pediatric patient with severe, therapy-refractory Crohn's disease (CD), all of whom failed widely used biologic therapies, including the approved treatments infliximab and adalimumab, as well as off-label use of vedolizumab and ustekinumab. Despite these first- and second-line interventions, patients' diseases remained active. Following the initiation of upadacitinib, an off-label Janus kinase (JAK) inhibitor with positive results in adult UC and CD patients, four children experienced rapid symptom improvement and achieved clinical remission. The rising incidence of pediatric inflammatory bowel disease (IBD) and the limited number of approved therapies underscore the need for additional treatment options in pediatric gastroenterology. Infliximab remains one of the only biologic therapies approved for pediatric UC and CD, forcing clinicians to rely on off-label medications, such as vedolizumab, ustekinumab, and upadacitinib, when standard treatments fail. To address this gap, it is crucial to include pediatric patients in clinical trials of new therapies, expanding the range of approved medications and improving outcomes for children with IBD.