A Review of the Current State of Off-Label Therapies for Pediatric Inflammatory Bowel Disease

Abstract

Off-label use of biologic therapies in patients with pediatric inflammatory bowel disease (IBD) has seen an increase in utilization. In this paper, we review the current state of off-label therapies in the pediatric IBD population. Real-world use of ustekinumab (UST), vedolizumab (VDZ), upadacitinib (UPA), tofacitinib, and ozanimod in the adult population could prove positive outcomes in the pediatric population. Established off-label therapies inch closer to comparable safety, efficacy, and outcomes in pediatric IBD use. Outcomes and use of newer biologic therapies in patients with pediatric IBD have improved with increased rates of steroid-free clinical remission (SFCR). Novel therapies, including Janus kinase (JAK) inhibitors and sphingosine-1-phosphate receptor (S1Pr) modulators, require further studies but could also prove effective.

Keywords

biologic therapy / Crohn’s disease (CD) / pediatric inflammatory bowel disease / small molecule / ulcerative colitis (UC)

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2025 The Author(s). Pediatric Discovery published by John Wiley & Sons Australia, Ltd on behalf of Children's Hospital of Chongqing Medical University.