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), upa...

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Main Authors: Yassin EL‐Najjar, Mary‐Joe Touma, Shuai Tan, Xiaoqin Zhou, Qi Liu
Format: Article
Language:English
Published: Wiley 2025-06-01
Series:Pediatric Discovery
Subjects:
Online Access:https://doi.org/10.1002/pdi3.70011
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author Yassin EL‐Najjar
Mary‐Joe Touma
Shuai Tan
Xiaoqin Zhou
Qi Liu
author_facet Yassin EL‐Najjar
Mary‐Joe Touma
Shuai Tan
Xiaoqin Zhou
Qi Liu
author_sort Yassin EL‐Najjar
collection DOAJ
description 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.
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institution Kabale University
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publisher Wiley
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series Pediatric Discovery
spelling doaj-art-9bd8fd593a3a4b6fbc27bb1e27bc23a92025-08-20T03:27:36ZengWileyPediatric Discovery2835-558X2835-55982025-06-0132n/an/a10.1002/pdi3.70011A Review of the Current State of Off‐Label Therapies for Pediatric Inflammatory Bowel DiseaseYassin EL‐Najjar0Mary‐Joe Touma1Shuai Tan2Xiaoqin Zhou3Qi Liu4Department of Internal Medicine University of Texas Southwestern Medical Center Dallas Texas USADepartment of Internal Medicine University of Texas Southwestern Medical Center Dallas Texas USADepartment of Internal Medicine University of Texas Southwestern Medical Center Dallas Texas USADepartment of Gastroenterology Children's Hospital of Chongqing Medical University National Clinical Research Center for Child Health and Disorders Ministry of Education Key Laboratory of Child Development and Disorders China International Science and Technology Cooperation Base of Child Development and Critical Disorders Chongqing Key Laboratory of Child Rare Diseases in Infection and Immunity Chongqing ChinaDepartment of Internal Medicine University of Texas Southwestern Medical Center Dallas Texas USAABSTRACT 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.https://doi.org/10.1002/pdi3.70011biologic therapyCrohn’s disease (CD)pediatric inflammatory bowel disease small moleculeulcerative colitis (UC)
spellingShingle Yassin EL‐Najjar
Mary‐Joe Touma
Shuai Tan
Xiaoqin Zhou
Qi Liu
A Review of the Current State of Off‐Label Therapies for Pediatric Inflammatory Bowel Disease
Pediatric Discovery
biologic therapy
Crohn’s disease (CD)
pediatric inflammatory bowel disease 
small molecule
ulcerative colitis (UC)
title A Review of the Current State of Off‐Label Therapies for Pediatric Inflammatory Bowel Disease
title_full A Review of the Current State of Off‐Label Therapies for Pediatric Inflammatory Bowel Disease
title_fullStr A Review of the Current State of Off‐Label Therapies for Pediatric Inflammatory Bowel Disease
title_full_unstemmed A Review of the Current State of Off‐Label Therapies for Pediatric Inflammatory Bowel Disease
title_short A Review of the Current State of Off‐Label Therapies for Pediatric Inflammatory Bowel Disease
title_sort review of the current state of off label therapies for pediatric inflammatory bowel disease
topic biologic therapy
Crohn’s disease (CD)
pediatric inflammatory bowel disease 
small molecule
ulcerative colitis (UC)
url https://doi.org/10.1002/pdi3.70011
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