Related Experiment Video
Updated: Jun 26, 2025

Scaled-Up Preparation of an Intermediate of Upatinib, ACT051-3
Published on: April 7, 2023
Rescue therapy with upadacitinib in medically refractory pediatric ulcerative colitis
Maria Miller1,2, Ashish S Patel1,2, Brad Pasternak1,2
1Division of Pediatric Gastroenterology Phoenix Children's Hospital Phoenix Arizona USA.
Insights
Janus kinase (JAK) inhibitors may offer a new treatment for pediatric inflammatory bowel disease (IBD). A 13-year-old with refractory ulcerative colitis (UC) achieved remission with a JAK inhibitor, avoiding colectomy.
Area of Science:
- Gastroenterology
- Immunology
- Pediatric Medicine
Background:
- Pediatric inflammatory bowel disease (IBD) treatment options are limited.
- Janus kinase (JAK) inhibitors are approved for adult IBD but their efficacy in pediatric populations is not well-established.
Observation:
- A 13-year-old female patient presented with severe ulcerative colitis (UC) refractory to multiple therapies including 5-aminosalicylate, azathioprine, adalimumab, vedolizumab, and ustekinumab.
- Despite extensive treatment, the patient experienced persistent pancolitis and required hospitalization, with the family declining colectomy.
Findings:
- The patient was initiated on upadacitinib, a JAK inhibitor.
- Within one week of starting upadacitinib, her symptoms resolved, leading to a steroid-free remission.
Implications:
- This case suggests that JAK inhibitors may be a viable advanced therapy option for pediatric patients with extensively refractory UC.
- Early consideration of JAK inhibitors could potentially prevent the need for colectomy in select pediatric IBD cases.
Abstract:
Approved options for advanced therapy in pediatric inflammatory bowel disease (IBD) are limited. Although Janus kinase (JAK) inhibitors are approved in adult IBD, their benefit in pediatric populations is not yet delineated. We present a 13-year-old female patient with ulcerative colitis (UC) refractory to numerous therapies and courses of prednisone that ultimately responded to a JAK inhibitor. Initial treatment consisted of 5-aminosalicylate and azathioprine. This was changed to adalimumab due to persistent symptoms. Repeat colonoscopy revealed pancolitis, thus she was transitioned to vedolizumab. She was hospitalized twice for uncontrolled symptoms on vedolizumab and subsequent scope showed continued pancolitis. As a result, she transitioned to ustekinumab without symptomatic relief after adjusting to monthly dosing. The family declined colectomy, opting to exhaust all medical therapies. Upadacitinib was started and her symptoms resolved within 1 week, and she remains in steroid-free remission. This case illustrates the possible role of JAK inhibitors in extensively refractory pediatric UC patients before colectomy.
More Related Videos
Related Concept Videos
Drugs for Treatment of Ulcerative Colitis in IBD
Drugs for Treatment of Crohn's Disease in IBD Using Immunomodulatory Agents
Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids
Inflammatory Bowel Disease V: Surgical Management
Here are some common surgical interventions for IBD:
Inflammatory Bowel Disease IV: Pharmacological Management
Pharmacologic...
Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF

