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Upadacitinib for Induction of Remission in Paediatric Crohn's Disease: An International Multicentre Retrospective

Shlomi Cohen1, Elizabeth A Spencer2, Michael T Dolinger2

  • 1Pediatric Gastroenterology Institute, "Dana-Dwek" Children's Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel and the Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv, Israel.

Alimentary Pharmacology & Therapeutics
|February 8, 2025
PubMed

Insights

Upadacitinib shows effectiveness in treating paediatric Crohn's disease (CD), with significant clinical response and remission rates observed. However, potential adverse events require careful consideration alongside its benefits.

Area of Science:

  • Pediatric Gastroenterology
  • Immunomodulatory Therapies
  • Inflammatory Bowel Disease Research

Background:

  • Limited data exists on upadacitinib's use in pediatric Crohn's disease (CD).
  • Assessing upadacitinib's role in pediatric CD is crucial for treatment advancement.

Purpose of the Study:

  • To evaluate the effectiveness of upadacitinib as an induction therapy in children with active Crohn's disease.
  • To assess the safety profile of upadacitinib in this pediatric population.

Main Methods:

  • Multicenter retrospective study involving 100 children with active CD across 30 global centers.
  • Data collected included demographics, clinical outcomes, laboratory markers, and adverse events at 8 weeks post-induction.
  • Intention-to-treat (ITT) analysis was used for primary outcome assessment.

Main Results:

  • Upadacitinib induction therapy achieved clinical response in 75% and clinical remission in 56% of pediatric CD patients.
  • Significant improvements were noted in C-reactive protein normalization (68%) and fecal calprotectin levels (<150 mcg/g in 58%).
  • Adverse events occurred in 24% of patients, with acne being the most common; two severe events led to discontinuation.

Conclusions:

  • Upadacitinib demonstrates efficacy as an induction therapy for refractory pediatric Crohn's disease.
  • The benefits of upadacitinib should be balanced against potential adverse events.
  • Further research may explore long-term outcomes and safety in pediatric CD patients.
Abstract

Keywords:
JAK inhibitorschildreninflammatory bowel disease

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