A219 SHORT-TERM OUTCOMES OF UPADACITINIB IN PEDIATRIC IBD: A RETROSPECTIVE REVIEW

TL;DR

Upadacitinib, a drug for adult IBD, shows promise in children, with most achieving symptom remission and reduced steroid use in a small study.

Contribution

First retrospective analysis of upadacitinib's short-term outcomes in pediatric IBD patients.

Findings

85.7% of pediatric IBD patients achieved clinical remission at 8 weeks with upadacitinib.

Significant reductions in symptoms and biomarkers like CRP and calprotectin were observed.

Most remissions were steroid-free, and no serious adverse events were reported.

Abstract

Pediatric inflammatory bowel disease (IBD) requires innovative treatments to improve symptoms and reduce steroid use. Upadacitinib, a selective JAK-1 inhibitor, has shown efficacy in adults but pediatric data remain limited

To assess clinical remission at 8 weeks in pediatric IBD patients treated with upadacitinib. Secondary aims include evaluating clinical response, steroid reduction, symptom improvement, biomarker changes, and adverse events

This retrospective review included pediatric UC and CD patients treated with upadacitinib (45 mg daily for 8-12 weeks). Clinical remission was defined as wPCDAI/PUCAI ≤10 at 8 weeks. Secondary outcomes included clinical response (wPCDAI decrease >17.5 for CD, PUCAI ≥20 for UC), steroid reduction, symptom scores (abdominal pain, urgency, rectal bleeding, stool frequency), biomarkers (CRP, calprotectin), and adverse events. The Naranjo scale assessed adverse event probability

Seven patients (5 CD, 2 UC) with complete 8-week data were analyzed. Median age was 16 years (IQR: 14-17), with a 3:4 male-to-female ratio

At baseline, 4 patients were on both upadacitinib and steroids, and 3 on upadacitinib alone

At 8 weeks, 3/4 on steroids achieved remission, with 1 fully weaned

All 3 patients on upadacitinib alone achieved steroid-free remission

85.7% (6/7) achieved remission at 8 weeks, p = 0.01

33.3% (2/6) of remissions remained on steroids, and 66.7% (4/6) were steroid-free

Secondary outcomes:

85.7% (6/7) achieved response, with wPCDAI/PUCAI scores improving from 40 (IQR: 30-52.5) to 5 (IQR: 0-15) CD scores improved from 52.5 (IQR: 40-60) to 7.5 (IQR: 0-25), and PUCAI from 32.5 (IQR: 25-40) to 0 (IQR: 0-0), p = 0.01

4 patients were on steroids at baseline, with 1/4 fully weaned by 8 weeks

Baseline prednisone: 40 mg (IQR: 30-50) vs. 25 mg (IQR: 10-30) at 8 weeks, p = 0.02

Abdominal pain: 2 (IQR: 1-3) to 0 (IQR: 0-0), p = 0.03

Urgency NRS: 5 (IQR: 3-6) to 0 (IQR: 0-2), p = 0.01

Rectal bleeding: 2 (IQR: 0-3) to 0 (IQR: 0-0), p = 0.04

Stool frequency: 2 (IQR: 1-3) to 0 (IQR: 0-1), p = 0.02

CRP: 25 mg/L (IQR: 3.1-39) to 10 mg/L (IQR: 2-18), p = 0.03

Calprotectin: 2000 µg/g (IQR: 580-5449) to 800 µg/g (IQR: 74-1378), p = 0.04

71.4% (5/7) experienced acne, 28.6% (2/7) headaches, and 28.6% (2/7) nausea

No serious adverse events were reported. Median Naranjo score: 2

Upadacitinib improved symptoms and reduced steroid use in pediatric IBD, with 85.7% (6/7) achieving remission at 8 weeks. Of these, 66.7% (4/6) achieved steroid-free remission. Clinical response was achieved in 85.7% (6/7), with significant improvements in both UC and CD. Mild adverse events were reported, supporting further exploration of upadacitinib in pediatric IBD. Additional patients will be recruited in the coming months to expand this dataset

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