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Multicenter Study
. 2025 Dec 1;31(12):3320-3326.
doi: 10.1093/ibd/izaf166.

Real-World Experience with Upadacitinib for Pediatric Acute Severe Ulcerative Colitis: An International Multicenter Retrospective Study from the Pediatric IBD Porto Group of ESPGHAN

Anat Yerushalmy-Feler  1   2 , Elizabeth A Spencer  3 , Suzannah Bergstein  3   4 , Katarina Mitrova  5 , Ondrej Hradsky  5 , Matteo Bramuzzo  6 , Magdalena Wlazlo  7 , Christine Olbjørn  8 , Christine Rungoe  9 , Nathaniel Weil  10 , Dan Turner  11 , Shlomi Cohen  1   2
Affiliations
Multicenter Study

Real-World Experience with Upadacitinib for Pediatric Acute Severe Ulcerative Colitis: An International Multicenter Retrospective Study from the Pediatric IBD Porto Group of ESPGHAN

Anat Yerushalmy-Feler et al. Inflamm Bowel Dis. .

Abstract

Background: Data on upadacitinib therapy for pediatric acute severe ulcerative colitis (ASC) are scarce. We aimed to evaluate the effectiveness and safety of upadacitinib as a salvage therapy in pediatric ASC.

Methods: Children and adolescents with ASC who were treated with upadacitinib for the induction of remission were enrolled in this retrospective multicenter study. Demographic, clinical, and laboratory data as well as adverse events (AEs) were recorded after the 8-week induction period and throughout 26 weeks of therapy. Analyses were based on the intention-to-treat principal.

Results: Twenty-two patients were included (median age 15.7 [interquartile range 13.5-16.6] years, 12 hospitalized), all with anti-tumor necrosis factor (TNF) therapy refractory disease. Ten patients were treated with corticosteroids at baseline, and upadacitinib was added to an ongoing biologic therapy in five patients. At week 8 of therapy, 11 (50%) patients of the cohort remained colectomy-free and in corticosteroid-free clinical remission (CFR), and 17 (77%) patients remained colectomy-free. Normal C-reactive protein (CRP) was achieved in 9 of 11 (82%) patients who were in CFR, and fecal calprotectin <150 mcg/g in 4 of 6 (67%) patients with available data. By week 26, 14 (64%) were in CFR and 16 (73%) patients remained colectomy-free. All these patients had normal CRP levels, and 4 of 7 patients with available data had fecal calprotectin <150 mcg/g. Twelve patients reported AEs, including two serious AEs of an appendiceal neuroendocrine tumor and cytomegalovirus colitis.

Conclusion: Upadacitinib is an effective induction therapy for children and adolescents with ASC after failing anti-TNF.

Keywords: JAK inhibitors; acute severe colitis; children and adolescents.

Plain language summary

This multicenter study describes 22 children with ASC treated with upadacitinib. Clinical remission was observed in 12 (55%) patients, and 11 (50%) were in colectomy-free, corticosteroid-free remission (CFR) after 8 weeks. By week 26, 14 (64%) patients were in CFR.

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Conflict of interest statement

These authors disclose the following: Last 3 years D.T. received consultation fee, research grant, royalties, or honorarium from Janssen, Pfizer, Shaare Zedek Medical Center, Hospital for Sick Children, Ferring, Abbvie, Takeda, Prometheus Biosciences, Lilly, SorrisoPharma, Boehringer Ingelheim, Galapagos, BMS, AlfaSigma, Merck. Last 3 years M.B. received consultation free from Pfizer, Pharming. E.A.S.: Nothing to disclose. Supported by NIH 5K23DK125760. O.H. received lectures/congress fees/consultancy from MSD, Abbvie, Takeda, Nutricia, Sandoz, Lilly, Pfizer.

References

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