Upadacitinib for Induction of Remission in Pediatric Ulcerative Colitis: An International Multi‑center Study.
Yerushalmy-Feler A., Spencer EA., Dolinger MT., Suskind DL., Mitrova K., Hradsky O., Conrad MA., Kelsen JR., Uhlig HH., Tzivinikos C., Ancona S., Wlazlo M., Hackl L., Shouval DS., Bramuzzo M., Urlep D., Olbjorn C., D'Arcangelo G., Pujol-Muncunill G., Yogev D., Kang B., Gasparetto M., Rungø C., Kolho K-L., Hojsak I., Norsa L., Rinawi F., Sansotta N., Magen Rimon R., Granot M., Scarallo L., Trindade E., Velasco Rodríguez-Belvís M., Turner D., Cohen S.
BACKGROUND AND AIMS: Data on upadacitinib therapy in children with ulcerative colitis (UC) or unclassified inflammatory bowel disease (IBD-U) are scarce. We aimed to evaluate the effectiveness and safety of upadacitinib as an induction therapy in pediatric UC or IBD-U. METHODS: In this multicenter retrospective study, children treated with upadacitinib for induction of remission of active UC or IBD-U from 30 centers worldwide were enrolled. Demographic, clinical and laboratory data as well as adverse events (AEs) were recorded at week 8 post induction. RESULTS: One hundred children were included (90 UC and 10 IBD-U, median age 15.6 [interquartile range 13.3-17.1] years). Ninety-eight were previously treated with biologic therapies, and 76 were treated with ≥2 biologics. At the end of the 8-week induction period, clinical response, clinical remission, and corticosteroid-free clinical remission (CFR) were observed in 84%, 62%, and 56% of the children, respectively. Normal C-reactive protein and fecal calprotectin (FC) <150 mcg/g were achieved in 75% and 50%, respectively. Combined CFR and FC remission was observed in 18/46 (39%) children with available data at 8 weeks. AEs were recorded in 37 children, including one serious AE of an appendiceal neuroendocrine tumor. The most frequent AEs were hyperlipidemia (n=13), acne (n=12), and infections (n=10, five of whom with herpes viruses). CONCLUSION: Upadacitinib is an effective induction therapy for refractory pediatric UC and IBD-U. Efficacy should be weighed against the potential risks of AEs.