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. Adverse events were recorded in 37 children, including 1 serious AE of an appendiceal neuroendocrine tumor. The most frequent AEs were hyperlipidemia (n = 13), acne (n = 12), and infections (n = 10, 5 of whom with herpes viruses). CONCLUSIONS: Upadacitinib is an effective induction therapy for refractory pediatric UC and IBD-U. Efficacy should be weighed against the potential risks of AEs.

Al Jalila Children's Specialty Hospital Dubai United Arab Emirates

Biomedical Research Centre University of Oxford Oxford UK

Centre of Human Genetics University of Oxford Oxford UK

Children's Hospital Zagreb University of Zagreb Medical School Zagreb Croatia

Department of Gastroenterology Hepatology and Nutrition University Children's Hospital Ljubljana Ljubljana Slovenia

Department of Gastroenterology Hepatology Feeding Disorders and Pediatrics The Children's Memorial Health Institute Warsaw Poland

Department of Paediatric and Adolescence Medicine Copenhagen University Hospital Amager and Hvidovre Hvidovre Denmark

Department of Paediatric and Adolescent Medicine Akershus University Hospital Lørenskog Norway

Department of Paediatric Gastroenterology and Nutrition Royal Hospital for Children and Young People Edinburgh Scotland

Department of Paediatric Gastroenterology Children's Hospital HUS and University of Helsinki Helsinki Finland

Department of Paediatrics 2nd Faculty of Medicine Charles University and Motol University Hospital Prague Czech Republic

Department of Paediatrics University of Oxford Oxford UK

Department of Pediatric Gastroenterology and Nutrition Hospital Infantil Universitario Niño Jesús Madrid Spain

Department of Pediatric Gastroenterology Hepatology and Nutrition Hospital Sant Joan de Déu Barcelona Spain

Department of Pediatrics 1 Medical University Innsbruck Innsbruck Austria

Department of Pediatrics School of Medicine Kyungpook National University Daegu Korea

Division of Gastroenterology Hepatology and Nutrition Children's Hospital of Philadelphia Philadelphia PA USA

Division of Pediatric Gastroenterology Susan and Leonard Feinstein IBD Clinical Center Icahn School of Medicine at Mount Sinai New York NY USA

Faculty of Medical and Health Sciences Tel Aviv University Tel Aviv Israel

Faculty of Medicine Tel Aviv University Tel Aviv Israel

Gastroenterology and Nutrition Unit Meyer Children's Hospital Florence Italy

Institute for Maternal and Child Health IRCCS Burlo Garofolo Trieste Italy

Institute of Gastroenterology Nutrition and Liver Diseases Schneider Children's Medical Center of Israel Petach Tikva Israel

Norfolk and Norwich University Hospital Jenny Lind Children's Hospital and University of East Anglia Norwich Medical School Norwich UK

Pediatric Department Children's Hospital Vittore Buzzi University of Milan Milan Italy

Pediatric Gastroenterology and Liver Unit Maternal and Child Health Department Sapienza University of Rome Rome Italy

Pediatric Gastroenterology and Nutrition Unit Centro Hospitalar Universitário São João Porto Portugal

Pediatric Gastroenterology Institute Dana Dwek Children's Hospital Tel Aviv Sourasky Medical Center Tel Aviv Israel

Pediatric Gastroenterology Unit and Faculty of Medicine Technion Haifa Emek Medical Centre Afula Israel

Pediatric Gastroenterology Unit Edmond and Lily Safra Children's Hospital Sheba Medical Center Ramat Gan Israel

Pediatric Hepatology Gastroenterology and Transplantation ASST Papa Giovanni XXIII Bergamo Italy

Rambam Health Care Campus and the Faculty of Medicine Pediatric Gastroenterology and Nutrition Institute Ruth Rappaport Children's Hospital Technion Israel Institute of Technology Haifa Israel

Seattle Children's Hospital IBD Center Seattle WA USA

The Juliet Keidan Institute of Paediatric Gastroenterology and Nutrition The Eisenberg R and D Authority Shaare Zedek Medical Center The Hebrew University of Jerusalem Jerusalem Israel

Translational Gastroenterology Unit University of Oxford Oxford UK

University of Washington Medical School Seattle WA USA

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