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

S. Cohen, EA. Spencer, MT. Dolinger, DL. Suskind, K. Mitrova, O. Hradsky, MA. Conrad, JR. Kelsen, HH. Uhlig, C. Tzivinikos, P. Henderson, M. Wlazlo, L. Hackl, DS. Shouval, M. Bramuzzo, D. Urlep, C. Olbjørn, G. D'Arcangelo, G. Pujol-Muncunill, D....

. 2025 ; 61 (8) : 1372-1380. [pub] 20250208

Jazyk angličtina Země Anglie, Velká Británie

Typ dokumentu časopisecké články, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc25009352

BACKGROUND: There are scarce data available on upadacitinib in children with Crohn's disease (CD). AIM: To evaluate the effectiveness and safety of upadacitinib as an induction therapy in paediatric CD. METHODS: This was a multicentre retrospective study between 2022 and 2024 of children treated with upadacitinib for induction of remission of active CD conducted in 30 centres worldwide affiliated with the IBD Interest and Porto group of the ESPGHAN. We recorded demographic, clinical and laboratory data and adverse events (AEs) at week 8 post-induction. The analysis of the primary outcome was based upon the intention-to-treat (ITT) principle. RESULTS: We included 100 children (median age 15.8 [interquartile range 14.3-17.2]). All were previously treated with biologic therapies including 89 with ≥ 2 biologics. At the end of the 8-week induction period, we observed clinical response, clinical remission and corticosteroid- and exclusive enteral nutrition-free clinical remission (CFR) in 75%, 56% and 52%, respectively. By the end of induction, 68% had achieved normalisation of C-reactive protein, and 58% had faecal calprotectin (FC) < 150 mcg/g. There was combined CFR and FC remission in 13/31 children with available data at 8 weeks (13% of the ITT population). AEs were recorded in 24 children; the most frequent was acne in 12. Two AEs (severe acne and hypertriglyceridemia) led to discontinuation of therapy. CONCLUSION: Upadacitinib is an effective induction therapy for refractory paediatric CD. Efficacy should be weighed against the potential risks of AEs.

Al Jalila Children's Specialty Hospital Dubai UAE

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 UK

Department of Paediatric Gastroenterology Children's Hospital HUS 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 Perelman School of Medicine at the University of Pennsylvania Philadelphia Pennsylvania USA

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

Faculty of Medicine Technion Haifa Israel

Faculty of Medicine Technion Israel Institute of Technology Haifa Israel

Faculty of Medicine Tel Aviv University Tel Aviv Israel

Gastroenterology and Nutrition Unit Meyer Children's Hospital IRCCS 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

Jenny Lind Children's Hospital Norfolk and Norwich University Hospitals Norwich UK

Norwich Medical School University of East Anglia 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 Institute Ruth Rappaport Children's Hospital Rambam Health Care Campus Israel Institute of Technology Haifa Israel

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

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

Pediatric Gastroenterology Unit Emek Medical Centre Afula Israel

Pediatric Hepatology Gastroenterology and Transplantation ASST Papa Giovanni XXIII Bergamo Italy

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

The Juliet Keidan Institute of Pediatric Gastroenterology and Nutrition Shaare Zedek Medical Center The Hebrew University of Jerusalem Jerusalem Israel

Translational Gastroenterology Unit University of Oxford Oxford UK

University of Helsinki Helsinki Finland

University of Washington Medical School Seattle Washington USA

Citace poskytuje Crossref.org

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