Upadacitinib Maintenance Therapy in Pediatric Ulcerative Colitis: 52-Week Multicenter Study From the Porto Group of the European Society for Paediatric Gastroenterology, Hepatology, and Nutrition

Background & 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 a maintenance therapy in pediatric UC. Methods Children treated with upadacitinib for maintenance of remission of active UC or IBD-U from 35 centers affiliated with the Porto group of the European Society for Paediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN) were enrolled in this retrospective study. Data on demographic, clinical, laboratory, endoscopic, imaging, and adverse events (AEs) were recorded over 52 weeks of follow-up. Results A total of 105 children were included (95 with UC and 10 with IBD-U; mean age, 14.6 ± 3.3 years). Prior to upadacitinib, 103 of 105 children (98%) were treated with biologic therapies and 79 (75%) with ≥2 biologics. Clinical remission and corticosteroid-free clinical remission (CFR) were observed after 8 weeks in 61 (58%) and 53 (51%) children, respectively. By week 52, 75 children (71%) achieved clinical remission and 73 (70%) achieved CFR. Sustained CFR was recorded in 63 children (60%). CFR with normal C-reactive protein was observed in 56% of children, and CFR with fecal calprotectin levels <150 mcg/g was observed in 38%, by week 52. Fifty-two children (50%) experienced AEs, 2 of which were serious (an appendiceal neuroendocrine tumor and cytomegalovirus colitis). The most frequent AEs were hyperlipidemia (n = 20), infections (n = 18), and acne (n = 14). Conclusions Upadacitinib is an effective induction and maintenance therapy for refractory pediatric UC and IBD-U. Effectiveness should be weighed against the potential risks of AEs.