Upadacitinib Maintenance Therapy in Pediatric Ulcerative Colitis: 52-Week Multicenter Study From the Porto Group of
Anat Yerushalmy-Feler1, Elizabeth A Spencer2, Marla C Dubinsky2
1Tel Aviv Sourasky Medical Center, Pediatric Gastroenterology Institute, "Dana-Dwek" Children's Hospital, Tel Aviv, Israel; The Gray Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv, Israel.
Insights
Upadacitinib effectively maintains remission in pediatric ulcerative colitis (UC) and inflammatory bowel disease unclassified (IBD-U). While generally safe, potential adverse events require careful consideration.
Area of Science:
- Pediatric Gastroenterology
- Immunology
- Pharmacology
Background:
- Limited data exist on upadacitinib for pediatric ulcerative colitis (UC) and unclassified inflammatory bowel disease (IBD-U).
- Evaluating upadacitinib's efficacy and safety as maintenance therapy is crucial for this population.
Purpose of the Study:
- To assess the effectiveness of upadacitinib in maintaining remission in pediatric patients with UC or IBD-U.
- To evaluate the safety profile of upadacitinib in this cohort over a 52-week period.
Main Methods:
- Retrospective study of 105 children (95 UC, 10 IBD-U) from 35 centers.
- Data collected on demographics, clinical, laboratory, endoscopic, imaging, and adverse events over 52 weeks.
- Focus on maintenance of remission in patients previously treated with biologics.
Main Results:
- High rates of clinical remission (71%) and corticosteroid-free clinical remission (CFR) (70%) by week 52.
- Sustained CFR achieved in 60% of patients.
- Common adverse events included hyperlipidemia, infections, and acne; serious events were rare.
Conclusions:
- Upadacitinib demonstrates effectiveness as maintenance therapy for refractory pediatric UC and IBD-U.
- The benefits of upadacitinib should be balanced with its potential adverse events.
- Further research may explore long-term outcomes and specific AE management strategies.
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.
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