Search

Background and Objectives: Current data on ustekinumab therapy in children with ulcerative colitis (UC) or unclassified inflammatory bowel disease (IBDU) are limited. We aimed to evaluate the effectiveness and safety of ustekinumab in pediatric UC and IBDU., Methods: This multicenter retrospective study included 16 centers affiliated with the IBD Interest and Porto groups of ESPGHAN. Children with UC or IBDU treated with ustekinumab were enrolled. Demographic, clinical, laboratory, endoscopic, and imaging data as well as adverse events were recorded. Analyses were all based on the intention-to-treat principle., Results: Fifty-eight children (39 UC and 19 IBDU, median age 14.5 [IQR 11.5-16.5] years) were included. All had failed biologic therapies, and 38 (66%) had failed two or more biologics. Corticosteroid-free clinical remission (CFR) was observed in 27 (47%), 33 (57%), and 37 (64%) children at 16, 26, and 52 weeks, respectively. Normalization of C-reactive protein and calprotectin < 150 μg/g were achieved in 60% and 52%, respectively, by 52 weeks. Endoscopic and radiologic remissions were reached in 8% and 23%, respectively. The main predictors of CFR were diagnosis of UC compared with IBDU (hazard ratio [HR] 2.2, 95% CI 1.03-4.85; p = 0.041) and no prior vedolizumab therapy (HR 2.1, 95% CI 1.11-4.27; p = 0.023). Ustekinumab serum levels were not associated with disease activity. Adverse events were recorded in six (10%) children, leading to discontinuation of the drug in three., Conclusion: Based on these findings, ustekinumab appears as an effective therapy for pediatric refractory UC and IBDU. The potential efficacy should be weighed against the risks of serious adverse events., (© 2024. The Author(s).)

Objective: The aim was to assess the awareness and real-life use of biosimilars in inflammatory bowel disease (IBD) among the members of the Italian Society of Pediatric Gastroenterology, Hepatology and Nutrition (SIGENP)., Methods: An anonymous web survey involving all SIGENP IBD units which can prescribe biosimilars was conducted between July 1st and December 1st, 2020. The questionnaire included 18 questions addressing the most relevant aspects of biosimilars in pediatric IBD, i.e., advantages, disadvantages, costs, traceability, general knowledge, and real-life use. A descriptive analysis of responses was performed., Results: Responses came from 26 pediatric IBD units in Italy, with representation of the North, the Center, and the South of Italy. The majority of participants (n = 20) had spent > 10 years caring for pediatric IBD patients, and worked in a center which had between 100 and 500 registered pediatric IBD patients (n = 14). Most participants (n = 18) reported they were aware that biosimilars have similar efficacy and safety to those of the originator, and all regarded cost-sparing as the main advantage of biosimilars. Most respondents (n = 20) reported they switch from originator to biosimilar in their everyday clinical practice, mostly during the maintenance phase. Most respondents (n = 20) registered no acute adverse events. Nearly all participants felt totally or very confident in using biosimilars., Conclusions: A few years after the introduction of the first biosimilar into the market, real-life data coming from the major IBD units in Italy confirm a favorable and confident position on the use of biosimilars in pediatric IBD., (© 2021. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)