TY - JOUR
T1 - Effectiveness and Safety of Ustekinumab in Pediatric Ulcerative Colitis
T2 - A Multi-center Retrospective Study from the Pediatric IBD Porto Group of ESPGHAN
AU - Cohen, Shlomi
AU - Rolandsdotter, Helena
AU - Kolho, Kaija Leena
AU - Turner, Dan
AU - Tzivinikos, Christos
AU - Bramuzzo, Matteo
AU - Pujol-Muncunill, Gemma
AU - Scarallo, Luca
AU - Urlep, Darja
AU - Rinawi, Firas
AU - Granot, Maya
AU - Kang, Ben
AU - Longueville, Ylva
AU - Rodríguez-Belvís, Marta Velasco
AU - Weintraub, Yael
AU - Navas-López, Víctor Manuel
AU - Yerushalmy-Feler, Anat
N1 - Publisher Copyright:
© The Author(s) 2024.
PY - 2024/9
Y1 - 2024/9
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85193937210&partnerID=8YFLogxK
U2 - 10.1007/s40272-024-00631-z
DO - 10.1007/s40272-024-00631-z
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C2 - 38780740
AN - SCOPUS:85193937210
SN - 1174-5878
VL - 26
SP - 609
EP - 617
JO - Paediatric Drugs
JF - Paediatric Drugs
IS - 5
ER -