Effectiveness and safety of biologics in pediatric inflammatory bowel disease: Real-life data from the Sicilian Network

ABSTRACT Introduction. The incidence and prevalence of pediatric inflammatory bowel disease (IBD) are rising worldwide, with a steep increase in children under 5 years of age. Compared to adult IBD, pediatric IBD presents with a more severe, aggressive phenotype and unique complications, notably growth impairment. The advent of anti–tumor necrosis factor (TNF) α agents has radically modified the management and disease course of IBD, resulting in greater remission and mucosal healing rates, fewer surgeries and hospitalizations, improved quality of life, and, notably for children, correction of growth failure, all while limiting drug toxicities. Objective: The aim of the study was to describe real-world experience with biologics, focusing on their effectiveness and safety, in pediatric IBD patients. Material and methods: Statistical analyses of the multicenter registry data from the Sicilian Network for Inflammatory Bowel Disease (SN-IBD) were performed for patients receiving biologics, with at least a follow up period of 26 weeks. The study population consisted of 93 children, divided into the study groups separately: 87 children (63CD, 24 UC) aged 7-17 years and 6 children (1 CD, 3 UC, 2 IC) who received biologics before the age of 7. Clinical benefit and safety were evaluated for each biologic agent used (Infliximab — IFX, Adalimumab — ADA, Golimumab — GOL). Results and conclusion: The research focused on 101 biologic treatments performed in the group 7—17 years children (63 Crohn’s disease [CD], 24 Ulcerative colitis [UC]), who received, 74 biologic treatments for CD, evaluated at 26, 52 and 104 weeks, that showed clinical benefit rates of 84.2%, 93.3%, and 66.7% with IFX (n = 38), and 88.9%, 84.4%, 65.2% with ADA (n= 36). Biologic treatments (n = 27) evaluated in the UC group at 26, 52, 104 weeks, led to clinical benefit rates of 85.7%, 83.3%, 50% in IFX subgroup (n = 21) and 40%, 50%, 33% in the ADA subgroup (n = 5), respectively. One patient treated with GOL showed 100% clinical benefit at 26 and 52 weeks. Overall adverse events (AEs) rate in this group of children was 9.25%. Effects of other 8 biologic treatments were studied in six younger children, aged < 6 years, (4 ADA, 4 IFX), who presented a clinical remission rate of 75% at 12 weeks and 25% at 52 weeks. AEs rate was 25% in this group. Conclusion: Our data show that biologic therapy in children, even at a younger age, is effective in allowing long-term remission with a good safety profile.