P0668Real-World Effectiveness & Safety of Risankizumab Induction and Maintenance Therapy in Paediatric Patients with Crohn’s Disease: A Multicenter Retrospective Analysis

Abstract Background Risankizumab, a selective interleukin-23 (p19) inhibitor, is approved for adults with Crohn’s disease (CD)1-2, but paediatric evidence is limited. We assessed real-world effectiveness, durability, safety and dosing of risankizumab in children with CD across multiple international centres. Methods Retrospective, multicentre cohort of patients 18 years initiating risankizumab monotherapy (2022–2025) at ten centres in North America, Europe, and the Middle East. The primary endpoint was corticosteroid- and exclusive enteral nutrition (EEN)-free clinical remission (CFR; weighted Paediatric Crohn’s Disease Activity Index wPCDAI 12.5) after induction (Week 12) among those with baseline active disease (wPCDAI ≥12.5). Secondary endpoints: clinical response (≥17.5-point wPCDAI decrease), C-reactive protein (CRP) remission (5 mg/L), combined CFR+CRP remission, CFR at Weeks 30/54, intestinal ultrasound (IUS) remission, treatment durability (on-treatment persistence; Kaplan–Meier), and adverse events (AEs). Logistic and Cox models explored predictors of post-induction CFR and durability. Results Ninety-six children were included (Table, median age 15 IQR 13–16 years; 51% male; 70% ileocolonic; 38% previously exposed to ustekinumab). Most (93/96, 97%) received adult induction; three 30 kg received 383 IQR 339–397 mg/m². At baseline, 68/96 (71%) had clinically active disease. Post-induction outcomes (active-disease cohort): CFR 28/68 (41%); clinical response 37/68 (54%); CRP remission 45/68 (66%); combined CFR+CRP 22/68 (32%). Maintenance (active-disease cohort): CFR 22/41 (54%) at Week 30 and 13/27 (48%) at Week 54; among post-induction remitters, 16/17 (94%) and 10/13 (77%) remained in remission at Weeks 30 and 54, respectively. Full cohort: median follow-up 33 weeks IQR 14–57; 84/96 (88%) remained on treatment. Durability was greater with post-induction CFR (Figure, log-rank p = 0.0019); adjusted hazard ratio for discontinuation 0.14 (95% CI 0.03–0.69; p = 0.015). Lower baseline erythrocyte sedimentation rate predicted higher odds of CFR (OR 0.97 per mm/hr; 95% CI 0.94–0.99; p = 0.01). Objective measures: IUS remission occurred in 6/15 (40%) with baseline IUS activity and aligned with CFR (p = 0.03). Safety: No serious infections, malignancies, or thromboembolic events. Conclusion In this first global paediatric cohort, risankizumab achieved meaningful induction and maintenance effectiveness with high on-treatment durability and a favourable safety profile. Baseline inflammatory burden influenced early response, and achieving post-induction CFR strongly predicted persistence. Findings support risankizumab as an effective option for children with moderate-to-severe CD and inform prospective paediatric IL-23 studies. References: 1. Ferrante M, Panaccione R, Baert F, et al. Risankizumab as maintenance therapy for moderately to severely active crohn’s disease: Results from the multicentre, randomised, double-blind, placebo-controlled, withdrawal phase 3 fortify maintenance trial. Lancet 2022;399:2031-46. 2. D’Haens G, Panaccione R, Baert F, et al. Risankizumab as induction therapy for crohn’s disease: Results from the phase 3 advance and motivate induction trials. Lancet 2022;399:2015-30. Conflict of interest: Dr. Spencer, Elizabeth: Advisory Board for Celltrion. Buck, Quentin: No conflict of interest Collen, Lauren: No conflict of interest Stein, Ronen: Research funding from Pfizer Babinski, Tyler: No conflict of interest Shouval, Dror: Lecturing fee - Takeda SAB - Tracells Rinawi, Firas: No conflict of interest Russell, Richard K.: Grant: Nestec Other: Abbvie, Celltrion, Janssen, Lilly, Nestle, Pharmacosmos, Pfizer Zifman, Eyal: No conflict of interest Topf Olivestone, Chani: No conflict of interest Tzvinikos, Christos: No conflict of interest Slae, Mordechai: No conflict of interest Lev Zion, Raffi: No conflict of interest Turner, Dan: Consultation fee: Janssen, Pfizer, Ferring, Abbvie, Takeda, Prometheus Biosciences, Lilly, SorrisoPharma, Boehringer Ingelheim, Galapagos, BMS, AlfaSigma, Merck, Gentech Research support: Janssen, Abbvie, Takeda, Pfizer Royalties: Shaare Zedek Medical Center, Hospital for Sick Children Dubinsky, Marla C: Personal Fees: Consultant or Advisory Board: Abbvie, Abivax, Astra Zeneca, BMS, Celltrion, Gilead, Genentech, Janssen, Johnson and Johnson, Lilly, Merck, Pfizer, Prometheus Biosciences, Sanofi, Spyre, Target RWE, Takeda Other: Shareholder, Co-founder, Board of Directors of Trellus Health Co-Founder Mi Test Health Assa, Amit: No conflict of interest