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

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