P1063 Comparison of all paediatric patients and young adults with Crohn’s Disease treated with ustekinumab in the REALITI Real-World Evidence Effectiveness Study

Abstract

Abstract Background Treatment of paediatric patients (pts) with Crohn’s disease (CD) can offer some challenges to providers. There is an urgent need to inform prescribers about paediatric efficacy, safety, and dosing. REALITI evaluated the effectiveness and safety of ustekinumab (UST) routine clinical care in paediatric pts (age 2 to <18) regardless of disease severity or baseline corticosteroid use. Reference data are shown for young adults (age 18 to 25) with CD, for whom UST is approved. Data were obtained from the ImproveCareNow (ICN) Registry. Methods Data from pts from ICN with CD initiating treatment with UST between 10 January 2010 and 29 February 2020 were evaluated. A supplemental, retrospective chart review collected data not in ICN. The primary endpoint was clinical remission (Short Paediatric CD Activity Index [sPCDAI] score ≤10) at Week (Wk) 52. Data were summarized descriptively, and the proportion of pts achieving clinical remission and associated 2-sided 95% confidence intervals (CI) were calculated along with rates of inflammatory bowel disease (IBD)-related hospitalization and surgeries and serious/opportunistic infections. Results Overall, 479 pts in ICN with CD were treated with UST. We report an analysis of paediatric pts (n=348) compared to young adults (n=131). Most paediatric pts (98.9%) and young adults (95.4%) had received prior biologic therapy, with approximately half (47.1% and 50.4%, respectively) receiving corticosteroids at baseline. Slightly higher proportions of pts had moderate to severe disease (sPCDAI ≥30) among paediatric pts (49.7%) vs young adults (44.8%). Clinical remission at Wk52 was achieved in 30.2% of paediatric pts (105/348; 95% CI: [25.6%, 35.2%]) vs 28.2% of young adults (37/131; 95% CI: [21.2%, 36.5%]) (Figure 1A). Clinical remission rates decreased slightly over time from Wk 20-52 (Figure 1B); discontinuation rates through WK 52 remained low and were similar between groups (21.0%, 22.9%, respectively). Overall, 31.9% of paediatric pts and 19.8% of young adults had IBD-related hospitalizations; IBD-related surgery was reported in 16.1% and 9.2% of pts, respectively. Serious infections occurred in 7.5% of paediatric pts and 3.8% of young adults. Rates of opportunistic infections were low (1.4%, 0%, respectively). No events of tuberculosis, malignancy, or anaphylaxis requiring UST discontinuation occurred. One death, deemed by investigators as unrelated to IBD or UST treatment, was reported among paediatric pts; no deaths were reported among young adults. Conclusion Using real-world data from the ICN Registry, the remission rates for using UST to treat CD were found to be generally comparable between paediatric pts and young adults. No new safety signals were identified.