Effectiveness and safety of ustekinumab in pediatric Crohn's disease: Results of the REALITI study

OBJECTIVES: Few approved treatments exist for children with Crohn's disease (CD). The REALITI study retrospectively assessed the effectiveness and safety of ustekinumab in real-world clinical settings for children with CD. METHODS: Data were collected from the prospective ImproveCareNow (ICN) registry for pediatric patients (≥ 2 to <18 years old) and young adult patients (≥ 18 to <26 years old), regardless of baseline CD severity. Additional analyses were conducted for a subset of patients who had moderately-to-severely active CD (short pediatric CD activity index [sPCDAI] ≥30). Key Week-52 endpoints included clinical remission (sPCDAI ≤10) and corticosteroid-free (CF) clinical remission. Safety events of interest were assessed at Week 52. RESULTS: Overall, 479 patients with CD were treated with ustekinumab, 348 pediatric patients and 131 young adults; most were biologic-exposed (pediatric, 98.9%; young adult, 95.4%). At Week 52 (observed case; excluding patients without Week 52 data), clinical remission was achieved by 47.3% (125/264) of pediatric patients and 44.8% (39/87) of young adults, and CF clinical remission by 41.3% (109/264) and 39.1% (34/87), respectively. At Week 52 (observed case), among patients with moderately-to-severely active CD, clinical remission was achieved by 36.9% (41/111) of pediatric patients and 34.3% (12/35) of young adults, and CF clinical remission by 31.5% (35/111) and 28.6% (10/35), respectively. Ustekinumab was well tolerated, with no new safety signals identified. CONCLUSIONS: In the REALITI study of real-world data from the ICN registry, the effectiveness and safety of ustekinumab treatment through 52 weeks were similar in pediatric and young adult patients with CD. TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT05242458; https://clinicaltrials.gov/study/NCT05242458.