OP18The UNITI Jr Study: Safety and efficacy results of ustekinumab in paediatric patients with Crohn’s Disease

Abstract Background Ustekinumab therapy induced and maintained response and remission in adult patients with moderate-to-severe Crohn’s disease (CD) in the IM-UNITI program.1,2 The UNITI Jr study (NCT04673357) evaluated efficacy and safety of ustekinumab in paediatric participants with moderately-to-severely-active CD. Methods 101 participants (≥2 – 18 years; PCDAI score 30; inadequate response/intolerant to conventional/biologic therapy or corticosteroid-dependent) received 1 open-label intravenous ustekinumab (Induction) dose. At Week (W)8, 97 participants were randomized (1:1) stratified by baseline body weight (40kg/≥40kg) and response status (response: PCDAI decline ≥12.5 points with a total PCDAI score not 30/non-responder PCDAI decline 12.5) to receive blinded subcutaneous ustekinumab maintenance therapy every 8- or 12-weeks (Q8W/Q12W) for 44-weeks. Ustekinumab dosing was based on body-surface-area (for participants 40kg) or weight-tiered (for participants ≥40kg). Primary endpoints were clinical remission (PCDAI ≤10) at W8, and at W52 among randomized, induction responders. Analysis was modified intent-to-treat (only includes responders at W8 before randomization). Results In UNITI Jr, 101 participants were enrolled (59.4% male; median IQR age 14.0 12.0-15.0 years; 42.6% biologic-naive; median IQR PCDAI score 40.0 35.0-45.0); at W8, 47 (46.5%) participants achieved clinical remission. Among participants who were in clinical remission at W8, 32/47 (68.1%) maintained clinical remission at W52. At W8, 85 (84.2%) achieved clinical response, of whom at W52, 46 (54.1% 95%CI: 43.6%-64.3%) achieved clinical remission and 45 (52.9% 95% CI: 42.4%-63.2%) achieved corticosteroid-free clinical remission. Not having previously failed biologic therapy was associated with higher W52 remission rates (Figure 1A). Remission rates were similar from W8 to W52 in all weight subgroups (Figure 1B). During maintenance, the efficacy was similar between Q8W and Q12W groups. Adverse events (AE)/serious adverse events (SAE) rates were similar between Q8W/Q12W groups. SAE occurred in 16.8% (17/101) of participants, of which CD exacerbation was most frequent (5/17); serious infections occurred in 5.9%. Conclusion Ustekinumab was well-tolerated with no new safety signals. Induction and maintenance therapy with ustekinumab is effective in treating moderate-to-severe paediatric CD. References: 1.Feagan BJ, et al. N Engl J Med. 2016;375(20):1946-1960. 2. Sandborn WJ, et al. Clin Gastroenterol Hepatol. 2022;20(3):578-590. Conflict of interest: Turner, Dan: Consultation fee, research grant, royalties, or honorarium from Johnson & Johnson, Pfizer, Shaare Zedek Medical Center, Hospital for Sick Children, Ferring, Abbvie, Takeda, Prometheus Biosciences, Lilly, SorrisoPharma, Boehringer Ingelheim, Galapagos, BMS, AlfaSigma, and Merck, received medical writing assistance from Johnson & Johnson De Greef, Elisabeth: Advisory Board J&J, received medical writing assistance from Johnson & Johnson Kierkuś, Jarosław: Grant: Nestle for the research conducted, received medical writing assistance from Johnson & Johnson Korczowski, Bartosz: A grant was received from Johnson & Johnson to conduct scientific research, received medical writing assistance from Johnson & Johnson Meglicka, Monika: Consultation fees, royalties from Sandoz and Ferring, received medical writing assistance from Johnson & Johnson Russell, Richard K.: Consultation fees, research grant, royalties, or honorarium from Johnson & Johnson, Pfizer, Ferring, Celltrion, Lilly, & AlfaSigma, received medical writing assistance from Johnson & Johnson Cohen, Stanley: Consultant, Janssen, Kate Farms research grants last 3 years, Janssen, Abbvie, principal, Nutrition4Kids, LLC, received medical writing assistance from Johnson & Johnson Hyams, Jeffrey: Abbvie: Advisory Board Janssen: Advisory Board Roche/Genentech: Consultant Takeda: Consultant, received medical writing assistance from Johnson & Johnson Griffiths, Anne: Consultation fee, research grant, royalties or honorarium from Abbvie, Alfasigma, Janssen, Lilly, Merck, Pfizer, Shaare Zedek Medical Centre, SickKids Hospital, Takeda, received medical writing assistance from Johnson & Johnson Rosh, Joel: Served/serves as an advisor and consultant for Abbvie serves/served as an advisor, consultant, and medical monitor for Janssen, received medical writing assistance from Johnson & Johnson Strauss, Richard: Employee of Johnson & Johnson and may hold stock or stock options in Johnson & Johnson, received medical writing assistance from Johnson & Johnson Van Limbergen, Els: Employee of Johnson & Johnson and may hold stock or stock options in Johnson & Johnson, received medical writing assistance from Johnson & Johnson Adedokun, Omoniyi: Employee of Johnson & Johnson and may hold stock or stock options in Johnson & Johnson, received medical writing assistance from Johnson & Johnson Kim, Lilianne: I’m an employee of Johnson and Johnson and I hold stocks in the company, received medical writing assistance from Johnson & Johnson Volger, Sheri: Employee of Johnson & Johnson and may hold stock or stock options in Johnson & Johnson, received medical writing assistance from Johnson & Johnson