Ustekinumab for children and adolescents with inflammatory bowel disease at a tertiary children's hospital in Japan

Abstract

Background and AimUstekinumab is a human monoclonal antibody targeting the p40 subunit of both interleukin‐12 and interleukin‐23 with reported efficacy to treat Crohn's disease. However, few studies have reported the use of ustekinumab for pediatric inflammatory bowel disease. This study aimed to assess the clinical efficacy and safety of ustekinumab in children and adolescents with inflammatory bowel disease.MethodsMedical records of patients aged under 20 years with Crohn's disease or Crohn's disease‐like inflammatory bowel disease who had received ustekinumab at a Japanese pediatric inflammatory bowel disease center were retrospectively reviewed for efficacy and safety. The primary outcome was the steroid‐free clinical remission rate at weeks 26 and 52. The steroid‐free remission rate beyond week 52 was also evaluated. Weighted pediatric Crohn's disease activity index and simple endoscopic score for Crohn's disease were used to assess disease activity.ResultsSeventeen patients were included (male : female = 8:9, A1a [diagnosed < 10 years old]:A1b [diagnosed ≥ 10 years old] = 8:9). All patients were on ustekinumab at week 26, and 9/10 continued treatment over 1 year. The steroid‐free clinical remission rates were 59% at week 26, 50% at week 52, and 70% over 1 year. Three of eight children who underwent endoscopy after ustekinumab introduction achieved endoscopic remission. No serious adverse events were recorded during the study period.ConclusionsUstekinumab may be an effective and safe treatment option for pediatric and adolescent Crohn's disease and Crohn's disease‐like inflammatory bowel disease patients having nonresponse or adverse reactions to anti‐tumor necrosis factor agents.

Ustekinumab as the First Biological Agent for Crohn’s Disease in a 10-Year-Old Girl

“…We assessed the wPCDAI, FC, and CS over time; slightly active findings were noted in the ileum at week 20, after which the interval of UST administration was shortened, and no findings were observed at week 75 when dietary restrictions were relaxed. Our patient achieved clinical remission (wPCDAI 0) at week 16 and remains in steroidfree clinical remission, which is the primary outcome in the report by Takeuchi et al (2021), even at week 75. Although Takeuchi et al (2021) did not evaluate FC in their study, the FC level in our patient was assessed.…”

“…Our patient achieved clinical remission (wPCDAI 0) at week 16 and remains in steroidfree clinical remission, which is the primary outcome in the report by Takeuchi et al (2021), even at week 75. Although Takeuchi et al (2021) did not evaluate FC in their study, the FC level in our patient was assessed. FC level decreased after UST administration at week 20 and subsequently remained low.…”

“…The safety of UST has been reported in terms of adverse events, such as susceptibility to infection (Papp et al 2015) or infusion reaction, compared to IFX. No significant adverse events were observed in the studies demonstrating the effectiveness of UST (Chavannes et al 2019;Dayan et al 2019;Takeuchi et al 2021). In this study, our patient experienced no adverse events over the 75 weeks of treatment.…”

“…Although only a few reports have assessed the effectiveness of UST as the first biological agent in both the induction and maintenance phases in the management of pediatric CD, the efficacy of UST for pediatric CD refractory to anti-tumor necrosis factor (TNF) therapy has been reported (Chavannes et al 2019;Dayan et al 2019;Takeuchi et al 2021). In our case, although PSL was administered along with UST in the induction phase, clinical and endoscopic remission was maintained after the discontinuation of PSL; hence, the patient's diet was increased.…”

“…The effectiveness of ustekinumab (UST) -a human monoclonal antibody that inhibits interleukin (IL)-12 and IL-23 functions by blocking the p40 subunit -has been reported in adult patients with IBD (Feagan et al 2016). However, except for a report by Takeuchi et al (2021) who describe the effectiveness and safety of UST in pediatric patients with IBD, there are very few reports regarding the effectiveness and safety of UST in pediatric patients with IBD, especially those who are biologically naive. Herein, we describe a pediatric case of Crohn's disease (CD) wherein UST was administered as the first biological agent.…”