Takeuchi Ichiro, Arai Katsuhiro, Kyodo Reiko, Sato Takuro, Tokita Kazuhide, Hirano Yuri, Shimizu Hirotaka
Center for Pediatric Inflammatory Bowel Disease, Division of Gastroenterology, National Center for Child Health and Development, Tokyo, Japan.
J Gastroenterol Hepatol. 2021 Jan;36(1):125-130. doi: 10.1111/jgh.15128. Epub 2020 Jun 29.
Ustekinumab 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.
Medical 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.
Seventeen 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.
Ustekinumab 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.
乌司奴单抗是一种靶向白细胞介素-12和白细胞介素-23的p40亚基的人源单克隆抗体，据报道对治疗克罗恩病有效。然而，很少有研究报道乌司奴单抗用于儿童炎症性肠病的情况。本研究旨在评估乌司奴单抗在儿童和青少年炎症性肠病患者中的临床疗效和安全性。
回顾性分析日本一家儿童炎症性肠病中心收治的20岁以下克罗恩病或克罗恩病样炎症性肠病患者接受乌司奴单抗治疗的病历，评估其疗效和安全性。主要结局指标为第26周和第52周的无激素临床缓解率。还评估了第52周后的无激素缓解率。采用加权儿童克罗恩病活动指数和克罗恩病简易内镜评分评估疾病活动度。
共纳入17例患者（男∶女 = 8∶9，A1a[诊断年龄＜10岁]∶A1b[诊断年龄≥10岁]= 8∶9）。所有患者在第26周时均接受乌司奴单抗治疗，其中9/10例持续治疗超过1年。第26周、第52周和1年时的无激素临床缓解率分别为59%、50%和70%。8例在使用乌司奴单抗后接受内镜检查的儿童中有3例实现内镜缓解。研究期间未记录到严重不良事件。
对于对抗肿瘤坏死因子药物无反应或有不良反应的儿童和青少年克罗恩病及克罗恩病样炎症性肠病患者，乌司奴单抗可能是一种有效且安全的治疗选择。
