Department of Pediatrics, Susan and Leonard Feinstein IBD Clinical Center, Icahn School of Medicine at Mount Sinai, New York, NY.
J Pediatr Gastroenterol Nutr. 2019 Jul;69(1):61-67. doi: 10.1097/MPG.0000000000002362.
Ustekinumab is an effective therapy for Crohn disease currently approved for adults. Off-label use in the pediatric population is increasing, but its effectiveness in this age group has not been reported.
The aim of the study was to describe real-world experience with ustekinumab at a tertiary care pediatric inflammatory bowel disease (IBD) center.
As part of an ongoing observational cohort study of biologic-treated pediatric IBD patients initiated in October 2014, data on demographics, disease behavior, location and activity, treatment, and surgical history were collected for all patients receiving ustekinumab. Disease activity was assessed using the Harvey Bradshaw index or partial Mayo score. Primary outcome was steroid-free remission at 52 weeks. Descriptive statistics summarized the safety and efficacy outcomes, and univariate analyses were performed to examine associations of clinical characteristics with efficacy.
Fifty-two children and young adults initiating ustekinumab were analyzed; 81% Crohn Disease, 8% ulcerative colitis, and 11% IBD-unspecified. Median [IQR] age at induction was 16.8 [14-18] years. Patients were followed for a minimum of 12 months. Most patients (81%) failed >1 anti-TNF, and 37% failed anti-TNF and vedolizumab; 10 patients were biologic-naïve. At week 52, 75% were still on ustekinumab, and 50% (bio-exposed) and 90% (bio-naïve) were in steroid-free remission. Two infusion reactions and neither serious adverse events nor serious infections were observed.
Our results suggest that ustekinumab is efficacious and safe in pediatric patients with IBD. Controlled clinical trial data are needed to confirm these observations.
乌司奴单抗是一种有效的克罗恩病治疗药物，目前已获批准用于成人。该药在儿科人群中的超适应证使用正在增加，但尚未有其在该年龄组中有效性的报告。
本研究旨在描述三级儿童炎症性肠病（IBD）中心的乌司奴单抗真实世界应用经验。
作为 2014 年 10 月启动的生物制剂治疗儿童 IBD 患者的一项正在进行的观察性队列研究的一部分，我们收集了所有接受乌司奴单抗治疗的患者的人口统计学、疾病行为、部位和活动度、治疗和手术史数据。疾病活动度采用 Harvey Bradshaw 指数或部分 Mayo 评分进行评估。主要结局为 52 周时无激素缓解。采用描述性统计汇总安全性和疗效结局，并进行单变量分析以评估临床特征与疗效的相关性。
共分析了 52 例开始使用乌司奴单抗的儿童和青少年患者；81%为克罗恩病，8%为溃疡性结肠炎，11%为 IBD 未特指类型。诱导时的中位[IQR]年龄为 16.8[14-18]岁。患者的随访时间至少为 12 个月。大多数患者（81%）曾接受过>1 种抗 TNF 治疗失败，37%曾接受过抗 TNF 和维得利珠单抗治疗失败，10 例患者为生物制剂初治。在第 52 周时，75%的患者仍在使用乌司奴单抗，50%（生物制剂暴露组）和 90%（生物制剂初治组）达到无激素缓解。观察到 2 例输液反应，但未发生严重不良事件或严重感染。
我们的结果表明，乌司奴单抗在儿童 IBD 患者中是有效且安全的。需要开展对照临床试验来证实这些观察结果。
