Mitchel Elana B, Dolinger Michael T, Constant Brad, Wang Zi, Guisado Daniela, Gao Michael, Fusillo Steven, Baldassano Robert N, Kelsen Judith, Dubinsky Marla, Huang Jing, Albenberg Lindsey
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA.
J Pediatr Gastroenterol Nutr. 2025 Apr;80(4):653-663. doi: 10.1002/jpn3.12452. Epub 2025 Jan 31.
Real-world data on ustekinumab for the treatment of pediatric Crohn's disease (CD) are limited. This study sought to evaluate the effectiveness, long-term durability, and safety of ustekinumab in the treatment of children with CD.
A retrospective longitudinal cohort study of children with CD treated with ustekinumab from two large centers between 2015 and 2020 was performed. The primary outcome was frequency of steroid-free clinical remission at 1 year. Secondary outcomes included time to steroid-free clinical remission, frequency of clinical and biochemical remission, drug escalation and discontinuation, serum level data, and adverse events. Standard descriptive and comparative statistics were performed. Logistic regression was used to identify factors associated with steroid-free remission at 1 year. Kaplan-Meier curves were used to visualize time-to-event relationships for outcomes.
A total of 101 patients were included. Median follow-up time on ustekinumab was 16.6 months (interquartile range [IQR]: 8.71-31.2) with drug failure in 28% at 1 year. Fifty-nine patients were in steroid-free clinical remission at 1 year. Higher baseline disease activity (odds ratio [OR]: 0.91 (95% confidence interval [CI]: 0.84-0.97), p = 0.01) and stricturing/penetrating disease phenotype (OR: 0.14 (95% CI: 0.03-0.65), p = 0.02) were associated with decreased likelihood of steroid-free clinical remission at 1-year. Ustekinumab drug escalation occurred in 70% of patients, and after escalation, 50 (70%) achieved clinical remission, and 49 (69%) achieved steroid-free remission at the last follow-up. Adverse events were rare and did not require therapy discontinuation.
Ustekinumab is effective and safe in the treatment of children with CD. Escalation of therapy occurs frequently but results in sustained durability.
关于优特克单抗治疗儿童克罗恩病（CD）的真实世界数据有限。本研究旨在评估优特克单抗治疗儿童CD的有效性、长期疗效及安全性。
对2015年至2020年间在两个大型中心接受优特克单抗治疗的CD患儿进行回顾性纵向队列研究。主要结局为1年时无类固醇临床缓解的频率。次要结局包括无类固醇临床缓解时间、临床和生化缓解频率、药物升级和停药情况、血清水平数据及不良事件。进行了标准的描述性和比较性统计分析。采用逻辑回归确定与1年时无类固醇缓解相关的因素。使用Kaplan-Meier曲线直观显示结局的事件发生时间关系。
共纳入101例患者。优特克单抗治疗的中位随访时间为16.6个月（四分位间距[IQR]：8.71 - 31.2），1年时28%的患者治疗失败。59例患者在1年时处于无类固醇临床缓解状态。较高的基线疾病活动度（比值比[OR]：0.91（95%置信区间[CI]：0.84 - 0.97），p = 0.01）和狭窄/穿透性疾病表型（OR：0.14（95%CI：0.03 - 0.65），p = 0.02）与1年时无类固醇临床缓解的可能性降低相关。70%的患者发生了优特克单抗药物升级，升级后，50例（70%）在最后一次随访时达到临床缓解，49例（69%）达到无类固醇缓解。不良事件罕见，无需停药治疗。
优特克单抗治疗儿童CD有效且安全。治疗升级频繁发生，但疗效持久。
