Abstract：
Ustekinumab is an effective therapy for adult Crohn\'s disease (CD), but data in paediatric CD patients are scarce. The aim of the study was to describe the real-life effectiveness and safety of ustekinumab in paediatric CD. This is a multicentre review of children with Crohn\'s disease treated with ustekinumab. The aim of our study was to describe the effectiveness and safety of ustekinumab in paediatric real-life practice. This is a study of the Paediatric IBD (inflammatory bowel disease) Porto group of ESPGHAN. Corticosteroid (CS)- and exclusive enteral nutrition (EEN)-free remission, defined as weighted Paediatric Crohn\'s Disease Activity Index (wPCDAI) < 12.5, and physician global assessment (PGA) were determined at weeks 12 and 52. A total of 101 children were included at a median age of 15.4 years (IQR 12.7-17.2) with a median follow-up of 7.4 months (IQR 5.6-11.8). Ninety-nine percent had received prior anti-TNF, 63% ≥ 2 anti-TNFα therapies and 22% vedolizumab. Baseline median wPCDAI was 39 (IQR 25-57.5) (71 (70%) patients with moderate-severe activity). Weeks 12 and 52 CS- and EEN-free remission were both 40.5%. Clinical response at week 6, iv induction route and older age at onset of ustekinumab treatment were predictive factors associated with clinical remission at week 12. Seven minor adverse events probably related to ustekinumab were reported. One patient died from an unrelated cause. Conclusion: Our results suggest that ustekinumab is effective and safe in children with chronically active or refractory CD. What is Known: • Ustekinumab is an effective therapy for adult moderate to severe Crohn\'s disease (CD). • Off-label use of ustekinumab in children is increasing especially in anti-TNF refractory CD. What is New: • Is the largest cohort of real-world use of ustekinumab in paediatric CD to date. • Clinical response at week 6, iv induction and older age at onset of ustekinumab were predictive factors associated with clinical response at week 12.
摘要：
Ustekinumab是成人克罗恩病（CD）的有效疗法，但是儿科CD患者的数据很少。该研究的目的是描述ustekinumab在儿科CD中的实际有效性和安全性。这是对使用ustekinumab治疗的克罗恩病儿童的多中心综述。我们研究的目的是描述ustekinumab在儿科现实生活中的有效性和安全性。这是ESPGHAN的儿科IBD（炎症性肠病）波尔图组的研究。无皮质类固醇（CS）和专有肠内营养（EEN）缓解，定义为加权儿科克罗恩病活动指数（wPCDAI）<12.5，并在第12周和第52周确定医师全球评估（PGA）。共纳入101名儿童，中位年龄为15.4岁（IQR12.7-17.2），中位随访时间为7.4个月（IQR5.6-11.8）。99%的人以前接受过抗肿瘤坏死因子，63%≥2种抗TNFα治疗和22%维多珠单抗。基线中位数wPCDAI为39（IQR25-57.5）（71（70％）患有中重度活动的患者）。第12周和第52周无CS和EEN缓解率均为40.5%。第6周的临床反应、静脉内诱导途径和ustekinumab治疗开始时的年龄是与第12周的临床缓解相关的预测因素。报告了7个可能与ustekinumab相关的轻微不良事件。一名患者死于无关的原因。结论：我们的结果表明，ustekinumab对患有慢性活动性或难治性CD的儿童是有效和安全的。已知:•Ustekinumab是成人中度至重度克罗恩病(CD)的有效疗法。•在儿童中使用ustekinumab的标签外使用正在增加，尤其是在抗TNF难治性CD中。新增内容：•是迄今为止在儿科CD中使用ustekinumab的最大现实队列。•第6周时的临床反应、静脉注射诱导和ustekinumab发作时的年龄较大是与第12周时的临床反应相关的预测因素。
