Department of Pediatrics, Children's Health Medical Center Dallas, Dallas, TX, 75235, USA.
Department of Pediatric Gastroenterology, UT Southwestern Medical Center, Dallas, TX, 75235, USA.
F1000Res. 2020 Apr 30;9:316. doi: 10.12688/f1000research.22673.2. eCollection 2020.
Ustekinumab is a monoclonal antibody that inhibits interleukins 12 and 23. It is approved for treatment of Crohn's disease (CD) in adults; however, there is a paucity of data regarding its use in pediatric CD. We describe our experience using ustekinumab in anti-TNF refractory CD pediatric patients. We performed a retrospective chart review on pediatric patients with CD who were started on ustekinumab from January 2016 to November 2018. We collected patient's clinical history, previous treatment history, surgeries related to CD, disease severity, as measured by abbrPCDAI, and endoscopic severity as recorded by SES-CD before and after ustekinumab. We identified 10 patients with CD who were started on ustekinumab due to non-response to currently approved agents. Seven patients needed augmented maintenance dosing every 4-6 weeks to achieve clinical response or remission. Six of these seven patients had therapeutic drug monitoring during the course of treatment, with five patients showing subtherapeutic drug levels of <4.5 μg/mL while on standard maintenance dosing every 8 weeks, and four patients showing therapeutic drug levels of >4.5 μg/mL on augmented dosing interval. The remaining three patients were on standard maintenance dosing for the duration of treatment. In this retrospective chart review, 7 out of 10 patients with anti-TNF refractory pediatric-onset CD required augmented maintenance doses of ustekinumab to achieve clinical response or remission. A prospective study is needed to define appropriate ustekinumab dosing and interval in management of pediatric CD.
乌司奴单抗是一种单克隆抗体，可抑制白细胞介素 12 和 23。它被批准用于治疗成人克罗恩病（CD）；然而，关于其在儿科 CD 中的应用的数据很少。我们描述了我们在抗 TNF 难治性 CD 儿科患者中使用乌司奴单抗的经验。我们对 2016 年 1 月至 2018 年 11 月期间开始使用乌司奴单抗的 CD 儿科患者进行了回顾性图表审查。我们收集了患者的临床病史、先前的治疗史、与 CD 相关的手术、疾病严重程度，采用 abbrPCDAI 进行评估，以及使用 SES-CD 记录治疗前后的内镜严重程度。我们确定了 10 名因对目前批准的药物无反应而开始使用乌司奴单抗的 CD 患者。7 名患者需要每 4-6 周增强维持剂量以实现临床缓解或缓解。这 7 名患者中有 6 名在治疗过程中进行了治疗药物监测，5 名患者在标准维持剂量每 8 周时显示低于 4.5μg/mL 的治疗药物水平，而 4 名患者在增强剂量间隔时显示高于 4.5μg/mL 的治疗药物水平。其余 3 名患者在整个治疗期间接受标准维持剂量。在这项回顾性图表审查中，10 名抗 TNF 难治性儿科发病 CD 患者中有 7 名需要增强乌司奴单抗的维持剂量以实现临床缓解或缓解。需要进行前瞻性研究来确定儿科 CD 管理中乌司奴单抗的适当剂量和间隔。
