Treister Alison D, Lio Peter A
Department of Dermatology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois.
Pediatr Dermatol. 2019 Jan;36(1):85-88. doi: 10.1111/pde.13697. Epub 2018 Oct 18.
BACKGROUND/OBJECTIVES: Clinical trials of dupilumab have shown efficacy in treating moderate-to-severe atopic dermatitis (AD) in adult patients. While a phase 2 trial of dupilumab has shown efficacy and safety in children, the medication awaits Food and Drug Administration (FDA) approval, and off-label use is limited by dosing currently available to adults. We present this case series to describe the efficacy and safety profile of off-label dupilumab use in six pediatric patients treated by one provider in a private practice.
We conducted a retrospective chart review of all patients under the age of 18 receiving dupilumab for AD. Two dosing regimens were used as follows: adult dose for patients ≥ 40 kg and half dose for those < 40 kg. We recorded the investigator's global assessment (IGA) and body surface area (BSA) prior to dupilumab initiation and following treatment. All patients were warned of potential side effects including injection site reaction, conjunctivitis, increased risk of infection, and lack of information about use in children.
Six pediatric patients were identified with an average age of 10.8 years (range: 7-15). All patients had a decrease in IGA of at least 2 points using biweekly 300 or 150 milligram (mg) doses for an average treatment duration of 8.5 months (range: 6-11). Three patients (50%) had an IGA of 1 after treatment. No side effects were reported.
Dupilumab appears to be an effective treatment for AD in pediatric patients. Optimal dose and delivery have yet to be determined.
背景/目的：度普利尤单抗的临床试验已显示其在治疗成年患者中重度特应性皮炎（AD）方面的疗效。虽然度普利尤单抗的2期试验已显示其在儿童中的疗效和安全性，但该药物尚待美国食品药品监督管理局（FDA）批准，且目前针对儿童的非标签用药因成人可用剂量而受到限制。我们呈现这个病例系列，以描述在一名私人诊所医生治疗的6例儿科患者中，度普利尤单抗非标签用药的疗效和安全性概况。
我们对所有18岁以下接受度普利尤单抗治疗AD的患者进行了回顾性病历审查。使用了两种给药方案如下：体重≥40kg的患者使用成人剂量，体重<40kg的患者使用半剂量。我们记录了开始度普利尤单抗治疗前及治疗后的研究者整体评估（IGA）和体表面积（BSA）。所有患者均被告知潜在的副作用，包括注射部位反应、结膜炎、感染风险增加以及儿童用药信息不足。
确定了6例儿科患者，平均年龄为10.8岁（范围：7 - 15岁）。所有患者每两周使用300或150毫克（mg）剂量，平均治疗持续时间为8.5个月（范围：6 - 11个月），IGA至少降低2分。3例患者（50%）治疗后IGA为1。未报告副作用。
度普利尤单抗似乎是治疗儿科患者AD的有效药物。最佳剂量和给药方式尚未确定。
