背景:发作性睡病的首发症状大多出现在儿童时期。儿童的药物管理选择有限,也是由于批准状态。Pitolisant是一种反组胺3受体激动剂,已被EMA和FDA批准用于治疗有或没有猝倒的成人发作性睡病。临床经验表明,在儿童和青少年中也有有益的用途。我们的目标是在现实世界中评估pitolisant在发作性睡病儿童/青少年中的作用和耐受性。
方法:这项多中心回顾性观察研究包括来自三个国际发作性睡病中心的55例发作性睡病患者(德国,法国和意大利)接受了pitolisant治疗。如果患者至少6岁并被诊断为1型或2型发作性睡病,则符合资格。人口统计学和临床特征,问卷,收集睡眠医学和实验室数据。
结果:55名儿童/青少年(25名女孩,45.45%,30个男孩,54.55%)6-18岁,发作性睡病(1型=92.7%,类型2=7.3%),用pitolisant治疗。平均pitolisant剂量为34.1mg/d。治疗对白天过度嗜睡(EDS)和猝倒有效:小儿Epworth嗜睡量表(ESS)评分从19降至13.5(p<0.001),每周猝倒频率从基线时的7.9提高到5.2(p<0.001)。pitolisant治疗耐受性良好。副作用轻微,大多是短期的。失眠是最常见的报告(5.5%)。
结论:第一个现实世界的结果表明,pitolisant治疗可有效改善发作性睡病儿童的EDS和猝倒,也是很好的耐受性。
BACKGROUND: First symptoms of narcolepsy mostly present during childhood. Pharmacological management options in children are limited, also due to approval status.
Pitolisant is an inverse histamine 3 receptor agonist and has been approved for the treatment of adult narcolepsy with or without cataplexy by EMA and FDA. Clinical experience indicates for a beneficial use also in children and adolescents. Our goal was to evaluate the effects and tolerability of
pitolisant in narcolepsy children/adolescents in a real-world setting.
METHODS: This multicentre retrospective observational study included 55 patients with narcolepsy from three international narcolepsy centers (Germany, France and Italy) who were treated with pitolisant. Patients were eligible if they were at least 6 years old and diagnosed with narcolepsy type 1 or 2. Demographic and clinical characteristics, questionnaires, sleep medicine and laboratory data were collected.
RESULTS: 55 children/adolescents (25 girls, 45.45%, 30 boys, 54.55%) aged 6-18 years, with narcolepsy (type 1 = 92.7%, type 2 = 7.3%), were treated with pitolisant. The mean
pitolisant dose was 34.1 mg/d. Treatment was effective for excessive daytime sleepiness (EDS) and cataplexy: the pediatric Epworth Sleepiness Scale (ESS) score decreased from 19 to 13.5 (p < 0.001) and the weekly cataplexy frequency improved from 7.9 at baseline to 5.2 (p < 0.001). Treatment with
pitolisant was well tolerated. Side effects were mild and mostly short-term. Insomnia was reported most frequently (5.5%).
CONCLUSIONS: First real-world results suggest that
pitolisant treatment is effective in improving EDS and cataplexy in children with narcolepsy, and also is well tolerated.