Center for Narcolepsy and Hypersomnias, Clinical Sleep and Neuroimmunology, Institute of Immunology, University of Witten/Herdecke, Witten, Germany.
Department of Biomedical Science and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.
Sleep Med. 2023 Mar;103:62-68. doi: 10.1016/j.sleep.2023.01.015. Epub 2023 Jan 24.
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.
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.
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%).
First real-world results suggest that pitolisant treatment is effective in improving EDS and cataplexy in children with narcolepsy, and also is well tolerated.
嗜睡症的首发症状大多出现在儿童时期。儿童患者的药物治疗选择有限，这也与药物的批准状况有关。羟丁酸钠是一种反向组胺 3 受体激动剂，已在欧盟和美国获得批准，可用于治疗成人伴有或不伴有猝倒的嗜睡症。临床经验表明，该药对儿童和青少年也有有益的作用。我们的目的是评估羟丁酸钠在真实世界环境中治疗儿童和青少年嗜睡症的疗效和耐受性。
这是一项多中心回顾性观察研究，纳入了来自德国、法国和意大利的三个国际嗜睡症中心的 55 例嗜睡症患儿（年龄 6-18 岁），这些患儿均接受了羟丁酸钠治疗。如果患者年龄至少 6 岁，且被诊断为 1 型或 2 型嗜睡症，则有资格入组。收集了患者的人口统计学和临床特征、问卷调查、睡眠医学和实验室数据。
55 例儿童和青少年（女 25 例，占 45.45%；男 30 例，占 54.55%）患有嗜睡症（1 型 92.7%，2 型 7.3%），接受了羟丁酸钠治疗。羟丁酸钠的平均剂量为 34.1mg/d。治疗对日间过度嗜睡（EDS）和猝倒均有效：儿童嗜睡多相睡眠潜伏期试验（MSLT）评分从 19 分降至 13.5 分（p<0.001），每周猝倒频率从基线时的 7.9 次改善至 5.2 次（p<0.001）。羟丁酸钠治疗耐受性良好。不良反应轻微，大多为短期。失眠报告最为常见（5.5%）。
初步的真实世界研究结果表明，羟丁酸钠治疗可有效改善儿童嗜睡症患者的 EDS 和猝倒，且耐受性良好。
