Neurology Unit, Ospedale dell'Angelo , Venezia Mestre, Italy.
Neurology Unit, Department of Neurosciences, University Hospital of Udine , Udine, Italy.
Expert Opin Pharmacother. 2021 Feb;22(2):155-162. doi: 10.1080/14656566.2020.1817387. Epub 2020 Sep 17.
Narcolepsy is a chronic sleep disorder characterized by a pentad of excessive daytime sleepiness (EDS), cataplexy, sleep paralysis, hypnagogic/hypnopompic hallucinations, and disturbed nocturnal sleep. Treatment of narcolepsy remains challenging and current therapy is strictly symptomatically based.
The present manuscript is based on an extensive Internet and PubMed search from 1990 to 2020. It is focused on the clinical and pharmacological properties of pitolisant in the treatment of narcolepsy.
Currently there is no cure for narcolepsy. Although efforts have been made, current treatments do not always allow to obtain an optimal control of symptoms. Pitolisant is an antagonist/inverse agonist of the histamine H3 autoreceptor. Its mechanism of action is novel and distinctive compared to the other available therapies for narcolepsy. Clinical trials suggest that pitolisant administered at a dose of ≤36 mg/day is an effective treatment option for narcolepsy, reducing EDS and cataplexy. Pitolisant is available as oral tablets and offers a convenient once-daily regimen. Pitolisant is generally well tolerated and showed minimal abuse potential in animals and humans. Long-term studies comparing the effectiveness and tolerability of pitolisant with active drugs (e.g. modafinil, sodium oxybate) are needed.
发作性睡病是一种慢性睡眠障碍，其特征是白天过度嗜睡（EDS）、猝倒、睡眠瘫痪、催眠/清醒幻觉和夜间睡眠紊乱。发作性睡病的治疗仍然具有挑战性，目前的治疗方法严格基于症状。
本文基于 1990 年至 2020 年的广泛互联网和 PubMed 搜索。它主要关注吡咯烷酮治疗发作性睡病的临床和药理学特性。
目前尚无治愈发作性睡病的方法。尽管已经做出了努力，但目前的治疗方法并不总是能使症状得到最佳控制。吡咯烷酮是组胺 H3 自身受体的拮抗剂/反向激动剂。与其他可用的发作性睡病治疗方法相比，其作用机制新颖独特。临床试验表明，吡咯烷酮的剂量为≤36mg/天，是治疗发作性睡病的有效选择，可减少 EDS 和猝倒。吡咯烷酮有口服片剂，提供方便的每日一次的方案。吡咯烷酮通常具有良好的耐受性，在动物和人类中显示出最小的滥用潜力。需要进行长期研究，比较吡咯烷酮与活性药物（如莫达非尼、羟丁酸钠）的有效性和耐受性。
