Sleep-Wake Disorders Unit, Department of Neurology, Gui-de-Chauliac Hospital, CHU Montpellier, Montpellier, France.
National Reference Centre for Orphan Diseases, Narcolepsy, Idiopathic Hypersomnia, and Kleine-Levin Syndrome, Montpellier, France.
J Sleep Res. 2022 Aug;31(4):e13631. doi: 10.1111/jsr.13631. Epub 2022 May 27.
This article addresses the clinical presentation, diagnosis, pathophysiology and management of narcolepsy type 1 and 2, with a focus on recent findings. A low level of hypocretin-1/orexin-A in the cerebrospinal fluid is sufficient to diagnose narcolepsy type 1, being a highly specific and sensitive biomarker, and the irreversible loss of hypocretin neurons is responsible for the main symptoms of the disease: sleepiness, cataplexy, sleep-related hallucinations and paralysis, and disrupted nocturnal sleep. The process responsible for the destruction of hypocretin neurons is highly suspected to be autoimmune, or dysimmune. Over the last two decades, remarkable progress has been made for the understanding of these mechanisms that were made possible with the development of new techniques. Conversely, narcolepsy type 2 is a less well-defined disorder, with a variable phenotype and evolution, and few reliable biomarkers discovered so far. There is a dearth of knowledge about this disorder, and its aetiology remains unclear and needs to be further explored. Treatment of narcolepsy is still nowadays only symptomatic, targeting sleepiness, cataplexy and disrupted nocturnal sleep. However, new psychostimulants have been recently developed, and the upcoming arrival of non-peptide hypocretin receptor-2 agonists should be a revolution in the management of this rare sleep disease, and maybe also for disorders beyond narcolepsy.
本文主要探讨了 1 型和 2 型发作性睡病的临床表现、诊断、病理生理学和治疗,重点介绍了最新研究发现。脑脊液中低水平的下丘脑泌素-1/食欲素-A 足以诊断 1 型发作性睡病,是一种高度特异性和敏感性的生物标志物,下丘脑泌素神经元的不可逆性丧失是导致该病主要症状(如嗜睡、猝倒、睡眠相关幻觉和瘫痪以及夜间睡眠紊乱)的原因。破坏下丘脑泌素神经元的过程高度疑似为自身免疫或免疫失调。在过去的二十年中,随着新技术的发展,人们对这些机制有了更深入的了解,取得了显著的进展。相反,2 型发作性睡病是一种定义不明确的疾病,其临床表现和演变具有多样性,目前发现的可靠生物标志物很少。人们对这种疾病的了解还很匮乏,其病因仍不清楚,需要进一步研究。目前发作性睡病的治疗仍仅针对症状,包括嗜睡、猝倒和夜间睡眠紊乱。然而,最近开发出了一些新型的精神兴奋剂,即将问世的非肽类下丘脑泌素受体-2 激动剂将成为治疗这种罕见睡眠疾病的一场革命,也许也将为发作性睡病以外的疾病带来新的治疗选择。