Biscarini Francesco, Barateau Lucie, Pizza Fabio, Plazzi Giuseppe, Dauvilliers Yves
Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy.
IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.
J Sleep Res. 2025 Oct;34(5):e70118. doi: 10.1111/jsr.70118. Epub 2025 Jun 18.
Central disorders of hypersomnolence (CDH) are rare neurological conditions lumped by excessive daytime sleepiness (EDS) as primary complaint mostly arising at young age, including narcolepsy type 1 (NT1), narcolepsy type 2 (NT2), idiopathic hypersomnia (IH), and Kleine-Levin syndrome (KLS). Advances in clinical and translational research have deepened our understanding of NT1, particularly the loss of hypothalamic hypocretin/orexin-producing neurons, establishing hypocretin deficiency as a reliable disease specific biomarker, although the exact mechanisms of neuronal loss remain unknown. Conversely, the pathophysiological mechanisms underlying NT2, IH, and KLS are still poorly understood, as well as their natural course. Standard diagnostic evaluation primarily relies on clinical symptoms, polysomnography and the multiple sleep latency test, with alternative neurophysiological markers (long term polysomnographic recordings), quantitative signal analysis, and wearables technologies being explored as potential innovative tools. Management remains symptomatic, combining pharmacological treatments such as stimulants, sodium oxybate, and emerging hypocretin/orexin receptor agonists, with nonpharmacological strategies tailored to improve patient quality of life. Notably, new therapies targeting orexin signalling offer promising avenues for transforming treatment approaches, particularly in NT1. Looking ahead, advancing precision medicine approaches and addressing unmet needs in CDH are essential to improve patient outcomes. This review summarises current knowledge and highlights future research directions in CDH pathophysiology, diagnostic approaches and pharmacological management.
中枢性过度嗜睡症(CDH)是一类罕见的神经系统疾病,主要表现为以日间过度嗜睡(EDS)为首要症状,多见于年轻人,包括1型发作性睡病(NT1)、2型发作性睡病(NT2)、特发性嗜睡症(IH)和克莱恩-莱文综合征(KLS)。临床和转化研究的进展加深了我们对NT1的理解,尤其是下丘脑分泌食欲素/orexin的神经元缺失,确立了食欲素缺乏作为一种可靠的疾病特异性生物标志物,尽管神经元缺失的确切机制仍不清楚。相反,NT2、IH和KLS的病理生理机制以及它们的自然病程仍知之甚少。标准的诊断评估主要依赖于临床症状、多导睡眠图和多次睡眠潜伏期试验,同时也在探索替代神经生理标志物(长期多导睡眠图记录)、定量信号分析和可穿戴技术作为潜在的创新工具。治疗仍以对症治疗为主,将兴奋剂、γ-羟基丁酸钠等药物治疗与旨在改善患者生活质量的非药物策略相结合。值得注意的是,针对orexin信号通路的新疗法为改变治疗方法提供了有前景的途径,尤其是在NT1方面。展望未来,推进精准医学方法并满足CDH未满足的需求对于改善患者预后至关重要。本综述总结了目前关于CDH病理生理学、诊断方法和药物治疗的知识,并强调了未来的研究方向。