Department of Child Neurology, Geisinger Medical Center, Janet Weis Children's Hospital, Danville, Pennsylvania; Department of Sleep Medicine, Geisinger Medical Center, Janet Weis Children's Hospital, Danville, Pennsylvania.
Division of Pediatric Pulmonology and Cystic Fibrosis Center, Cohen Children's Medical Center, Lake Success, New York; Pediatric Neurology Service Line for Northwell Health, Department of Pediatrics & Neurology, Zucker School of Medicine at Hofstra/Northwell, Cohen Children's Medical Center, Lake Success, New York.
Pediatr Neurol. 2019 Apr;93:39-42. doi: 10.1016/j.pediatrneurol.2018.10.020. Epub 2018 Nov 22.
Narcolepsy is a disabling sleep-wake disorder characterized by the pentad symptoms of excessive daytime sleepiness, sleep paralysis, sleep fragmentation, sleep-related hallucinations, and cataplexy. There is no curative therapy for narcolepsy. Treatment is therefore symptom directed. Symptom management is generally directed at improving excessive daytime sleepiness, sleep fragmentation, and cataplexy. First-line treatment for excessive daytime sleepiness is typically daily use of wake-promoting agents, such as modafinil or armodafinil, or stimulant therapy, such as methylphenidate or amphetamines. Alternatively, sodium oxybate can be used nightly for improved cataplexy, sleep consolidation, and following day wakefulness. These therapies can be limited in some patients because of inadequate efficacy, poor tolerability, or side effects.
We describe five narcolepsy patients with severe excessive daytime sleepiness who had an inadequate response or experienced side effects with the initial therapies but had a positive response to treatment with baclofen.
These patients reported subjective improvement in sleep maintenance without fragmentation and daytime sleepiness. Average Epworth Sleepiness Scale assessment before treatment was 15.8 with post-treatment assessment being 10.4 (P < 0.05).
Baclofen may be an effective treatment for excessive daytime sleepiness and sleep fragmentation in narcolepsy and warrants further study.
发作性睡病是一种使人丧失能力的睡眠-觉醒障碍，其特征是出现日间过度嗜睡、睡眠瘫痪、睡眠碎片化、睡眠相关幻觉和猝倒这五组症状。目前尚无针对发作性睡病的治愈性疗法。因此，治疗主要针对症状。症状管理通常旨在改善日间过度嗜睡、睡眠碎片化和猝倒。针对日间过度嗜睡的一线治疗通常是每日使用促觉醒药物，如莫达非尼或阿莫达非尼，或兴奋剂治疗，如哌甲酯或安非他命。或者，也可以每晚使用羟丁酸钠来改善猝倒、睡眠巩固和次日的清醒度。由于疗效不足、耐受性差或副作用，这些疗法在某些患者中可能受到限制。
我们描述了五例患有严重日间过度嗜睡的发作性睡病患者，他们对初始治疗的反应不足或出现副作用，但对巴氯芬治疗有积极反应。
这些患者报告说，睡眠维持方面的主观改善，没有碎片化和日间嗜睡。治疗前的平均 Epworth 嗜睡量表评估为 15.8，治疗后的评估为 10.4（P<0.05）。
巴氯芬可能是发作性睡病日间过度嗜睡和睡眠碎片化的有效治疗方法，值得进一步研究。
