儿童发作性睡病药物治疗及进展

doi:10.12372/jcp.2025.24e1345

摘要/Abstract

摘要：

发作性睡病是一种以日间过度思睡、猝倒、夜间睡眠紊乱、睡眠瘫痪及睡眠幻觉为主要表现的神经系统罕见病，通常起病于儿童或青少年时期。其治疗以改善临床症状为主，包括缓解日间过度思睡和猝倒等症状的药物治疗。尽管食欲素替代疗法、食欲素受体激动剂正处于研究阶段，但发作性睡病的药物在儿童中大多数属于超说明书用药，因此，仍需开展更多的临床研究以证实其在儿童中的安全性及有效性。

关键词: 儿童, 发作性睡病, 药物治疗

Abstract:

Narcolepsy is a rare neurological disorder characterized by excessive daytime sleepiness, cataplexy, disturbed night sleep, sleep paralysis and sleep hallucinations. It typically onsets during childhood or adolescence. The treatment is focused on ameliorating clinical symptoms, encompassing drug therapy to alleviate symptoms such as excessive daytime sleepiness and cataplexy. Despite the fact that orexin replacement therapy and orexin receptor agonists are in the research stage, the majority of drugs for narcolepsy used in children are off-label. Hence, more clinical studies are requisite to validate their safety and efficacy in children.

Key words: child, narcolepsy, pharmacologic treatment

中图分类号:

张镱馨, 洪思琦. 儿童发作性睡病药物治疗及进展[J]. 临床儿科杂志, 2025, 43(6): 475-482.

ZHANG Yixin, HONG Siqi. Pharmacologic and upcoming treatment of pediatric narcolepsy[J]. Journal of Clinical Pediatrics, 2025, 43(6): 475-482.

图/表 1

表1

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药物	作用机制	药代动力学	作用症状	用法用量	不良反应
替洛利生	H3受体拮抗剂/反向激动剂	T_max 3.5小时 T_1/2 20小时	EDS、猝倒、睡眠幻觉、睡眠瘫痪	口服，每天早上1次成人：9~36 mg·d^-1，起始剂量9 mg·d^-1 儿童（6岁及以上）：4.5~36 mg·d^-1，起始剂量4.5 mg·d^-1	失眠、头痛、恶心、焦虑、易怒、头晕、抑郁、震颤、睡眠障碍、疲劳、呕吐、眩晕、消化不良等
莫达非尼	多巴胺再摄取抑制剂	T_max 2小时 T_1/2 9~14小时	EDS	口服，每天早上1次成人：200~400 mg·d^-1，起始剂量100 mg·d^-1 儿童：无推荐	头痛、恶心、腹泻、失眠等，严重不良反应：Stevens-Johnson综合征、重症多形性红斑
羟丁酸钠	GABA-B受体激动剂	T_max 0.5~1.25小时 T_1/2 0.5~1小时	EDS、猝倒、睡眠幻觉、睡眠瘫痪、夜间睡眠紊乱	口服，每晚2次（睡前和半夜）成人：6~9 g/晚，起始剂量4.5 g/晚儿童（7岁及以上）：最大推荐剂量6~9 g/晚，起始剂量≤4.5 g/晚	遗尿、恶心、呕吐、头痛、体重减轻、食欲下降、鼻咽炎、头晕等，严重不良反应：急性精神症状、自杀意念，大剂量引起呼吸抑制
索安非妥	多巴胺和去甲肾上腺素再摄取抑制剂	T_max 2~3小时 T_1/2 7小时	EDS	口服，每天早上1次成人：75~150 mg·d^-1，起始剂量75 mg·d^-1 儿童：无推荐	头痛、食欲下降、失眠、恶心等
哌甲酯（缓释片）	多巴胺和去甲肾上腺素再摄取抑制剂	T_max 6~8小时 T_1/2 3.5小时	EDS	口服，每天早上1次成人：18~54 mg·d^-1，起始剂量18或36 mg·d^-1 儿童（6岁以上）：18~54 mg·d^-1，起始剂量18 mg·d^-1	失眠、紧张、头痛、食欲减退等，罕见不良反应：精神疾病，避免在患心脏病患者中使用