Idiopathic hypersomnia: does first to approval mean first-line treatment?
Idiopathic hypersomnia: does first to approval mean first-line treatment?
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DOI：
10.1016/s1474-4422(21)00419-1
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发表时间：
2022-01
期刊：
影响因子：
48
通讯作者：
Trotti, Lynn Marie
中科院分区：
文献类型：
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作者：
Trotti, Lynn Marie
特发性睡眠过度是一种慢性睡眠-觉醒障碍，尽管夜间睡眠持续时间正常或长，但仍会导致白天过度嗜睡，通常伴有从夜间睡眠或午睡中醒来时的明显睡眠惯性。有几种治疗特发性嗜睡症的药物是标签外的，包括莫达非尼、羟丁酸钠和传统的精神兴奋剂。1羟丁酸盐药物是根据FDA风险评估和缓解策略（REMS）计划在美国处方和分发的，并带有几个严重副作用的“黑框”警告。2021年8月，美国食品药品监督管理局（FDA）批准钙、镁、钾、羟丁酸钠（简称低羟丁酸钠）用于治疗成人特发性嗜睡症。2对于特发性睡眠过度症患者来说，这一批准是一个重要的里程碑，使低羟丁酸钠成为FDA批准的第一种也是唯一一种用于治疗这种疾病的药物。FDA批准低羟丁酸钠是否意味着这种药物现在是特发性睡眠过度症治疗的一线药物？Yves Dauvilliers及其同事在《柳叶刀神经病学》上发表的一篇文章的结论中提出了这一观点，3他们提出了一项在特发性嗜睡症成人中进行的低羟丁酸钠随机戒断试验的结果。在这项试验中，154名参与者进入了低羟丁酸钠治疗的开放标签阶段，大约16周后，115名参与者被随机分配继续低羟丁酸钠（n= 56）或在双盲戒断期转换为安慰剂（n= 59）。本研究未与任何其他活性药物进行头对头比较。此外，该人群并不局限于未接受过治疗的参与者;相反，超过一半的入组参与者正在服用促进觉醒的药物，并使用低羟丁酸钠作为辅助治疗。
Idiopathic hypersomnia is a chronic sleep-wake disorder that causes excessive daytime sleepiness despite normal or long nocturnal sleep durations, often accompanied by pronounced sleep inertia when awakening from overnight sleep or naps. Several treatments are prescribed off-label for idiopathic hypersomnia, including modafinil, sodium oxybate, and traditional psychostimulants. 1 Oxybate medications are prescribed and dispensed in the USA under an FDA Risk Evaluation and Mitigation Strategy (REMS) program and carry several “black box” warnings for serious side effects. In August 2021, the US Food and Drug Administration (FDA) approved calcium, magnesium, potassium, sodium oxybate (known as lower-sodium oxybate) for the treatment of adults with idiopathic hypersomnia. 2 This approval is an important milestone for people living with idiopathic hypersomnia, making lower-sodium oxybate the first and only medication FDA-labelled for the treatment of this disorder.Should the FDA-approval of lower-sodium oxybate be interpreted to mean that this medication is now first-line for idiopathic hypersomnia treatment? This notion is suggested in the conclusion of an Article in The Lancet Neurology, by Yves Dauvilliers and colleagues, 3 who present the findings of a randomized withdrawal trial of lower-sodium oxybate in adults with idiopathic hypersomnia. In this trial, 154 participants entered an open-label phase of treatment with lower-sodium oxybate and, after a period of about 16 weeks, 115 were randomly assigned either to continue lower-sodium oxybate (n= 56) or switch to placebo (n= 59) in a double-blind withdrawal phase. No head-to-head comparison with any other active medication was done in this study. Furthermore, the population was not restricted to treatment-naive participants; rather, more than half of enrolled participants were taking wake-promoting medications and using lower-sodium oxybate as an adjunct treatment.