Department of Neurology, Sleep-Wake Disorders Center, Gui-de-Chauliac Hospital, Institute for Neurosciences of Montpellier INM, INSERM, University of Montpellier, Montpellier, France.
Sleep Disorders & Research Center, Henry Ford Health System, Detroit, MI, USA.
J Comp Eff Res. 2024 Sep;13(9):e240031. doi: 10.57264/cer-2024-0031. Epub 2024 Aug 1.
WHAT IS THIS SUMMARY ABOUT?: This is a plain language summary of a published article in the journal . Narcolepsy is a sleep condition that has 2 different subtypes: narcolepsy type 1 and narcolepsy type 2. These are called NT1 and NT2 for short. Sodium oxybate (SXB) is approved to treat excessive daytime sleepiness (EDS) and cataplexy. People with NT1 and NT2 both have EDS, but cataplexy is only present in people with NT1. Limited information is available about how SXB works in people with NT2. This is because previous trials have included only people with NT1 or people with unspecified narcolepsy. For more than 20 years, the only available formulation of this medicine had to be given twice during the night. Many people with narcolepsy find that chronically waking up in the middle of the night for a second dose of SXB is disruptive to themselves or others in their household. People have also reported sleeping through alarm clocks, missing their second dose, and feeling worse the next day. Some people have accidentally taken the second dose too early, putting them at risk for serious adverse effects. These adverse effects may include slow breathing, low blood pressure, or sedation. The US Food and Drug Administration (FDA) approved a medicine called LUMRYZ (sodium oxybate) for extended-release oral suspension in May 2023. LUMRYZ is a once-nightly formulation of SXB (ON-SXB for short) and is taken as a single dose before bedtime. This medicine treats EDS and muscle weakness (also known as cataplexy) in people with narcolepsy. A clinical trial called REST-ON studied ON-SXB to find out if it was better at treating narcolepsy symptoms than a medicine with no active ingredients (placebo). This summary describes a study that tested whether ON-SXB was better than placebo at treating narcolepsy symptoms in people with NT1 or NT2.
WHAT WERE THE RESULTS?: This study showed that compared to people who took placebo, people who took ON-SXB were able to stay awake longer during the day, felt less sleepy during the daytime, had less cataplexy, and had more improvements in their symptoms overall than people who took placebo.
WHAT DO THE RESULTS MEAN?: ON-SXB has been proven effective for people with NT1 or NT2. Unlike prior formulations of SXB, ON-SXB is taken once at bedtime, without requiring waking up in the middle of the night for a second dose.
这是一篇发表在期刊上的文章的通俗易懂的摘要。发作性睡病有两种不同的亚型：发作性睡病 1 型和发作性睡病 2 型。这两种类型分别简称为 NT1 和 NT2。羟丁酸钠（SXB）被批准用于治疗白天过度嗜睡（EDS）和猝倒。NT1 和 NT2 患者都有 EDS，但只有 NT1 患者会出现猝倒。关于 SXB 在 NT2 患者中的作用机制，信息有限。这是因为之前的试验只包括 NT1 患者或未特指的发作性睡病患者。20 多年来，这种药物唯一可用的制剂必须在夜间分两次给药。许多发作性睡病患者发现，为了第二剂 SXB 而在半夜频繁醒来，会干扰自己或家中其他人的睡眠。人们还报告说，他们会睡过闹钟，错过第二剂，第二天感觉更糟糕。有些人不小心过早地服用了第二剂，使他们面临严重不良反应的风险。这些不良反应可能包括呼吸缓慢、血压降低或镇静。美国食品和药物管理局（FDA）于 2023 年 5 月批准了一种名为 LUMRYZ（羟丁酸钠）的药物，用于延长释放口服混悬剂。LUMRYZ 是 SXB 的一种每晚一次的制剂（简称 ON-SXB），在睡前服用一剂。这种药物可治疗发作性睡病患者的 EDS 和肌肉无力（也称为猝倒）。一项名为 REST-ON 的临床试验研究了 ON-SXB，以确定它在治疗发作性睡病症状方面是否优于无活性成分的药物（安慰剂）。本摘要描述了一项研究，该研究测试了 ON-SXB 在治疗 NT1 或 NT2 患者的发作性睡病症状方面是否优于安慰剂。
结果如何？这项研究表明，与服用安慰剂的患者相比，服用 ON-SXB 的患者白天保持清醒的时间更长，白天感觉更不困倦，猝倒发作更少，整体症状改善更多。
结果意味着什么？ON-SXB 已被证明对 NT1 或 NT2 患者有效。与之前的 SXB 制剂不同，ON-SXB 只需在睡前服用一次，无需在半夜醒来服用第二剂。
