每晚一次的氧酸钠(FT218)治疗1型和2型发作性睡病的疗效:来自3期REST-ON试验的事后分析

IF 5.3 2区 医学 Q1 CLINICAL NEUROLOGY
Sleep Pub Date : 2023-11-08 DOI:10.1093/sleep/zsad152
Yves Dauvilliers, Thomas Roth, Richard Bogan, Michael J Thorpy, Anne Marie Morse, Asim Roy, Jordan Dubow, Jennifer Gudeman
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引用次数: 0

摘要

研究目的:3期REST-ON试验的事后分析评估了每晚一次的缓释氧酸钠(ON-SXB;FT218)与安慰剂对1型(NT1)和2型(NT2)发作性睡病患者白天嗜睡和夜间睡眠中断的影响。方法:参与者按发作性睡病类型分层,按1:1随机分配ON-SXB (4.5 g,第1周;6克,2-3周;7.5 g,第4-8周;9克(第9-13周)或安慰剂。评估包括NT1和NT2亚组中清醒维持测试(MWT)和临床总体印象改善(gi - i)评分的平均睡眠潜伏期(主要终点)和睡眠阶段变化、夜间觉醒、患者报告的睡眠质量、睡眠的清新性和Epworth嗜睡量表(ESS)评分(次要终点)。结果:修正意向治疗人群包括190名参与者(NT1, n = 145;NT2, n = 45)。与安慰剂相比,ON-SXB在NT1(所有剂量,p < 0.001)和NT2(6和9 g, p < 0.05)亚组的睡眠潜伏期有显著改善。与安慰剂相比,两个亚组中更大比例的参与者使用ON-SXB的CGI-I评分得到了很大/非常大的改善。两个亚组的睡眠阶段变化和睡眠质量都有显著改善(所有剂量与安慰剂相比,p < 0.001)。与安慰剂相比,所有ON-SXB剂量在NT1的睡眠清新性(p < 0.001)、夜间唤醒(p < 0.05)和ESS评分(p≤0.001)方面均有显著改善,而NT2则有方向性改善。结论:单一ON-SXB睡前剂量对NT1和NT2患者的日间嗜睡和DNS有临床意义的改善,对有限NT2亚组的作用较小。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Efficacy of once-nightly sodium oxybate (FT218) in narcolepsy type 1 and type 2: post hoc analysis from the Phase 3 REST-ON Trial.

Study objectives: Post hoc analyses from the phase 3 REST-ON trial evaluated efficacy of extended-release once-nightly sodium oxybate (ON-SXB; FT218) vs placebo for daytime sleepiness and disrupted nighttime sleep in narcolepsy type 1 (NT1) and 2 (NT2).

Methods: Participants were stratified by narcolepsy type and randomized 1:1 to ON-SXB (4.5 g, week 1; 6 g, weeks 2-3; 7.5 g, weeks 4-8; and 9 g, weeks 9-13) or placebo. Assessments included mean sleep latency on Maintenance of Wakefulness Test (MWT) and Clinical Global Impression-Improvement (CGI-I) rating (coprimary endpoints) and sleep stage shifts, nocturnal arousals, and patient-reported sleep quality, refreshing nature of sleep, and Epworth Sleepiness Scale (ESS) score (secondary endpoints) separately in NT1 and NT2 subgroups.

Results: The modified intent-to-treat population comprised 190 participants (NT1, n = 145; NT2, n = 45). Significant improvements were demonstrated with ON-SXB vs placebo in sleep latency for NT1 (all doses, p < .001) and NT2 (6 and 9 g, p < .05) subgroups. Greater proportions of participants in both subgroups had CGI-I ratings of much/very much improved with ON-SXB vs placebo. Sleep stage shifts and sleep quality significantly improved in both subgroups (all doses vs placebo, p < .001). Significant improvements with all ON-SXB doses vs placebo in refreshing nature of sleep (p < .001), nocturnal arousals (p < .05), and ESS scores (p ≤ .001) were reported for NT1 with directional improvements for NT2.

Conclusions: Clinically meaningful improvements of a single ON-SXB bedtime dose were shown for daytime sleepiness and DNS in NT1 and NT2, with less power for the limited NT2 subgroup.

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来源期刊
Sleep
Sleep 医学-临床神经学
CiteScore
10.10
自引率
10.70%
发文量
1134
审稿时长
3 months
期刊介绍: SLEEP® publishes findings from studies conducted at any level of analysis, including: Genes Molecules Cells Physiology Neural systems and circuits Behavior and cognition Self-report SLEEP® publishes articles that use a wide variety of scientific approaches and address a broad range of topics. These may include, but are not limited to: Basic and neuroscience studies of sleep and circadian mechanisms In vitro and animal models of sleep, circadian rhythms, and human disorders Pre-clinical human investigations, including the measurement and manipulation of sleep and circadian rhythms Studies in clinical or population samples. These may address factors influencing sleep and circadian rhythms (e.g., development and aging, and social and environmental influences) and relationships between sleep, circadian rhythms, health, and disease Clinical trials, epidemiology studies, implementation, and dissemination research.
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