A Survey of People Living with Narcolepsy in the USA: Path to Diagnosis, Quality of Life, and Treatment Landscape from the Patient's Perspective.

IF 7.4 2区医学 Q1 CLINICAL NEUROLOGY

CNS drugs Pub Date : 2025-03-01 Epub Date: 2025-03-20 DOI:10.1007/s40263-024-01142-8

Luis E Ortiz, Anne Marie Morse, Lois Krahn, Maggie Lavender, Matthew Horsnell, Dianna Cronin, Beth Schneider, Jennifer Gudeman

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Abstract

Background: Narcolepsy is a chronic, burdensome neurologic disorder that significantly impacts the daily life of people with narcolepsy (PWN). Real-world perspectives from PWN can help address their unique experiences and treatment needs. PWN were surveyed to examine the path to a narcolepsy diagnosis, the breadth of symptom burden experienced by PWN, and current trends in treatment.

Methods: A 15-min online survey was sent by email to 3959 US members of MyNarcolepsyTeam (February 2022). The survey was divided into three sections (screening [patient characteristics], diagnosis/symptoms, and patient quality of life) for a total of 27 questions.

Results: In total, 110 members completed the survey. Of these, most were female (84%) and nearly half (48%) were diagnosed with narcolepsy type 1 (with cataplexy). Approximately one-third (31%) of members reported receiving a definitive diagnosis ≥ 10 years after first speaking with a clinician; most were previously diagnosed with depression (73%). Excessive daytime sleepiness (EDS, 93%) and fatigue (84%) were the most frequently reported symptoms that prompted respondents to seek a diagnosis or feel that something was wrong. Additionally, EDS was reported as the most troubling symptom (92%). Respondents' most desired treatment outcome was to stop sleeping during the day (77%). Most (76%) indicated an extremely or very severe impact on daily life. One in eight respondents were not taking any medication for their narcolepsy. Of those taking medication, 58% received polypharmacy to address narcolepsy symptoms.

Conclusions: These survey findings further characterize the diagnostic delay, symptom burden, and treatment needs of PWN. Understanding the breadth of impact of narcolepsy from the patients' perspective could improve shared decision-making between PWN and their treating clinicians.

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对美国发作性睡病患者的调查：从患者的角度看诊断、生活质量和治疗前景。

背景：嗜睡症是一种慢性、繁重的神经系统疾病，严重影响嗜睡症患者的日常生活。来自PWN的现实世界视角可以帮助解决他们独特的经历和治疗需求。对PWN进行调查，以检查发作性睡病诊断的途径、PWN所经历的症状负担的广度以及目前的治疗趋势。方法：通过电子邮件向MyNarcolepsyTeam的3959名美国成员发送15分钟的在线调查（2022年2月）。调查分为三个部分（筛查[患者特征]、诊断/症状和患者生活质量），共有27个问题。结果：共有110名成员完成了调查。其中，大多数是女性（84%），近一半（48%）被诊断为1型嗜睡症（伴猝倒）。大约三分之一（31%）的患者报告在首次与临床医生交谈后≥10年接受了明确诊断；大多数人之前被诊断患有抑郁症（73%）。白天过度嗜睡（EDS, 93%）和疲劳（84%）是最常报告的症状，促使受访者寻求诊断或感觉出了问题。此外，据报道EDS是最令人困扰的症状（92%）。受访者最期望的治疗结果是白天停止睡觉（77%）。大多数人（76%）表示对日常生活的影响极其严重或非常严重。八分之一的受访者没有服用任何治疗发作性睡病的药物。在服用药物的患者中，58%的人接受了多种药物治疗，以缓解发作性睡病症状。结论：这些调查结果进一步表征了PWN的诊断延迟、症状负担和治疗需求。从患者的角度了解发作性睡病影响的广度可以改善PWN及其治疗临床医生之间的共同决策。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

CNS drugs 医学-精神病学

CiteScore

12.00

自引率

3.30%

发文量

审稿时长

6-12 weeks

期刊介绍： CNS Drugs promotes rational pharmacotherapy within the disciplines of clinical psychiatry and neurology. The Journal includes: - Overviews of contentious or emerging issues. - Comprehensive narrative reviews that provide an authoritative source of information on pharmacological approaches to managing neurological and psychiatric illnesses. - Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. - Adis Drug Reviews of the properties and place in therapy of both newer and established drugs in neurology and psychiatry. - Original research articles reporting the results of well-designed studies with a strong link to clinical practice, such as clinical pharmacodynamic and pharmacokinetic studies, clinical trials, meta-analyses, outcomes research, and pharmacoeconomic and pharmacoepidemiological studies. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in CNS Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.