Abstract Introduction Narcolepsy type 1 (NT1) is a chronic neurological disorder of hypersomnolence, characterized by excessive daytime sleepiness (EDS), cataplexy, disrupted nighttime sleep, hallucinations, sleep paralysis, and cognitive symptoms. NT1 can negatively affect multiple aspects of life (education, work participation, family, quality of life). Large-scale burden of illness studies in NT1, related to disease severity, are lacking. This study examined the impact on various life domains across the spectrum of disease severity among people with NT1 in the US. Methods Cross-sectional online survey of people with NT1, narcolepsy type 2, or idiopathic hypersomnia was conducted in the US. Participants were recruited from clinical sites, patient panels, and patient associations. Questions covered demographics, symptoms, Epworth Sleepiness Scale (ESS), 5-level EQ-5D (mapped to health utilities using a US tariff), education, and employment. NT1 group findings are reported here. Results Overall, 247 participants (female: 74.9%) with NT1, median (IQR) age 40 (31—49) years, were recruited. Median (IQR) ESS total score, 16 (12—19), indicated severe EDS. Comorbidities included back pain (56.2%), anxiety (55.7%), depression (53.1%), and headache (44.3%). Participants (87.2%) had taken narcolepsy medication in the past 30 days. Overall, mean (SD) EQ-5D utility score was 0.61 (0.30); for lower normal daytime sleepiness (ESS 0—5): 0.64 (0.29) and for severe EDS (ESS 16—24): 0.53 (0.32). Overall, 45.3% had a university degree (37.5%, ESS 0—5; 41.9%, ESS 16—24) and 55.3% were employed (75.0%, ESS 0—5; 47.2%, ESS 16—24), of whom 81.5% reported work disruption due to NT1: 50.0% (ESS 0—5); 40.7% (ESS 16—24). Primary reasons for work disruption were fatigue (89.1%), sleepiness (87.3%), and difficulty concentrating (73.6%). In the past 3 months, 35.6% of those employed took sick leave because of their narcolepsy (25.0%, ESS 0—5; 22.8%, ESS 16—24). Of those unemployed, 45.9% received disability benefits. Overall, 57.0% required caregiver assistance. Conclusion Despite current management, NT1 imposes substantial burden beyond clinical outcomes, limiting work capacity and increasing reliance on informal caregivers and disability benefits. Improved treatment efficacy has the potential to improve overall functioning and quality of life. Support (if any) Study was funded by Takeda Development Center Americas, Inc. Medical writing was funded by Takeda Pharmaceuticals U.S.A., Inc.
Jönsson et al. (Fri,) studied this question.