2025 American Academy of Neurology Abstract Website

Objective:

To understand the patient experience and identify unmet needs, a survey of patients with narcolepsy type 1 (narcolepsy with cataplexy [NT1]) was conducted.

Background:

Narcolepsy is a lifelong disease with a significant burden of illness. Patients exhibit a broad spectrum of symptoms, which often require multiple treatments.

Design/Methods:

CRESCENDO was a survey of adults diagnosed with NT1 and currently on an FDA-approved medication for the condition, conducted from October-December 2023. The survey assessed symptom burden and impact on quality of life and was developed and executed in partnership with Narcolepsy Network. A third-party research firm conducted the survey and ensured patient privacy.

Results:

Survey respondents comprised 203 people with NT1. The most used narcolepsy treatments were wake promoting agents (53%), oxybates (47%), and stimulants (42%). The average age respondents reported first experiencing symptoms was 18 years; average age of diagnosis was 28 (range 8-67) years. Depression (37%), anxiety (37%), sleep apnea (31%), and obesity (24%) were the most frequently reported diagnosed co-occurring conditions. Most respondents reported persistent cataplexy (77%). Sixty-five percent of respondents reported that cataplexy burdened their professional life, 60% their social life, and 50% their day-to-day life; 64% and 68% reporting being embarrassed by falling down and slurred speech, respectively. Additional persistent symptoms were also observed: EDS (Epworth Sleepiness Scale; score >10) in 64% of respondents, cognitive impairment (British Columbia-Cognitive Complaints Inventory; score ≥5) in 74%, and depressive symptoms (Patient Health Questionnaire-8; score ≥5) in 78%.

Conclusions:

The CRESCENDO survey provides valuable patient-generated insights into the burden of illness in persons with NT1. Despite ongoing treatment for narcolepsy, survey respondents reported a wide range of persistent symptoms including cataplexy, EDS, and cognitive impairment, with significant impact on quality of life. These results can be used to better address the significant unmet needs of this patient population.