Abstract Details

Title Residual Symptom Burden in Patients With Narcolepsy Satisfied With Treatment: Subgroup Analysis From the CRESCENDO Survey

Topic Sleep

Presentation(s) P6 - Poster Session 6 (5:00 PM-6:00 PM)

Poster/Presentation
Number 14-009

Objective To analyze residual symptom burden in a subgroup of respondents from the CRESCENDO survey of patients with narcolepsy type 1 (NT1) who reported satisfaction with their current narcolepsy medication.

Background People with NT1 often experience breakthrough symptoms, despite taking multiple medications. The CRESCENDO survey examined the patient experience in NT1, including a detailed characterization of symptom burden.

Design/Methods CRESCENDO was conducted in 2023 in adults with NT1 taking FDA-approved medication for narcolepsy. The survey included assessments of symptom burden and quality of life impact and was conducted in partnership with patient advocacy organization Narcolepsy Network. Satisfaction was determined on a categorical scale.

Results Of 203 respondents, 60.6% were taking multiple classes of narcolepsy medication. A majority (63.1%) reported satisfaction with their current treatment(s), 20.2% were neutral, and 16.7% were dissatisfied. Reasons for satisfaction included symptom improvement (70%) and the ability to complete more activities (37%). Of satisfied respondents, 71.9% reported breakthrough cataplexy (10.9% experienced ≥1 attack per day, and 22.8% experienced multiple attacks per week). Additionally, 53.3% reported cataplexy burdens their professional lives, 49.3% their social lives, and 38.7% their day-to-day lives. Other breakthrough symptoms included excessive daytime sleepiness (89.1%), brain fog (73.4%), difficulty concentrating (71.9%), depression (67.2%; Patient Health Questionnaire-8 score ≥5), and cognitive complaints (64.8%; British Columbia Cognitive Complaints Inventory score ≥5).

Conclusions Respondents satisfied with their current NT1 medication continued to experience substantial symptom burden. These findings reveal a disconnect between patient-reported treatment satisfaction and symptom resolution, possibly reflecting limitations of current therapies, symptom underreporting, or diminished patient expectations. The results suggest a need for enhanced assessment of residual symptoms and novel treatments.

Authors/Disclosures
Michael J. Thorpy, MD (Montefiore Medical Center) PRESENTER	Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avadel. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for alkermes. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Harmony. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for centessa. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Axsome. Dr. Thorpy has received publishing royalties from a publication relating to health care.
Amy Kant	Amy Kant has nothing to disclose.
Keith Harper	Keith Harper has nothing to disclose.
Eileen Leary, PhD (Axsome Therapeutics)	Dr. Leary has received personal compensation for serving as an employee of Centessa Pharmaceuticals. Dr. Leary has received personal compensation for serving as an employee of Axsome Therapeutics. Dr. Leary has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clinical Partners Group. Dr. Leary has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pulse Infoframe. Dr. Leary has or had stock in Jazz Pharmaceuticals.Dr. Leary has or had stock in Axsome Therapeutics.Dr. Leary has or had stock in Centessa Pharmaceuticals.
Graham M. Eglit, PhD	Dr. Eglit has received personal compensation for serving as an employee of Axsome Therapeutics Inc. Dr. Eglit has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Developmental Neuropsychology: A Life-Span Developmental Journal. Dr. Eglit has stock in Axsome Therapeutics.
Samantha Floam, DMD (Axsome Therapeutics)	Dr. Floam has received personal compensation for serving as an employee of Axsome Therapeutics.
Herriot Tabuteau (Axsome Therapeutics)	Herriot Tabuteau has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Axsome Therapeutics.

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