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.