Abstract Introduction Narcolepsy type 1 (NT1; narcolepsy with cataplexy) is a chronic disease which often requires multiple treatments to address its symptoms. Although cataplexy is a core symptom of narcolepsy, it often goes underrecognized and can cause limitations to daily life. The CRESCENDO survey evaluated the experience of treated patients with NT1. Here we conducted a subgroup analysis of respondents reporting residual cataplexy to better understand the burden and impact on quality of life (QoL). Methods CRESCENDO was developed in partnership with the Narcolepsy Network, conducted (October–December 2023) by a third-party research firm, and included adults diagnosed with NT1 currently taking FDA-approved narcolepsy medication. Results Patients reporting residual cataplexy (n=156; 76.8%) were older (median age 42.0 vs 38.0 yrs) and more likely to identify as White (76.3% vs 51.1%) and female (70.5% vs 42.6%), compared with those reporting no residual cataplexy (n=47). Polypharmacy was more common in the residual cataplexy group (65.4%) compared with the no residual cataplexy group (44.7%). The residual cataplexy group was more likely to be currently treated with antidepressants (42.9 % vs 23.4%) and less likely to be currently treated with oxybates (42.3% vs 61.7%). Patients with residual cataplexy reported greater subjective cognitive impairment (mean BC-CCI score 9.1 vs 5.2), depressive symptoms (mean PHQ-8 score 10.5 vs 7.0), and EDS (mean ESS score 13.6 vs 8.9). Patients with residual cataplexy also reported greater burden related to cognitive (any difficulty with work/school 92.9% vs 83.0%, family/friends relationship 80.8% vs 61.7%, or social activity enjoyment 88.5% vs 66.0%) and depressive (48.7% vs 34.0% reporting very/extreme difficulty with work/home/social life) symptoms. Other residual symptoms were more frequently self-reported in the residual cataplexy group (EDS, 94.9% vs 78.7%; brain fog 87.2% vs 44.7%; depression 50.0% vs 29.8%). Conclusion Despite current treatment, residual cataplexy was common among patients with NT1, affecting a majority of NT1 patient survey respondents. These patients reported a substantial symptom burden and reduced QoL. There is prevailing unmet need for additional treatment options to treat cataplexy and improve outcomes in this population. Support (if any) Axsome Therapeutics Inc.
Thorpy et al. (Fri,) studied this question.