0693 Demographics and Disease Characteristics Among Pediatric Patients with Narcolepsy

Abstract Introduction Narcolepsy is a chronic disorder, with symptom onset frequently occurring between ages 10-25 years. Clinical and demographic information is lacking in pediatric patients with narcolepsy. It is particularly important to understand the path to diagnosis for pediatric patients with narcolepsy, as missed or delayed diagnosis may have adverse effects on quality of life and impact social and academic development. This study was conducted to better understand this population, including the prevalence of narcolepsy subtypes, time from symptom onset to diagnosis, and patterns of medication use. Methods A retrospective review of Pulse Infoframe registry data of patients aged ≤18 years with a diagnosis of narcolepsy type 1 (NT1) or 2 (NT2) from 22 US hospitals between 2009-2017 was conducted. All patients included in the analysis had undergone a clinical evaluation, an overnight polysomnographic study, and a Multiple Sleep Latency Test. Data were analyzed descriptively. Results In total, 471 pediatric patients were identified in the dataset (NT1, 70.3%; NT2, 29.5%; unknown narcolepsy type, 0.2%; female, 47.6%; white, 48.2%; Black, 41.2%; non-Hispanic, 89.4%). The most reported Tanner stage classifications were stage 1 (n=79 [16.8%]) and stage 5 (n=55 [11.7%]); 48.9% (n=235) of classifications were not available. The median (IQR) ages at the onset of excessive daytime sleepiness (EDS) and at diagnosis were 9.0 (6.9-12.5) years and 12.0 (8.5-15.0) years, respectively. Mean (SD) Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) scores in patients before treatment were 17.8 (4.5). Over half of the patients reported restless sleeping (62.6%), sleep maintenance problems (58.4%), sleep talking (57.3%), and snoring (54.4%). The most frequently reported comorbidity was excessive weight gain (48.4%). Prior treatments included stimulants (87.9%), wake-promoting agents (69.4%), and antidepressants (50.7%); 30.6% had tried sodium oxybate. Conclusion In this pediatric dataset, most patients reported a delay of ~3 years from symptom onset to narcolepsy diagnosis, with NT1 being a more prevalent diagnosis than NT2, and a wide range of pubertal developmental stages at diagnosis was observed. ESS-CHAD scores reflected substantial levels of EDS; sleep-related symptoms were also common in these patients. These findings emphasize the need for timely diagnosis and access to effective treatments for these patients. Support (if any) Avadel Pharmaceuticals