Demographics and Disease Characteristics Among Pediatric Patients With Narcolepsy
Kiran Maski1, Anne Morse2, Sally Ibrahim3, Rakesh Bhattacharjee4, Judith Owens5, Luis Ortiz6, Gary Feldman7, Narong Simakajornboon8, Joshua Henderson9, Jennifer Gudeman10, Brian Abaluck10, Femida H. Gwadry Sridhar9
1Boston Children'S Hospital, 2Geisinger Medical Center, 3University Hospitals Cleveland Medical Center, 4University of California, 5Boston Children's Hospital, Harvard Medical School, 6Johns Hopkins Medical Institutions, Johns Hopkins All Children’s Hospital, 7Ocean Sleep Medicine, 8Cincinnati Children’s Hospital Medical Center, 9Pulse Infoframe, 10Avadel Pharmaceuticals
Objective:
To better understand the subtype prevalence, time to diagnosis, and medication use of pediatric patients with narcolepsy.
Background:
Narcolepsy is a chronic disorder with symptom onset frequently occurring between age 10-25 years. Clinical and demographic information is lacking in pediatric patients with narcolepsy.
Design/Methods:
A retrospective review of 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 were assessed. 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, 0.2%; female, 47.6%; white, 48.2%; black, 41.2%; non-Hispanic, 89.4%). The most common reported Tanner stage score classifications were stage 1 (n=79 [16.8%]) and stage 5 (n=55 [11.7%]; not available, n=235 [48.9%]). 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 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.
Conclusions:
In this 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.
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