https://orcid.org/0000-0003-1743-0704
![Sample our Health and Social Care journals, sign in here to start your FREE access for 14 days]({"type":"image" , "src" : "/sda/5938/TOC-Subject-Sample-2021-Health-Social-Care.jpg"})
Abstract
Narcolepsy is a neurological disorder of the sleep-wake cycle characterized by excessive daytime sleepiness (EDS), cataplexy, nighttime sleep disturbances, and REM-sleep-related phenomena (sleep paralysis, hallucinations) that intrude into wakefulness. Dysfunction of the hypocretin/orexin system has been implicated as the underlying cause of narcolepsy with cataplexy. In most people with narcolepsy, symptom onset occurs between the ages of 10 and 35 years, but because the disorder is underrecognized and testing is complex, delays in diagnosis and treatment are common. Narcolepsy is treated with a combination of lifestyle modifications and medications that promote wakefulness and suppress cataplexy. Treatments are often effective in improving daytime functioning for individuals with narcolepsy, but side effects and/or lack of efficacy can result in suboptimal management of symptoms and, in many cases, significant residual impairment. Additionally, the psychosocial ramifications of narcolepsy are often neglected. Recently two new pharmacologic treatment options, solriamfetol and pitolisant, have been approved for adults, and the indication for sodium oxybate in narcolepsy has been expanded to include children. In recent years, there has been an uptick in patient-centered research, and promising new diagnostic and therapeutic options are in development. This paper summarizes current and prospective pharmacological therapies for treating both EDS and cataplexy, discusses concerns specific to children and reproductive-age women with narcolepsy, and reviews the negative impact of health-related stigma and efforts to address narcolepsy stigma.
Disclosure
Dr. Emily C. Barker is a medical writer and has worked as an independent contractor generating reports and summaries for medical advisory board meetings and round-table discussions for Jazz Pharmaceuticals and has also received speaker honoraria from Jazz Pharmaceuticals. Julie Flygare, JD is employed as the President & CEO of Project Sleep, a 501(c)(3) non-profit organization dedicated to raising awareness about sleep health and sleep disorders. As a patient-perspective expert, Ms. Flygare has consulted on patient-centricity and health communications best practices with drug developers including Harmony Biosciences, Jazz Pharmaceuticals, Avadel Pharmaceuticals and Takeda Pharmaceuticals. She also receives royalties from sales of her book, Wide Awake & Dreaming: A Memoir of Narcolepsy. Dr. Shalini Paruthi is a clinical educator and sleep medicine physician, with focus in pediatrics. She gives professional lectures and receives honoraria from conference organizers and royalties from UpToDate. Dr. Paruthi is an uncompensated member of the Board of Directors of the Restless Legs Syndrome Foundation. Dr. Katherine M. Sharkey is a sleep medicine physician; she recently participated in an early-access patient program for pitolisant sponsored by Harmony Biosciences. As an academic physician, she gives professional talks and receives honoraria from conference organizers and royalties from UpToDate. Her research is funded through grant support from NIH and the Hassenfeld Institute. Dr. Sharkey is an uncompensated founding member of TIMES UP Healthcare. The authors report no other conflicts of interest in this work.