title: Tolerance and efficacy of sodium oxybate in childhood narcolepsy with cataplexy: a retrospective study.
abstract: Narcolepsy with cataplexy is a sleep disorder characterized by excessive daytime sleepiness, irresistible sleep episodes, and sudden loss of muscle tone (cataplexy) mostly triggered by emotions. Narcolepsy with cataplexy is a disabling lifelong disorder frequently arising during childhood. Pediatric narcolepsy often results in severe learning and social impairment. Improving awareness about this condition increases early diagnosis and may allow patients to rapidly access adequate treatments, including pharmacotherapy and/or non-medication-based approaches. Even though children currently undergo pharmacotherapy, data about safety and efficacy in the pediatric population are scarce. Lacking international guidelines as well as drugs registered for childhood narcolepsy with cataplexy, physicians have no other alternative but to prescribe in an off-label manner medications identical to those recommended for adults. We retrospectively evaluated 27 children ranging from 6 to 16 years old, suffering from narcolepsy with cataplexy, who had been treated with off-label sodium oxybate and had been followed in a clinical setting. Throughout a semi-structured interview, we documented the good efficacy and tolerability of sodium oxybate in the majority of the patients. This study constitutes a preliminary step towards a further randomized controlled trial in childhood narcolepsy with cataplexy.
authors.affiliation: Pediatric Sleep Center, National Reference Network for Orphan Diseases-Narcolepsy and Idiopathic Hypersomnia, CHU Robert-Debrée, Paris VII University, Paris, France. michel.lecendreux@rdb.aphp.fr
authors.forename: Michel
authors.initials: M
authors.lastname: Lecendreux
authors.forename: Francesca
authors.initials: F
authors.lastname: Poli
authors.forename: Delphine
authors.initials: D
authors.lastname: Oudiette
authors.forename: Fatima
authors.initials: F
authors.lastname: Benazzouz
authors.forename: Claire E H M
authors.initials: CE
authors.lastname: Donjacour
authors.forename: Christian
authors.initials: C
authors.lastname: Franceschini
authors.forename: Elena
authors.initials: E
authors.lastname: Finotti
authors.forename: Fabio
authors.initials: F
authors.lastname: Pizza
authors.forename: Oliviero
authors.initials: O
authors.lastname: Bruni
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Plazzi
mesh_terms: D000293:Adolescent; D002648:Child; D004361:Drug Tolerance; D005260:Female; D006801:Humans; D006993:Hypnotics and Sedatives / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D009290:Narcolepsy / Q000188:drug therapy*; D012189:Retrospective Studies; D012978:Sodium Oxybate / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D006993:Hypnotics and Sedatives; D012978:Sodium Oxybate