AI Article Synopsis
- Narcolepsy typically starts in childhood, but there are limited medication options for young patients; Pitolisant, approved for adults, shows potential benefits for children and adolescents with narcolepsy.
- A study involving 55 patients aged 6-18 from three European centers assessed the effects of Pitolisant on symptoms, finding significant reductions in excessive daytime sleepiness and cataplexy frequency.
- The treatment was generally well-tolerated, with mild side effects, primarily short-term insomnia, indicating that Pitolisant could be a promising option for managing narcolepsy in younger patients.
Article Abstract
Background: First symptoms of narcolepsy mostly present during childhood. Pharmacological management options in children are limited, also due to approval status. Pitolisant is an inverse histamine 3 receptor agonist and has been approved for the treatment of adult narcolepsy with or without cataplexy by EMA and FDA. Clinical experience indicates for a beneficial use also in children and adolescents. Our goal was to evaluate the effects and tolerability of pitolisant in narcolepsy children/adolescents in a real-world setting.
Methods: This multicentre retrospective observational study included 55 patients with narcolepsy from three international narcolepsy centers (Germany, France and Italy) who were treated with pitolisant. Patients were eligible if they were at least 6 years old and diagnosed with narcolepsy type 1 or 2. Demographic and clinical characteristics, questionnaires, sleep medicine and laboratory data were collected.
Results: 55 children/adolescents (25 girls, 45.45%, 30 boys, 54.55%) aged 6-18 years, with narcolepsy (type 1 = 92.7%, type 2 = 7.3%), were treated with pitolisant. The mean pitolisant dose was 34.1 mg/d. Treatment was effective for excessive daytime sleepiness (EDS) and cataplexy: the pediatric Epworth Sleepiness Scale (ESS) score decreased from 19 to 13.5 (p < 0.001) and the weekly cataplexy frequency improved from 7.9 at baseline to 5.2 (p < 0.001). Treatment with pitolisant was well tolerated. Side effects were mild and mostly short-term. Insomnia was reported most frequently (5.5%).
Conclusion: First real-world results suggest that pitolisant treatment is effective in improving EDS and cataplexy in children with narcolepsy, and also is well tolerated.
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| http://dx.doi.org/10.1016/j.sleep.2023.01.015 | DOI Listing |
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