Real-world treatment of pediatric narcolepsy with pitolisant: A retrospective, multicenter study.

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.

Adjustment to lockdown in children and adolescents with narcolepsy in France.

STUDY OBJECTIVES: The goal of the current study was to explore the effect of a 2-month quarantine on children and adolescents with narcolepsy and to describe the changes in their sleep, mood, and metabolism during this period. METHODS: The study involved 77 patients aged 7 to 23 years with a diagnosis of narcolepsy. Participants had to answer multiple-choice questions to characterize the changes and modifications in their own habits and state of health. RESULTS: Participants reported changes in daytime sleep, nighttime sleep schedules, treatment intake, food intake, weight, and amount of screen time. Most patients reported deterioration in their memory and attention abilities as well as a decrease in their work efficiency. Mood appeared to be less affected, although it deteriorated in less than one-third of the participants who reported feeling sad more often. CONCLUSIONS: The prolonged and complete lockdown seemed to have an effect on children and adolescents with narcolepsy, and changes are often considered by the participants to depend on or to determine an overall quality of adaptation to the situation. This study highlights the importance of maintaining and strengthening time markers in individuals with narcolepsy and should help to establish guidelines that would apply in future quarantine situations. CITATION: de Laclause AP, Konofal E, Bokov P, Delclaux C, Lecendreux M. Adjustment to lockdown in children and adolescents with narcolepsy in France. J Clin Sleep Med. 2022;18(9):2247-2252.

Short Research Article: Impact of a prolonged lockdown on the symptoms of paediatric ADHD and common associated disorders.

OBJECTIVE: Our objective is to explore the change in the severity of ADHD, ODD and anxiety during a two-month lockdown among children in France and the moderating role of behavioural regulation. METHOD: In 235 children with ADHD, the symptom severity of ADHD, ODD and anxiety was investigated one and two months after the beginning of lockdown, and one month after its end. Behavioural regulation skills were estimated with the Behaviour Regulation Index. RESULTS: ADHD, ODD and anxiety scores were increasing or decreasing depending on BRI. CONCLUSION: Baseline behavioural regulation skills may act as a moderating factor for the persistence of ADHD, ODD and anxiety symptoms related to the lockdown.