講師資料
Talks：
the Wake promoting agent with or without psychostimulant properties
Name：
Dr. Christian CAUSSÉ
Position：
Medical Affairs Director
Affiliation：
Bioprojet Pharma
Email：
Photo：
Research Interests：
Sleep disorders, narcolepsy, Obstructive Sleep Anea
Selected Publications：
1. Dauvilliers, Yves et al. “Safety and efficacy of pitolisant in children aged 6 years or older with narcolepsy with or without cataplexy: a double-blind, randomised, placebo-controlled trial.” The Lancet. Neurology vol. 22,4 (2023): 303-311. doi:10.1016/S1474-4422(23)00036-4
2. Pépin, Jean-Louis et al. “Long-Term Efficacy and Safety of Pitolisant for Residual Sleepiness Due to OSA.” Chest vol. 165,3 (2024): 692-703. doi:10.1016/j.chest.2023.11.017
3. Craig, Sonya et al. “Investigation and management of residual sleepiness in CPAP-treated patients with obstructive sleep apnoea: the European view.” European respiratory review : an official journal of the European Respiratory Society vol. 31,164 210230. 25 May. 2022, doi:10.1183/16000617.0230-2021
Abstract：
Background and Aims
Narcolepsy is a rare disorder occurring from childhood and persisting through adulthood. Pitolisant, a selective histamine H3-receptor inverse agonist, obtained an EMA/FDA approval for the treatment of narcolepsy in adult. We assessed the pitolisant efficacy and safety on Excessive Daytime Sleepiness (EDS) and cataplexy in children.
Methods
This is a double-blind, multicenter, randomized, placebo-controlled study including children with narcolepsy with a Pediatric Daytime Sleepiness Scale (PDSS) score ≥ 15 who were randomized to pitolisant/placebo (2:1) once-a-day for 4-week flexible dosing (5-40mg pitolisant) followed by 4-week stable dosing. The primary endpoint was the improvement of the Ullanlinna Narcolepsy Scale (UNS), a 11-item score measuring cataplexy and EDS in various situations. Main secondary endpoints were changes in PDSS, UNScatapexy subscore (UNSctp), cataplexy episodes per week (WRC), maintenance of wakefulness test (MWT) and safety.
Results
Among 115 selected patients, 110 were randomized (72 pitolisant, 38 placebo). The UNS reduction score was significantly higher in the pitolisant group (from 24.63(7.8) to 18.23(8.14)) versus placebo (from 23.68(9.08) to 21.77(9.25)), difference:-3.69 (1.37), P=0.0073. PDSS reduced from 20.16(3.64) to 14.57(5.37) with Pitolisant vs 20.00(3.49) to 17.96(5.6) with placebo (P=0.0015). The UNSctp also decreased with Pitolisant (-2.88) vs placebo (-1.12) (P=0.029). The improvement of WRC and MWT were better with Pitolisant. The most frequent adverse events for Pitolisant were headache (19.2%) and insomnia (6.8%) versus (8.1%) and (2.7%) for placebo.
Conclusions
In Narcolepsy children above 6 years old, Pitolisant 5 to 40 mg/day demonstrates significant efficacy in reducing Excessive Daytime Sleepiness and cataplexy and is well tolerated.
2024年會：
10/13 11:50 the Wake promoting agent with or without psychostimulant properties. [會議室2]