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

Autor: Triller A; Center for Narcolepsy and Hypersomnias, Clinical Sleep and Neuroimmunology, Institute of Immunology, University of Witten/Herdecke, Witten, Germany., Pizza F; Department of Biomedical Science and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy., Lecendreux M; Pediatric Sleep Centre, Robert Debre Hospital, Paris, France; Reference Center for Narcolepsy and Hypersomnias, Robert Debre Hospital, Paris, France., Lieberich L; Center for Narcolepsy and Hypersomnias, Clinical Sleep and Neuroimmunology, Institute of Immunology, University of Witten/Herdecke, Witten, Germany., Rezaei R; Center for Narcolepsy and Hypersomnias, Clinical Sleep and Neuroimmunology, Institute of Immunology, University of Witten/Herdecke, Witten, Germany., Pech de Laclause A; Pediatric Sleep Centre, Robert Debre Hospital, Paris, France; Reference Center for Narcolepsy and Hypersomnias, Robert Debre Hospital, Paris, France., Vandi S; Department of Biomedical Science and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy., Plazzi G; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy; Department of Biomedical, Metabolic and Neural Sciences, University of Modena and Reggio Emilia, Modena, Italy., Kallweit U; Center for Narcolepsy and Hypersomnias, Clinical Sleep and Neuroimmunology, Institute of Immunology, University of Witten/Herdecke, Witten, Germany. Electronic address: ulf.kallweit@uni-wh.de.
Jazyk: angličtina
Zdroj: Sleep medicine [Sleep Med] 2023 Mar; Vol. 103, pp. 62-68. Date of Electronic Publication: 2023 Jan 24.
DOI: 10.1016/j.sleep.2023.01.015
Abstrakt: 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.
Competing Interests: Declaration of competing interest UK reports receiving research grants from Bioprojet Pharma, Jazz Pharmaceuticals, and serving as a consultant or advisory board member for AOP Orphan Pharmaceuticals, Bioprojet Pharma, Harmony Biosciences, Jazz Pharmaceuticals, Takeda Pharmaceutical Co., Ltd., and UCB Pharma. ML reports serving as a consultant and participating in advisory boards for NLS Pharma, Jazz Pharmaceuticals, UCB Pharma, and Bioprojet; and receiving grant support from Shire. APdL reports no conflict of interest. LL reports no conflict of interest. GP reports participating in advisory boards for UCB, Jazz Pharmaceuticals, and Bioprojet. FP received fee from Jazz for symposia, support for attending congress from Bioprojet, and participated to advisory board for Takeda. RR reports no conflict of interest. AT reports no conflict of interest. SV reports no conflict of interest.
(Copyright © 2023 Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
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