TY - JOUR
T1 - Interim analysis of a post-authorization safety study of pitolisant in treating narcolepsy
T2 - A real-world European study
AU - Plazzi, Giuseppe
AU - Mayer, Geert
AU - Bodenschatz, Ralf
AU - Bonanni, Enrica
AU - Cicolin, Alessandro
AU - Della Marca, Giacomo
AU - Dolso, Pierluigi
AU - Strambi, Luigi Ferini
AU - Ferri, Raffaele
AU - Geisler, Peter
AU - Happe, Svenja
AU - Heidbreder, Anna
AU - Herold, Jürgen
AU - Kallweit, Ulf
AU - Leclair-Visonneau, Laurène
AU - Lederer, Katharina
AU - Liguori, Claudio
AU - Meurling, Johan
AU - Parrino, Liborio
AU - Proserpio, Paola
AU - Puligheddu, Monica
AU - Quera Salva, Maria Antonia
AU - Remi, Jan
AU - Romigi, Andrea
AU - Rupprecht, Sven
AU - Savarese, Maria Antonietta
AU - Schaff, Jean-Luc
AU - Terzaghi, Michele
AU - Winter, Yaroslav
AU - Caussé, Christian
AU - Collin, Irène
AU - Lecomte, Isabelle
AU - Dauvilliers, Yves
N1 - Copyright © 2025 The Authors. Published by Elsevier B.V. All rights reserved.
PY - 2025/5
Y1 - 2025/5
N2 - BACKGROUND: Narcolepsy is a chronic sleep disorder characterized mainly by excessive daytime sleepiness (EDS) and cataplexy in the case of narcolepsy type 1 (NT1). Pitolisant is a histamine 3 receptor antagonist/inverse agonist that reduces EDS and cataplexy in patients with narcolepsy.METHODS: We performed a prospective 5-year follow-up, non-interventional study of adults with NT1 and NT2 receiving pitolisant. The primary objectives were to collect information on the long-term safety of pitolisant and analyze the utilization patterns of pitolisant. The secondary objectives were to assess clinical benefit, adherence, impact on patients' quality of life, disease burden, and patient satisfaction. We reported the results of an interim analysis after 42.6 months.RESULTS: The population comprised 370 patients (mean age, 40 ± 15 years; 51.4 % women; NT1, 71.4 %; NT2, 28.6 %); 364 received ≥1 dose of pitolisant. Data were available for 356 patients (97.8 %). Most patients (68.4 %) had ≥1 comorbidity (obesity [BMI≥30], 31.9 %; neuropsychiatric, 31 %; and cardiovascular, 22.8 %). Forty-eight patients (13.2 %) had received no prior narcoleptic treatment, while 98 (31 %) were taking a previous therapy, which was switched to pitolisant. Treatment was combined with pitolisant in 218 (69 %) patients. Pitolisant was discontinued by 131 patients (35.4 %), mainly for safety reasons (14.3 %), lack of response (8.7 %), and patient decision (7.6 %). Overall, 355 treatment-emergent adverse events (3 serious) were reported by 156 patients (42.9 % of safety population), with 218 possibly treatment-related (61.4 %) in 109 patients (29.9 %). Improvements were observed in EDS, cataplexy, and quality of life.CONCLUSIONS: Pitolisant was generally safe and well tolerated in patients with NT1 and NT2 and can be used in both types. Improvements were found in EDS, cataplexy, and quality of life, with good adherence and satisfaction.
AB - BACKGROUND: Narcolepsy is a chronic sleep disorder characterized mainly by excessive daytime sleepiness (EDS) and cataplexy in the case of narcolepsy type 1 (NT1). Pitolisant is a histamine 3 receptor antagonist/inverse agonist that reduces EDS and cataplexy in patients with narcolepsy.METHODS: We performed a prospective 5-year follow-up, non-interventional study of adults with NT1 and NT2 receiving pitolisant. The primary objectives were to collect information on the long-term safety of pitolisant and analyze the utilization patterns of pitolisant. The secondary objectives were to assess clinical benefit, adherence, impact on patients' quality of life, disease burden, and patient satisfaction. We reported the results of an interim analysis after 42.6 months.RESULTS: The population comprised 370 patients (mean age, 40 ± 15 years; 51.4 % women; NT1, 71.4 %; NT2, 28.6 %); 364 received ≥1 dose of pitolisant. Data were available for 356 patients (97.8 %). Most patients (68.4 %) had ≥1 comorbidity (obesity [BMI≥30], 31.9 %; neuropsychiatric, 31 %; and cardiovascular, 22.8 %). Forty-eight patients (13.2 %) had received no prior narcoleptic treatment, while 98 (31 %) were taking a previous therapy, which was switched to pitolisant. Treatment was combined with pitolisant in 218 (69 %) patients. Pitolisant was discontinued by 131 patients (35.4 %), mainly for safety reasons (14.3 %), lack of response (8.7 %), and patient decision (7.6 %). Overall, 355 treatment-emergent adverse events (3 serious) were reported by 156 patients (42.9 % of safety population), with 218 possibly treatment-related (61.4 %) in 109 patients (29.9 %). Improvements were observed in EDS, cataplexy, and quality of life.CONCLUSIONS: Pitolisant was generally safe and well tolerated in patients with NT1 and NT2 and can be used in both types. Improvements were found in EDS, cataplexy, and quality of life, with good adherence and satisfaction.
KW - Humans
KW - Narcolepsy/drug therapy
KW - Male
KW - Female
KW - Adult
KW - Piperidines/therapeutic use
KW - Prospective Studies
KW - Middle Aged
KW - Quality of Life
KW - Europe
KW - Patient Satisfaction
KW - Treatment Outcome
KW - Follow-Up Studies
UR - https://www.scopus.com/pages/publications/85217949520
U2 - 10.1016/j.sleep.2025.02.012
DO - 10.1016/j.sleep.2025.02.012
M3 - Article
C2 - 39978240
SN - 1878-5506
VL - 129
SP - 20
EP - 30
JO - Sleep Medicine
JF - Sleep Medicine
ER -
SDG 3 Good Health and Well-being