[Real-life WAKE study in narcolepsy patients with cataplexy treated with pitolisant and unresponsive to previous treatments].

Autor: Del Río-Villegas R; Hospitales Vithas, Madrid, España., Martínez-Orozco FJ; Hospital Clínico San Carlos, 28040 Madrid, España., Romero-Santo Tomás O; Hospital Universitario Vall d´hebron, Barcelona, España., Yébenes-Cortés M; Pharmacoeconomics and Outcomes Research Iberia, Pozuelo de Alarcón, España., Gómez-Barrera M; Pharmacoeconomics and Outcomes Research Iberia, Pozuelo de Alarcón, España., Gaig-Ventura C; Hospital Clínic de Barcelona, 08036 Barcelona, España.
Jazyk: Spanish; Castilian
Zdroj: Revista de neurologia [Rev Neurol] 2022 Oct 01; Vol. 75 (7), pp. 165-171.
DOI: 10.33588/rn.7507.2022090
Abstrakt: Introduction: Type 1 narcolepsy is a disabling disease that requires continuous treatment, which is not always effective. Pitolisant is a new drug with a different mechanism of action that offers a new treatment option. The objective of the study was to analyse the effectiveness and safety of pitolisant in patients with type 1 narcolepsy that did not respond to or tolerate previous standard treatments.
Patients and Methods: Real-life multicentre descriptive observational study that included patients diagnosed with type 1 narcolepsy who did not respond to or tolerate previous treatments and started treatment with pitolisant. The study evaluated three different moments: the start of treatment, the stabilization of treatment with pitolisant and the three months after.
Results: In 32 patients included (mean age, 44 years; 37.5% women) the mean of the Epworth Sleepiness Scale was reduced from 17.1 to 13.5; 47.8% of the patients improved from their cataplexy; 65% of the patients improved their clinical global impression at the physician's and at the patient's discretion and the mean number of medications consumed was reduced from 2.0 to 1.4. The most frequent adverse effect was insomnia in 43.8% of patients. Of the 32 patients, 23 continued with the treatment during the 3-month follow-up period.
Conclusions: In patients with type I narcolepsy who do not respond to or do not tolerate the available treatments, pitolisant can improve their clinical situation and reduce their medication consumption. Studies with a higher level of evidence are needed to confirm these results.
Databáze: MEDLINE