Clinical characteristics of a large cohort of patients with narcolepsy candidate for pitolisant: a cross-sectional study from the Italian PASS Wakix® Cohort

Abstract

Introduction Narcolepsy is a chronic and rare hypersomnia of central origin characterized by excessive daytime sleepiness and a complex array of symptoms as well as by several medical comorbidities. With growing pharmacological options, polytherapy may increase the possibility of a patient-centered management of narcolepsy symptoms. The aims of our study are to describe a large cohort of Italian patients with narcolepsy who were candidates for pitolisant treatment and to compare patients’ subgroups based on current drug prescription (drug-naïve patients in whom pitolisant was the first-choice treatment, switching to pitolisant from other monotherapy treatments, and adding on in polytherapy). Methods We conducted a cross-sectional survey based on Italian data from the inclusion visits of the Post Authorization Safety Study of pitolisant, a 5-year observational, multicenter, international study. Results One hundred ninety-one patients were enrolled (76.4% with narcolepsy type 1 and 23.6% with narcolepsy type 2). Most patients (63.4%) presented at least one comorbidity, mainly cardiovascular and psychiatric. Pitolisant was prescribed as an add-on treatment in 120/191 patients (62.8%), as switch from other therapies in 42/191 (22.0%), and as a first-line treatment in 29/191 (15.2%). Drug-naive patients presented more severe sleepiness, lower functional status, and a higher incidence of depressive symptoms. Conclusion Our study presents the picture of a large cohort of Italian patients with narcolepsy who were prescribed with pitolisant, suggesting that polytherapy is highly frequent to tailor a patient-centered approach.

Long-Term Treatment of Narcolepsy and Idiopathic Hypersomnia with Low-Sodium Oxybate

“…1,[15][16][17][18][19] The prevalence of narcolepsy in 2016 was estimated at 44.3 per 100,000 persons. 7 In association with the sleep/wake state instability and EDS of narcolepsy, the following are common: decreased functioning and ability to pursue activities, 4,[20][21][22] decreased QoL, 4,5,23,24 increased usage of medical resources and associated health-care costs, 25 and reduced productivity and increased associated costs (eg, short-term disability incidents and days). 25 Idiopathic hypersomnia is also characterized by EDS, 1 but in contrast to narcolepsy, patients emphasize virtually continuous cognitive aspects of EDS (eg, impaired alertness and difficulty sustaining attention; "brain fog") as being especially burdensome.…”

“…87,88 In the study of LXB in narcolepsy, 89 31.8% and 35.3% of participants were overweight or obese, respectively, at study entry, consistent with the well-established increased risk of obesity in narcolepsy. 21,36,37 At the end of the SDP, with a total of 14 weeks of LXB treatment, mean (SD) change in body weight in the overall population was −1.6 (3.5) kg. 89 Mean weight loss was numerically greater in oxybate-naive participants compared with participants who had been taking SXB at study entry; among oxybate-naive participants, mean weight loss was also numerically greater in participants who were overweight or obese compared with normal weight.…”

Sleep and Stroke: Opening Our Eyes to Current Knowledge of a Key Relationship

“…Also, an increased risk of cardiovascular comorbidity is observed in patients with narcolepsy type 1 [6], a central hypersomnia characterized by orexin deficiency. Patients with narcolepsy show lack of nocturnal blood pressure dipping, and disrupted nighttime sleep, and other comorbidities (e.g., obesity, diabetes, and mood disorders) that may concur to raise the cardiovascular risk [54]; furthermore, drugs used to manage narcoleptic symptoms may concur to increase cardiovascular risk [99].…”

Is pitolisant safe for clinical use? A retrospective pharmacovigilance study focus on the post‐marketing safety

“…Different from traditional stimulants, pitolisant works by enhancing the release of histamine, promoting wakefulness and reducing excessive sleepiness 14 . The robust efficacy of pitolisant for the reduction in both EDS and cataplexy has been demonstrated 15–18 . In addition, pitolisant has been found to be well‐tolerated and has a low risk of abuse.…”