Update on the treatment of narcolepsy: clinical efficacy of pitolisant

Calik, Michael W.

doi:10.2147/nss.s103462

Cited by 41 publications

(17 citation statements)

References 26 publications

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“…Narcolepsy with cataplexy is a chronic neurological sleep‐wake disorder. The narcolepsy prevalence has been estimated in the range 0.02‐0.05 5% . The age of onset varies between individuals and has a bimodal pattern, peaking at the age of 15 and 36 .…”

Section: Introductionmentioning

confidence: 99%

“…The narcolepsy prevalence has been estimated in the range 0.02-0.05 5%. [1][2][3][4] The age of onset varies between individuals and has a bimodal pattern, peaking at the age of 15 and 36. 5 A recent review demonstrated that the relative risk for developing narcolepsy after Pandemrix vaccination increased fivefold to 14-fold in children and adolescents and twofold to sevenfold in adults, during the first year after vaccination.…”

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confidence: 99%

“…20 Moreover, dose administration of sodium oxybate is impractical, since it is given in the night in order to suppress intense night mares and poor sleep. 3,21 The new drug, pitolisant, has a mechanism of action different from those found among other pharmaceuticals used for treating narcolepsy symptoms, which is important in view of the common apprehension among clinicians that there is a need for improved pharmacotherapy in narcolepsy. Many patients have daily symptoms of the disease although the medication has been optimized.…”

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confidence: 99%

“…) vs the treatment effect reported for group(3). Similarly, the treatment effect of pitolisant as an add-on treatment to standard treatment, compared with standard treatment, was approximated with the comparison of treatment effects between groups (2) and(1) (see, for instance, Drummond, 2015, p. 271).…”

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confidence: 99%

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The cost utility of pitolisant as narcolepsy treatment

Bolin

Niska²,

Pirhonen

et al. 2020

Acta Neurol Scand

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Objectives The cost‐effectiveness of available pharmacological treatments for narcolepsy is largely unknown. Available pharmacological treatments are associated with tolerability, abuse, and adherence issues. Pitolisant is the first inverse agonist of the histamine H3 receptor to be prescribed for the treatment of narcolepsy with and without cataplexy. Studies suggest that pitolisant is both as effective as previously introduced drugs and is associated with fewer adverse effects. The objective in this study was to estimate the cost‐effectiveness of pitolisant as monotherapy, and pitolisant as an adjunctive treatment to modafinil, compared with standard treatment. Materials & Methods Calculations were performed using a Markov model with a 50‐year time horizon. Healthcare utilization and quality‐adjusted life years (QALYs) for each treatment alternative were calculated assuming no treatment effect on survival. Probabilistic sensitivity analyses were performed for treatment effectiveness and healthcare cost parameters. Results The cost per additional quality‐adjusted life year was estimated at SEK 356 337 (10 SEK ≈ 1 Euro) for pitolisant monotherapy, and at SEK 491 128 for pitolisant as an adjunctive treatment, as compared to standard treatment. The cost‐effectiveness measure was demonstrated to be particularly sensitive to the assumptions made concerning indirect effects on total healthcare utilization and the pitolisant treatment cost. Conclusions The incremental cost‐effectiveness ratios were below the unofficial willingness‐to‐pay threshold at SEK 500 000. The estimated costs per additional QALY obtained here are likely to overestimate the true cost‐effectiveness ratio since significant potential indirect effects—pertaining both to labor‐market and household‐related productivity—of treatment are not taken into account.

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Section: Introductionmentioning

confidence: 99%

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confidence: 99%

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confidence: 99%

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confidence: 99%

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The cost utility of pitolisant as narcolepsy treatment

Bolin

Niska²,

Pirhonen

et al. 2020

Acta Neurol Scand

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“…Pitolisant is a new drug with a specific action that increases hypothalamic levels of histamine by antagonising histamine (H3) auto-receptors in the tuberomammillary nucleus, the origin of wake-promoting histaminergic neurones 30. Recent placebo-controlled studies have confirmed a modest positive effect on cataplexy symptoms with improved wakefulness in people with narcolepsy 31.…”

Section: Treatmentmentioning

confidence: 99%

Cataplexy

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2018

Pract Neurol

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Remarkably and almost invariably, the clinical phenomenon of cataplexy results from the loss of around 40 000 hypocretin-containing neurones in the lateral hypothalamus in the context of narcolepsy type 1. Cataplexy reflects the dysregulation of rapid-eye-movement (REM) sleep, such that REM-sleep atonia intrudes inappropriately into wakefulness as brief episodes of either focal or total paralysis of voluntary muscle. The semiology of cataplexy differs between adults and children. A defining and enigmatic aspect is that certain emotional stimuli usually trigger the episodes. Cataplexy can be the most disabling symptom of the narcolepsy syndrome, severely limiting normal activities of daily living. Antidepressant drug therapy at relatively low doses is the traditional treatment; these most likely work through inhibiting REM sleep, predominantly by increasing brain monoamine concentrations. Sodium oxybate is probably the most effective drug for severe cataplexy, taken before overnight sleep and once through the night; its precise mechanism of action remains obscure. Pitolisant is a new agent for treating the excessive daytime sleepiness of narcolepsy that also helps cataplexy control by increasing histamine concentrations in the hypothalamus. Further understanding of the neurobiology of cataplexy and how it relates to hypocretin deficiency should improve our understanding of the brain’s emotional processing and provide insights into REM sleep and its control.

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