Update on treatment for idiopathic hypersomnia

ABSTRACT

Introduction: Idiopathic hypersomnia (IH) is a poorly characterized orphan central disorder of hypersomnolence responsible for excessive daytime sleepiness (EDS), prolonged nighttime sleep and sleep inertia that often require long-term symptomatic stimulant medication. To date, no drug has currently the authorization for the treatment of IH patients worldwide.

Areas covered: The authors reviewed data on pharmacological treatment of IH obtained from published literature (Medline/PubMed/Web of Science) and Clinicaltrial.gov database from 1997 to 2017. Most of data on treatment of IH derived from observational studies and case series with only three well-designed clinical trials available.

Expert opinion: In two recent randomized, double-blind, placebo-controlled trials, modafinil improves EDS in IH. Most of other wakefulness-promoting agents labeled for narcolepsy have similar efficacy in cases series of IH patients. Pitolisant and sodium oxybate show promising results in two retrospective studies. The efficacy of γ-aminobutyric acid-A receptor antagonists on objective EDS needs to be clarified. All these medications are used off-label for the management of EDS in IH. Specific clinical instruments and objective tests are required in IH to better evaluate the severity of EDS and responsiveness to medications, but also prolonged sleep and sleep inertia, to optimize the whole management of IH patients.

KEYWORDS:

• GABA
• idiopathic hypersomnia
• Modafinil
• Sodium oxybate
• WAKEFULNESS-promoting agents

Article highlights

• Little is known about the pathophysiology, clinical characterization and treatment response of IH.

• Due to insufficient level of evidences, no treatment has currently an indication for the treatment of EDS in IH.

• Two recent well-designed studies confirmed the efficacy of modafinil for EDS in IH.

• Pitolisant and sodium oxybate are promising medications in IH

• More specific tools are needed to better assess the severity of the symptoms of IH and the treatment responsiveness

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Declaration of interest

Y. Dauvilliers served as consultant for, and received research/grant support from Bioprojet Pharma, Flamel Technologies, Jazz Pharmaceuticals, Theranexus, Takeda and UCB. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.