Update on the treatment of idiopathic hypersomnia: Progress, challenges, and expert opinion.

Idiopathic hypersomnia is a central hypersomnolence disorder of unknown origin characterized by excessive daytime sleepiness despite normal or long sleep time, and frequent severe sleep inertia. Management strategies have been largely derived from expert consensus, due to a lack of disease-specific assessments and reliance on case series and rare randomized controlled studies. Guidelines recommend treatment with off-label medications. Modafinil, which was approved for idiopathic hypersomnia until 2011 in Europe, is the most commonly used treatment and improved sleepiness in two recent randomized placebo-controlled trials. In 2021, low-sodium oxybate (LXB) was approved in the United States for idiopathic hypersomnia. In a placebo-controlled, double-blind, randomized withdrawal study, LXB reduced daytime sleepiness and sleep inertia, and improved daily functioning. Here, treatment options are reviewed considering the authors' professional experience, current guidelines, and the latest research developments. The choice of pharmacotherapy should be guided by symptom profile, age, comorbidities (eg, depressive symptoms, cardiovascular problems), and concomitant medications (eg, oral contraceptives). Nonpharmacologic approaches have a role in management. An instrument (idiopathic hypersomnia severity scale) has been validated in idiopathic hypersomnia specifically, opening a path to better assessment of symptoms, impact, and response to treatment. Continued research on idiopathic hypersomnia is needed to support treatment algorithms.
Competing Interests: Declaration of competing interest Isabelle Arnulf has participated in advisory boards for UCB Pharma, Idorsia (2020), Ono Pharma (2019), and Roche Pharma (2019). Robert Thomas has patents and licenses for ECG-spectrogram, licensed to MyCardio, LLC through the Beth Israel Deaconess Medical Center, and for auto-CPAP algorithm, licensed by BIDMC to DeVilbiss-Drive; holds an unlicensed patent for a CO(2) device for central/complex sleep apnea; is a general sleep medicine consultant for GLG Councils and Guidepoint Global; and is a consultant for and has participated in advisory boards for Jazz Pharmaceuticals. Asim Roy has received consultancy fees from Jazz Pharmaceuticals and Harmony Biosciences. Yves Dauvilliers is a consultant for and has participated in advisory boards for Jazz Pharmaceuticals, UCB Pharma, Avadel, Idorsia, Takeda, Harmony Biosciences, and Bioprojet.
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