Individualizing Narcolepsy Care: Real-World Oxybate Outcomes

Laura Herpel, MD, discussed clinical interpretations of the REFRESH study, a real-world investigation of once-nightly sodium oxybate in patients with narcolepsy, emphasizing the correspondence between real-world and randomized clinical trial data and the broader functional benefits observed beyond sleepiness reduction.¹

Herpel noted that the improved outcomes observed in oxybate-treated patients were consistent with prior literature, and that real-world tolerability and adverse event profiles in REFRESH closely matched those reported in randomized clinical trials.² She characterized the disability and quality-of-life findings as particularly notable, emphasizing that meaningful narcolepsy management extends beyond improvement in sleepiness scores alone to encompass functional participation in work and social domains.

A key finding Herpel highlighted involved the 7-item patient-rated impact questionnaire, which captured improvements not only in core narcolepsy symptoms but also in commonly underrecognized comorbidities, including anxiety, depression, brain fog, and fatigue. She explained that excessive daytime sleepiness, typically measured using the Epworth Sleepiness Scale, often co-occurs with disrupted nighttime sleep, and that improved nighttime sleep stability may confer benefits not achieved with wake-promoting agents or stimulants alone. She noted with particular interest the reduction in stimulant use observed in both oxybate-naive patients and those switching from twice-nightly oxybate formulations.

Herpel acknowledged the inherent analytic challenges of real-world data, including confounding variables and a likely selection bias toward patients motivated to switch therapies for specific reasons. Despite these limitations, she stated: "to know that we have to have individualized therapy for our patients and if something is not working for them, there's other options and we have ways to continue to make improvements in care." She also noted the added value of incorporating partner-reported observations regarding ease of use and perceived patient improvement.

Dr Herpel practices at Bogan Sleep Consultants in Columbia, South Carolina, and is adjunct faculty at the Medical University of South Carolina in Charleston.

References

1. Meskill S, Meskill G, Herpel L, et al. REFRESH: prospective, observational study design of once-nightly sodium oxybate for narcolepsy in clinical practice. Sleep. 2025;48(Suppl 1):A372.

2. Ibrahim S, Harsh J, Corser B, et al. Long-term safety and tolerability of once-nightly sodium oxybate: a post hoc analysis from RESTORE. Sleep. 2025;48(Suppl 1):A361.