Real-World Outcomes With Sodium Oxybate in Narcolepsy

CONFERENCE REPORTER

Laura Herpel, MD, presented findings from the REFRESH study, a real-world, multisite investigation of once-nightly sodium oxybate in patients with type 1 or type 2 narcolepsy.¹

Herpel described that REFRESH enrolled patients across 13 clinical sites who were initiating sodium oxybate therapy, including both patients naive to oxybate therapy and those switching from twice-nightly oxybate formulations. Unlike a randomized controlled trial with a fixed titration schedule, REFRESH employed real-world titration, in which clinicians adjusted dosing according to standard clinical practice. Patients continued concomitant medications as clinically indicated, including stimulants, antidepressants, and antihypertensives. Herpel noted that the study population reflected typical clinical demographics for narcolepsy, with a predominance of female patients and an age distribution consistent with real-world practice patterns.

Patients completed questionnaires at baseline and at 4-week intervals through 4 months of therapy. Outcome measures included sleepiness scores, narcolepsy severity scores, and the Sheehan Disability Scale, which assesses work and life functioning. A 0-to-10 self-rated impairment scale was also administered at baseline and study end to capture the impact of comorbid factors commonly associated with narcolepsy, including depression, anxiety, quality of life, fatigue, and brain fog. For patients who switched, the study additionally captured qualitative data on the specific factors motivating the switch, with input solicited from patients' partners as well. These real-world tolerability findings build on earlier long-term safety data from the RESTORE study, an open-label switch study in patients with narcolepsy.²

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.