A randomized trial evaluating the effectiveness of sodium oxybate therapy on quality of life in narcolepsy.

Objective: To evaluate the efficacy of sodium oxybate versus placebo to improve quality of life in patients with narcolepsy.

Methods: A multicenter, double-blind, placebo-controlled trial. Methods: Outpatient facility of 42 sleep centers in the United States, Canada, and Europe. Methods: Study participants were 285 patients with narcolepsy, 16 to 75 years of age, with a median Epworth Sleepiness Scale score of 18, a Maintenance of Wakefulness Test sleep latency of 9.56 minutes, and experiencing symptoms of narcolepsy, including cataplexy and excessive daytime sleepiness with recurrent sleep episodes almost daily for at least 3 months at the time of enrollment. Methods: Subjects were gradually withdrawn from narcolepsy medications used for cataplexy, including antidepressants. Subsequently, participants were randomly assigned to receive 4.5, 6.0, or 9.0 g per day of sodium oxybate or placebo taken in two equally divided doses upon retiring to bed and again 2.5 to 4 hours later for 4 weeks during the stable dosing phase.

Results: The change in quality of life following the administration of sodium oxybate was measured with the Functional Outcomes of Sleep Questionnaire. The nightly administration of sodium oxybate produced significant dose-related improvements in the Total Functional Outcomes of Sleep Questionnaire score, as well as in the Activity Level, General Productivity, Vigilance, and Social Outcomes subscales.

Conclusions: The nocturnal administration of sodium oxybate in patients with narcolepsy was associated with statistically significant and clinically relevant improvements in functional status, an important component of quality of life.