OBJECTIVES: To examine the clinical (safety, effectiveness) and humanistic (QoL and other PROs) outcomes associated with sodium oxybate (SXB) in the management of narcolepsy in real-world settings.

METHODS: We searched PubMed for relevant records on 10th November 2022, using appropriate search strategies. Publication details, demographics, intervention, comparator, effectiveness, safety and humanistic outcomes were extracted from eligible records.

RESULTS: Of the 425 records screened, 21 publications from 17 studies containing 2,228 unique participants (females: 1,418, males: 810; adults (10 studies): 2,047; children/ adolescents (7 studies): 181) were included in analysis. Mean age ranged from 34.3 to 44.1 years in adults, 11.0 to 15.3 years in children. In 11 studies, SXB was administered as per recommended dosage. Of the 17 included studies, effectiveness, safety, and humanistic outcomes were evaluated by 17, 5, and 2 studies respectively. SXB treatment was associated with a reduction in Epworth Sleepiness Score, total sleep time, proportion of REM sleep, wakefulness after sleep onset, awakenings, and cataplexy. SXB increased sleep efficiency, duration of slow wave sleep (night and total), duration of daytime wakefulness, and sleep latency. SXB also improved humanistic outcomes including sleep quality, daytime functioning scores, SF-36 scores (physical component, mental component, and all 8 subscales) and patient-reported outcomes (presenteeism, overall work and activity impairment, and risk of motor vehicle accidents). No new safety signals were noted. There was a lack of uniformity in the outcomes assessed by included studies.

CONCLUSIONS: SXB is safe and effective in narcolepsy patients treated in real-world settings. Future real-world studies need to evaluate standard outcomes consistently to enable better analysis of data on effectiveness and safety.