Treatment Effective for Severe Narcolepsy Symptoms

Pitolisant showed robust efficacy for reducing excessive daytime sleepiness and cataplexy in adults with severe narcolepsy symptoms, according to study findings published in Sleep.

The finding stemmed from a post hoc analysis of pooled data from a pair of randomized, placebo-controlled, 7- and 8-week trials of pitolisant in adults with narcolepsy.

Researchers gauged the clinical impact of the treatment using 2 effect size metrics: (1) Cohen’s d, which assesses the standardized mean difference of an effect; and (2) number needed to treat (NNT), which determines the number of patients that would need to be treated to achieve a specific outcome for one person.

The study specifically focused on the treatment’s impact in 3 subgroups of participants: (1) those with baseline scores of 16 or higher on the Epworth Sleepiness Scale; (2) those with sleep latency of 8 minutes or less on the Maintenance of Wakefulness Test; and (3) those with 15 or more cataplexy attacks a week.

Cohen’s d effect size values for pitolisant compared with placebo were .80 among patients with higher Epworth Sleepiness Scale scores, .31 among patients with short sleep latency, and 1.31 among patients with at least 15 cataplexy attacks per week, according to study authors.

The NNT for pitolisant was 3 for an Epworth Sleepiness Scale score reduction of 3 or more points from baseline, or a final score of 10 or less, among patients in the first subgroup.

For a 50% or more drop in the weekly rate of cataplexy in patients with 15 or more weekly attacks, the NNT was 2, reported researchers.

For “much” or “very much” improved Clinical Global Impression of Change, the NNT was 5 in the excessive daytime sleepiness group, 4 in the short sleep latency subgroup, and 3 in the cataplexy subgroup.

Bioprojet Pharma and Harmony Biosciences supported the study.

Reference:
Meskill G, Davis C, Zarycranski D, Doliba M, Schwartz JC, Dayno J. Clinical impact of pitolisant on excessive daytime sleepiness and cataplexy in adults with high burden of narcolepsy symptoms. Sleep. 2022;45(suppl 1): abstract 0383. Presented at Sleep 2022.