Date of Award
Selective Evidence-Based Medicine Review
Master of Science in Health Sciences - Physician Assistant
Physician Assistant Studies
Laura Levy, DHSc, PA-C
Objective: The objective of this selective EBM review is to determine whether or not pitolisant is effective in reducing excessive daytime sleepiness and cataplexy in adults with narcolepsy.
Study Design: Review of two randomized control trials (RCTs) published in 2013 and 2017, and one prospective, placebo-controlled, single-blind study published in 2008.
Data Sources: All articles were published in English and taken from peer-reviewed journals, which were found using the PubMed database.
Outcomes Measured: The outcomes of investigation measured include excessive daytime sleepiness (EDS) assessed by change in Epworth Sleepiness Scale (ESS) score, and cataplexy rate calculated from recorded cataplexy attacks in patients’ sleep diaries.
Results: Dauvilliers, et al. found pitolisant was more effective in reducing mean ESS scores from baseline compared to placebo (-5.8 vs -3.4; p=0.024). A decrease in ESS score indicates improved EDS. Also, in post-hoc analyses, Dauvilliers et al. found that pitolisant was superior to placebo in reducing daily cataplexy rate from baseline (0.38 vs 0.92; p=0.034). Szakacs, et al. found pitolisant to be effective in reducing weekly cataplexy rate (WCR) by 75% from baseline compared to placebo (38% decrease in WCR), p <0.0001; they also report a significant decrease in ESS score from baseline in the pitolisant group compared with placebo (-5.4 vs -1.9; p= 0.0001). In a single-blind, placebo-controlled, prospective study, Lin et al. found that tiprolisant (currently called pitolisant) showed a significant reduction in ESS score from baseline compared to placebo (5.9 vs 1.0; p<0.001).
Conclusions: Pitolisant was shown to be efficacious in reducing EDS and cataplexy in adults with narcolepsy.
Michael, Christina M., "Is Pitolisant Effective in Reducing Excessive Daytime Sleepiness and Cataplexy in Adults with Narcolepsy?" (2019). PCOM Physician Assistant Studies Student Scholarship. 487.