替洛利生治疗中国发作性睡病患者有效性及安全性的研究OACSTPCD

Objective The efficacy and safety of pitolisant in the treatment of adult narcolepsy have been con-firmed in clinical trials abroad,but there is a lack of data on the application of pitolisant in Chinese patients.The aim of this study is to investigate the efficacy and safety of pitolisant in the treatment of adult narcolepsy in China.Methods A to-tal of 30 subjects were enrolled in this study and were given individualized titration once a day for 8 weeks.Epworth Som-nolence Scale(EES)and daily cataplexy rate(DCR)were used as the primary outcome measures,and Clinical Global Impression-Severity scale(CGI-S)and Clinical Global Impression of Change Scale(CGI-C)were used as the secondary outcome measures;safety indicators included adverse event(AE)records and laboratory examination.Results The results showed that there were significant improvements in ESS score and number of cataplexy attacks after medication.At the end of treatment,ESS score was reduced by(7.63±4.79)(P<0.001).For all subjects,the average daily number of cataplexy attacks was 0.83 at baseline,which was reduced significantly to(0.39±0.82)after 3 weeks of pitolisant treatment and(0.38±0.79)after 5 weeks of pitolisant treatment(P<0.05).After the treatment ended,the median CGI-S score of exces-sive daytime sleepiness(EDS)improved from"severe"at baseline to"mild"(P<0.001),and the CGI-S score of cataplexy improved from"mild"at baseline to"basically normal"(P<0.001).Most AEs were mild and did not receive any drug treat-ment,and there were no significant changes in other laboratory markers used to monitor the general condition of the sub-jects before and after treatment.Conclusion Pitolisant has good efficacy and safety in the treatment of EDS and cata-plexy in Chinese adults with narcolepsy.