Observational studyNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2026
A real-world study on the treatment of pediatric narcolepsy with pitolisant in China.
Observational study in Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
objectiveThis real-world study evaluated the effectiveness and safety of pitolisant, a histamine H₃ receptor inverse agonist, in Chinese pediatric patients with narcolepsy.
methodsThis retrospective, observational, single-center study enrolled 40 pediatric patients treated with pitolisant between May and August 2024. Data collection encompassed demographic profiles, sleep questionnaires (Pediatric Epworth Sleepiness Scale [PESS] and Ullanlinna Narcolepsy Scale [UNS]), polysomnographic parameters, concomitant medications, and laboratory evaluations. Efficacy endpoints included changes in PESS and UNS scores from baseline, and caregiver-reported improvement in core narcolepsy manifestations other than excessive daytime sleepiness (EDS). Safety was assessed through adverse event monitoring.
resultsThe cohort had a mean age at onset of 9.1 ± 1.9 years, a median disease duration of 6 (IQR: 2, 24)months and a mean follow-up period of 5.6 months (range:4.5–7months). Patients received either pitolisant monotherapy (n = 20, mean dose: 12.8 mg) or add-on therapy (n = 20, mean dose: 9.4 mg). Post-treatment, significant reductions were observed in both PESS (14.3 ± 2.8 vs. 10.7 ± 2.6, P < 0.001) and UNS scores (22.8 ± 5.8 vs. 17.0 ± 3.4, P < 0.001). Subgroup analysis demonstrated a greater PESS improvement in patients with baseline scores > 13 compared to those with scores ≤ 13 (P < 0.005). Additionally, 73.7% reported reduced cataplexy frequency, and 75% exhibited improved nocturnal sleep quality. Adverse events were mild, headache (15%), insomnia (12.5%), and somnolence (10%) as the most frequent.
conclusionPitolisant demonstrates efficacy in alleviating excessive daytime sleepiness, cataplexy, and nocturnal disturbances in Chinese children with narcolepsy. The achieved efficacy at lower doses (mean 9.4–12.8 mg/day) than typically required in Western populations (18–36 mg/day), suggests potential ethnic variations in effective dosing, with a favorable safety profile.
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