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Objective <p>This real-world study evaluated the effectiveness and safety of pitolisant, a histamine H₃ receptor inverse agonist, in Chinese pediatric patients with narcolepsy.</p> Methods <p>This 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.</p> Results <p>The cohort had a mean age at onset of 9.1 ± 1.9 years, a median disease duration of 6 <b>(</b>IQR: 2, 24)months and a mean follow-up period of 5.6 months (range:4.5–7months). Patients received either pitolisant monotherapy (<i>n</i> = 20, mean dose: 12.8&#xa0;mg) or add-on therapy (<i>n</i> = 20, mean dose: 9.4&#xa0;mg). Post-treatment, significant reductions were observed in both PESS (14.3 ± 2.8 vs. 10.7 ± 2.6, <i>P</i> &lt; 0.001) and UNS scores (22.8 ± 5.8 vs. 17.0 ± 3.4, <i>P</i> &lt; 0.001). Subgroup analysis demonstrated a greater PESS improvement in patients with baseline scores &gt; 13 compared to those with scores ≤ 13 (<i>P</i> &lt; 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.</p> Conclusion <p>Pitolisant 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&#xa0;mg/day) than typically required in Western populations (18–36&#xa0;mg/day), suggests potential ethnic variations in effective dosing, with a favorable safety profile.​</p>

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A real-world study on the treatment of pediatric narcolepsy with pitolisant in China

  • 期刊论文

  • 发表日期：

    2026年1月27日

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  • Shen Zhang,
  • Yun Wu,
  • Weihua Zhang,
  • Jiuwei Li,
  • Hui Xiong,
  • Zhifei Xu,
  • Changhong Ding,
  • Tongli Han,
  • Fang Fang

摘要

Objective

This real-world study evaluated the effectiveness and safety of pitolisant, a histamine H₃ receptor inverse agonist, in Chinese pediatric patients with narcolepsy.

Methods

This 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.

Results

The 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.

Conclusion

Pitolisant 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.​