Wang Han, Chen Hong, Ding Xin, Cao Xueyan, Mai Jiahui, Zou Huafang, Zou Dongfang, Zhu Yanwei, Liao Jianxiang, Cao Dezhi
Department of Neurology, Shenzhen Children's Hospital, Shenzhen, China.
Epilepsia Open. 2025 Aug;10(4):1043-1053. doi: 10.1002/epi4.70058. Epub 2025 May 24.
To investigate the efficacy and tolerability of perampanel (PER) on Dravet syndrome in China by a prospective real-world study.
We prospectively enrolled children with Dravet syndrome from the neurology clinic of Shenzhen Children's Hospital from September 2020 to October 2021. The average monthly seizure frequency within 2 months before enrollment was recorded as the baseline. PER was administered after enrollment. The response rate (the rate of seizure reduction ≥50% compared to the baseline) was regarded as the major index. The adverse events and retention rates were regarded as the minor index. All indexes were calculated after 3 and 6 months' follow-up, respectively. The outcome-predictive value of various clinical factors was analyzed.
A total of 21 children were enrolled, including 15 boys and 6 girls, with a median age of 4 years and 8 months (1 year and 3 months to 15 years and 6 months). Averagely, 2.57 ASMs had been administrated and failed to control seizures before enrollment. The response rates were 52.4% (11/21) and 47.6% (10/21) after 3 and 6 months of PER administration, respectively. Adverse events were observed in 8 patients (38.1%), including irritability (3/8, 37.5%), fatigue (3/8, 37.5%), unstable walking (2/8, 25%), somnolence (2/8, 25%), and sluggish response (2/8, 25%); most of them were mild and temporary. The retention rate was 90.5% (19/21) at 6 months' follow-up. Two patients discontinued PER due to failure of seizure control, and no patients discontinued due to adverse events.
This study provides a comprehensive analysis of PER's effectiveness and tolerability in treating DS, highlighting its importance as an additional treatment option. It broadens the range of anti-seizure medications (ASMs) that may be effective for patients with DS.
In the neurology clinic of Shenzhen Children's Hospital, 21 children with Dravet syndrome were administered perampanel. Perampanel, as a novel anti-seizure medication approved for focal and generalized epilepsies as add-on therapy, showed sufficient efficacy and a satisfactory safety and tolerability profile in this study.
通过一项前瞻性真实世界研究，探讨吡仑帕奈（PER）在中国治疗Dravet综合征的疗效和耐受性。
我们于2020年9月至2021年10月在深圳儿童医院神经内科前瞻性招募Dravet综合征患儿。将入组前2个月内的平均每月癫痫发作频率记录为基线。入组后给予PER治疗。将缓解率（与基线相比癫痫发作减少≥50%的比例）作为主要指标，不良事件和保留率作为次要指标。分别在随访3个月和6个月后计算所有指标，并分析各种临床因素对结局的预测价值。
共纳入21例患儿，其中男15例，女6例，中位年龄为4岁8个月（1岁3个月至15岁6个月）。入组前平均使用过2.57种抗癫痫药物（ASM）且未能控制癫痫发作。PER治疗3个月和6个月后的缓解率分别为52.4%（11/21）和47.6%（10/21）。8例患者（38.1%）出现不良事件，包括易激惹（3/8，37.5%）、疲劳（3/8，37.5%）、行走不稳（2/8，25%）、嗜睡（2/8，25%）和反应迟钝（2/8，25%）；大多数为轻度且为暂时性。6个月随访时的保留率为90.5%（19/21）。2例患者因癫痫发作控制不佳停用PER，无患者因不良事件停药。
本研究全面分析了PER治疗Dravet综合征的有效性和耐受性，突出了其作为一种附加治疗选择的重要性。它拓宽了可能对Dravet综合征患者有效的抗癫痫药物（ASM）范围。
在深圳儿童医院神经内科，对21例Dravet综合征患儿给予了吡仑帕奈治疗。吡仑帕奈作为一种被批准用于局灶性和全身性癫痫附加治疗的新型抗癫痫药物，在本研究中显示出足够的疗效以及令人满意的安全性和耐受性。
