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辅助性佩南滨在 4-19 岁癫痫患儿中的长期疗效和安全性:一项真实世界研究。

Long-term efficacy and safety of adjunctive perampanel in pediatric patients aged 4-19 years with epilepsy: a real-world study.

机构信息

Department of Pediatrics, Yonsei University College of Medicine, Division of Pediatric Neurology, Severance Children's Hospital, 50-1 Yonsei-ro, Seodaemun-gu, Seoul, 03722, Republic of Korea.

Department of Pediatrics, Kangdong Sacred Heart Hospital, Hallym University College of Medicine, 150 Seongan-ro, Gangdong-gu, Seoul, 05355, Republic of Korea.

出版信息

Sci Rep. 2023 Sep 1;13(1):14369. doi: 10.1038/s41598-023-40594-8.

Abstract

This study determined the 24-month outcomes of perampanel treatment in children and adolescents with epilepsy. The percentage of ≥ 50% responders was 47.3% (139/294) at 12 months and 49.0% (144/294) at 24 months. A 100% reduction in seizures for more than 12 months was observed in 12.2% (36/294). Discontinuation occurred in 39.8% (117/294). The most common reason for discontinuation was adverse events (29.1%, 34/117). Baseline seizure frequency was higher in children aged < 12 years than in patients aged ≥ 12 years; however, the percentage of seizure reduction and ≥ 50% responders did not significantly differ between the two groups. The rate of early discontinuation was higher (p < 0.001) and the duration of perampanel treatment was shorter in children aged < 12 years (p = 0.001). Most children aged < 12 years discontinued PER due to inadequate effectiveness, while adverse event was the most common reason in patients aged ≥ 12 years (p = 0.045). Only slow titration was significantly associated with ≥ 50% of responders. In conclusion, this study showed that perampanel can be utilized effectively and safely for a prolonged period in pediatric patients aged 4 to < 12 years, as well as in patients aged 12 years and older.

摘要

本研究旨在评估吡仑帕奈治疗儿童和青少年癫痫患者的 24 个月的疗效。12 个月和 24 个月时,应答率≥50%的患者比例分别为 47.3%(139/294)和 49.0%(144/294)。12 个月时,100%的患者癫痫发作减少持续超过 12 个月。39.8%(117/294)的患者停药。最常见的停药原因是不良事件(29.1%,34/117)。基线发作频率在<12 岁的儿童中高于≥12 岁的患者,但两组的发作减少率和应答率≥50%无显著差异。早期停药率较高(p<0.001),<12 岁儿童的吡仑帕奈治疗持续时间较短(p=0.001)。大多数<12 岁的儿童因疗效不足而停止使用 PER,而≥12 岁的患者因不良事件停药最常见(p=0.045)。仅缓慢滴定与应答率≥50%显著相关。总之,本研究表明,吡仑帕奈可有效、安全地用于 4 至<12 岁的儿童患者,以及 12 岁及以上的患者,持续时间较长。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5f92/10474112/c9c7220ce8cc/41598_2023_40594_Fig1_HTML.jpg

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