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Introduction <p>This study aimed to evaluate the effectiveness, tolerability and safety of perampanel (PER) as an add-on therapy for southern Chinese patients aged ≥ 12&#xa0;years with focal epilepsy.</p> Methods <p>This prospective cohort study enrolled consecutive patients with focal epilepsy treated between January 2023 and January 2024. Patients received PER as add-on therapy, with medication adjustments, seizure frequency and adverse events (AEs) monitored at 3, 6, 9 and 12&#xa0;months. Logistic regression analyzed factors influencing 6- and 12-month treatment outcomes.</p> Results <p>Among 196 patients (full analysis set), 169 (86.2%) had drug-resistant epilepsy. Of these, 73.5% (144/196) received PER ≤ 4&#xa0;mg/day. The 50% response rates at 6 and 12&#xa0;months were 79.7% (114/143) and 86.0% (86/100), respectively. Retention rates at 6 and 12&#xa0;months were 78.3% (148/189) and 59.3% (102/172), with cumulative retention rates of 71.8% and 63.3%, respectively. AEs occurred in 42 patients (21.4%), primarily dizziness and psychiatric symptoms. Logistic regression analysis identified disease duration of &lt; 5&#xa0;years (OR = 15.893, 95% CI = 1.418–178.158, <i>P</i> &lt; 0.05) and unknown etiology (OR = 14.528, 95% CI = 2.508–84.140, <i>P</i> &lt; 0.05) as predictors of higher long-term response rates.</p> Conclusion <p>PER demonstrated good effectiveness and safety as an add-on therapy for focal epilepsy in southern Chinese patients aged ≥ 12&#xa0;years. Lower doses of PER (≤ 4&#xa0;mg/day) may achieve satisfactory effectiveness in PER-sensitive populations, while shorter disease duration and unknown etiology were associated with better long-term outcomes. These findings support PER’s utility in managing focal epilepsy, particularly in drug-resistant cases.</p>

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Perampanel as Add-on in Patients Aged ≥ 12 Years with Focal Epilepsy: A Prospective Real-World Observational Study from Southern China

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年6月4日

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  • Xiaoli Shi,
  • Xiangru Lu,
  • Lixia Li,
  • Yanting Lu,
  • Lang Shen,
  • Jinou Zheng,
  • Yuan Wu,
  • Lu Yu

摘要

Introduction

This study aimed to evaluate the effectiveness, tolerability and safety of perampanel (PER) as an add-on therapy for southern Chinese patients aged ≥ 12 years with focal epilepsy.

Methods

This prospective cohort study enrolled consecutive patients with focal epilepsy treated between January 2023 and January 2024. Patients received PER as add-on therapy, with medication adjustments, seizure frequency and adverse events (AEs) monitored at 3, 6, 9 and 12 months. Logistic regression analyzed factors influencing 6- and 12-month treatment outcomes.

Results

Among 196 patients (full analysis set), 169 (86.2%) had drug-resistant epilepsy. Of these, 73.5% (144/196) received PER ≤ 4 mg/day. The 50% response rates at 6 and 12 months were 79.7% (114/143) and 86.0% (86/100), respectively. Retention rates at 6 and 12 months were 78.3% (148/189) and 59.3% (102/172), with cumulative retention rates of 71.8% and 63.3%, respectively. AEs occurred in 42 patients (21.4%), primarily dizziness and psychiatric symptoms. Logistic regression analysis identified disease duration of < 5 years (OR = 15.893, 95% CI = 1.418–178.158, P < 0.05) and unknown etiology (OR = 14.528, 95% CI = 2.508–84.140, P < 0.05) as predictors of higher long-term response rates.

Conclusion

PER demonstrated good effectiveness and safety as an add-on therapy for focal epilepsy in southern Chinese patients aged ≥ 12 years. Lower doses of PER (≤ 4 mg/day) may achieve satisfactory effectiveness in PER-sensitive populations, while shorter disease duration and unknown etiology were associated with better long-term outcomes. These findings support PER’s utility in managing focal epilepsy, particularly in drug-resistant cases.