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. 2025 Aug;14(4):1521-1537.
doi: 10.1007/s40120-025-00760-8. Epub 2025 Jun 4.

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

Xiaoli Shi #  1   2 , Xiangru Lu #  1 , Lixia Li  1 , Yanting Lu  1 , Lang Shen  1 , Jinou Zheng  1 , Yuan Wu  1 , Lu Yu  3
Affiliations

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

Xiaoli Shi et al. Neurol Ther. 2025 Aug.

Abstract

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.

Keywords: Affect factors; Effectiveness; Focal epilepsy; Perampanel; Safety.

Plain language summary

This study evaluated the effectiveness, safety, and tolerability of perampanel (PER) as an adjunctive therapy for southern Chinese patients aged ≥ 12 years with focal epilepsy. A total of 196 patients, including 86.2% with drug-resistant epilepsy, were prospectively followed for 12 months. Most patients (73.5%) received a low PER dose (≤ 4 mg/day), which demonstrated comparable effectiveness to higher doses. At 6 and 12 months, 79.7% and 86.0% of patients, respectively, achieved a ≥ 50% reduction in seizure frequency. Retention rates were 78.3% at 6 months and 59.3% at 12 months. Adverse events, primarily dizziness and psychiatric symptoms, occurred in 21.4% of patients but were generally manageable. Logistic regression analysis identified shorter disease duration (< 5 years) and unknown etiology as significant predictors of better long-term outcomes. These findings support PER as an effective and well-tolerated adjunctive therapy for focal epilepsy, particularly in drug-resistant cases, with lower doses showing similar efficacy to higher doses.

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Conflict of interest statement

Declarations. Conflict of Interest: Xiaoli Shi, Xiangru Lu, Lixia Li, Yanting Lu, Lang Shen, Jinou Zheng, Yuan Wu and Lu Yu declare that they have no conflict of interest. Ethical Approval: The study received approval from the Medical Ethics Committee of the First Affiliated Hospital of Guangxi Medical University and met the medical ethics requirements (2023-E350-01). Informed consent was obtained from all individual participants included in the study.

Figures

Fig. 1
Fig. 1
All patient dispositions
Fig. 2
Fig. 2
Patient distribution of the dose of the addition of PER. a Patient distribution of the starting dosage of the addition of PER; b patient distribution of the maximum dosage of the addition of PER
Fig. 3
Fig. 3
Analysis of effectiveness of PER treatment during the follow-up period. a Median percent change in the seizure frequency per 28 days after the addition of PER relative to baseline (%), *p < 0.001 versus baseline; b the response rate to PER treatment. p < 0.05 versus 1–3 months, **< 0.05 versus 4–6 months
Fig. 4
Fig. 4
Retention rates of patients who received PER as an add-on therapy. a Retention rate on perampanel add-on therapy; b cumulative retention
See this image and copyright information in PMC

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