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Effectiveness and tolerability of perampanel as first add-on treatment in pediatric patients with focal and generalized epilepsy

Year: 2025
Type: article
Study design: Observational Study
Abstract: PURPOSE: To compare the effectiveness and safety of perampanel as first add-on vs. late add-on therapy in children with focal or generalized epilepsy. METHODS: Retrospective, single-center study conducted between March 2022 and April 2023, including children ≥ 4 years with focal an generalized epilepsy who started treatment with perampanel in November 2020 or later. Patients were classified into two groups: perampanel as first and as late add-on treatment. Effectiveness and safety data were collected at 3, 6, and 12 months after starting treatment. Primary outcomes were responder rates, seizure-freedom rates, and worsening. RESULTS: 57 patients (45.6 % women) with a mean (SD) age of 8.8 (±3.5) years were included; 27 in the first and 30 in the late add-on group. Effectiveness was consistently higher throughout visits in patients receiving perampanel as first vs. late add-on treatment, with higher response rates (12 months, 95.0 % vs. 55.6 %, p = 0.003), higher seizure-freedom rates (12 months, 65 % vs. 37.0 %, p = 0.058), and fewer patients with worsening (12 months, 0 % vs. 12.8 %, p = 0.031). Retention rates remained high in both groups throughout visits. Sixteen patients discontinued treatment. Reasons were lack of effectiveness, which was more frequent in the late vs. first add-on group, with few patients discontinuing due to adverse events. CONCLUSION: The improved effectiveness outcomes of perampanel used as first vs. late add-on therapy in a pediatric population and the favorable safety outcomes support its use as first add-on therapy in pediatric patients with focal and generalized epilepsy.
Language: English
Field: Medicine
Open Access status: hybrid
Estimated APC paid: $2,880
OpenAlex ID: W4415503131
FWCI:
Cites: 29
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