ArticleAnnals of clinical and translational neurology2023
Efficacy of perampanel in pediatric epilepsy with known and presumed genetic etiology.
Article in Annals of clinical and translational neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed, 8 citations in OpenAlex.
- Efficacy and Safety of Adjunctive Perampanel in Young Children (7-46 Months) With Drug-Resistant Epilepsy: A Real-World Study.CNS neuroscience & therapeutics · 2026Article
- [Efficacy and safety of perampanel add-on therapy in children with epilepsy of genetic etiology].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2025Article
- Clinical factors associated with perampanel retention, response, seizure freedom and tolerability: real-world evidence from the PERMIT Extension study.Therapeutic advances in neurological disorders · 2025Article
- Response to amoxicillin and perampanel in infantile Alexander disease.Epilepsia open · 2024Article
- The Therapeutic Role of Perampanel in Treating Pediatric Patients With Dravet Syndrome: A Scoping Review.Cureus · 2024Article
- Initial therapeutic target attainment of perampanel in pediatric patients with epilepsy.Frontiers in pharmacology · 2023Article
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Authors and funding
10 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveThe efficacy of perampanel (PER) in pediatric epilepsy with specific etiologies has not been well established. Here, we investigated outcome and predictors of PER treatment in a pediatric cohort with known and presumed genetic etiology.
methodsWe included pediatric patients with potential genetic epilepsy who received PER treatment and underwent whole-exome sequencing (WES) from January 2020 to September 2021. All patients were followed up for >12 months.
resultsA total of 124 patients were included. Overall response rates were 51.6% and 49.6% at 6 months and 12 months, respectively. Pathogenic or likely pathogenic variants in 27 multiple genes were detected among 58 patients (46.8%) by WES. On performing multivariate logistic regression analysis, only developmental delay (OR = 0.406, P = 0.042) was a negative predictor of treatment response. However, the seizure onset age, positive WES results, and number of ASMs before PER administration were not significantly. Thirteen carriers with variants in the SCN1A gene showed a better response compared to eight patients with other sodium channels (P = 0.007), and to the other 45 patients with positive WES results (OR = 7.124, 95% CI = 1.306-38.860, P = 0.023). Adverse events were only reported in 23 patients, the most common being emotional problems.
interpretationPER is safe and efficacious in pediatric patients with known and presumed genetic etiology. The response rate is comparable to that reported in other pediatric populations, and lower among those with developmental delay. A gene-specific response to PER is found along with better efficacy links to pathogenic variants in the SCN1A gene.
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