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Efficacy and Safety of Adjunctive Perampanel in Young Children (7-46 Months) With Drug-Resistant Epilepsy: A
Qiao Zeng1,2,3, Xueqian Xia1, Hao Zheng1,2
1Department of Neurology, Children's Hospital of Chongqing Medical University, National Clinical Research Center for Child Health and Disorders, Ministry of Education Key Laboratory of Child Development and Disorders, Chongqing, China.
Insights
Adjunctive perampanel (PER) showed significant efficacy in treating drug-resistant epilepsy (DRE) in young children. This study highlights PER as a potential broad-spectrum antiseizure medication for this vulnerable population.
Area of Science:
- Pediatric Neurology
- Epileptology
- Pharmacology
Background:
- Drug-resistant epilepsy (DRE) poses significant challenges in young children.
- Evaluating novel therapeutic options for DRE in pediatric populations is crucial.
Purpose of the Study:
- To assess the efficacy and safety of adjunctive perampanel (PER) in children aged 7-46 months with DRE.
- To identify predictors of treatment response in a real-world setting.
Main Methods:
- A real-world, open-label, single-arm study involving 87 children with DRE.
- PER was administered as adjunctive therapy to existing antiseizure medications (ASMs).
- Primary endpoint: responder rate (≥50% seizure reduction) at 3, 6, 9, and 12 months; secondary endpoints included seizure freedom, retention, and adverse events.
Main Results:
- Responder rates ranged from 39.5% to 44.4% over 12 months.
- Higher response rates were observed in Dravet syndrome (50.0%) and Lennox-Gastaut syndrome (50.0%), and in children with genetic epilepsies (51.7%).
- Perinatal brain injury predicted favorable response, while concomitant use of three ASMs predicted poorer outcomes. Treatment retention was 55.2% at 12 months. TEAEs occurred in 23.0%.
Conclusions:
- Adjunctive perampanel demonstrates clinically meaningful efficacy in young children with DRE.
- PER exhibits a favorable safety profile, supporting its use as a broad-spectrum ASM in this age group.
Background:
To evaluate the efficacy and safety of adjunctive perampanel (PER) in young children with drug-resistant epilepsy (DRE) aged 7-46 months, and to identify predictors of treatment response in real-world clinical practice.
Methods:
We conducted a nonrandomized, open-label, single-arm, self-controlled real-world study at the Children's Hospital of Chongqing Medical University between December 2020 and August 2024. Eighty-seven children with DRE received PER as adjunctive therapy to existing antiseizure medications (ASMs). The primary endpoint was the responder rate (≥ 50% reduction in seizure frequency) at 3, 6, 9, and 12 months. Secondary endpoints included seizure freedom, treatment retention, and treatment-emergent adverse events (TEAEs).
Results:
Responder rates were 39.5%, 46.9%, 43.2%, and 44.4% at 3, 6, 9, and 12 months, respectively. Responder rates were 50.0% in Dravet syndrome, 50.0% in Lennox-Gastaut syndrome, and 34.8% in infantile epileptic spasms syndrome. Children with genetic etiologies had a 51.7% responder rate, including 60.0% in those with SCN1A variants. Multivariable logistic regression identified perinatal brain injury as an independent predictor of favorable response, while concomitant use of three ASMs predicted poorer outcomes. Treatment retention rates were 87.4%, 69.0%, 59.8%, and 55.2% at 3, 6, 9, and 12 months. TEAEs occurred in 23.0% of patients, most commonly somnolence (11.5%) and irritability/aggressive behavior (9.2%); 4.6% discontinued due to TEAEs.
Conclusion:
Adjunctive PER demonstrated clinically meaningful efficacy and a favorable safety profile in young children with DRE, supporting its potential role as a broad-spectrum ASM in this age group.
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