Efficacy and Safety of Adjunctive Perampanel in Young Children (7–46 Months) With Drug‐Resistant Epilepsy: A Real‐World Study
Source: PubMed Central Open Access, NCBI / U.S. National Library of Medicine
ABSTRACT 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. cns70998-sec-0001 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). cns70998-sec-0002 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 withvariants. 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/aggress
Abstract
ABSTRACT 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. cns70998-sec-0001 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). cns70998-sec-0002 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 withvariants. 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. cns70998-sec-0003 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. cns70998-sec-0004 This real‐world study evaluates adjunctive perampanel (PER) in young children (7–46 months) with drug‐resistant epilepsy. Results show clinically meaningful efficacy, with responder rates of up to 50% in certain syndromes. PER demonstrates a favorable safety profile, supporting its role as an effective treatment option in this age group. graphical