Skuteczność i bezpieczeństwo perampanelu jako leku uzupełniającego u małych dzieci (7-46 miesięcy) z lekooporną epilepsją - badanie w warunkach rzeczywistej praktyki
Efficacy and Safety of Adjunctive Perampanel in Young Children (7-46 Months) With Drug-Resistant Epilepsy: A Real-World Study
W skrócie
Badanie oceniało, czy lek zwany perampanel, stosowany dodatkowo do innych leków przeciwpadaczkowych, pomaga małym dzieciom (od 7 do 46 miesięcy) z trudną do leczenia epilepsją. Wyniki pokazały, że u około 40-45 procent dzieci liczba napadów padaczkowych zmniejszyła się o co najmniej połowę, a najlepsze rezultaty osiągnęły dzieci z określonymi genetycznymi przyczynami epilepsji. Lek był ogólnie bezpieczny, najczęściej powodując senność i drażliwość, a większość dzieci kontynuowała leczenie przez cały rok obserwacji.
Oryginalny abstract (angielski)
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.