Pediatric developmental epileptic encephalopathies treated with cenobamate: Real-world outcomes across etiologies, syndromes, and seizure types.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
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Aledo-Serrano Ángel, Valls-Carbó Adrián, González-Alguacil Elena, García-Peñas Juan José, Sariego Jamardo Andrea, Aznar-Laín Gemma, Ibáñez-Micó Salvador, Alarcón Martínez Helena, Buenache-Espartosa Raquel, Rekarte-García Saray, Lorenzo-Dieguez Manuel, Domínguez-Carral Jana, López López María, García-Ribes Ainhoa, Martínez-González María Jesús, Arias Eva, García-Ron Adrián, Boronat Susana, Turón Viñas Eulalia, Casellas Dolors, Navarro Virginia, Conejo David, Miravet Elena, Gil-Nagel Rein Antonio, Sánchez-Miranda Román Irene, Lamagrande Nuria, Muñoz Cabeza María, Smeyers Patricia, Soto-Insuga Víctor
Аннотация
METHODS: A retrospective multicenter cohort of children (≤18 years old) with DEEs were treated with CNB at 17 Spanish hospitals. Primary outcomes were retention, response (≥50% reduction), and seizure freedom at 3, 6, and 12 months. Mixed-effects logistic and ordinal models were adjusted for age, syndrome, etiology, seizure type, and concomitant medication.
RESULTS: Among 152 children (median age = 12 years), 27.6% had Lennox-Gastaut syndrome (LGS) and 58.6% unspecified DEE, with a median of 9 prior antiseizure medications. Retention was 88%, 90%, and 93% and responder rates 64%, 73%, and 79% at 3, 6, and 12 months; seizure freedom was 8%, 12%, and 18% (evaluable n = 152, 105, and 57 at 3, 6, and 12 months, respectively). LGS and other DEEs reached identical 12-month responder rates (both 79%), whereas Dravet syndrome showed limited sustained benefit. By etiology, structural cases had the highest 12-month responder rate (93% vs. 68% in nonstructural, p = .04), but no etiology was independently associated with response. By seizure type, responder rates were highest for tonic (81%) and bilateral tonic-clonic (76%) and lowest for absences (54%, adjusted odds ratio [OR] = .43, p = .032). Treatment-emergent seizure worsening occurred in 10.5%. Sodium channel blockers were the main risk factor for adverse events (OR = 2.10); 10.5% discontinued CNB due to adverse events.
SIGNIFICANCE: CNB was associated with sustained effectiveness and acceptable tolerability across pediatric DEEs. Slow weight-based titration and proactive simplification of sodium channel blockers and clobazam may optimize benefit-risk.