Long-term safety and efficacy of adjunctive brivaracetam in pediatric patients with epilepsy: An open-label,follow-up trial |
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| Authors: | Lieven Lagae Kerstin Alexandra Klotz András Fogarasi Florin Floricel Christoph Reichel Jan-Peer Elshoff Sofia Fleyshman Harriet Kang |
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| Affiliation: | 1. Department of Development and Regeneration, Pediatric Neurology Section, University Hospitals Katholieke Universiteit Leuven, Leuven, Belgium;2. Department of Neuropediatrics and Muscle Disorders, Medical Center, University of Freiburg, Freiburg, Germany;3. Department of Neuropediatrics, Bethesda Children's Hospital, Budapest, Hungary;4. UCB Pharma, Monheim am Rhein, Germany;5. UCB Pharma, Morrisville, North Carolina, USA;6. Department of Neurology, Icahn School of Medicine at Mount Sinai Hospital, New York, New York, USA |
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| Abstract: | Objective This study was undertaken to evaluate the long-term safety, tolerability, and efficacy of adjunctive brivaracetam (BRV) treatment in pediatric patients with epilepsy. Methods A phase 3, open-label, multicenter, long-term follow-up trial (N01266; NCT01364597) was conducted on patients (aged 1 month to <17 years at core trial entry; direct enrollers aged 4 to <17 years) treated with BRV. Outcomes included treatment-emergent adverse events (TEAEs), behavior assessments (Achenbach Child Behavior Checklist [CBCL], Behavior Rating Inventory of Executive Function [BRIEF]/BRIEF-Preschool version [BRIEF-P]), and efficacy outcomes (percent change in focal seizure frequency, 50% responder rate for all seizure types for patient subgroups <2 years and ≥2 years of age using daily record card data). Results Of 257 patients with ≥1 dose of BRV (141 [54.9%] male; mean age = 8.0 years [SD = 4.5]), 36 patients were <2 years of age, and 72.0% of patients had a history of focal seizures. Mean BRV exposure was 3.2 patient-years. At least one TEAE occurred in 93.4% patients, and 32.3% had serious TEAEs. Seven patients died during the trial; no deaths were considered treatment-related. Patients ≥2 years of age had a median decrease in 28-day adjusted focal seizure frequency of 62.9%, and 50.9% had a ≥50% response in all seizures. Patients <2 years of age had a median decrease in 28-day adjusted focal seizure frequency of 96.9%, and 68.2% had a ≥50% response in all seizures. Kaplan–Meier estimated treatment retention was 72.7%, 64.5%, 57.8%, 53.3%, 50.1%, and 44.8% at 1, 2, 3, 4, 5, and 6 years, respectively. Mean changes (baseline to last evaluation) for all Achenbach CBCL and BRIEF-P/BRIEF subscale scores were negative, reflecting stability/slight improvement. Significance Long-term adjunctive BRV treatment was generally well tolerated and efficacious in reducing seizure frequency, and had high retention rates, with generally stable cognitive/behavioral scores in pediatric patients with epilepsy. |
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| Keywords: | antiseizure medication adolescents children focal seizures |
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