Adjunctive Use of Cenobamate in Paediatric Drug-Resistant Epilepsy: A Real-World, Single-Centre Experience

Background/Objectives: Epilepsy represents one of the most common chronic neurological disorders in children, with a considerable proportion of patients exhibiting resistance to pharmacotherapy despite the advent of novel antiseizure medications (ASMs) in recent decades. This retrospective study assesses the off-label administration of cenobamate—a newly approved antiseizure medication (ASM) for focal seizures in adults—in a cohort of paediatric patients with drug-resistant epilepsy at a single neurology centre. Methods: Clinical outcomes were reviewed retrospectively for 18 children who received cenobamate for at least 6 months. Results: Eighteen paediatric patients with drug-resistant epilepsy received cenobamate therapy at a neurology centre. The mean age was 164.6 months, and each patient had previously trialled an average of 8.7 antiseizure medications. During a follow-up period of up to 29 months, 39% of participants achieved complete seizure freedom, while five additional patients experienced a seizure reduction exceeding 80%. Concomitant clobazam use was common among the cohort. Adverse events were reported in 78% of patients, predominantly somnolence, though these were generally transient or manageable. One patient developed a temporary exacerbation of seizures, which resolved following a dosage adjustment. Many patients were able to reduce or discontinue other ASMs during the observation period. Conclusions: Cenobamate demonstrated acceptable tolerability in this paediatric cohort, and seizure improvements were observed in a subset of patients. Further clinical trials are warranted to comprehensively evaluate the efficacy and safety profile of cenobamate in this patient population.