Cenobamate use for drug-resistant epilepsy in children: A real-world single-center study.

BACKGROUND

Cenobamate was approved in 2020 by the EMA for the adjunctive treatment of focal-onset seizures in adults. Based on data from clinical trials and early experience in adult patients, we used cenobamate in children whose seizures were not controlled with available options.

METHODS

We prospectively collected clinical data in patients who started treatment with cenobamate and report dosage, efficacy, retention rate, and adverse effects.

RESULTS

Overall, 50 patients (median age 12.2 years, range 3.1-18.5, 19 patients below age 10 years; 44% female, median concomitant antiseizure medications 2) were treated with cenobamate for a median duration of 16 months (range 3-28). After gradually increasing cenobamate to clinical effect at a reduced dosage schedule, 60% (29/48) of children were responders (≥50% reduction) and 12% were free of major motor seizures. Patients were treated with an average daily dose of 3.7 mg/kg. During cenobamate treatment, 8 (16%) patients were able to discontinue at least one concomitant antiseizure medication. The retention rate at 12 months was 82%. The most common adverse events were somnolence (42%), nausea/vomiting (4%), and ataxia (4%).

CONCLUSIONS

The findings of this single-center real-world study are in line with prior controlled studies and real-world experience in adult patients, as well as with the limited published experience in children. The efficacy and lack of serious side effects found in this study supports the use of cenobamate as a treatment for epilepsy also in children and infants.