Efficacy and safety of cenobamate in the treatment of drug-resistant epilepsy: a systematic review and meta-analysis in adult, pediatric, and combined populations.

In 30 seconds

This systematic review and random-effects meta-analysis assessed cenobamate efficacy and safety for drug-resistant epilepsy in adults, children, and mixed-age populations. It included 36 studies: 22 adult studies, six pediatric studies, and eight mixed-age studies without separable age-specific data. The pooled proportion with at least 50% seizure reduction was 0.65 in adults (95% CI: 0.58-0.71) and 0.60 in children (95% CI: 0.39-0.78). Pediatric estimates were numerically similar to adult estimates, but the comparison was indirect and the pediatric evidence was limited.

Key findings

• In adults, the pooled responder proportion (≥ 50% seizure reduction) was 0.65 (21 studies, 3,871 patients; 95% CI: 0.58-0.71).
• In pediatric patients, the preliminary response rate was 0.60 (6 studies, 256 patients; 95% CI: 0.39-0.78).
• Pooled seizure freedom rates were 0.15 in adults (95% CI: 0.10-0.21) and 0.20 in pediatric patients (95% CI: 0.15-0.26).
• The incidence of adverse events was 0.63 in adults and 0.48 in pediatric patients; pediatric serious adverse events were estimated at 0.03 (95% CI: 0.01-0.12).

Why it matters

The review brings together reported cenobamate outcomes across age groups, including preliminary pediatric estimates that may inform understanding of its potential role in drug-resistant epilepsy. However, numerical similarity between age groups does not establish comparable efficacy or safety: the subgroup comparison was indirect, and the pediatric evidence base was small. These findings highlight the need for stronger age-specific evidence.

What to keep in mind

Most included studies were retrospective observational studies, and pediatric samples were small. Mixed-age studies without separable age-specific data could not contribute to subgroup comparisons; pediatric estimates therefore remain preliminary.