Clobazam therapy of refractory epilepsy in tuberous sclerosis complex

Summary Clobazam (CLB) was recently approved by the FDA, but has not been evaluated in tuberous sclerosis complex (TSC). We retrospectively reviewed a cohort of patients with TSC and refractory epilepsy who started CLB over a 5-year period. Clinical characteristics and number of tubers on MRI were assessed. Duration of therapy, therapeutic response and adverse events were recorded. CLB was prescribed in 29 adults and children of whom 72% were cognitively impaired, with a median age at seizure onset of 5 months. Mean duration of CLB therapy was 17.3 months with a 12 and 24-month estimated retention rate of 82% and 68%, respectively. Twenty patients (69%) reported a good response (>50% seizure reduction) at the end of the titration, and six patients (21%) remained good responders after 12 months of CLB therapy. Adverse events occurred in 13 patients, predominantly somnolence and behavioral disorders. One quarter of the responders reported improvement in behavior. No predictive factor for a good response could be identified. CLB appears to be a well-tolerated and valuable option for treatment of refractory epilepsy in TSC.