Preventive antiepileptic treatment in tuberous sclerosis complex: long-term, prospective trial

Abstract Backgroud. Drug-resistant epilepsy is the main risk factor for future intellectual disability in patients with tuberous sclerosis complex (TSC). Clinical epileptic seizures are often preceded by electroencephalographic changes that provide an opportunity for preventive treatment. The study aimed to evaluate neuropsychological and epilepsy outcomes at school age in tuberous sclerosis complex children who received preventive antiepileptic treatment in infancy. Methods A prospective, non-randomized clinical trial was performed for 14 infants diagnosed with TSC in whom serial electroencephalographic recordings were performed, and preventive treatment with vigabatrin initiated when active epileptic discharges were detected. An age-matched control group consisted of 31 infants with TSC in whom treatment with vigabatrin was given only after onset of clinical seizures. Results of clinical assessment of epilepsy and cognitive outcomes were analysed. Results All patients in the preventive group (n=14) and 25 of 31 patients in the standard treatment group were followed through minimum age 5, median 8.8 and 8.0 years in the preventive and standard groups, respectively. The median IQ was 94 for the preventive group in comparison to 46 for the standard group (P Conclusions This study provides evidence that preventive antiepileptic treatment in infants with TSC improves long-term epilepsy control and cognitive outcome at school age.