Abstracts

RATIONALE: Most patients (80%-90%) with tuberous sclerosis complex (TSC) have epilepsy characterized by infantile spasms, partial and/or generalized seizures. However, response to AEDs may differ from that of other patients without TSC. Therefore, the effectiveness of AEDs should be specifically evaluated in TSC-associated seizures. Because of its effectiveness in partial-onset and generalized seizures in children, topiramate (TPM) was evaluated as adjunctive therapy in children with TSC. METHODS: Following a baseline period of ?2 weeks, TPM was initiated at 1-3 mg/kg/day and titrated in 1-3 mg/kg increments every 3-7 days to a maximum dose of 60 mg/kg/day. When patients were seizure free 1 week, patients entered a stabilization phase. If seizure control was not improved with 60 mg/kg/day, TPM was tapered and discontinued. RESULTS: Fourteen children with TSC (7 boys/7 girls; age, 2-12 yrs) received 10-60 mg/kg/day TPM. At the end of 6 months of TPM treatment, seizures were reduced >50% in 64% (9/14); 3 patients had no seizures for 6 months. Number or location of tuberosis lesions, age at onset, or EEG abnormalities did not predict TPM response. Side effects included akathisia and cognitive slowing in 4 patients, who discontinued TPM (1 due to cognitive slowing and 3 due to inadequate response). CONCLUSIONS: These results suggest that TPM is beneficial in the treatment of TSC-associated seizures and warrant a well-controlled study.