To evaluate the efficacy and tolerability of
topiramate (TPM) as add-on
therapy in children less than 12 years of age with
refractory epilepsy, according to
epilepsy syndromes, we conducted an open, prospective, pragmatic and multicenter study in France. Efficacy was assessed, especially according to
epilepsy syndromes, as well as tolerability. We included 207 children (41 of whom were less than 4 years of age). TPM was effective (responders with >50% decrease in seizure frequency) in 50% of 128 patients with
partial epilepsy, and in 44% of 79 patients with
generalized epilepsy. In case of
generalized epilepsy, responders more frequently had generalized symptomatic
epilepsy, severe
myoclonic epilepsy and myoclono-astatic
epilepsy, whereas response rate was mild in both
infantile spasms and
Lennox-Gastaut syndrome (LGS). Improvement was well maintained in all patients during the treatment period (median 5.6 months). Seizure frequency/severity increased (worsening) in 13% of patients with
partial epilepsy and 17% with
generalized epilepsy (particularly in those with
infantile spasms), and resulted in withdrawal of TPM for 8%. The most frequently reported adverse events were moderate neurobehavioral and
gastrointestinal disorders. Adverse events led to withdrawal of TPM from 13.5% of patients. Children less than 4 years of age had particularly good tolerability. Results confirm that TPM is effective and well tolerated in children under 12 years of age in a broad range of
epilepsy syndromes, including refractory
partial epilepsy, and symptomatic and myoclonic
generalized epilepsy. Use of TPM should be considered in children under 4 years of age, and slow and progressive titration is important.