[P3-39] Efficacy of Stiripentol in Patients with Dravet Syndrome
[Introduction]
Dravet syndrome is one of the most pharmacoresistant epilepsy syndrome. Stiripentol is the only compound that proved its efficacy in Dravet syndrome through randomized placebo-controlled trials.
[Methodology]
Medical records of patients with Dravet syndrome who visited the institutions were surveyed to examine the effect of antiepileptic drugs on generalized clonic or tonic-clonic seizures. Patients older than one year of age treated with at least one conversion antiepileptic drug and more than four generalized tonic-clonic seizures per month were invited to participate in the Stiripentol study. A lower than initial dose was used in those patients. Seizure status and adverse effects during the first 4 weeks of Stiripentol add-on therapy and during long-term treatment were compared with baseline.
[Results]
With Stiripentol, generalized tonic-clonic seizures were reduced more than 50% in 10 of 14 patients (71%). Moreover, duration of seizures was shortened in 10 patients and status epilepticus decreased in 10. These effects continued in the long-term although to a lesser degree. Adverse effects (loss of appetite, sleep disturbance, ataxia, hyperactivity/irritability) were few and they could resolve after dose modification.
[Conclusions]
Our data suggest that an introduction of Stiripentol will result in substantial patient benefit.
Dravet syndrome is one of the most pharmacoresistant epilepsy syndrome. Stiripentol is the only compound that proved its efficacy in Dravet syndrome through randomized placebo-controlled trials.
[Methodology]
Medical records of patients with Dravet syndrome who visited the institutions were surveyed to examine the effect of antiepileptic drugs on generalized clonic or tonic-clonic seizures. Patients older than one year of age treated with at least one conversion antiepileptic drug and more than four generalized tonic-clonic seizures per month were invited to participate in the Stiripentol study. A lower than initial dose was used in those patients. Seizure status and adverse effects during the first 4 weeks of Stiripentol add-on therapy and during long-term treatment were compared with baseline.
[Results]
With Stiripentol, generalized tonic-clonic seizures were reduced more than 50% in 10 of 14 patients (71%). Moreover, duration of seizures was shortened in 10 patients and status epilepticus decreased in 10. These effects continued in the long-term although to a lesser degree. Adverse effects (loss of appetite, sleep disturbance, ataxia, hyperactivity/irritability) were few and they could resolve after dose modification.
[Conclusions]
Our data suggest that an introduction of Stiripentol will result in substantial patient benefit.