title: Stiripentol: efficacy and tolerability in children with epilepsy.
abstract: OBJECTIVE
  Stiripentol (STP) is a new antiepileptic drug (AED) that inhibits cytochrome P450, resulting in increased plasma concentrations of concomitant AEDs. The efficacy and tolerability of STP as an add-on therapy in children were assessed.
  
  
  METHODS
  Two hundred twelve patients with refractory epilepsy, aged from 1 month to 20.5 years, received STP either in a single-blind, placebo-controlled trial (108 patients) or in a further open trial (104 other patients selected by epilepsy syndrome for possible efficacy based on the results of the previous trial).
  
  
  RESULTS
  Among the 97 patients who could be analyzed for efficacy in the placebo-controlled study, the median seizure frequency was lower at 3 months with STP than with the placebo (p<0.0001); 49% responded to the drug, including 10% who became seizure free. Patients with partial epilepsy had the highest response rate (57%). Results were confirmed in the open study where 68% of the 91 patients receiving STP responded at 3 months. These patients were mainly those with partial epilepsy (73%) who were receiving carbamazepine (CBZ) (75%) as comedication (p<0.001). Ten of the 20 children with severe myoclonic epilepsy in infancy also responded with clobazam (CLB) as comedication. Efficacy was sustained long term in 74% of the 94 patients still receiving STP at a mean 30-month follow-up. Adverse events were reported in 48% of the 212 patients, mainly anorexia and loss of weight, but these events required STP discontinuation in only nine cases. Side effects were minimized in the open trial by optimizing the dose of comedication.
  
  
  CONCLUSIONS
  STP seems to be a promising add-on drug, particularly when combined with CBZ in patients with partial childhood epilepsy refractory to vigabatrin (VGB) and with CLB in patients with severe myoclonic epilepsy in infancy.
authors.affiliation: Neuropediatric Department and INSERM U 29, Hospital Saint Vincent de Paul, Paris, France.
authors.forename: J
authors.initials: J
authors.lastname: Perez
authors.forename: C
authors.initials: C
authors.lastname: Chiron
authors.forename: C
authors.initials: C
authors.lastname: Musial
authors.forename: E
authors.initials: E
authors.lastname: Rey
authors.forename: H
authors.initials: H
authors.lastname: Blehaut
authors.forename: P
authors.initials: P
authors.lastname: d'Athis
authors.forename: J
authors.initials: J
authors.lastname: Vincent
authors.forename: O
authors.initials: O
authors.lastname: Dulac
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000927:Anticonvulsants / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004148:Dioxolanes / Q000009:adverse effects / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D004831:Epilepsies, Myoclonic / Q000188:drug therapy; D004828:Epilepsies, Partial / Q000188:drug therapy; D004827:Epilepsy / Q000188:drug therapy*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D010919:Placebos; D016037:Single-Blind Method; D016896:Treatment Outcome
chemical_list: D000927:Anticonvulsants; D004148:Dioxolanes; D010919:Placebos; C021092:stiripentol