title: Audit of use of stiripentol in adults with Dravet syndrome.
abstract: OBJECTIVE
  There are very few data available in the literature on the use of stiripentol in adults with Dravet syndrome (DS). DS cases are increasingly recognized in adulthood, and more children with DS now survive to adulthood. The aim of the study was to document the effectiveness and tolerability of stiripentol in adults with DS.
  
  
  METHODS
  We conducted an observational clinical audit in the epilepsy service of the National Hospital for Neurology and Neurosurgery, London (UK).
  
  
  RESULTS
  We included 13 adult subjects with DS (eight females, five males). The responder (defined as more than 50% reduction in all seizure types) rate was 3/13 (23%) at 36 months. The following other outcomes were reported: seizure exacerbation (3/13, 23%), no change (3/13, 23%), less than 50% reduction in seizures (2/13, 15%), more than 50% reduction in generalized tonic-clonic seizures but no other seizure types (1/13, 8%), undefined response (1/13, 8%). The retention rate was 62% after 1 year and 31% after 5 years. Adverse effects were reported in 7/13 (54%): the most frequent were anorexia, weight loss, unsteadiness and tiredness. Withdrawal due to adverse effects occurred in 3/13 (23%).
  
  
  CONCLUSIONS
  Compared with previous studies on children with DS, our results show a lower responder rate and a similar tolerability profile. Stiripentol can be effective with a good tolerability profile. Our audit is small, but supports the use of stiripentol in adults with DS when first-line treatments are ineffective or not tolerated, in keeping with published guidelines.
authors.affiliation: Department of Clinical and Experimental Epilepsy, NIHR University College London Hospitals Biomedical Research Centre, UCL Institute of Neurology, London, UK.|Epilepsy Society, Chalfont-St-Peter, Buckinghamshire, UK.|Neuroscience Department, Polytechnic University of Marche, Ancona, Italy.
authors.forename: S
authors.initials: S
authors.lastname: Balestrini
authors.affiliation: Department of Clinical and Experimental Epilepsy, NIHR University College London Hospitals Biomedical Research Centre, UCL Institute of Neurology, London, UK.|Epilepsy Society, Chalfont-St-Peter, Buckinghamshire, UK.
authors.forename: S M
authors.initials: SM
authors.lastname: Sisodiya
mesh_terms: D000293:Adolescent; D000328:Adult; D000927:Anticonvulsants / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004148:Dioxolanes / Q000009:adverse effects / Q000627:therapeutic use*; D004831:Epilepsies, Myoclonic / Q000188:drug therapy* / Q000235:genetics; D005260:Female; D006801:Humans; D008297:Male; D009154:Mutation; D062550:NAV1.1 Voltage-Gated Sodium Channel / Q000235:genetics
chemical_list: D000927:Anticonvulsants; D004148:Dioxolanes; D062550:NAV1.1 Voltage-Gated Sodium Channel; C568242:SCN1A protein, human; C021092:stiripentol
keywords: antiepileptic drugs; epilepsy; seizures; stiripentol