Journal article

Stiripentol efficacy and safety in Dravet syndrome: a 12-year observational study

Kenneth A Myers, Paul Lightfoot, Shekhar G Patil, J Helen Cross, Ingrid E Scheffer



AIM: To assess long-term safety and efficacy of stiripentol as an antiepileptic medication for people with Dravet syndrome. METHOD: A prospective, observational open-label study (2003-2015) of the efficacy and long-term safety of stiripentol in patients with Dravet syndrome and ongoing seizures. Frequency of generalized tonic-clonic seizures, focal seizures, status epilepticus, and adverse events were recorded. RESULTS: Forty-one patients started stiripentol, with median age at enrolment 5 years 7 months (range 11mo-22y) and median duration of treatment 37 months (range 2-141mo). Twenty out of 41 patients had greater than or equal to 50% long-term reduction in frequency of generalized tonic-..

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University of Melbourne Researchers


Awarded by Australian National Health and Medical Research Council (NHMRC)

Awarded by Citizens United for Research in Epilepsy (CURE)

Awarded by NHMRC

Awarded by National Health and Medical Research Council of Australia

Funding Acknowledgements

This study was supported by funding from an Australian National Health and Medical Research Council (NHMRC) Program Grants (628952, 1091593). IES has a NHMRC Practitioner Fellowship (1104831). KAM holds a Taking Flight Award from Citizens United for Research in Epilepsy (CURE; grant number 439534). JHC is supported by the National Institute for Health and Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London and is a NIHR Senior Investigator. None of the funders had any role in study design, data collection, data analysis, manuscript preparation, or publication decisions. The authors have stated that they had no interests that might be perceived as posing a conflict or bias.