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Title: Stiripentol efficacy and safety in Dravet syndrome: a 12-year observational study.
Austin Authors: Myers, Kenneth A;Lightfoot, Paul;Patil, Shekhar G;Cross, J Helen;Scheffer, Ingrid E 
Affiliation: Epilepsy Research Centre, Department of Medicine, The University of Melbourne, Austin Health, Heidelberg, Victoria, Australia
Apollo Paediatric Centre, Apollo Hospital, Navi Mumbai, India
UCL Institute of Child Health & Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK
Issue Date: 2018 2018-02-23
Publication information: Developmental medicine and child neurology 2018; 60(6): 574-578
Abstract: To assess long-term safety and efficacy of stiripentol as an antiepileptic medication for people with Dravet syndrome. 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. 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-clonic seizures. Frequency of focal seizures was decreased by greater than or equal to 50% in 11 out of 23 patients over the long-term. Frequency of status epilepticus was decreased by 50% or more in 11 out of 26 patients. The most common adverse events were anorexia, weight loss, sedation, and behavioural changes. One patient had worsening of absence and myoclonic seizures. Another developed recurrent pancreatitis on concurrent valproate. Stiripentol improves long-term seizure frequency in approximately 50% of patients with Dravet syndrome, when used as part of unrestricted polytherapy. Long-term use appears safe. In more than 40% of patients, episodes of status epilepticus markedly decrease after stiripentol initiation. What this paper adds Frequency of status epilepticus is reduced in 40% of patients with Dravet syndrome after stiripentol initiation. Stiripentol is effective for generalized tonic-clonic and focal seizures. Stiripentol can be safely used with a range of antiepileptic drugs.
DOI: 10.1111/dmcn.13704
ORCID: 0000-0001-7831-4593
PubMed URL: 29473155
Type: Journal Article
Appears in Collections:Journal articles

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