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

Myers, KA; Lightfoot, P; Patil, SG; Cross, JH; Scheffer, IE; (2018) Stiripentol efficacy and safety in Dravet syndrome: a 12-year observational study. Developmental Medicine and Child Neurology , 60 (6) pp. 574-578. 10.1111/dmcn.13704. Green open access

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Abstract

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‐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. INTERPRETATION: 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.

Type: Article
Title: Stiripentol efficacy and safety in Dravet syndrome: a 12-year observational study
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/dmcn.13704
Publisher version: https://doi.org/10.1111/dmcn.13704
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10061031
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