ArticleJournal of clinical neurology (Seoul, Korea)2018
Efficacy of Stiripentol in Dravet Syndrome with or without SCN1A Mutations.
Article in Journal of clinical neurology (Seoul, Korea), 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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Who cites it
9 citing papers in PubMed, 20 citations in OpenAlex.
- Early-Onset and Syndromic Pediatric Epilepsy in Kazakhstan: Clinical, Molecular, and Phenotypic Spectrum.Journal of clinical medicine · 2026Article
- Efficacy and Safety of Long-Term Treatment with Stiripentol in Children and Adults with Drug-Resistant Epilepsies: A Retrospective Cohort Study of 196 Patients.Drugs - real world outcomes · 2022Article
- Genetic Landscape ofMolecular syndromology · 2022Article
- Fever-Associated Seizures or Epilepsy: An Overview of Old and Recent Literature Acquisitions.Frontiers in pediatrics · 2022Review
- Genomic Investigation of Infantile and Childhood Epileptic Encephalopathies in Kazakhstan: An Urgent Priority.Frontiers in neurology · 2021Article
- Stiripentol: A Review in Dravet Syndrome.Drugs · 2019Review
- Re-annotation of 191 developmental and epileptic encephalopathy-associated genes unmasks de novo variants inNPJ genomic medicine · 2019Article
- Dravet Syndrome in Lebanon: First Report on Cases withCase reports in medicine · 2019Article
- Benign and severe early-life seizures: a round in the first year of life.Italian journal of pediatrics · 2018Review
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
background and purposeThe aim of this study was to determine the effectiveness of stiripentol (STP) add-on therapy to valproate and clobazam in patients with Dravet syndrome (DS) according to the presence of mutations in the sodium channel alpha-1 subunit gene (SCN1A).
methodsWe performed direct sequencing to analyze SCN1A mutations in 32 patients with clinically confirmed with DS, and classified them into mutation (pathogenic or likely pathogenic) and nonmutation groups based on American College of Medical Genetics and Genomics guidelines. We compared the efficacy of STP in reducing the seizure frequency between the two groups.
resultsThe 32 patients comprised 15 patients in the mutation group (with definite SCN1A mutations) and 17 patients in the nonmutation group with variants of unknown significance or benign variants. The clinical profile did not differ significantly between the mutation and nonmutation groups. The seizure frequency relative to baseline reduced by 72.53±23.00% (mean±SD) in the mutation group versus 50.58±40.14% in the nonmutation group (p=0.004). The efficacy of STP was better in DS patients with missense mutations that in those with truncation mutations, and was not favorable in patients with mutations at linkers between domains (DII-DIII), linkers between segments of domain I (DI S1-S2), or splice sites, although the small number of patients prevented statistical analyses.
conclusionsThe efficacy of STP was significantly better in DS patients with definite SCN1A mutations than in those without mutations.
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