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Journal article
Stiripentol in Dravet syndrome: Results of a retrospective US study
Epilepsia (Copenhagen), v 54(9), pp 1595-1604
01 Sep 2013
PMID: 23848835
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Abstract
Purpose: To review the efficacy and tolerability of stiripentol in the treatment of U.S. children with Dravet syndrome.
Methods: U.S. clinicians who had prescribed stiripentol for two or more children with Dravet syndrome between March 2005 and 2012 were contacted to request participation in this retrospective study. Data collected included overall seizure frequency, frequency of prolonged seizures, and use of rescue medications and emergency room (ER)/hospital visits in the year preceding stiripentol initiation, and with stiripentol therapy. We separately assessed efficacy in the following treatment groups: group A, stiripentol without clobazam or valproate; group B, stiripentol with clobazam but without valproate; group C, stiripentol with valproate but without clobazam; and group D, stiripentol with clobazam and valproate. In addition, adverse effects were recorded.
Key Findings: Thirteen of 16 clinicians contacted for study participated and provided data on 82 children. Stiripentol was initiated a median of 6.0years after seizure onset and 1.2years after diagnosis of Dravet syndrome. Compared to baseline, overall seizure frequency was reduced in 2/6 in group A, 28/35 in group B, 8/14 in group C, and 30/48 in group D. All children with prolonged seizure frequency greater than quarterly during the baseline period experienced a reduction in this frequency on the various treatment arms with stiripentol. Similarly, 2/4 patients in group A, 25/25 in group B, 5/10 in group C, and 26/33 in group D experienced reduction in frequency of rescue medication use and 1/1 in group A, 12/12 in group B, 3/5 in group C, and 18/19 in group D had reduction in frequency of ER/hospital visits. Adverse effects were reported in 38, most commonly sedation and reduced appetite. Four patients (5%) discontinued stiripentol for adverse effects and two (2%) for lack of efficacy.
Significance: Stiripentol is an effective and well-tolerated therapy that markedly reduced frequency of prolonged seizures in Dravet syndrome.
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Details
- Title
- Stiripentol in Dravet syndrome: Results of a retrospective US study
- Creators
- Elaine C. Wirrell - Divisions of Child and Adolescent Neurology, Department of Neurology, Mayo Clinic, Rochester, Minnesota, U.S.ALinda Laux - Division of Neurology, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Lurie Children's Hospital, Chicago, Illinois, U.S.ADavid N. Franz - University of CincinnatiJoseph Sullivan - University of California, San FranciscoRussell P. Saneto - University of WashingtonRichard P. Morse - Dartmouth CollegeOrrin Devinsky - New York UniversityHarry Chugani - Wayne State UniversityAngel Hernandez - Cook Children's Health Care SystemLorie Hamiwka - Nationwide Children's HospitalMohamad A. Mikati - Duke UniversityIgnacio Valencia - Drexel UniversityMarie-Emmanuelle Le Guern - BiocodexLaurent Chancharme - BiocodexMarcio Sotero de Menezes - Swedish Med Ctr, Dept Pediat, Div Neurol, Seattle, WA USA
- Publication Details
- Epilepsia (Copenhagen), v 54(9), pp 1595-1604
- Publisher
- Wiley
- Number of pages
- 10
- Grant note
- CURE Cyberonics Biocodex Pfizer GlaxoSmithKline Novartis Cubist National Institute of Neurological Disorders and Stroke (NINDS); United States Department of Health & Human Services; National Institutes of Health (NIH) - USA; NIH National Institute of Neurological Disorders & Stroke (NINDS) UCB; UCB Pharma SA Dravet.org Lundbeck; Lundbeck Corporation Athena
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Pediatrics
- Web of Science ID
- WOS:000324029400017
- Scopus ID
- 2-s2.0-84883742045
- Other Identifier
- 991019169711104721
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- Collaboration types
- Domestic collaboration
- International collaboration
- Web of Science research areas
- Clinical Neurology