Affiliation:
1. Pediatric Neurology and French Reference Center for Rare Epilepsies (CRéER) APHP, Necker‐Enfants Malades Hospital Paris France
2. INSERM U1141 and Institut Neurospin Paris France
3. Biocodex Compiègne France
4. INSERM U1163, Institut Imagine, chaire GEEN‐DS Université Paris cité Paris France
Abstract
AbstractAimTo evaluate the safety and efficacy of stiripentol initiated before 2 years of age in patients with Dravet syndrome.MethodThis was a 30‐year, real‐world retrospective study. We extracted the data of the 131 patients (59 females, 72 males) who initiated stiripentol before 2 years of age between 1991 and 2021 from the four longitudinal databases of Dravet syndrome available in France.ResultsStiripentol was added to valproate and clobazam (93%) at 13 months and a median dose of 50 mg/kg/day. With short‐term therapy (<6 months on stiripentol, median 4 months, median age 16 months), the frequency of tonic–clonic seizures (TCS) lasting longer than 5 minutes decreased (p < 0.01) and status epilepticus (>30 minutes) disappeared in 55% of patients. With long‐term therapy (last visit on stiripentol <7 years of age, median stiripentol 28 months, median age 41 months), the frequency of long‐lasting TCS continued to decline (p = 0.03). Emergency hospitalizations dropped from 91% to 43% and 12% with short‐ and long‐term therapies respectively (p < 0.001). Three patients died, all from sudden unexpected death in epilepsy. Three patients discontinued stiripentol for adverse events; 55% reported at least one adverse event, mostly loss of appetite/weight (21%) and somnolence (11%). Stiripentol was used earlier, at lower doses, and was better tolerated by patients in the newest database than in the oldest (p < 0.01).InterpretationInitiating stiripentol in infants with Dravet syndrome is safe and beneficial, significantly reducing long‐lasting seizures including status epilepticus, hospitalizations, and mortality in the critical first years of life.
Subject
Neurology (clinical),Developmental Neuroscience,Pediatrics, Perinatology and Child Health
Cited by
5 articles.
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