Should Off Label Antiepileptic Drugs Be Used for Treatment of Infancy and Childhood Epilepsy? Discussions Based on a Dravet Syndrome Case Report

JOURNAL OF PEDIATRIC EPILEPSY(2020)

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摘要
Epilepsy represents a burdensome neurological disorder with higher incidence before the age of 18 years. The treatment is medical and involves long-term administration of antiepileptic drugs (AED). There are known high-resistant syndromes with onset in infancy and childhood, Dravet syndrome, being one of them. It is a well-known fact that early seizures treatment prevents associated comorbidities, cognitive and motor disabilities, and improve long-term prognosis. There are several AEDs available but not all of them are approved for use in infants. This is due to the need for additional toxicology studies at this age and for development of suitable formulations. A 14-month-old girl with Dravet syndrome was presented here. Prompt diagnosis was made based on clinical features and confirmed by the genetic tests. She partially responded to valproate and clobazam but continued to have prolonged febrile seizures. We added stiripentol after consulting reports of studies in infants younger than 2 years and after obtaining family consent. She responded well with decrease in episodes of status epilepticus and improvement in psychomotor development and stiripentol was tolerated well. Off label use of certain AEDs can benefit infants when there are no major pharmacokinetic differences in comparison to older children.
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off label,antiepileptic drugs,Dravet syndrome
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