About 25% of children with epilepsy cannot control their seizures after combined treatment with two well tolerated antiepileptic drugs, which is known as drug-resistant epilepsy. Stiripentol (STP) is a second-generation antiepileptic drug with unique pharmacological mechanisms and pharmacokinetic properties. It exerts direct anticonvulsant effects by acting on the gamma aminobutyric acid (GABA) system, and can also enhance the efficacy of other antiepileptic drugs through hepatic enzyme inhibition properties, exerting indirect anticonvulsant effects. Its inhibition of lactate dehydrogenase (LDH) and neuroprotective effects have also been confirmed by research. Currently, STP has been approved both domestically and internationally as an adjunct therapy to first-line treatment drugs for Dravet syndrome, based on chlorpromazine and valproic acid. Clinical studies have shown that STP, as an adjunct therapy for refractory atypical absence epilepsy and partial epileptic seizures in children, may be effective and well tolerated. Short-term adverse events mainly include neurological reactions (30%) and gastrointestinal reactions (29%), while appetite loss (16%) and weight loss (6%) are common long-term adverse events of STP. These adverse events are dose-dependent and can be relieved by reducing the dose.

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