Main indications and clinical application directions of Stiripentol

Stiripentol (Stiripentol) is an oral anti-epileptic drug, mainly suitable for epileptic seizures related to Dravet Syndrome (DS). Dravet syndrome is a rare genetic epilepsy disorder that usually presents with persistent seizures in infancy, accompanied by symptoms such as febrile epilepsy, complex partial seizures, and generalized convulsions. Episodes of this disease often respond poorly to conventional antiepileptic drugs, so there is an urgent clinical need for specific treatment options. Stipentol is used as an adjuvant drug, usually in combination with clobazole or other anti-epileptic drugs to enhance seizure control.

In clinical application, the advantage of stiripentol is that it can significantly reduce the frequency of attacks in patients with Dravet syndrome and improve their quality of life. Research and clinical practice have shown that patients over 6 months old and weighing at least 7 kg can achieve more obvious effects when combined with clobazole. It should be emphasized that there is currently insufficient data to support the use of stiripentol alone in the treatment of Dravet syndrome, and combined use is still the clinical norm. This medication strategy also reflects the complexity of the treatment of Dravet syndrome, that is, it is often difficult to completely control symptoms with a single drug, making multi-drug combination therapy a realistic option.

From the perspective of pharmacological mechanism, stiripentol exerts anti-epileptic effects by enhancingGABAergic neuroinhibition and inhibiting the drug metabolizing enzyme CYP3A4 in the liver, thereby prolonging the blood concentration of other anti-epileptic drugs and achieving synergistic effects. Overseas experts pointed out that this mechanism gives stiripentol unique advantages in the treatment of refractory childhood epilepsy, but it also requires strict monitoring of possible drug interactions and adverse reactions caused by combined use.

In clinical practice, doctors will adjust the dose according to the patient's weight, attack frequency and drug tolerance, and combine life intervention, attack trigger management and regular neurological follow-up to achieve comprehensive management.

Reference materials:https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/206709s000,207223s000lbl.pdf