a University of Colorado Denver Anschutz School of Medicine , Children's Hospital Colorado , Aurora , CO , USA.
Expert Rev Clin Pharmacol. 2019 May;12(5):379-388. doi: 10.1080/17512433.2019.1605904. Epub 2019 Apr 24.
Dravet syndrome is an early childhood-onset epilepsy syndrome characterized by drug-resistant seizures, frequent episodes of status epilepticus, and the development of neurocognitive impairment. Seizure freedom in this condition is rare and there is a higher rate of sudden unexplained death in epilepsy patients (SUDEP) than other epilepsy syndromes. Stiripentol is a recently approved medication with an indication specifically for the treatment of seizures in children with Dravet syndrome. Areas covered: Review of relevant literature including the current and emerging treatment of seizures in children with Dravet syndrome, with a focus on stiripentol. This includes a review of the literature regarding the mechanism of action, clinical efficacy, and safety/tolerability of stiripentol. Expert opinion: Stiripentol has been available through expanded access programs resulting in a reduction of seizures and episodes of status epilepticus. With the Federal Drug Administration (FDA) approval, this treatment option will be more readily available to the Dravet syndrome population in the United States. The approval comes at a time of other treatment options also receiving approval (cannabidiol) and several products in ongoing studies (fenfluramine, TAK-935) providing additional treatment options and hope on the horizon for those impacted by this severe epilepsy syndrome.
德拉维特综合征是一种儿童期起病的癫痫综合征，其特征为耐药性癫痫发作、频繁癫痫持续状态发作以及神经认知障碍的发展。该病症很少能达到无癫痫发作，且癫痫猝死率（SUDEP）高于其他癫痫综合征。司替戊醇是一种最近获得批准的药物，专门用于治疗德拉维特综合征儿童的癫痫发作。
对包括治疗儿童德拉维特综合征癫痫发作的当前和新兴治疗方法的相关文献进行综述，重点关注司替戊醇。这包括对司替戊醇的作用机制、临床疗效以及安全性/耐受性的文献综述。
通过扩大准入计划，司替戊醇已经可用于临床，这导致癫痫发作和癫痫持续状态发作减少。随着美国食品和药物管理局（FDA）的批准，这种治疗选择将更容易为美国的德拉维特综合征患者所获得。该批准恰逢其他治疗选择也获得批准（大麻二酚）以及几项正在进行研究的产品（芬氟拉明、TAK-935）之际，为受这种严重癫痫综合征影响的人群提供了更多的治疗选择和希望。
