Clinic of Child and Adolescent Neuropsychiatry, Medical School, University of Salerno, Italy.
Neuropsychiatr Dis Treat. 2011;7:399-407. doi: 10.2147/NDT.S13910. Epub 2011 Jul 5.
Rufinamide is an orally active, structurally novel compound (1-[(2,6-difluorophenil1) methyl1]-1 hydro 1,2,3-triazole-4 carboxamide), which is structurally distinct from other anticonvulsant drugs. It was granted orphan drug status for the adjunctive treatment of Lennox-Gastaut syndrome (LGS) in the United States in 2004, and released for use in Europe in 2007. In January 2009, rufinamide was approved by the United States Food and Drug Administration for treatment of LGS in children 4 years of age and older. It is also approved for adjunctive treatment for partial seizures in adults and adolescents. Rufinamide's efficacy mainly against atonic/tonic seizures in patients with LGS seems nowadays indubitable and has been confirmed both in randomized controlled trial and in open label extension studies. More recently, rufinamide was evaluated for the adjunctive treatment of childhood-onset epileptic encephalopathies and epileptic syndromes other than LGS, including epileptic spasms, multifocal epileptic encephalopathy with spasm/tonic seizures, myoclonic-astatic epilepsy, Dravet syndrome and malignant migrating partial seizures in infancy. This review updates the existing literature data on the efficacy and safety/tolerability of rufinamide in childhood-onset epilepsy syndromes.
氨己烯酸是一种具有口服活性的、结构新颖的化合物(1-[(2,6-二氟苯基)甲基]-1 氢-1,2,3-三唑-4 羧酰胺),与其他抗惊厥药物在结构上不同。2004 年,它在美国被授予孤儿药地位,用于辅助治疗 Lennox-Gastaut 综合征(LGS),并于 2007 年在欧洲上市。2009 年 1 月,氨己烯酸被美国食品和药物管理局批准用于治疗 4 岁及以上儿童的 LGS。它也被批准用于辅助治疗成人和青少年的部分性癫痫发作。氨己烯酸对 LGS 患者的强直/阵挛性发作的疗效似乎是毋庸置疑的,这一点已在随机对照试验和开放标签扩展研究中得到证实。最近,氨己烯酸被评估用于辅助治疗儿童期起病的癫痫性脑病和除 LGS 以外的其他癫痫综合征,包括癫痫性痉挛、多灶性癫痫性脑病伴痉挛/强直发作、肌阵挛-失张力性癫痫、Dravet 综合征和婴儿期恶性游走性部分性癫痫发作。本综述更新了氨己烯酸在儿童期起病的癫痫综合征中的疗效和安全性/耐受性的现有文献数据。