Coppola Giangennaro, Besag Frank, Cusmai Raffaella, Dulac Olivier, Kluger Gerhard, Moavero Romina, Nabbout Rima, Nikanorova Marina, Pisani Francesco, Verrotti Alberto, von Stülpnagel Celina, Curatolo Paolo
Child and Adolescent Neuropsychiatry, Medical School, University of Salerno, Italy.
South Essex Partnership University NHS Foundation Trust, Bedfordshire, United Kingdom.
Eur J Paediatr Neurol. 2014 Nov;18(6):685-90. doi: 10.1016/j.ejpn.2014.05.008. Epub 2014 May 28.
The literature on the efficacy and safety of rufinamide in childhood-onset epilepsy syndromes currently includes approximately 600 paediatric patients. This paper summarizes the views of a panel of experienced European epileptologists with regard to the current role of rufinamide in the treatment of childhood epilepsies.
Rufinamide is effective in decreasing the seizure frequency in the Lennox-Gastaut syndrome (LGS), especially tonic and atonic seizures. It might consequently be preferred to other drugs as a second-line treatment for LGS when drop-attacks are frequent. The mean responder rate in the published studies is 38% with seizure freedom achieved in 2.4% of patients. Rufinamide has shown some efficacy in epileptic encephalopathies other than LGS. It can be also effective as adjunctive therapy in children and adolescents with drug-resistant partial seizures. The available data suggest that rufinamide has an acceptable risk/benefit ratio with quite a low risk of aggravating seizures. Common adverse effects (somnolence, nausea and vomiting) are usually mild and self-limiting; they are more frequently observed during titration than in the maintenance phase, suggesting that low escalation rates might be associated with fewer adverse effects. Rufinamide appears to have a favourable cognitive profile compared with other antiepileptic drugs.
Rufinamide is only approved for adjunctive treatment of seizures associated with LGS in children 4 years of age and older. There are very few data on rufinamide treatment at the onset of LGS or early in the course of the disorder; whether early treatment will improve outcome has yet to be determined.
目前关于卢非酰胺治疗儿童期癫痫综合征有效性和安全性的文献纳入了约600例儿科患者。本文总结了一组经验丰富的欧洲癫痫专家对于卢非酰胺在儿童癫痫治疗中当前作用的观点。
卢非酰胺可有效降低Lennox-Gastaut综合征(LGS)的癫痫发作频率,尤其是强直发作和失张力发作。因此,当跌倒发作频繁时,作为LGS的二线治疗药物,它可能比其他药物更受青睐。已发表研究中的平均缓解率为38%,2.4%的患者实现无癫痫发作。卢非酰胺在LGS以外的癫痫性脑病中也显示出一定疗效。在耐药性部分性发作的儿童和青少年中,它作为辅助治疗也可能有效。现有数据表明,卢非酰胺的风险/效益比可接受,加重癫痫发作的风险相当低。常见不良反应(嗜睡、恶心和呕吐)通常较轻且为自限性;在滴定过程中比维持阶段更常观察到,这表明低滴定速率可能与较少的不良反应相关。与其他抗癫痫药物相比,卢非酰胺似乎对认知功能有有利影响。
卢非酰胺仅被批准用于4岁及以上儿童LGS相关癫痫发作的辅助治疗。关于LGS发病时或疾病早期使用卢非酰胺治疗的数据非常少;早期治疗是否会改善预后尚待确定。