Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois, U.S.A.
Epilepsia. 2013 Sep;54 Suppl 6(0 6):89-92. doi: 10.1111/epi.12288.
Benzodiazepine-refractory status epilepticus (established status epilepticus, ESE) is a relatively common emergency condition with several widely used treatments. There are no controlled, randomized, blinded clinical trials to compare the efficacy and tolerability of currently available treatments for ESE. The ESE treatment trial is designed to determine the most effective and/or the least effective treatment of ESE among patients older than 2 years by comparing three arms: fosphenytoin (fPHT) levetiracetam (LVT), and valproic acid (VPA). This is a multicenter, randomized, double-blind, Bayesian adaptive, phase III comparative effectiveness trial. Up to 795 patients will be randomized initially 1:1:1, and response-adaptive randomization will occur after 300 patients have been recruited. Randomization will be stratified by three age groups, 2-18, 19-65, and 66 and older. The primary outcome measure is cessation of clinical seizure activity and improving mental status, without serious adverse effects or further intervention at 60 min after administration of study drug. Each subject will be followed until discharge or 30 days from enrollment. This trial will include interim analyses for early success and futility. This trial will be considered a success if the probability that a treatment is the most effective is >0.975 or the probability that a treatment is the least effective is >0.975 for any treatment. Proposed total sample size is 795, which provides 90% power to identify the most effective and/or the least effective treatment when one treatment arm has a true response rate of 65% and the true response rate is 50% in the other two arms.
苯二氮䓬类药物难治性癫痫持续状态(已确立的癫痫持续状态,ESE)是一种较为常见的紧急情况,有多种广泛应用的治疗方法。目前尚无对照、随机、双盲临床试验来比较 ESE 现有治疗方法的疗效和耐受性。ESE 治疗试验旨在通过比较三种药物(苯妥英钠、左乙拉西坦和丙戊酸钠)来确定 2 岁以上患者中最有效和/或最无效的 ESE 治疗方法。这是一项多中心、随机、双盲、贝叶斯适应性、III 期比较有效性试验。最初将有 795 名患者随机分组,1:1:1,在招募了 300 名患者后,将进行反应适应性随机分组。随机分组将按三个年龄组(2-18 岁、19-65 岁和 66 岁及以上)分层。主要结局指标为临床痫性发作停止和精神状态改善,无严重不良事件或进一步干预,在给予研究药物后 60 分钟。每个受试者将被随访至出院或入组后 30 天。该试验将包括早期成功和无效的中期分析。如果一种治疗方法的有效性概率>0.975,或者任何一种治疗方法的最无效性概率>0.975,则该试验将被视为成功。建议的总样本量为 795 例,当一个治疗组的真实反应率为 65%,而其他两个组的真实反应率为 50%时,该试验具有 90%的效力来识别最有效和/或最无效的治疗方法。