Stiripentol in childhood partial epilepsy: Randomized placebo-controlled trial with enrichment and withdrawal design
Stiripentol in childhood partial epilepsy: Randomized placebo-controlled trial with enrichment and withdrawal design
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DOI：
10.1177/08830738060210062101
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发表时间：
2006-06-01
影响因子：
1.9
通讯作者：
Pons, Gerard
中科院分区：
文献类型：
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作者：
Chiron, Catherine;Tonnelier, Sylvie;Pons, Gerard
抑制细胞色素P450酶的新型抗癫痫药物Stiripentol与卡马西平联合治疗儿童难治性部分性癫痫的开放试验显示了一定的疗效。我们的目的是在安慰剂对照试验中检验这些结果。为了限制纳入的患者数量，我们使用了富集和撤回设计。在67名儿童中，在1个月的单盲安慰剂基线后，进入了4个月的开放式添加司替戊醇研究，32名应答者被随机分配2个月，要么继续司替戊醇（n = 17），要么退出安慰剂（n = 15）。如果与基线相比，随机分组后癫痫发作增加至少50%，则患者退出（主要终点）：有6名患者服用司替戊醇，8名患者服用安慰剂（不显著）。然而，与基线（次要终点）相比，司替戊醇组（-75%）的癫痫发作频率下降幅度大于安慰剂组（-22%）（P
Stiripentol, a new antiepileptic drug inhibiting cytochrome P450-enzymes, suggested some efficacy when combined with carbamazepine in an open trial in refractory partial epilepsy of childhood. Our objective was to test these results in a placebo-controlled trial. To limit the number of patients included, we used an enrichment and withdrawal design. Among the 67 children entered in a 4-month open add-on stiripentol study following a 1-month single-blind placebo baseline, the 32 responders were randomized for 2 months either to continue stiripentol (n = 17) or to withdraw to placebo (n = 15). If seizures increased by at least 50% after randomization compared with baseline, the patients dropped out (primary end point): there were six patients on stiripentol and eight patients on placebo (not significant). However, a decrease in seizure frequency compared with baseline (secondary end point) was greater on stiripentol (-75%) than on placebo (-22%) (P