Stiripentol 在 Dravet 综合征中的应用：一项美国回顾性研究的结果。
Stiripentol in Dravet syndrome: results of a retrospective U.S. study.
机构信息
Divisions of Child and Adolescent Neurology, Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA.
出版信息
Epilepsia. 2013 Sep;54(9):1595-604. doi: 10.1111/epi.12303. Epub 2013 Jul 12.
PURPOSE
To review the efficacy and tolerability of stiripentol in the treatment of U.S. children with Dravet syndrome.
METHODS
U.S. clinicians who had prescribed stiripentol for two or more children with Dravet syndrome between March 2005 and 2012 were contacted to request participation in this retrospective study. Data collected included overall seizure frequency, frequency of prolonged seizures, and use of rescue medications and emergency room (ER)/hospital visits in the year preceding stiripentol initiation, and with stiripentol therapy. We separately assessed efficacy in the following treatment groups: group A, stiripentol without clobazam or valproate; group B, stiripentol with clobazam but without valproate; group C, stiripentol with valproate but without clobazam; and group D, stiripentol with clobazam and valproate. In addition, adverse effects were recorded.
KEY FINDINGS
Thirteen of 16 clinicians contacted for study participated and provided data on 82 children. Stiripentol was initiated a median of 6.0 years after seizure onset and 1.2 years after diagnosis of Dravet syndrome. Compared to baseline, overall seizure frequency was reduced in 2/6 in group A, 28/35 in group B, 8/14 in group C, and 30/48 in group D. All children with prolonged seizure frequency greater than quarterly during the baseline period experienced a reduction in this frequency on the various treatment arms with stiripentol. Similarly, 2/4 patients in group A, 25/25 in group B, 5/10 in group C, and 26/33 in group D experienced reduction in frequency of rescue medication use and 1/1 in group A, 12/12 in group B, 3/5 in group C, and 18/19 in group D had reduction in frequency of ER/hospital visits. Adverse effects were reported in 38, most commonly sedation and reduced appetite. Four patients (5%) discontinued stiripentol for adverse effects and two (2%) for lack of efficacy.
SIGNIFICANCE
Stiripentol is an effective and well-tolerated therapy that markedly reduced frequency of prolonged seizures in Dravet syndrome.
目的
回顾美国患有德拉维综合征的儿童使用司替戊醇的疗效和耐受性。
方法
2005 年 3 月至 2012 年间，联系了曾为两名或两名以上患有德拉维综合征的儿童开具司替戊醇处方的美国临床医生，以请求参与这项回顾性研究。收集的数据包括司替戊醇治疗前一年和治疗期间的总体癫痫发作频率、持续性癫痫发作频率、以及急救药物和急诊室（ER）/医院就诊的使用情况。我们分别评估了以下治疗组的疗效：A 组，无氯巴占或丙戊酸的司替戊醇；B 组，无丙戊酸但有氯巴占的司替戊醇；C 组，无氯巴占但有丙戊酸的司替戊醇；D 组，有氯巴占和丙戊酸的司替戊醇。此外，还记录了不良反应。
主要发现
16 名联系参与研究的临床医生中有 13 名提供了 82 名儿童的数据。司替戊醇的起始治疗时间中位数为癫痫发作后 6.0 年和诊断为德拉维综合征后 1.2 年。与基线相比，A 组的 2/6 例、B 组的 28/35 例、C 组的 8/14 例和 D 组的 30/48 例总体癫痫发作频率降低。在基线期间持续性癫痫发作频率大于每季度的所有儿童，在各种司替戊醇治疗组中均观察到频率降低。同样，A 组的 2/4 例、B 组的 25/25 例、C 组的 5/10 例和 D 组的 26/33 例患者减少了急救药物的使用频率，A 组的 1/1 例、B 组的 12/12 例、C 组的 3/5 例和 D 组的 18/19 例患者减少了急诊室/医院就诊的频率。报告了 38 例不良反应，最常见的是镇静和食欲减退。4 名患者（5%）因不良反应和 2 名患者（2%）因疗效不佳而停止使用司替戊醇。
意义
司替戊醇是一种有效且耐受性良好的治疗方法，可显著降低德拉维综合征患者持续性癫痫发作的频率。
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