Audit of use of stiripentol in adults with Dravet syndrome.
Audit of use of stiripentol in adults with Dravet syndrome.
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DOI：
10.1111/ane.12611
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发表时间：
2017-01
影响因子：
3.5
通讯作者：
Sisodiya SM
中科院分区：
文献类型：
--
作者：
Balestrini S;Sisodiya SM
文献中关于司替戊醇用于Dravet综合征（DS）成人的数据很少。DS病例在成年期越来越多地被认识到，现在有更多的DS儿童存活到成年。该研究的目的是记录司替戊醇在DS成人中的有效性和耐受性。我们在伦敦（英国）国立神经病学和神经外科医院的癫痫服务中进行了观察性临床审计。我们纳入了13名成年DS受试者（8名女性，5名男性）。36个月时，应答者（定义为所有癫痫发作类型减少50%以上）率为3/13（23%）。报告了以下其他结局：癫痫发作加重（3/13，23%），无变化（3/13，23%），癫痫发作减少50%以下（2/13，15%），全身强直阵挛性癫痫发作减少50%以上，但无其他癫痫发作类型（1/13，8%），未确定缓解（1/13，8%）。1年后的留存率为62%，5年后为31%。7/13例（54%）报告了不良反应：最常见的是厌食、体重减轻、不稳定和疲劳。3/13例（23%）患者因不良反应退出研究。与以前对DS儿童的研究相比，我们的结果显示了较低的应答率和相似的耐受性。Stiripentol可以是有效的，具有良好的耐受性。我们的审计规模较小，但支持在一线治疗无效或不耐受的DS成人中使用司替戊醇，与已发布的指南保持一致。
There are very few data available in the literature on the use of stiripentol in adults with Dravet syndrome (DS). DS cases are increasingly recognized in adulthood, and more children with DS now survive to adulthood. The aim of the study was to document the effectiveness and tolerability of stiripentol in adults with DS. We conducted an observational clinical audit in the epilepsy service of the National Hospital for Neurology and Neurosurgery, London (UK). We included 13 adult subjects with DS (eight females, five males). The responder (defined as more than 50% reduction in all seizure types) rate was 3/13 (23%) at 36 months. The following other outcomes were reported: seizure exacerbation (3/13, 23%), no change (3/13, 23%), less than 50% reduction in seizures (2/13, 15%), more than 50% reduction in generalized tonic‐clonic seizures but no other seizure types (1/13, 8%), undefined response (1/13, 8%). The retention rate was 62% after 1 year and 31% after 5 years. Adverse effects were reported in 7/13 (54%): the most frequent were anorexia, weight loss, unsteadiness and tiredness. Withdrawal due to adverse effects occurred in 3/13 (23%). Compared with previous studies on children with DS, our results show a lower responder rate and a similar tolerability profile. Stiripentol can be effective with a good tolerability profile. Our audit is small, but supports the use of stiripentol in adults with DS when first‐line treatments are ineffective or not tolerated, in keeping with published guidelines.
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影响因子：
5.6
作者：
Guerrini, R;Dravet, C;Dulac, O
通讯作者：
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