Department of Neurology, Hospital Ruber Internacional, Madrid, Spain.
Department of Neurology, Hospital Universitario La Fe, Valencia, Spain.
Epilepsia Open. 2024 Feb;9(1):164-175. doi: 10.1002/epi4.12847. Epub 2023 Nov 8.
To assess efficacy and tolerability of stiripentol (STP) as adjunctive treatment in Dravet syndrome and non-Dravet refractory developmental and epileptic encephalopathies (DREEs).
Retrospective observational study of all children and adults with DREE and prescribed adjunctive STP at Hospital Ruber Internacional from January 2000 to February 2023. Outcomes were retention rate, responder rate (proportion of patients with ≥50% reduction in total seizure frequency relative to baseline), seizure freedom rate, responder rate for status epilepticus, rate of adverse event and individual adverse events, reported at 3, 6, and 12 months and at final visit. Seizure outcomes are reported overall, and for Dravet and non-Dravet subgroups.
A total of 82 patients (55 Dravet syndrome and 27 non-Dravet DREE) were included. Median age was 5 years (range 1-59 years), and median age of epilepsy onset was younger in the Dravet group (4.9 [3.6-6] months) than non-Dravet (17.9 [6-42.3], P < 0.001). Median follow-up time STP was 24.1 months (2 years; range 0.3-164 months) and was longer in the Dravet group (35.9 months; range 0.8-164) than non-Dravet (17 months range 0.3-62.3, P < 0.001). At 12 months, retention rate, responder rate and seizure free rate was 68.3% (56/82), 65% [48-77%] and 18% [5.7-29%], respectively. There were no statistically significant differences between groups on these seizure outcomes. Adverse events were reported in 46.3% of patients (38/82), without differences between groups.
In this population of patients with epileptic and developmental encephalopathies, outcomes with adjunctive STP were similar in patients with non-Dravet DREE to patients with Dravet syndrome.
评估司替戊醇（STP）作为添加治疗在德拉维特综合征和非德拉维特难治性发育性和癫痫性脑病（DREE）中的疗效和耐受性。
这是一项对 2000 年 1 月至 2023 年 2 月期间在 Hospital Ruber Internacional 接受辅助 STP 治疗的所有 DREE 儿童和成人的回顾性观察性研究。结局包括保留率、应答率（与基线相比，总发作频率减少≥50%的患者比例）、无发作率、癫痫持续状态的应答率、不良反应发生率和个别不良反应发生率，分别在 3、6 和 12 个月以及最后一次就诊时报告。发作结局总体报告，并按德拉维特和非德拉维特亚组报告。
共纳入 82 例患者（55 例德拉维特综合征和 27 例非德拉维特 DREE）。中位年龄为 5 岁（范围 1-59 岁），癫痫发作的中位年龄在德拉维特组（4.9[3.6-6]个月）较非德拉维特组（17.9[6-42.3]岁）年轻（P<0.001）。STP 的中位随访时间为 24.1 个月（2 年；范围 0.3-164 个月），在德拉维特组（35.9 个月；范围 0.8-164）较非德拉维特组（17 个月，范围 0.3-62.3）长（P<0.001）。在 12 个月时，保留率、应答率和无发作率分别为 68.3%（56/82）、65%[48-77%]和 18%[5.7-29%]。在这些发作结局方面，两组之间无统计学差异。46.3%的患者（38/82）报告了不良反应，两组之间无差异。
在这组患有癫痫和发育性脑病的患者中，非德拉维特 DREE 患者与德拉维特综合征患者接受辅助 STP 治疗的结局相似。
