Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies.

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2025-06-01 Epub Date: 2025-05-03 DOI:10.1007/s40120-025-00755-5

Víctor Soto-Insuga, Elena González-Alguacil, María Ballarà-Petitbò, Nuria Lamagrande-Casanova, Anna Duat-Rodríguez, Cristina Benítez-Provedo, Elena Cardenal-Muñoz, Juan José García-Peñas

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引用次数: 0

Abstract

Introduction: Stiripentol is approved as an add-on therapy with clobazam and/or valproate for seizures associated with Dravet syndrome (DS). Evidence of stiripentol efficacy in other drug-resistant epilepsies is limited.

Methods: This was a single-centre, retrospective, observational study of patients aged ≤ 15 years with non-Dravet epilepsy or DS who initiated stiripentol treatment in Spain.

Results: The study included 18 patients with DS and 17 with non-Dravet epilepsy; 76.5% of the latter had a developmental and epileptic encephalopathy. Median (range) age at stiripentol initiation was 52 (4-180) months. Three months of add-on stiripentol provided overall improvement in seizures (number, duration and/or intensity) for 76.5% of the non-Dravet and 61.1% of the DS patients (p = 0.30), all of whom were drug-resistant to prior antiseizure medications (ASMs). Stiripentol reduced seizure frequency by ≥ 50% in 58.8% of the non-Dravet patients and 44.4% of the DS cohort (p = 0.40); 20% of all patients became seizure-free. Stiripentol reduced all seizure types in both cohorts. Kaplan-Meier survival analysis found a higher probability of sustained stiripentol efficacy in the DS cohort (120 months) than the non-Dravet cohort (16 months; p = 0.012). Stiripentol improved cognition and Clinical Global Impression scale scores in approximately 60% of all patients; sleep improved for 19.2%. Acute stiripentol treatment (maximum dose 6.7-100 mg/kg/day) initiated in five patients (four with non-Dravet epilepsy and one with DS) during refractory status epilepticus (SE) successfully resolved SE over a median 0.5 days. Adverse events, mainly mild-to-moderate, occurred in 47.1% and 41.2% of patients in the non-Dravet and DS cohorts, respectively. Six patients (35.3%) with non-Dravet epilepsy discontinued ≥ 1 other ASMs after stiripentol initiation.

Conclusion: Add-on stiripentol provides overall improvement in different seizure types and non-seizure manifestations for paediatric patients with drug-resistant epilepsy, including epileptic syndromes besides DS, and appeared effective in acute treatment of SE. Stiripentol was generally well tolerated.

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斯立哌醇治疗德拉韦综合征的疗效：对耐药癫痫患者的回顾性病历回顾。

斯立哌醇被批准作为氯巴唑仑和/或丙戊酸盐的附加治疗与Dravet综合征（DS）相关的癫痫发作。斯立哌妥对其他耐药癫痫的疗效证据有限。方法：这是一项单中心、回顾性、观察性研究，研究对象是西班牙年龄≤15岁的非德拉韦癫痫或退行性癫痫患者，这些患者开始使用斯特里哌酮治疗。结果：本研究纳入18例DS患者和17例非dravet癫痫患者；后者有发育性和癫痫性脑病的占76.5%。施曲妥醇起始时的中位（范围）年龄为52（4-180）个月。对于76.5%的非达拉韦组和61.1%的退行性痴呆患者（p = 0.30）， 3个月的施曲妥醇治疗可使癫痫发作（次数、持续时间和/或强度）得到总体改善，这些患者均对既往抗癫痫药物（ASMs）具有耐药性。在58.8%的非dravet患者和44.4%的DS患者中，斯立哌醇使癫痫发作频率降低了≥50% (p = 0.40)；20%的患者不再癫痫发作。斯立喘醇降低了两组患者的所有癫痫发作类型。Kaplan-Meier生存分析发现，DS组（120个月）比非dravet组(16个月；p = 0.012)。斯立喷醇改善了大约60%的患者的认知和临床总体印象量表得分；19.2%的人睡眠得到改善。5例难治性癫痫持续状态（SE）患者（4例为非德拉韦癫痫，1例为DS）接受急性斯立哌醇治疗（最大剂量6.7- 100mg /kg/天），在中位0.5天内成功缓解SE。在非draft组和DS组中，不良事件发生率分别为47.1%和41.2%，主要为轻中度。6例（35.3%）非曲哌酮癫痫患者在施曲哌醇启动后停止了≥1种其他asm。结论：施替妥醇对小儿耐药癫痫患者的不同发作类型和非发作表现均有全面改善，包括除DS外的癫痫综合征，对SE的急性治疗效果明显。斯立喷醇的耐受性一般良好。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Neurology and Therapy CLINICAL NEUROLOGY-

CiteScore

5.40

自引率

8.10%

发文量

103

审稿时长

6 weeks

期刊介绍： Aims and Scope Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice. Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. 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