辅助治疗成人和儿童难治性lenox -胃综合征：回顾性图表回顾。

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2025-06-19 DOI:10.1007/s40120-025-00779-x

Karen Keough, Alec Romick

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引用次数: 0

摘要

lenox - gastaut综合征（LGS）是一种特别严重的发育性癫痫性脑病（DEE），其特征是多种类型的耐药、失能性癫痫发作。尽管积极的治疗包括多种药物、手术、植入设备和饮食治疗，预后仍然很差，经常持续发作，有受伤和早期死亡的风险。Cenobamate （CNB）是一种抗癫痫药物（ASM），被批准用于治疗成人局灶性癫痫发作，但实际经验表明，dei患者的癫痫发作频率有希望降低。方法：回顾性分析36例成人和儿童LGS患者在一名医生的治疗下使用CNB的疗效和耐受性。结果：36例LGS患者（69%为男性，中位年龄15.5岁）中，86%的患者在添加CNB（中位治疗持续时间23个月）后癫痫发作频率降低，其中22例患者（61%）癫痫发作频率降低≥75%，5例患者癫痫发作自由（14%）。相当比例的患者（75%,n = 27）成功地减少了他们的伴随药物，包括19例患者减少或停止使用大麻二酚，21例患者减少或停止使用氯巴唑。三分之二的患者报告了不良事件，反映了最初批准试验中报告的相同症状，嗜睡是最常见的。结论：本综述为CNB治疗LGS的疗效提供了有希望的证据。进一步的前瞻性研究将有助于阐明CNB对LGS患者的疗效和安全性。

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Cenobamate for Adjunctive Treatment in Adult and Pediatric Patients with Refractory Lennox-Gastaut Syndrome: A Retrospective Chart Review.

Introduction: Lennox-Gastaut syndrome (LGS) is a particularly severe developmental epileptic encephalopathy (DEE) characterized by multiple types of drug-resistant, incapacitating seizures. Despite aggressive therapy including polypharmacy, surgery, implanted devices, and dietary therapy, the prognosis remains poor, with frequent ongoing seizures and risk of injury and early death. Cenobamate (CNB) is an antiseizure medication (ASM) approved for the treatment of focal seizures in adults, but real-world experience in patients with DEEs has shown promising reductions in seizure frequency.

Methods: This retrospective chart review determined the effectiveness and tolerability of CNB in 36 adult and pediatric patients with LGS under the treatment of one physician.

Results: Among 36 patients (69% male, median age 15.5 years) with LGS, 86% experienced a reduction in seizure frequency after the addition of CNB (median treatment duration 23 months), including ≥ 75% reduction in 22 patients (61%) and seizure freedom in 5 patients (14%). A substantial proportion of patients (75%, n = 27) successfully reduced their concomitant medications, including the lowering or discontinuation of cannabidiol in 19 patients and clobazam in 21 patients. Adverse events were reported in two-thirds of patients, reflecting the same symptoms reported in the original approval trials, with somnolence being the most common.

Conclusions: This chart review provides promising evidence for the efficacy of CNB in treating LGS. Additional prospective studies will help to clarify CNB's efficacy and safety profile for patients with LGS.

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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. The journal is read by a global audience and receives submissions from all over the world. Neurology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research. Rapid Publication The journal’s rapid publication timelines aim for a peer review decision within 2 weeks of submission. If an article is accepted, it will be published online 3-4 weeks from acceptance. These rapid timelines are achieved through the combination of a dedicated in-house editorial team, who closely manage article workflow, and an extensive Editorial and Advisory Board who assist with rapid peer review. 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