Keough Karen, Romick Alec
Pediatrix Medical Group-Neurology, 7940 Shoal Creek Blvd, Suite 100, Austin, TX, 78746, USA.
Texas A&M University Health Science Center-Round Rock, Round Rock, TX, USA.
Neurol Ther. 2025 Aug;14(4):1685-1694. doi: 10.1007/s40120-025-00779-x. Epub 2025 Jun 19.
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.
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.
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.
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.
伦诺克斯 - 加斯托综合征（LGS）是一种特别严重的发育性癫痫性脑病（DEE），其特征为多种类型的耐药性、致残性癫痫发作。尽管采用了包括联合用药、手术、植入设备和饮食疗法在内的积极治疗，但预后仍然很差，癫痫发作频繁，存在受伤和过早死亡的风险。司替戊醇（CNB）是一种获批用于治疗成人局灶性癫痫发作的抗癫痫药物（ASM），但在DEE患者中的实际应用经验显示癫痫发作频率有望降低。
这项回顾性病历审查确定了在一位医生治疗下的36例成年和儿童LGS患者中CNB的有效性和耐受性。
在36例LGS患者（69%为男性，中位年龄15.5岁）中，86%的患者在加用CNB后癫痫发作频率降低（中位治疗持续时间23个月），其中22例患者（61%）癫痫发作频率降低≥75%，5例患者（14%）癫痫发作停止。相当一部分患者（75%，n = 27）成功减少了他们的伴随用药，包括19例患者降低或停用了大麻二酚，21例患者降低或停用了氯巴占。三分之二的患者报告了不良事件，反映出与最初获批试验中报告的相同症状，嗜睡是最常见的。
这项病历审查为CNB治疗LGS的疗效提供了有前景的证据。更多的前瞻性研究将有助于阐明CNB对LGS患者的疗效和安全性概况。
