Friedo Anna-Lena, Greshake Benedikt, Makridis Konstantin L, Straub Hans-Beatus
Epilepsy Center Tabor, Bernau bei Berlin, Germany.
Department of Pediatric Neurology, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Front Neurol. 2023 Jul 17;14:1209487. doi: 10.3389/fneur.2023.1209487. eCollection 2023.
Epilepsy patients with intellectual disability often suffer from drug-resistant epilepsy (DRE), which severely affects patients' quality of life. Cenobamate (CNB) is a recently approved novel and effective ASM that can achieve high rates of seizure freedom in previously drug-resistant patients.
We performed a retrospective data analysis of the first patients treated with CNB at a single center. Outcome and treatment response were assessed at two different time points, and ASM burden was calculated.
A 12 patients (7 males and 5 females) began treatment at a median age of 43 years, six of whom had developmental and epileptic encephalopathies. Prior to treatment with CNB, patients had tried a median of 13 different ASM. At the start of CNB therapy, patients were taking a median of 3 ASM. Treatment outcomes were available for 11 patients. After the first follow-up period (median 9 months), 55% of patients showed a significant seizure reduction of more than 50%, with three patients showing a reduction of more than 75% (27%). One patient achieved complete seizure freedom, while one patient did not respond to treatment. These response rates were consistently maintained at second follow-up after a median of 22 months. Ten patients (83%) reported adverse events (AE), the most common of which were dizziness and fatigue. No cases of drug reactions with eosinophilia and systemic symptoms (DRESS) were observed. The majority of AEs were mild and resolved over time. In addition, most patients were able to reduce their concomitant ASM.
Cenobamate has been shown to be an effective ASM in patients with DRE and in patients with intellectual disabilities. After more than 1 year of treatment with CNB, close monitoring and management of drug-drug interactions may reduce enzyme-inducing ASMs and lead to better long-term outcomes. With CNB treatment, many patients can achieve a reduced overall drug burden while maintaining a reduction in seizures.
患有智力障碍的癫痫患者常患有药物难治性癫痫(DRE),这严重影响患者的生活质量。司替戊醇(CNB)是一种最近获批的新型有效抗癫痫药物(ASM),在既往耐药患者中可实现较高的无癫痫发作率。
我们对单中心首例接受CNB治疗的患者进行了回顾性数据分析。在两个不同时间点评估结局和治疗反应,并计算ASM负担。
12例患者(7例男性和5例女性)开始治疗的中位年龄为43岁,其中6例患有发育性和癫痫性脑病。在接受CNB治疗之前,患者平均尝试了13种不同的ASM。在CNB治疗开始时,患者平均服用3种ASM。11例患者有治疗结局数据。在第一个随访期(中位时间9个月)后,55%的患者癫痫发作显著减少超过50%,3例患者减少超过75%(27%)。1例患者实现完全无癫痫发作,而1例患者对治疗无反应。这些反应率在中位时间22个月后的第二次随访中持续保持。10例患者(83%)报告了不良事件(AE),最常见的是头晕和疲劳。未观察到伴有嗜酸性粒细胞增多和全身症状的药物反应(DRESS)病例。大多数AE为轻度且随时间缓解。此外,大多数患者能够减少其联用的ASM。
司替戊醇已被证明是一种对DRE患者和智力障碍患者有效的ASM。在用CNB治疗1年多后,密切监测和管理药物相互作用可能会减少酶诱导性ASM,并带来更好的长期结局。通过CNB治疗,许多患者可以在维持癫痫发作减少的同时降低总体药物负担。