Kork Epilepsy Center, Kehl-Kork, Germany.
Epilepsy Center Frankfurt Rhine-Main, Goethe University Frankfurt, Frankfurt am Main, Germany.
Epilepsia. 2021 Mar;62(3):785-794. doi: 10.1111/epi.16829. Epub 2021 Feb 3.
There is evidence that everolimus (EVE) significantly reduces seizure frequency in epilepsy patients with tuberous sclerosis complex (TSC). Given that TSC-related proliferative processes are more dynamic during brain development, seizure outcomes of patients treated with EVE may be age-related and may be less convincing in adult patients. The aim of this study was to assess the effectiveness and the safety profile of EVE in adults in clinical practice.
We performed a multicenter retrospective chart review of TSC subjects with active epilepsy who started EVE in adulthood (≥18 years of age) at seven German epilepsy centers. The primary endpoint was the retention rate after 6 months.
A total of 45 subjects with a mean age of 31.6 ± 11.1 years at EVE start fulfilled the inclusion criteria. Retention rate after 6 months was 98% (43/44 evaluable subjects). Response rate (seizure reduction ≥ 50%) was 33% (14/43 evaluable subjects; four completely seizure-free). We did not find a significant relationship between epilepsy outcome parameters and patient age at EVE start. Adverse events were reported in 19 subjects and were judged to be serious in six patients. Three patients died during the observation period.
Evidence suggests that EVE is an effective add-on treatment for epilepsy in adult TSC patients, surprisingly without any age limit to individual benefit. A strong age-dependent effect within the period of adulthood seems unlikely. Even if there was no proof of a causal relationship between deaths and EVE intake, patients with EVE should be carefully monitored, especially for infections and stomatitis.
有证据表明依维莫司（EVE）可显著降低结节性硬化症（TSC）癫痫患者的癫痫发作频率。鉴于 TSC 相关的增殖过程在大脑发育过程中更为活跃，接受 EVE 治疗的患者的癫痫发作结果可能与年龄相关，在成年患者中可能不太明显。本研究旨在评估 EVE 在临床实践中对成年患者的疗效和安全性。
我们对 7 家德国癫痫中心的 45 例成年（≥18 岁）开始接受 EVE 治疗的活跃性癫痫 TSC 患者进行了多中心回顾性图表审查。主要终点是 6 个月后的保留率。
共有 45 例患者符合纳入标准，EVE 起始时的平均年龄为 31.6±11.1 岁。6 个月后的保留率为 98%（44 例可评估患者中的 43 例）。反应率（癫痫发作减少≥50%）为 33%（43 例可评估患者中的 14 例；4 例完全无癫痫发作）。我们未发现癫痫结局参数与 EVE 起始时患者年龄之间存在显著关系。19 例患者报告了不良事件，其中 6 例被判断为严重不良事件。3 例患者在观察期间死亡。
有证据表明，EVE 是成年 TSC 患者癫痫的有效附加治疗方法，令人惊讶的是，它对个体获益没有年龄限制。在成年期内，年龄依赖性影响似乎不太可能。即使没有证据表明死亡与 EVE 摄入之间存在因果关系，但仍应密切监测服用 EVE 的患者，尤其是感染和口腔炎。
