Department of Neurology, Christian Doppler Medical Center and Center for Cognitive Neuroscience, Paracelsus Medical University, Salzburg, Austria.
Department of Mathematics, Paris Lodron University, Salzburg, Austria.
Epilepsia. 2018 Sep;59(9):1727-1739. doi: 10.1111/epi.14520. Epub 2018 Jul 25.
To pool observational data on the routine use of perampanel to obtain information on real-world outcomes and data in populations typically underrepresented in clinical trials.
Individual-level data of people with epilepsy treated with perampanel at 45 European centers were merged into a single dataset. Prespecified outcomes were: 1-year retention rate, 1-year seizure freedom rate (duration ≥6 months), and incidence of treatment-emergent adverse events (TEAEs). In addition, relationships were explored with logistic regression analyses.
The full analysis set comprised 2396 people: 95% had focal seizures; median epilepsy duration was 27 years; median number of concomitant antiepileptic drugs (AEDs) was 2; and median prior AEDs was 6. One-year retention rate was 48% (1117/2332; 95% confidence interval [CI] 46-50%), and 1-year seizure-free rate (≥6-month duration) was 9.2% (74/803; 95% CI 7-11%). Median treatment duration was 11.3 months (1832 patient-years); median dose was 8 mg. In 388 individuals with available data at 3, 6, and 12 months, responder rates were 42%, 46%, and 39%, respectively. During the first year, TEAEs were reported in 68% of participants (1317/1497; 95% CI 66-70%). Logistic regression found higher age at perampanel initiation was associated with higher seizure-free rate, and higher number of prior AEDs with lower seizure-free rate and lower rates of somatic TEAEs. In 135 individuals aged ≥65 years, 1-year retention rate was 48% and seizure-free rate was 28%.
Across a large, treatment-resistant population, add-on perampanel was retained for ≥1 year by 48% of individuals, and 9% were seizure-free for ≥6 months. TEAEs were in line with previous reports in routine clinical use, and less frequent than in the clinical trial setting. No new or unexpected TEAEs were seen. Despite the limitations of observational studies, our data indicate that some individuals may derive a marked benefit from the use of perampanel.
汇总有关仑帕奈常规应用的观察性数据，以获取临床试验中代表性不足人群的真实世界结局和数据信息。
将 45 家欧洲中心使用仑帕奈治疗的癫痫患者的个体水平数据合并到一个数据集。预先设定的结局为：1 年保留率、1 年无发作率（持续时间≥6 个月）和治疗中出现的不良事件（TEAEs）发生率。此外，还进行了逻辑回归分析以探索相关关系。
全分析集共纳入 2396 例患者：95%为局灶性发作；中位癫痫病程为 27 年；中位合用抗癫痫药物（AED）数为 2 种；中位既往 AED 数为 6 种。1 年保留率为 48%（2332/2396；95%置信区间[CI]为 46-50%），1 年无发作率（持续时间≥6 个月）为 9.2%（74/803；95%CI 为 7-11%）。中位治疗持续时间为 11.3 个月（1832 患者年）；中位剂量为 8mg。在 388 例有 3、6 和 12 个月数据的患者中，应答率分别为 42%、46%和 39%。在第一年中，68%（1317/1497；95%CI 为 66-70%）的患者报告了 TEAEs。逻辑回归发现，仑帕奈起始年龄较高与无发作率较高相关，而既往 AED 数量较多与无发作率较低和躯体 TEAEs 发生率较低相关。在 135 例年龄≥65 岁的患者中，1 年保留率为 48%，无发作率为 28%。
在一个大型、治疗抵抗的人群中，仑帕奈的加用治疗在 1 年内被 48%的患者保留，9%的患者无发作持续时间≥6 个月。TEAEs 与之前在常规临床应用中的报告一致，且比临床试验中的更少见。未观察到新的或意外的 TEAEs。尽管存在观察性研究的局限性，但我们的数据表明，某些个体可能从仑帕奈的使用中获得显著获益。
