Table_1_Efficacy and tolerability of perampanel: a Chinese real-world observational study in epilepsy.XLSX

PurposeTo investigate whether there exists a statistically significant distinction between the effectiveness and tolerance of perampanel (PER) and the number of antiseizure medications (ASMs) that were tried prior to administering PER.MethodA prospective, observational study was performed at West China Hospital of Sichuan University. The study included patients diagnosed with epilepsy who were prescribed PER and were monitored for a minimum of 6 months. The efficacy of PER was evaluated at 1, 3, 6, and 12-month intervals by examining the retention rate and the 50% response rate. All statistical analyses were conducted using IBM SPSS Statistics version 25 (IBM Corporation, Armonk, New York).ResultsA total of 1,025 patients were identified, of which 836 were included in the analysis. Seven hundred and eighty-nine patients (94.4%) were followed up for a year. The median age of the patients was 29.32 ± 14.06 years, with 45.81% of the patients being male and 17.0% being adolescents. The average duration of epilepsy was 11.22 ± 8.93 years. Overall, PER was discontinued in 49.5% of patients, with the most common reasons being inadequate therapeutic effect and treatment-emergent adverse events (TEAEs). At the 6-month follow-up, the retention rate was 54.2% (454/836), and 39.6% of patients had a 50% response. At the 12-month follow-up, the retention rate was 49.4% (340/789), and 44.5% of patients had a 50% response. Patients who received PER as monotherapy had the highest retention rates (P = 0.034) and 50% response rates (P < 0.001) at any follow-up point. TEAEs were reported in 32.0% of patients, and these led to discontinuation in 15.4% of patients. The most common TEAEs were dizziness and somnolence. There was no significant difference between subgroups (P = 0.57), but there was a significant difference between the dosage of PER and TEAEs (P < 0.001).Main findingsThe study concludes that PER is effective in treating both focal and generalized tonic-clonic seizures. Patients who had fewer previous exposures to ASMs exhibited higher response rates to PER. TEAEs related to PER dosage were more prevalent during the first 3 months of treatment and tended to improve with continued use, ultimately demonstrating favorable long-term tolerability.

研究目的：旨在探讨在给予佩马西平（PER）之前尝试的抗癫痫药物（ASMs）数量与PER的有效性和耐受性之间是否存在统计学上的显著差异。研究方法：在四川大学华西医院进行了一项前瞻性、观察性研究。研究纳入了被诊断为癫痫并接受PER治疗且至少接受6个月监测的患者。通过考察保留率和50%反应率，在1、3、6和12个月的时间点评估了PER的疗效。所有统计分析均使用IBM SPSS Statistics版本25（IBM公司，纽约阿蒙克）进行。研究结果：共确定了1,025名患者，其中836名患者纳入分析。789名患者（94.4%）接受了为期一年的随访。患者的中位年龄为29.32 ± 14.06岁，其中45.81%的患者为男性，17.0%为青少年。癫痫的平均病程为11.22 ± 8.93年。总体而言，49.5%的患者中断了PER治疗，最常见的原因是治疗效果不足和治疗相关不良事件（TEAEs）。在6个月随访时，保留率为54.2%（454/836），39.6%的患者达到了50%的反应率。在12个月随访时，保留率为49.4%（340/789），44.5%的患者达到了50%的反应率。接受PER单药治疗的患者在任何随访点均具有最高的保留率（P = 0.034）和50%的反应率（P < 0.001）。TEAEs在32.0%的患者中报告，导致15.4%的患者中断治疗。最常见的不良事件为眩晕和嗜睡。在各亚组之间没有显著差异（P = 0.57），但PER剂量与TEAEs之间存在显著差异（P < 0.001）。主要发现：研究结论表明，PER在治疗局灶性和全身性强直阵挛性癫痫发作方面均有效。既往接触ASMs较少的患者对PER的反应率更高。与PER剂量相关的TEAEs在治疗的前3个月更为普遍，并且随着治疗的持续使用而趋于改善，最终显示出良好的长期耐受性。