D'Aniello Alfredo, Giallonardo Anna Teresa, Tinuper Paolo, Mecarelli Oriano, Aguglia Umberto, Assenza Giovanni, Gambardella Antonio, Canevini Maria Paola, Scifo Renato, Meletti Stefano, De Giorgis Valentina, Bonanni Enrica, Michelucci Roberto, Cerminara Caterina, Romeo Antonino, Vigevano Federico, Operto Francesca Felicia, Tramacere Luciana, Di Gennaro Giancarlo, Ngo Leock Y, Patten Anna, Gentile Anna Lisa, Pecori Andrea, Goldman Samantha
Department of Epilepsy, IRCCS Istituto Neurologico Mediterraneo Neuromed, Pozzilli, Italy.
Department of Human Neurosciences, Epilepsy Centre, Sapienza University of Rome, Rome, Italy.
Epilepsia Open. 2025 Jun 4. doi: 10.1002/epi4.70068.
The AMPA study (Study 501; NCT04257604) was a multicenter, prospective, 12-month observational study in Italy that evaluated the effectiveness and safety of adjunctive perampanel in patients with focal-onset seizures (FOS), with or without focal to bilateral tonic-clonic seizures (FBTCS).
Patients aged ≥12 years with insufficiently controlled FOS, with or without FBTCS, receiving 1-3 anti-seizure medications (ASMs) were prescribed adjunctive perampanel per the approved indication. The primary endpoint was the median percent change in total seizure frequency per 28 days from baseline at Month 6. Baseline seizure frequency per 28 days was calculated using seizure diaries and/or medical records of seizures occurring in the 8 weeks prior to the baseline visit while patients were receiving 1-3 ASMs. Treatment-emergent adverse events (TEAEs), including serious TEAEs, were monitored for up to 12 months.
Of the 240 patients enrolled in the study, 234 were included in the Full and Safety Analysis Sets. Median age (minimum, maximum) was 36.0 years (12, 84) and 51.3% (n = 120/234) were female. The majority of patients (77.8% [n = 182/234]) received ≥2 concomitant ASMs at baseline, with the most common being carbamazepine (33.8% [n = 79/234]). The median percent reduction in total seizure frequency per 28 days from baseline (95% confidence interval) was 55.4% (46.7%-66.7%) at Month 6. Overall, the retention rate was 57.3% (n = 134/234) following 12 months of treatment. During the study, the overall incidence of TEAEs was 56.4% (n = 132/234), with the most frequently reported TEAE being dizziness/vertigo (21.8% [n = 51/234]). Serious TEAEs were experienced by 6.0% (n = 14/234) of patients and no deaths were reported during the 12-month treatment period.
Data from the AMPA study suggest that adjunctive perampanel is associated with improvement in seizure control and with good retention rates and tolerability in a real-world clinical setting. These findings further support the use of adjunctive perampanel as a suitable treatment option for adolescent and adult patients with epilepsy.
Our study looked at teenage and adult patients with epilepsy in Italy who took the study drug, called perampanel, as well as the epilepsy treatments they had already been prescribed. After 12 months, 134 out of 234 patients were still using perampanel. Patients taking perampanel had fewer seizures than they did before they started taking perampanel. Side effects occurred in 132 patients (most commonly dizziness/vertigo, irritability, and sleepiness) and caused 45 of them to withdraw from the study. Perampanel was a suitable treatment option for teenage and adult patients with epilepsy.
AMPA研究（研究501；NCT04257604）是一项在意大利进行的多中心、前瞻性、为期12个月的观察性研究，评估了辅助使用吡仑帕奈治疗局灶性发作（FOS）患者（伴或不伴有继发全面性强直-阵挛发作（FBTCS））的有效性和安全性。
年龄≥12岁、FOS控制不佳（伴或不伴有FBTCS）且正在接受1 - 3种抗癫痫药物（ASM）治疗的患者，根据批准的适应症给予辅助使用吡仑帕奈。主要终点是第6个月时每28天总发作频率相对于基线的中位数变化百分比。每28天的基线发作频率是使用发作日记和/或基线访视前8周内患者接受1 - 3种ASM治疗时发生的发作的病历记录来计算的。对治疗中出现的不良事件（TEAE），包括严重TEAE，进行长达12个月的监测。
该研究共纳入240例患者，其中234例纳入全分析集和安全性分析集。中位年龄（最小值，最大值）为36.0岁（12, 84），51.3%（n = 120/234）为女性。大多数患者（77.8% [n = 182/234]）在基线时接受≥2种联合ASM治疗，最常见的是卡马西平（33.8% [n = 79/234]）。第6个月时每28天总发作频率相对于基线的中位数降低百分比（95%置信区间）为55.4%（46.7% - 66.7%）。总体而言，治疗随访12个月后的保留率为57.3%（n = 134/234）。在研究期间，TEAE的总体发生率为56.4%（n = 132/234），最常报告的TEAE是头晕/眩晕（21.8% [n = 51/234]）。6.0%（n = 14/234）的患者经历了严重TEAE，在12个月的治疗期内未报告死亡病例。
AMPA研究的数据表明，在真实世界的临床环境中，辅助使用吡仑帕奈与改善癫痫控制、良好的保留率和耐受性相关。这些发现进一步支持将辅助使用吡仑帕奈作为青少年和成年癫痫患者的一种合适治疗选择。
我们的研究观察了意大利的青少年和成年癫痫患者，他们服用了名为吡仑帕奈的研究药物以及他们已经被处方的癫痫治疗药物。12个月后，234例患者中有134例仍在使用吡仑帕奈。服用吡仑帕奈的患者发作次数比开始服用吡仑帕奈前减少。132例患者出现了副作用（最常见的是头晕/眩晕、易怒和嗜睡），其中45例因此退出研究。吡仑帕奈是青少年和成年癫痫患者的一种合适治疗选择。
