Meridian Clinical Research, LLC, 6602 Waters Avenue, Building C, Savannah, GA, 31406, USA.
University of Tennessee Health Science Center, Le Bonheur Children's Hospital, 49 N Dunlap Street, Memphis, TN, 38105, USA.
Eur J Paediatr Neurol. 2024 May;50:23-30. doi: 10.1016/j.ejpn.2024.02.008. Epub 2024 Mar 5.
The non-interventional Phase IV PROVE study (NCT03208660) assessed retention, efficacy, safety and tolerability, and perampanel dosing in patients with epilepsy during routine clinical care. This analysis evaluated final data from patients aged <4 years and 4-<12 years.
Data were obtained retrospectively from medical/pharmacy records of patients in the United States initiating perampanel after January 1, 2014, according to treating clinician recommendations. Retention rate was the primary endpoint. Secondary assessments included median percent changes in seizure frequency, seizure-freedom rates, investigator impression of seizure effect, and safety and tolerability.
The Safety Analysis Set (SAS) included 41 patients (<4 years; mean maximum dose, 3.5 mg/day) and 203 patients (4-<12 years; mean maximum dose, 5.3 mg/day); 24-month retention rates were 35.7% (n = 5/14) and 42.0% (n = 47/112), respectively. In the Full Analysis Set, during Months 1-3, median percent reductions in seizure frequency were 33.3% (n = 8 [<4 years]) and 26.0% (n = 32 [4-<12 years]), and seizure-freedom rates were 12.5% in both groups (n = 1/8 and n = 4/32); patient numbers were low at later time points. Most patients showed improvements in seizure control (45.9% [<4 years] versus 52.4% [4-<12 years]) or no change (45.9% versus 34.5%) (SAS). Treatment-emergent adverse events (TEAEs) were reported in 12 (<4 years: 29.3%; most common, irritability [7.3%]) and 64 patients (4-<12 years: 31.5%; most common, aggression [6.9%]).
Perampanel was generally well tolerated with <21% of TEAEs leading to withdrawal at 24 months, had favorable retention rates (≥50% and >35% at 12 and 24 months, respectively), and sustained efficacy in pediatric patients during routine clinical care.
非干预性 IV 期 PROVE 研究（NCT03208660）评估了癫痫患者在常规临床护理期间的保留率、疗效、安全性和耐受性以及吡仑帕奈的剂量。本分析评估了年龄<4 岁和 4-<12 岁的患者的最终数据。
根据治疗临床医生的建议，从 2014 年 1 月 1 日起在美国开始使用吡仑帕奈的患者的医疗/药房记录中回顾性获得数据。保留率是主要终点。次要评估包括癫痫发作频率的中位数百分比变化、癫痫发作无发作率、研究者对癫痫发作效果的印象以及安全性和耐受性。
安全性分析集（SAS）包括 41 名（<4 岁；最大剂量平均值为 3.5mg/天）和 203 名（4-<12 岁；最大剂量平均值为 5.3mg/天）患者；24 个月的保留率分别为 35.7%（n=5/14）和 42.0%（n=47/112）。在全分析集中，在第 1-3 个月，癫痫发作频率的中位数百分比降低分别为 33.3%（n=8 [<4 岁]）和 26.0%（n=32 [4-<12 岁]），无癫痫发作率在两组中均为 12.5%（n=1/8 和 n=4/32）；在以后的时间点，患者人数较低。大多数患者的癫痫控制得到改善（45.9% [<4 岁]与 52.4% [4-<12 岁]）或无变化（45.9%与 34.5%）（SAS）。在 12 名（<4 岁：29.3%；最常见的是易激惹[7.3%]）和 64 名患者（4-<12 岁：31.5%；最常见的是攻击性行为[6.9%]）中报告了治疗期出现的不良事件（TEAEs）。
吡仑帕奈总体耐受性良好，<21%的 TEAEs 导致 24 个月时停药，保留率良好（分别在 12 个月和 24 个月时≥50%和>35%），并且在常规临床护理中对儿科患者具有持续疗效。
