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.

中文翻译：

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IV 期 PROVE 研究：吡仑帕奈在儿童癫痫患者的真实临床护理中的应用

非介入性 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、有效性、安全性和耐受性以及吡仑帕奈剂量。该分析评估了年龄 <4 岁和 4-<12 岁患者的最终数据。数据是根据临床医生的建议，从 2014 年 1 月 1 日之后开始使用吡仑帕奈的美国患者的医疗/药房记录中回顾性获得的。保留率是主要终点。次要评估包括癫痫发作频率的中位百分比变化、无癫痫发作率、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 名患者（<4 岁；平均最大剂量，3.5 毫克/天）和 203 名患者（4-<12 岁；平均最大剂量，5.3 毫克/天）； 24 个月保留率分别为 35.7% (n = 5/14) 和 42.0% (n = 47/112)。在完整分析集中，第 1-3 个月期间，癫痫发作频率减少的中位数百分比分别为 33.3%（n = 8 [<4 岁]）和 26.0%（n = 32 [4-<12 岁]），并且癫痫发作-两组的自由率均为 12.5%（n = 1/8 和 n = 4/32）；后来的时间点患者数量较低。大多数患者的癫痫发作控制情况有所改善（45.9% [<4 岁] 对比 52.4% [4-<12 岁]）或无变化（45.9% 对比 34.5%） (SAS)。 12 名患者（<4 岁：29.3%；最常见的是易怒 [7.3%]）和 64 名患者（4-<12 岁：31.5%；最常见的是攻击性 [6.9%]）报告了治疗引起的不良事件 (TEAE) ）。吡仑帕奈通常耐受性良好，24 个月时导致停药的 TEAE 数 <21%，保留率良好（12 个月和 24 个月分别≥50% 和 >35%），并且在常规临床护理期间对儿科患者持续有效。