目的:确定Perampanel(PER)作为4-12岁癫痫患儿的辅助治疗的有效性和安全性。
方法:我们进行了非随机,开放标签,安慰剂不受控制,现实世界的自我对照研究,包括2020年7月4日至2023年9月20日在重庆医科大学附属儿童医院接受PER作为辅助治疗的216名癫痫患儿(4-12岁)。
结果:(1)辅助PER治疗在3、6、9和12个月的有效率为62.8%,67.8%,65.3%,和61.2%,分别。PER显示出缓解局灶性癫痫发作的功效,全身性强直-阵挛性癫痫发作,肌阵挛性癫痫发作,和缺勤癫痫发作。变异型自限性癫痫伴中央颞部尖峰(SeLECTS)和Lennox-Gastaut综合征(LGS)的有效率分别为89.5%和66.7%,分别。(2)局灶性非运动性发作性癫痫发作有或没有意识受损,双侧强直阵挛性癫痫发作(FBTCS),LGS,SLECTS的变体,伴随的抗癫痫药物(ASM)的数量,有癫痫家族史,头颅磁共振成像局灶性病变是影响疗效的独立因素。PER添加的顺序不影响功效。3、6、9和12个月的保留率为90.7%,84.7%,74.7%,64.9%,分别。(3)45例患者出现不良反应(45/216,20.8%),最常见的是易怒/攻击行为(18/216,8.3%)和嗜睡(14/216,6.5%)。12例患者(12/216,5.6%)因不良反应退出研究。
结论:在中国年轻的癫痫患儿中,PER是有效的,安全,作为辅助疗法耐受性良好,使其成为与广谱ASM一起使用的可行选择。
OBJECTIVE: To determine the efficacy and safety of
perampanel (PER) as an adjunctive therapy in children aged 4-12 years with epilepsy.
METHODS: We performed a non-randomized, open-label, placebo-uncontrolled, real-world self-controlled study that included 216 young children (aged 4-12 years) with epilepsy who received PER as adjunctive therapy at the children\'s hospital affiliated with Chongqing Medical University from July 4, 2020, to September 20, 2023.
RESULTS: (1) The efficacy rates of adjunctive PER therapy at 3, 6, 9, and 12 months were 62.8%, 67.8%, 65.3%, and 61.2%, respectively. PER showed efficacy in alleviating focal seizures, generalized tonic-clonic seizures, myoclonic seizures, and absence seizures. The efficacy rates for variants of self-limited epilepsy with centrotemporal spikes (SeLECTS) and Lennox-Gastaut syndrome (LGS) were 89.5% and 66.7%, respectively. (2) Focal non-motor onset seizures with or without impaired awareness, focal to bilateral tonic-clonic seizures (FBTCS), LGS, variants of SeLECTS, the number of concomitant antiseizure medications (ASMs), a family history of epilepsy, and focal lesions on cranial magnetic resonance imaging were independent factors affecting efficacy. The order of PER addition did not affect efficacy. The retention rates at 3, 6, 9, and 12 months were 90.7%, 84.7%, 74.7%, 64.9%, respectively. (3) Adverse reactions occurred in 45 patients (45/216, 20.8%), with irritability/aggressive behavior (18/216, 8.3%) and somnolence (14/216, 6.5%) being the most common. Twelve patients (12/216, 5.6%) withdrew from the study because of adverse reactions.
CONCLUSIONS: In young Chinese children with epilepsy, PER is effective, safe, and well-tolerated as an adjunctive therapy, making it a viable option for use with broad-spectrum ASMs.