New York University Comprehensive Epilepsy Center, New York, New York, USA.
UCB Pharma, Morrisville, North Carolina, USA.
Epilepsia. 2022 Oct;63(10):2664-2670. doi: 10.1111/epi.17366. Epub 2022 Aug 12.
Drug treatment for children with epilepsy should, ideally, be governed by evidence from adequate and well-controlled clinical studies. However, these studies are difficult to conduct, and so direct evidence supporting the informed use of specific drugs is often lacking. The Research Roundtable for Epilepsy (RRE) met in 2020 to align on an approach to therapy development for focal seizures in children age 1 month <2 years of age.
The RRE reviewed the regulatory landscape, epidemiology, seizure semiology, antiseizure medicine pharmacology, and safety issues applicable to this population.
After reviewing evidence, the conclusion was that pediatric efficacy trials would be impracticable to conduct but a waiver of the regulatory requirement to conduct any study would lead to an absence of information to guide dosing in a critical population. Review of available data and discussion of RRE attendees led to the conclusion that the requirements for extrapolation of efficacy from older children down to infants from age 1 month to <2 years old appeared to be met. After the RRE, the US Food and Drug Administration (FDA) approved brivaracetam for use in children with focal epilepsy above the age of 1 month in August 2021 and lacosamide in October 2021, both based on the principle of extrapolation from data in older children.
These recommendations should result in more rapid accessibility of antiseizure medications for infants.
儿童癫痫的药物治疗应根据充分且良好对照的临床研究证据来指导。然而，这些研究很难开展，因此通常缺乏支持特定药物合理使用的直接证据。癫痫研究圆桌会议（RRE）于 2020 年开会，就儿童（年龄<2 岁，发病 1 个月以上）局灶性癫痫的治疗方法达成一致。
RRE 回顾了监管环境、流行病学、发作症状学、抗癫痫药物药理学和适用于该人群的安全性问题。
在审查了证据后，得出的结论是，儿科疗效试验难以开展，但如果放弃进行任何研究的监管要求，将导致缺乏信息来指导关键人群的用药剂量。对现有数据的审查和 RRE 与会者的讨论得出结论，似乎满足了从较大儿童外推疗效至 1 个月至<2 岁婴儿的要求。RRE 之后，美国食品和药物管理局（FDA）于 2021 年 8 月批准布立西坦用于 1 个月以上有局灶性癫痫的儿童，2021 年 10 月批准拉考沙胺用于该人群，两者均基于从较大儿童数据外推的原则。
这些建议应能使婴儿更快地获得抗癫痫药物。
