Prevention of Epilepsy in Infants with Tuberous Sclerosis Complex in the EPISTOP Trial.
Prevention of Epilepsy in Infants with Tuberous Sclerosis Complex in the EPISTOP Trial.
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DOI：
10.1002/ana.25956
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发表时间：
2021-03
影响因子：
11.2
通讯作者：
EPISTOP Investigators
中科院分区：
文献类型：
--
作者：
Kotulska K;Kwiatkowski DJ;Curatolo P;Weschke B;Riney K;Jansen F;Feucht M;Krsek P;Nabbout R;Jansen AC;Wojdan K;Sijko K;Głowacka-Walas J;Borkowska J;Sadowski K;Domańska-Pakieła D;Moavero R;Hertzberg C;Hulshof H;Scholl T;Benova B;Aronica E;de Ridder J;Lagae L;Jóźwiak S;EPISTOP Investigators
癫痫发生在70 - 90%的结节性硬化症（TSC）儿童中，并且通常对药物有抵抗力。最近，预防性抗癫痫治疗的概念，以修改癫痫的自然史已被提出。EPISTOP是一项临床试验，旨在比较TSC婴儿的预防性与常规抗癫痫治疗。在这项多中心研究中，94名无癫痫发作史的TSC婴儿每月进行视频脑电图（EEG）随访，并在首次脑电图或临床癫痫发作后开始接受氨己烯酸作为常规抗癫痫治疗，或在癫痫发作前检测到癫痫样EEG活动时进行预防性治疗。在6个研究中心，受试者在随机对照试验（RCT）中以1：1的比例随机分配至治疗组。在4个研究中心，治疗分配是固定的;这被称为开放标签试验（奥尔特）。受试者随访至2岁。主要终点是至首次临床癫痫发作的时间。在54名受试者中，癫痫样脑电图异常被确定在癫痫发作之前。27例被纳入RCT，27例被纳入奥尔特。预防性治疗组至首次临床癫痫发作的时间显著长于常规治疗组[RCT：364天（95% CI = 223-535）vs 124天（95% CI = 33-149）;奥尔特：426天（95% CI = 258-628）vs 106天（95% CI = 11-149）]。在24个月时，我们的汇总分析显示预防性治疗降低了临床癫痫发作（比值比[OR] = 0.21，p = 0.032）、耐药性癫痫（OR = 0.23，p = 0.022）和婴儿痉挛（OR = 0，p < 0.001）的风险。未观察到与预防性治疗相关的不良事件。氨己烯酸预防性治疗是安全的，并修改了TSC癫痫发作的自然史，降低了癫痫的风险和严重程度。神经网络2021;89：304-314
Epilepsy develops in 70 to 90% of children with tuberous sclerosis complex (TSC) and is often resistant to medication. Recently, the concept of preventive antiepileptic treatment to modify the natural history of epilepsy has been proposed. EPISTOP was a clinical trial designed to compare preventive versus conventional antiepileptic treatment in TSC infants. In this multicenter study, 94 infants with TSC without seizure history were followed with monthly video electroencephalography (EEG), and received vigabatrin either as conventional antiepileptic treatment, started after the first electrographic or clinical seizure, or preventively when epileptiform EEG activity before seizures was detected. At 6 sites, subjects were randomly allocated to treatment in a 1:1 ratio in a randomized controlled trial (RCT). At 4 sites, treatment allocation was fixed; this was denoted an open‐label trial (OLT). Subjects were followed until 2 years of age. The primary endpoint was the time to first clinical seizure. In 54 subjects, epileptiform EEG abnormalities were identified before seizures. Twenty‐seven were included in the RCT and 27 in the OLT. The time to the first clinical seizure was significantly longer with preventive than conventional treatment [RCT: 364 days (95% confidence interval [CI] = 223–535) vs 124 days (95% CI = 33–149); OLT: 426 days (95% CI = 258–628) vs 106 days (95% CI = 11–149)]. At 24 months, our pooled analysis showed preventive treatment reduced the risk of clinical seizures (odds ratio [OR] = 0.21, p = 0.032), drug‐resistant epilepsy (OR = 0.23, p = 0.022), and infantile spasms (OR = 0, p < 0.001). No adverse events related to preventive treatment were noted. Preventive treatment with vigabatrin was safe and modified the natural history of seizures in TSC, reducing the risk and severity of epilepsy. ANN NEUROL 2021;89:304–314
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结节性硬化症复杂诊断标准更新：2012年iinternation Tuberous硬化症复杂共识会议的建议。
影响因子：
3.8
作者：
Northrup H;Krueger DA;International Tuberous Sclerosis Complex Consensus Group
通讯作者：
International Tuberous Sclerosis Complex Consensus Group
影响因子：
3.1
作者：
Kotulska, Katarzyna;Jurkiewicz, Elzbieta;Jozwiak, Sergiusz
通讯作者：
Jozwiak, Sergiusz
影响因子：
5.6
作者：
Overwater, Iris E.;Bindels-de Heus, Karen;de Wit, Marie-Claire Y.
通讯作者：
de Wit, Marie-Claire Y.
影响因子：
5.6
作者：
Kwan, Patrick;Arzimanoglou, Alexis;French, Jacqueline
通讯作者：
French, Jacqueline
影响因子：
5.6
作者：
Fisher, Robert S.;Acevedo, Carlos;Wiebe, Samuel
通讯作者：
Wiebe, Samuel