Impact of Initial Therapy and Response on Long Term Outcome in Children with CAE

初始治疗和反应对 CAE 儿童长期结果的影响

• 批准号: 8334082
• 负责人: TRACY A GLAUSER
• 金额: $ 316.56万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2003
• 资助国家: 美国
• 起止时间: 2003-09-30 至 2014-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8334082
• 关键词: Absence Epilepsy; Academic achievement; Accounting; Address; Age; Attention; Behavioral; Body mass index; Child; Childhood; Clinical; Clinical Trials; Cognitive; Control Groups; Development; Disease remission; Dose; Double-Blind Method; Effectiveness; Electroencephalography; Emotional; Epilepsy; Ethosuximide; Evidence based treatment; Failure; Freedom; Growth; Growth and Development function; Individual; Intelligence; Kilogram; Knowledge; Measures; Memory; Monitor; Neuropsychological Tests; Newly Diagnosed; Outcome; Outcome Measure; Pharmaceutical Preparations; Pharmacotherapy; Principal Investigator; Quality of life; Randomized; Randomized Controlled Trials; Safety; Seizures; Syndrome; Tonic - clonic seizures; Treatment Failure; Valproic Acid; cognitive function; cohort; comparative effectiveness; comparative trial; executive function; follow-up; lamotrigine; neuropsychological; primary outcome; response

DESCRIPTION (provided by applicant): Childhood Absence Epilepsy (CAE) is the most common childhood onset epilepsy syndrome, accounting for 10-17% of all childhood onset epilepsy. In its first 6 years, the 32 center CAE trial successfully completed a double blind randomized controlled comparative trial (NS045911 and NS045803) comparing short term outcomes for ethosuximide, lamotrigine and valproic acid as initial therapy for children with newly diagnosed CAE characterized by only absence seizures. The CAE trial established ethosuximide as the optimal initial short term therapy for children with newly diagnosed CAE. However, it is clear that long term outcomes remain the crucial outcomes that should be used to determine optimal therapy. We propose to use this well characterized, carefully monitored cohort treated through a randomized controlled trial to determine if ethosuximide maintains its superior effectiveness (freedom from failure) over the long term along with better seizure freedom, seizure remission, cognitive and safety outcomes than lamotrigine or valproic acid in this well defined idiopathic epilepsy syndrome. Serial assessment of seizure control (including occurrence of generalized tonic clonic seizures), seizure remission, behavioral/emotional status, quality of life and growth will occur over the entire 4 years of the study. All CAE subjects and a cohort of 150 comparable healthy unrelated control subjects will be assessed using a new neuropsychological testing battery to better understand the CAE subjects' neuropsychological function at 6 years after start of therapy. This study's results will identify the optimal initial monotherapy for best long term outcomes. These results will address knowledge gaps about the long term comparative effectiveness of initial monotherapy for CAE and allow clinicians to make more evidence based treatment decisions for initial therapy to optimize both seizure and cognitive long term outcomes. RELEVANCE: Epilepsy (CAE) is the most common childhood epilepsy syndrome. Although a recent clinical trial of 446 children with CAE found ethosuximide as optimal initial short term therapy, is it not clear how short term results relate to long term outcomes. Optimal long term therapy can be determined by following these children for four more years.

描述（由申请人提供）：儿童缺勤癫痫（CAE）是最常见的儿童发作癫痫综合症，占所有儿童期癫痫的10-17％。在最初的6年中，32 Center CAE试验成功完成了一项双重盲随机对照比较试验（NS045911和NS045803），比较了Ethosuximide，Lamotrigine和Valproic Acid的短期结局作为仅缺席持有的新诊断为CAE的儿童的初始治疗的初始治疗。 CAE试验确立了新诊断为CAE儿童的最佳初始短期治疗。但是，很明显，长期结局仍然是确定最佳治疗的关键结果。我们建议使用通过随机对照试验进行的这种特征性，仔细监测的队列，以确定Ethosuximide长期保持其优势效率（免于失败），以及比羊角三酰胺或瓣膜酸更好的癫痫发作自由，癫痫发作的缓解，认知和安全性结果。癫痫发作控制的序列评估（包括发生普遍的隆隆声癫痫发作），癫痫发作，行为/情绪状况，生活质量和成长将在整个研究的整个研究中发生。将使用新的神经心理学测试电池评估所有CAE受试者和150名可比健康无关的对照受试者的队列，以更好地了解CAE受试者在治疗开始后6年的神经心理学功能。这项研究的结果将确定最佳长期结局的最佳初始单一疗法。这些结果将解决有关CAE初始单一疗法的长期比较有效性的知识差距，并允许临床医生做出更多基于证据的治疗决策，以优化癫痫发作和认知能力的长期结果。 相关性：癫痫（CAE）是最常见的儿童癫痫综合征。尽管最近446名CAE儿童的临床试验发现乙糖胺是最佳的初始短期治疗，但尚不清楚短期结果与长期结局如何相关。最佳的长期治疗可以通过跟随这些儿童多四年来确定。