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WONOEP评估:癫痫生物标志物的发展——我们能从患者身上学到什么?

WONOEP appraisal: Development of epilepsy biomarkers-What we can learn from our patients?

作者信息

Jozwiak Sergiusz, Becker Albert, Cepeda Carlos, Engel Jerome, Gnatkovsky Vadym, Huberfeld Gilles, Kaya Mehmet, Kobow Katja, Simonato Michele, Loeb Jeffrey A

机构信息

Department of Child Neurology, Medical University of Warsaw, Warsaw, Poland.

Department of Child Neurology, The Children's Memorial Health Institute, Warsaw, Poland.

出版信息

Epilepsia. 2017 Jun;58(6):951-961. doi: 10.1111/epi.13728. Epub 2017 Apr 7.

Abstract

OBJECTIVE

Current medications for patients with epilepsy work in only two of three patients. For those medications that do work, they only suppress seizures. They treat the symptoms, but do not modify the underlying disease, forcing patients to take these drugs with significant side effects, often for the rest of their lives. A major limitation in our ability to advance new therapeutics that permanently prevent, reduce the frequency of, or cure epilepsy comes from a lack of understanding of the disease coupled with a lack of reliable biomarkers that can predict who has or who will get epilepsy.

METHODS

The main goal of this report is to present a number of approaches for identifying reliable biomarkers from observing patients with brain disorders that have a high probability of producing epilepsy.

RESULTS

A given biomarker, or more likely a profile of biomarkers, will have both a quantity and a time course during epileptogenesis that can be used to predict who will get the disease, to confirm epilepsy as a diagnosis, to identify coexisting pathologies, and to monitor the course of treatments.

SIGNIFICANCE

Additional studies in patients and animal models could identify common and clinically valuable biomarkers to successfully translate animal studies into new and effective clinical trials.

摘要

目的

目前用于癫痫患者的药物仅对三分之二的患者有效。对于那些有效的药物,它们也只是抑制癫痫发作。它们只能治疗症状,却无法改变潜在疾病,这就迫使患者服用这些有显著副作用的药物,而且往往要服用终生。我们推进能够永久预防、减少癫痫发作频率或治愈癫痫的新疗法的能力存在一个主要限制,即对该疾病缺乏了解,同时也缺乏可靠的生物标志物来预测谁患有癫痫或谁将会患上癫痫。

方法

本报告的主要目标是提出一些方法,以便通过观察极有可能引发癫痫的脑部疾病患者来识别可靠的生物标志物。

结果

特定的生物标志物,或者更有可能是一组生物标志物,在癫痫发生过程中会有一个数量和时间进程,可用于预测谁会患上这种疾病、确诊癫痫、识别并存的病理状况以及监测治疗过程。

意义

在患者和动物模型中开展的更多研究可以识别出常见且具有临床价值的生物标志物,从而成功地将动物研究转化为新的有效临床试验。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1201/5806696/b902c1df02f3/nihms855024f1.jpg

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