多发性硬化的生物标志物。

Pablo Villoslada
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引用次数: 11

摘要

追求个性化医疗需要发展生物标志物来预测疾病病程,监测疾病演变,根据疾病活动对患者亚群进行分层,并预测和监测对治疗的反应。多发性硬化症(MS)是一种常见于年轻人的神经系统疾病,病程不可预测,可能会导致严重的残疾,降低患者的生活质量。目前,疾病预后是基于临床信息(复发率和残疾量表)和诊断测试(脑MRI或脑脊液中存在寡克隆带)。然而,基于这些信息,神经科医生做出准确预后的能力非常有限,而患者认为这是他们最关心的问题之一。尽管最近的许多研究已经确定了与MS病程相关的不同分子和成像技术,但在大多数情况下,这些技术的诊断准确性尚未得到适当的评估。这一缺陷部分是由于未能验证这些生物标志物,这阻碍了它们在临床实践中的应用。然而,最近针对Devic病的抗水通道蛋白-4抗体的验证和MS光学相干断层扫描的发展,都是MS生物标志物发展可以提供的好处的例子。事实上,为了促进转化研究和提高患者的生活质量,目前可能有必要纠正研究中对临床验证而不是发现的偏见。
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Biomarkers for multiple sclerosis.

The pursuit of personalized medicine requires the development of biomarkers to predict disease course, monitor disease evolution, stratify patient subgroups by disease activity and to predict and monitor response to therapies. Multiple sclerosis (MS) is a common neurological disease in young adults with an unpredictable course that may be associated with significant disability, diminishing the patient's quality of life. Currently, disease prognosis is based on clinical information (relapse rate and disability scales) and diagnostic tests (brain MRI or the presence of oligoclonal bands in the cerebrospinal fluid). However, the ability of neurologists to make an accurate prognosis is very limited based on such information, a situation perceived by patients as one of their biggest concerns. Although many recent studies have identified different molecules and imaging techniques associated with the course of MS, in most cases the diagnostic accuracy of such technologies has not been properly assessed. This shortcoming is partly due to the failure to validate such biomarkers, which impedes their application in clinical practice. However, the recent validation of anti-aquaporin-4 antibodies for Devic's disease and the development of optic coherent tomography for MS, are examples of the benefits that the development of MS biomarkers can offer. Indeed, it may currently be necessary to redress the bias in research towards clinical validation rather than discovery in order to promote translational research and improve patient's quality of life.

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来源期刊
Drug news & perspectives
Drug news & perspectives 医学-药学
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