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磁共振成像纹状体体积:亨廷顿舞蹈病临床试验的一种生物标志物。

Magnetic resonance imaging striatal volumes: a biomarker for clinical trials in Huntington's disease.

作者信息

Aylward Elizabeth H

机构信息

Seattle Children's Research Institute, Seattle, Washington, USA.

出版信息

Mov Disord. 2014 Sep 15;29(11):1429-33. doi: 10.1002/mds.26013. Epub 2014 Aug 27.

Abstract

An abundance of research shows that magnetic resonance imaging (MRI) striatal volumes decrease long before diagnosis of Huntington's disease (HD) and closely track disease progression. Additional research indicates that these volumetric measures meet important criteria for a biomarker that can be used in clinical trials: They are 1) objectively measureable; 2) able to predict known endpoints; and 3) associated with known mechanisms of pathology of the disease. Researchers should consider formal application to regulatory agencies for biomarker status of volumetric MRI striatal measures, because these measures are anticipated to contribute significantly in the assessment of treatment effectiveness in HD.

摘要

大量研究表明,在亨廷顿舞蹈症(HD)被诊断出来很久以前,磁共振成像(MRI)显示的纹状体体积就会减小,并且与疾病进展密切相关。更多研究表明,这些体积测量值符合可用于临床试验的生物标志物的重要标准:它们1)可客观测量;2)能够预测已知的终点;3)与该疾病已知的病理机制相关。研究人员应考虑正式向监管机构申请将MRI纹状体体积测量作为生物标志物的地位,因为预计这些测量值将在评估HD治疗效果方面做出重大贡献。

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