Feigin Andrew
Center for Neurosciences, North Shore-Long Island Jewish Research Institute, Manhasset, New York 11030, USA.
NeuroRx. 2004 Jul;1(3):323-30. doi: 10.1602/neurorx.1.3.323.
The use of physiological, anatomical, and other biological tests is commonplace in the practice of medicine. In neurology, objectively measured tests termed biomarkers (BMs) are playing an increasing role in diagnosis and management of disease, both in clinical practice and in experimental therapeutics. This article will discuss the various applications of BMs to the assessment of therapies for neurological diseases and will use examples from neurological diseases to elucidate the strengths and potential weaknesses of BMs. As the understanding of the pathophysiology of many neurological diseases has improved, new BMs have been developed, and efforts have been made to use these as proxies for clinical endpoints. A BM used in this manner is referred to as a surrogate endpoint (SE). There are many potential advantages and disadvantages of using SEs in the evaluation of new therapies, and these will be reviewed as well. Furthermore, the evidence required for the development of an SE and the nature of the evidence that can be derived from the use of BMs and SEs will be discussed.
在医学实践中，使用生理学、解剖学及其他生物学检测方法是很常见的。在神经病学领域，被称为生物标志物（BMs）的客观测量检测方法在疾病的诊断和管理中，无论是在临床实践还是实验治疗中，都发挥着越来越重要的作用。本文将讨论生物标志物在评估神经疾病治疗方法中的各种应用，并将通过神经疾病的实例来阐明生物标志物的优势和潜在不足。随着对许多神经疾病病理生理学的认识不断提高，新的生物标志物已被开发出来，并努力将其用作临床终点的替代指标。以这种方式使用的生物标志物被称为替代终点（SE）。在评估新疗法时使用替代终点有许多潜在的优点和缺点，本文也将对此进行综述。此外，还将讨论开发替代终点所需的证据以及使用生物标志物和替代终点可获得的证据的性质。
