First Department of Internal Medicine, School of Medicine, National University of Athens, 75 M Asias Street, 11527 Athens, Greece.
Nat Rev Rheumatol. 2011 Nov 1;7(12):708-17. doi: 10.1038/nrrheum.2011.157.
Biomarkers have an important influence on the clinical decision-making processes involved in diagnosis, assessment of disease activity, allocation of treatment, and determining prognosis. The clinical usefulness of a biomarker is dependant on demonstration of its validity. Ideally, biomarkers should provide information not available from currently available tests and should be tested as they would be used in clinical practice; however, potential biomarkers could be affected by many different clinical or patient variables-such as disease activity, therapeutic intervention, or the presence of comorbidities--and validation studies might not include all the design features that are required to ensure that the biomarker is a true measure of the clinical process it is intended to reflect. In this Review, we appraise studies that have been conducted to validate six promising new biomarkers for diagnosis, disease activity assessment, or prognosis in patients with systemic autoimmune diseases. We discuss the validity of these six biomarkers with particular reference to the features of the studies that lend weight to or distract from their findings. The intent of this discussion is to draw attention to elements of validation study design that should be considered when evaluating the robustness of a biomarker, which differ according to the marker's intended use.
生物标志物对涉及诊断、疾病活动评估、治疗分配和预后判断的临床决策过程有重要影响。生物标志物的临床实用性取决于其有效性的证明。理想情况下，生物标志物应该提供当前可用测试无法提供的信息，并且应该按照临床实践中的使用方式进行测试；然而，潜在的生物标志物可能会受到许多不同的临床或患者变量的影响，如疾病活动、治疗干预或合并症的存在，验证研究可能不包括所有确保生物标志物真实反映其预期临床过程所需的设计特征。在这篇综述中，我们评估了已进行的旨在验证六种有前途的新生物标志物在系统性自身免疫性疾病患者中的诊断、疾病活动评估或预后中的作用的研究。我们特别参考研究的特点讨论了这六种生物标志物的有效性，这些特点对其研究结果有支持或干扰作用。讨论的目的是提请注意在评估生物标志物的稳健性时应考虑的验证研究设计要素，这些要素根据标志物的预期用途而有所不同。
