National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: III. The 2014 Biomarker Working Group Report
National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: III. The 2014 Biomarker Working Group Report
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DOI：
10.1016/j.bbmt.2015.01.003
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发表时间：
2015-05-01
影响因子：
4.3
通讯作者：
Schultz, Kirk R.
中科院分区：
文献类型：
--
作者：
Paczesny, Sophie;Hakim, Frances T.;Schultz, Kirk R.
迫切需要基于生物学的标记物来确认或帮助诊断或预后异基因造血细胞移植后慢性移植物抗宿主病（GVHD）或监测其进展，以促进新疗法的评估。生物标志物被定义为客观测量和评估的任何特征，作为正常生物或致病过程或对治疗干预的药理学反应的指标。生物标志物在慢性GVHD临床试验或患者管理中的应用包括以下内容：（1）慢性GVHD疾病活动性的诊断和评估，包括区分不可逆损害与持续疾病活动性； (2) 发生慢性GVHD的预后风险； (3) 预测治疗反应。慢性 GVHD 生物标志物研究的样品采集应按照既定的质量控制指南进行详细记录，以按日历和事件驱动的间隔进行样品采集、处理、保存和测试。前瞻性临床试验最有可能提供支持生物标志物研究所需的一致的受试者治疗和标准化文件。迄今为止，还没有慢性 GVHD 生物标志物有资格在临床应用中使用。自 2005 年慢性 GVHD 生物标志物工作组报告以来，越来越多的慢性 GVHD 候选生物标志物可供进一步研究。本文提供了一个由 4 部分组成的生物标志物研究框架：识别、验证、鉴定和应用，其术语基于美国食品药品管理局和欧洲药品管理局指南。 (C) 2015 年美国血液和骨髓移植协会。
Biology-based markers to confirm or aid in the diagnosis or prognosis of chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic cell transplantation or monitor its progression are critically needed to facilitate evaluation of new therapies. Biomarkers have been defined as any characteristic that is objectively measured and evaluated as an indicator of a normal biological or pathogenic process, or of a pharmacologic response to a therapeutic intervention. Applications of biomarkers in chronic GVHD clinical trials or patient management include the following: (1) diagnosis and assessment of chronic GVHD disease activity, including distinguishing irreversible damage from continued disease activity; (2) prognostic risk to develop chronic GVHD; and (3) prediction of response to therapy. Sample collection for chronic GVHD biomarkers studies should be well documented following established quality control guidelines for sample acquisition, processing, preservation, and testing, at,intervals that are both calendar and event driven. The consistent therapeutic treatment of subjects and standardized documentation needed to support biomarker studies are most likely to be provided in prospective clinical trials. To date, no chronic GVHD biomarkers have been qualified for use in clinical applications. Since our previous chronic GVHD Biomarkers Working Group report in 2005, an increasing number of chronic GVHD candidate biomarkers are available for further investigation. This paper provides a 4-part framework for biomarker investigations: identification, verification, qualification, and application with terminology based on Food and Drug Administration and European Medicines Agency guidelines. (C) 2015 American Society for Blood and Marrow Transplantation.