Clinical trial design and evidence-based outcomes in the study of liver diseases
Clinical trial design and evidence-based outcomes in the study of liver diseases
复制标题
DOI：
10.1016/j.jhep.2009.01.005
复制
发表时间：
2009-04-01
影响因子：
25.7
通讯作者：
Kramer, Barnett S.
中科院分区：
文献类型：
--
作者：
Croswell, Jennifer M.;Kramer, Barnett S.
目前的医学培训通常不包括试验设计的正式研究，迫使临床医生独立获取这些知识。本文回顾了临床试验设计的基本要素。介绍了临床证据的总体层次，并讨论了主要研究设计类型的相对优势和局限性。针对试验结果提出了试验设计中证据层次的推论：“终点金字塔”。该金字塔代表了一系列结果，从有形的健康事件到对个人没有直接身体影响的中间标记。探讨了使用最终健康结果替代终点所固有的潜在优势和困难。提倡利用“硬”临床终点的随机对照试验作为直接评估治疗益处和危害的最有效和可靠的方法；然而，通过肝细胞癌治疗的案例研究，我们强调了即使是最高水平的证据也可能变得复杂的挑战。所有试验都有一个“信噪比”——本次综述强调需要仔细、深思熟虑地考虑每项研究的潜在局限性，并提供基本工具来帮助从业者识别临床试验的常见陷阱。由 Elsevier B.V. 代表欧洲肝脏研究协会出版。
Current medical training often does not include the formal study of trial design, forcing clinicians to acquire this knowledge independently. This article reviews the foundational elements of clinical trial design. An overarching hierarchy of clinical evidence is introduced, and the relative strengths and limitations of the major types of study designs are discussed. A corollary to the hierarchy of evidence in trial designs is proposed for trial outcomes: the "pyramid of endpoints." This pyramid represents a spectrum of outcomes from tangible health events to intermediate markers with no direct physical impact on an individual. The potential advantages and difficulties inherent in the use of surrogate endpoints for final health outcomes are explored. Randomized controlled trials utilizing "hard" clinical endpoints are advocated as the most efficient and reliable way to directly assess the benefits and harms of a therapy; however, using a case study of treatments for hepatocellular carcinoma, we highlight the challenges that can complicate even the highest levels of evidence. All trials have a "signal-to-noise" ratio - this review emphasizes the need for careful and deliberate consideration of the potential limitations of every study, and provides basic tools to assist the practitioner in identifying common pitfalls of clinical trials. Published by Elsevier B.V. on behalf of the European Association for the Study of the Liver.