A new and rapid scoring system to assess the scientific evidence from clinical trials.
A new and rapid scoring system to assess the scientific evidence from clinical trials.
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DOI：
10.1111/j.1540-8183.2006.00205.x
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发表时间：
2006-12-01
影响因子：
2.1
通讯作者：
Silber, Sigmund
中科院分区：
文献类型：
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作者：
Silber, Sigmund
指南基于随机对照试验（RCTs）、登记试验、简单登记、病例报告和工作组成员个人经验的现有数据的科学分析。此外，荟萃分析和亚组分析用于得出建议的优势。幸运的是，主要的心脏学会，即美国心脏病学会（ACC）、美国心脏协会（AHA）和欧洲心脏病学会（ESC），基本上使用相同的建议水平定义。然而，在不断扩大的心脏病学领域，压倒性的和越来越多的临床研究揭示了这些研究传统排名的局限性：应用ACC/AHA/ESC推荐水平标准定义，几乎每个PCI手术都很容易达到A级证据水平，即使有两个小的、不充分的研究和一个替代终点。虽然荟萃分析是创建主要诊断或治疗方式概述的重要工具，但它们必然存在严重的局限性。汇总几个不充分的小型试验可以产生统计上人为的“显著”结果。这一点尤其重要，因为只有两项包含几乎相同研究的荟萃分析才容易得出证据水平a。随机对照试验通常是根据功效计算设计和实施的，可以选择主要临床终点或替代终点。然而，替代终点并不一定与临床结果相关。医学史上充满了由替代终点的无效研究引起的错误。许多研究人员和公司试图梳理出特定亚群患者的治疗效果。这些亚群分析通常不够有力。ACC/AHA/ESC评分系统的另一个主要限制是，其定义既不包括研究的有效性，也不包括主要临床终点的选择。ACC/AHA/ESC分级系统的另一个限制是，两个“简单”的注册中心可能已经达到了证据b级。提出了一个新的评分系统，解决了大多数这些限制：一个主要临床终点得到3分，而以下所有终点都得到1分。双盲设计，主要终点评估间隔>或= 6个月，多中心（至少3个中心），独立数据和安全监测，主要终点达到>或= 80%，替代主要终点随访>或= 80%，临床主要终点随访>或= 95%。因此，最大可达到的分数是10分。该评分系统也可以应用于使用预定义对照组和功率计算的高质量注册控制试验。对于简单的登记研究和亚组分析，已经开发了一种改进的评分系统（最高可达到5分）。建议的新评分系统的优点是其透明度，可重复性和易用性，通过快速回答临床试验的关键质量问题。这里提出的新的评分系统应该有助于决定使用哪种治疗方法并激发讨论。
Guidelines are based on a scientific analysis from existing data of randomized controlled trials (RCTs), registry trials, simple registries, case reports, and the personal experience of the task force members. Furthermore, meta-analyses and subgroup analyses are used to derive the strengths of recommendations. Fortunately, the major cardiac societies, i.e., the American College of Cardiology (ACC), the American Heart Association (AHA), and the European Society of Cardiology (ESC), are essentially using the same definitions for the levels of recommendations. In the expanding field of cardiology, however, the overwhelming and increasing number of clinical studies reveals the limitations of the traditional ranking of these studies: Applying the standard definitions of the ACC/AHA/ESC criteria for the levels of recommendation, almost every PCI procedure would easily reach the level of evidence A, even with two small, underpowered studies and a surrogate endpoint. Although meta-analyses are important tools for creating an overview of major diagnostic or treatment modalities, they are bound to severe limitations. The compilation of several underpowered, small trials can generate a statistically artificial "significant" result. This is especially important because only two meta-analyses containing almost identical studies could easily yield an evidence level A. RCTs are usually designed and conducted according to a power calculation, for which a primary clinical or surrogate endpoint can be chosen. Surrogate endpoints, however, do not necessarily correlate with the clinical outcome. The history of medicine is full of errors introduced by underpowered studies with surrogate endpoints. Many investigators and companies attempt to tease out treatment effects in specific subpopulations of patients. These subgroup analyses are usually underpowered. Another major limitation of the ACC/AHA/ESC scoring system is that neither the power of a study nor the choice of a primary clinical endpoint is included in their definitions. Yet another limitation of the ACC/AHA/ESC grading system is that two "simple" registries may already lead to a level of evidence B. A new scoring system is presented addressing most of these limitations: a primary clinical endpoint receives three points, whereas all of the following receive one point: double-blind design, evaluation interval of primary endpoint > or = 6 months, multicenter (at least three centers), independent data and safety monitoring, power of > or = 80% for primary endpoint achieved, and follow-up > or = 80% for a surrogate primary endpoint or follow-up of > or = 95% for a clinical primary endpoint. Thus, the maximum achievable points is 10. This scoring system can also be applied for high-quality registry controlled trials using a predefined control group and power calculation. For simple registry studies and subgroup analyses, a modified scoring system has been developed (maximum achievable points is 5). The advantage of the suggested new scoring system is its transparency, reproducibility, and ease of use by quickly answering the key quality questions for clinical trials. The new scoring system suggested here should help make decisions regarding which treatment to use and stimulate discussions.