Harvard Medical School, Brigham and Women's/Faulkner Hospitals, Boston, MA 02130, USA.
Postgrad Med. 2010 May;122(3):222-9. doi: 10.3810/pgm.2010.05.2161.
Contemporary clinical decision making is well supported by a wide variety of information sources, including clinical practice guidelines, position papers, and insights from randomized controlled trials (RCTs). Much of our fundamental understanding of cardiovascular risk factors is based on multiple observations from major epidemiologic studies, such as The Seven Country Studies and the US-based Framingham Heart Study. These studies provided the framework for the development of clinical practice guidelines, including the National Cholesterol Education Program Adult Treatment Panel series. The objective of this article is to highlight the value of observational studies as a complement to clinical trial data for clinical decision making in real-world practice. Although RCTs are still the benchmark for assessing clinical efficacy and safety of a specific therapeutic approach, they may be of limited utility to practitioners who must then adapt the lessons learned from the trial into the patient care environment. The use of well-structured observational studies can improve our understanding of the translation of clinical trials into clinical practice, as demonstrated here with the example of statins. Although such studies have their own limitations, improved techniques for design and analysis have reduced the impact of bias and confounders. The introduction of the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) guidelines has provided more uniformity for such studies. When used together with RCTs, observational studies can enhance our understanding of effectiveness and utility in real-world clinical practice. In the examples of statin observational studies, the results suggest that relative effectiveness of different statins and potential impact of switching statins should be carefully considered in treating individual patients by practicing physicians.
当代临床决策得到了各种信息来源的广泛支持，包括临床实践指南、立场文件以及随机对照试验（RCT）的见解。我们对心血管危险因素的许多基本认识都是基于多项主要流行病学研究的观察结果，如七国研究和美国弗雷明汉心脏研究。这些研究为临床实践指南的制定提供了框架，包括国家胆固醇教育计划成人治疗专家组系列。本文的目的是强调观察性研究作为 RCT 数据的补充，对真实世界实践中的临床决策的价值。虽然 RCT 仍然是评估特定治疗方法临床疗效和安全性的基准，但对于必须将从试验中获得的经验教训应用于患者护理环境的医生来说，它们的实用性可能有限。本文以他汀类药物为例，展示了使用结构良好的观察性研究可以提高我们对临床试验转化为临床实践的理解。尽管这些研究存在自身的局限性，但设计和分析技术的改进已经降低了偏倚和混杂因素的影响。强化观察性研究报告的流行病学（STROBE）指南的引入为这类研究提供了更多的一致性。当与 RCT 一起使用时，观察性研究可以增强我们对真实世界临床实践中有效性和实用性的理解。在他汀类药物观察性研究的例子中，结果表明，在治疗个体患者时，临床医生应仔细考虑不同他汀类药物的相对有效性以及转换他汀类药物的潜在影响。
