Psaty B M, Siscovick D S, Weiss N S, Koepsell T D, Rosendaal F R, Lin D, Heckbert S R, Wagner E H, Furberg C D
Department of Medicine, University of Washington, Seattle, USA.
Am J Hypertens. 1996 Feb;9(2):178-83. doi: 10.1016/0895-7061(96)00015-5.
Outcomes research seeks to identify effective evidence-based methods of providing the best medical care. While randomized clinical trials (RCT) usually provide the clearest answers, they are often not done or not practicable. More than a decade after the introduction of calcium channel blockers and angiotensin converting enzyme (ACE) inhibitors, clinical trial data about their effect on major disease endpoints in patients with hypertension are still not available. The primary alternatives are the use of randomized trials that include surrogate endpoints, such as level of blood pressure or extent of carotid atherosclerosis, and the use of observational studies that include major disease endpoints. Both approaches, their strengths and limitations, are discussed in detail. The possibility of residual confounding limits the strength of inferences that can be drawn from observational studies. Similarly, the possibility of important drug effects, other than those involving the surrogate endpoint, limits the inferences that can be drawn from randomized trials that rely solely on surrogate outcomes as guides to therapy. In the absence of evidence from large clinical trials that include major disease endpoints, treatment decisions and guidelines need to synthesize the best available information from a variety of sources. Consistency of findings across various study designs, outcomes, and populations is critical to the practice of evidence-based medicine and the effort to maximize the health benefits of antihypertensive therapies.
结果研究旨在确定提供最佳医疗护理的有效循证方法。虽然随机临床试验（RCT）通常能给出最清晰的答案，但它们往往未开展或不具可行性。在钙通道阻滞剂和血管紧张素转换酶（ACE）抑制剂问世十多年后，关于它们对高血压患者主要疾病终点影响的临床试验数据仍未可得。主要的替代方法是使用包含替代终点（如血压水平或颈动脉粥样硬化程度）的随机试验，以及使用包含主要疾病终点的观察性研究。将详细讨论这两种方法及其优缺点。残余混杂的可能性限制了能从观察性研究得出的推断力度。同样，除涉及替代终点之外的重要药物效应的可能性，也限制了仅依赖替代结果作为治疗指导的随机试验所能得出的推断。在缺乏包含主要疾病终点的大型临床试验证据的情况下，治疗决策和指南需要综合来自各种来源的最佳可用信息。不同研究设计、结果和人群中的研究结果一致性，对于循证医学实践以及使抗高血压治疗的健康益处最大化的努力而言至关重要。
