Atar Dan, Ong Seleen, Lansberg Peter J
1Department of Cardiology, Division of Medicine B, Oslo University Hospital, Oslo, Norway; 2Pfizer Ltd, Walton Oaks, Surrey, United Kingdom; and 3Department of Vascular Medicine, Durrer Center for Cardiogenetic Research, Academic Medical Center, Amsterdam, The Netherlands.
Am J Ther. 2015 Sep-Oct;22(5):e141-50. doi: 10.1097/MJT.0b013e318245ce94.
It is widely accepted that randomized controlled trials (RCTs) are the gold standard for demonstrating the efficacy of a given therapy (results under ideal conditions). Observational studies, on the other hand, can complement this by demonstrating effectiveness (results under real-world conditions). To examine the role that observational studies can play in complementing data from RCTs, we reviewed published studies for statins, a class of drugs that have been widely used to reduce the risk of cardiovascular (CV) events by lowering low-density lipoprotein cholesterol levels. RCTs have consistently demonstrated the benefits of statin treatment in terms of CV risk reduction and have demonstrated that more intensive statin therapy has incremental benefits over less intensive treatment. Observational studies of statin use in 'real-world' populations have served to augment the evidence base generated from statin RCTs in preselected populations of patients who are often at high CV risk and have led to similar safety and efficacy findings. They have also raised questions about factors affecting medication adherence, under-treatment, switching between statins, and failure to reach low-density lipoprotein cholesterol target levels, questions for which the answers could lead to improved patient care.
人们普遍认为，随机对照试验（RCT）是证明特定疗法疗效（理想条件下的结果）的金标准。另一方面，观察性研究可以通过证明有效性（现实世界条件下的结果）来补充这一点。为了研究观察性研究在补充随机对照试验数据方面可以发挥的作用，我们回顾了已发表的关于他汀类药物的研究，他汀类药物是一类已被广泛用于通过降低低密度脂蛋白胆固醇水平来降低心血管（CV）事件风险的药物。随机对照试验一直证明他汀类药物治疗在降低心血管风险方面的益处，并表明更强化的他汀类药物治疗比不太强化的治疗有额外的益处。在“现实世界”人群中对他汀类药物使用的观察性研究有助于扩大在通常具有高心血管风险的预先选定患者人群中他汀类药物随机对照试验产生的证据基础，并得出了类似的安全性和有效性结果。它们还引发了关于影响药物依从性、治疗不足、他汀类药物之间的转换以及未能达到低密度脂蛋白胆固醇目标水平等因素的问题，这些问题的答案可能会改善患者护理。
