School of Public Health, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong Special Administrative Region, China.
Am J Epidemiol. 2013 May 15;177(10):1128-33. doi: 10.1093/aje/kws344. Epub 2013 Apr 12.
Prevention and treatment of common noncommunicable chronic diseases have been revolutionized by the development of therapies. Recently, several randomized controlled trials (RCTs) designed to assess the efficacy of new therapies targeted at well-established risk factors for noncommunicable chronic diseases have reported lower benefits than expected. Subsequent observational analysis of the same trial data has not clarified these unexpected findings. Mendelian randomization (MR) provides an approach for estimating causal effects from observational or trial data and thus provides information complementary to that from an RCT. An RCT assesses the efficacy of a therapy but does not usually confirm the underlying mechanistic pathway. In contrast, an MR study does not assess the efficacy of a therapy but rather assesses causal effects on an underlying mechanistic pathway. We suggest that incorporating an MR study into an RCT at the design stage would improve etiologic understanding of current therapies and enhance the search for therapies for the significant amount of noncommunicable chronic diseases that resists current treatments.
治疗方法的发展彻底改变了常见非传染性慢性病的预防和治疗。最近，几项旨在评估针对非传染性慢性病既定风险因素的新疗法疗效的随机对照试验（RCT）报告的益处低于预期。对同一试验数据的后续观察性分析并没有澄清这些意外发现。孟德尔随机化（MR）提供了一种从观察性或试验数据中估计因果效应的方法，因此提供了与 RCT 数据互补的信息。RCT 评估一种疗法的疗效，但通常不能证实潜在的机制途径。相比之下，MR 研究不评估一种疗法的疗效，而是评估对潜在机制途径的因果效应。我们建议在设计阶段将 MR 研究纳入 RCT，以提高对当前疗法的病因学理解，并增强对大量抵抗当前治疗方法的非传染性慢性病的治疗方法的研究。
