Vestbo Jørgen, Janson Christer, Nuevo Javier, Price David
Division of Infection, Immunity and Respiratory Medicine, School of Biological Sciences, University of Manchester, Manchester, UK.
Dept of Medical Sciences: Respiratory, Allergy and Sleep Research, Uppsala University, Uppsala, Sweden.
ERJ Open Res. 2020 Oct 13;6(4). doi: 10.1183/23120541.00044-2020. eCollection 2020 Oct.
Randomised controlled trials (RCTs) are the gold standard for evaluating treatment efficacy in patients with obstructive lung disease. However, due to strict inclusion criteria and the conditions required for ascertaining statistical significance, the patients included typically represent as little as 5% of the general obstructive lung disease population. Thus, studies in broader patient populations are becoming increasingly important. These can be randomised effectiveness trials or observational studies providing data on real-world treatment effectiveness and safety data that complement efficacy RCTs. In this review we describe the features associated with the diagnosis of asthma and chronic obstructive pulmonary disease (COPD) in the real-world clinical practice setting. We also discuss how RCTs and observational studies have reported opposing outcomes with several treatments and inhaler devices due to differences in study design and the variations in patients recruited by different study types. Whilst observational studies are not without weaknesses, we outline recently developed tools for defining markers of quality of observational studies. We also examine how observational studies are capable of providing valuable insights into disease mechanisms and management and how they are a vital component of research into obstructive lung disease. As we move into an era of personalised medicine, recent observational studies, such as the NOVEL observational longiTudinal studY (NOVELTY), have the capacity to provide a greater understanding of the value of a personalised healthcare approach in patients in clinical practice by focussing on standardised outcome measures of patient-reported outcomes, physician assessments, airway physiology, and blood and airway biomarkers across both primary and specialist care.
随机对照试验（RCT）是评估阻塞性肺病患者治疗效果的金标准。然而，由于严格的纳入标准和确定统计学显著性所需的条件，纳入的患者通常仅占阻塞性肺病总体人群的5%。因此，针对更广泛患者群体的研究变得越来越重要。这些研究可以是随机有效性试验或观察性研究，提供关于实际治疗效果和安全性的数据，以补充疗效RCT。在本综述中，我们描述了在实际临床实践环境中与哮喘和慢性阻塞性肺疾病（COPD）诊断相关的特征。我们还讨论了由于研究设计的差异以及不同研究类型招募的患者的差异，RCT和观察性研究如何报告了几种治疗方法和吸入装置的相反结果。虽然观察性研究并非没有弱点，但我们概述了最近开发的用于定义观察性研究质量标志物的工具。我们还研究了观察性研究如何能够提供对疾病机制和管理的宝贵见解，以及它们如何成为阻塞性肺病研究的重要组成部分。随着我们进入个性化医疗时代，最近的观察性研究，如新型观察性纵向研究（NOVELTY），有能力通过关注患者报告结果、医生评估、气道生理学以及初级和专科护理中的血液和气道生物标志物的标准化结果测量，更深入地了解个性化医疗方法在临床实践中的价值。
