Cazzola Mario, Hanania Nicola Alexander, MacNee William, Rüdell Katja, Hackford Claire, Tamimi Nihad
University of Rome 'Tor Vergata', Department of Medicine of Systems, Unit of Respiratory Clinical Pharmacology, Rome, Italy.
Section of Pulmonary, Critical Care and Sleep Medicine, Baylor College of Medicine, Houston, TX, USA.
Int J Chron Obstruct Pulmon Dis. 2015 Apr 7;10:725-38. doi: 10.2147/COPD.S77368. eCollection 2015.
Patient-reported outcome (PRO) measures that quantify disease impact have become important measures of outcome in COPD research and treatment. The objective of this literature review was to comprehensively evaluate psychometric properties of available PRO instruments and the ability of each of them to characterize pharmaceutical treatment effects from published clinical trial evidence. Identified in this study were several PRO measures, both those that have been used extensively in COPD clinical trials (St George's Respiratory Questionnaire and Chronic Respiratory Questionnaire) and new instruments whose full value is still to be determined. This suggests a great need for more information about the patient experience of treatment benefit, but this also may pose challenges to researchers, clinicians, and other important stakeholders (eg, regulatory agencies, pharmaceutical companies) who develop new treatment entities and payers (including but not limited to health technology assessment agencies such as the National Institute for Health and Care Excellence and the Canadian Agency for Drugs and Technologies in Health). The purpose of this review is to enable researchers and clinicians to gain a broad overview of PRO measures in COPD by summarizing the value and purpose of these measures and by providing sufficient detail for interested audiences to determine which instrument may be the most suitable for evaluating a particular research purpose.
量化疾病影响的患者报告结局(PRO)指标已成为慢性阻塞性肺疾病(COPD)研究和治疗中重要的结局指标。本综述的目的是全面评估现有PRO工具的心理测量特性,以及它们根据已发表的临床试验证据表征药物治疗效果的能力。本研究确定了几种PRO指标,既有在COPD临床试验中广泛使用的指标(圣乔治呼吸问卷和慢性呼吸问卷),也有其全部价值仍有待确定的新工具。这表明非常需要更多关于患者治疗获益体验的信息,但这也可能给开发新治疗实体的研究人员、临床医生和其他重要利益相关者(如监管机构、制药公司)以及支付方(包括但不限于像英国国家卫生与临床优化研究所和加拿大卫生技术评估局这样的卫生技术评估机构)带来挑战。本综述的目的是通过总结这些指标的价值和用途,并为感兴趣的读者提供足够的细节,以便他们确定哪种工具可能最适合评估特定的研究目的,从而使研究人员和临床医生能够全面了解COPD中的PRO指标。