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      International Journal of COPD (submit here)

      This international, peer-reviewed Open Access journal by Dove Medical Press focuses on pathophysiological processes underlying Chronic Obstructive Pulmonary Disease (COPD) interventions, patient focused education, and self-management protocols. Sign up for email alerts here.

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      A review of the most common patient-reported outcomes in COPD – revisiting current knowledge and estimating future challenges

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          Abstract

          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.

          Most cited references55

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          Measurement of health status. Ascertaining the minimal clinically important difference.

          In recent years quality of life instruments have been featured as primary outcomes in many randomized trials. One of the challenges facing the investigator using such measures is determining the significance of any differences observed, and communicating that significance to clinicians who will be applying the trial results. We have developed an approach to elucidating the significance of changes in score in quality of life instruments by comparing them to global ratings of change. Using this approach we have established a plausible range within which the minimal clinically important difference (MCID) falls. In three studies in which instruments measuring dyspnea, fatigue, and emotional function in patients with chronic heart and lung disease were applied the MCID was represented by mean change in score of approximately 0.5 per item, when responses were presented on a seven point Likert scale. Furthermore, we have established ranges for changes in questionnaire scores that correspond to moderate and large changes in the domains of interest. This information will be useful in interpreting questionnaire scores, both in individuals and in groups of patients participating in controlled trials, and in the planning of new trials.
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            The measurement of dyspnea. Contents, interobserver agreement, and physiologic correlates of two new clinical indexes.

            To improve the clinical measurement of dyspnea, we developed a baseline dyspnea index that rated the severity of dyspnea at a single state and a transition dyspnea index that denoted changes from that baseline. The scores in both indexes depend on ratings for three different categories: functional impairment; magnitude of task, and magnitude of effort. At the baseline state, dyspnea was rated in five grades from 0 (severe) to 4 (unimpaired) for each category. The ratings for each of the three categories were added to form a baseline focal score (range, 0 to 12). At the transition period, changes in dyspnea were rated by seven grades, ranging from -3 (major deterioration), to +3 (major improvement). The ratings for each of the three categories were added to form a transition focal score (range, -9 to +9). In 38 patients tested with respiratory disease, interobserver agreement was highly satisfactory for both indexes. The baseline focal score had the highest correlation (r = 0.60; P less than 0.001) with the 12-minute walking distance (12 MW), while significant, but lower, correlations existed for lung function. For the transition focal score, there was a significant correlation only with the 12 MW (r = 0.33; p = 0.04). These results indicate that dyspnea can receive a direct clinical rating that provides important information not disclosed by customary physiologic tests.
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              The COPD assessment test (CAT): response to pulmonary rehabilitation. A multicentre, prospective study.

              The COPD (chronic obstructive pulmonary disease) assessment test (CAT) is a recently introduced, simple to use patient-completed quality of life instrument that contains eight questions covering the impact of symptoms in COPD. It is not known how the CAT score performs in the context of clinical pulmonary rehabilitation (PR) programmes or what the minimum clinically important difference is. The introduction of the CAT score as an outcome measure was prospectively studied by PR programmes across London. It was used alongside other measures including the St George's Respiratory Questionnaire, the Chronic Respiratory Disease Questionnaire, the Clinical COPD Questionnaire, the Hospital Anxiety and Depression score, the Medical Research Council (MRC) dyspnoea score and a range of different walking tests. Patients completed a 5-point anchor question used to assess overall response to PR from 'I feel much better' to 'I feel much worse'. Data were available for 261 patients with COPD participating in seven programmes: mean (SD) age 69.0 (9.0) years, forced expiratory volume in 1 s (FEV(1)) 51.1 (18.7) % predicted, MRC score 3.2 (1.0). Mean change in CAT score after PR was 2.9 (5.6) points, improving by 3.8 (6.1) points in those scoring 'much better' (n=162), and by 1.3(4.5) in those who felt 'a little better' (n=88) (p=0.002). Only eight individuals reported no difference after PR and three reported feeling 'a little worse', so comparison with these smaller groups was not possible. The CAT score is simple to implement as an outcome measure, it improves in response to PR and can distinguish categories of response.
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                Author and article information

                Journal
                Int J Chron Obstruct Pulmon Dis
                Int J Chron Obstruct Pulmon Dis
                International Journal of COPD
                International Journal of Chronic Obstructive Pulmonary Disease
                Dove Medical Press
                1176-9106
                1178-2005
                2015
                07 April 2015
                : 10
                : 725-738
                Affiliations
                [1 ]University of Rome ‘Tor Vergata’, Department of Medicine of Systems, Unit of Respiratory Clinical Pharmacology, Rome, Italy
                [2 ]Section of Pulmonary, Critical Care and Sleep Medicine, Baylor College of Medicine, Houston, TX, USA
                [3 ]University of Edinburgh/MRC Centre for Inflammation Research, The Queen’s Medical Research Institute, Edinburgh, United Kingdom
                [4 ]Patient Reported Outcome Center of Excellence, Global Market Access, Primary Care Business Unit, Pfizer, Tadworth, United Kingdom
                Author notes
                Correspondence: Mario Cazzola, University of Rome ‘Tor Vergata’, Department of Medicine of Systems, Unit of Respiratory Clinical Pharmacology, Via Montpellier 1, Rome 00133, Italy, Tel +39 06 2090 0631, Email mario.cazzola@ 123456uniroma2.it
                Article
                copd-10-725
                10.2147/COPD.S77368
                4396518
                25897216
                cc6ce199-f84d-40f7-beb8-781cf1852bc7
                © 2015 Cazzola et al. This work is published by Dove Medical Press Limited, and licensed under Creative Commons Attribution – Non Commercial (unported, v3.0) License

                The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed.

                History
                Categories
                Review

                Respiratory medicine
                copd,patient reported outcome,health related quality of life,quality of life,psychometric properties

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