Dataset Information

Measuring respiratory symptoms in clinical trials of COPD: reliability and validity of a daily diary.

ABSTRACT:

Background

Although respiratory symptoms are characteristic features of COPD, there is no standardised method for quantifying their severity in stable disease.

Objective

To evaluate the EXACT-Respiratory Symptom (E-RS) measure, a daily diary comprising 11 of the 14 items in the Exacerbations of Chronic Pulmonary Disease Tool (EXACT).

Methods

Qualitative: patient focus group and interviews to address content validity. Quantitative: secondary data analyses to test reliability and validity.

Results

Qualitative: n=84; mean (SD) age 65 (10) years, FEV1 1.2(0.4) L; 44% male. Subject descriptions of their respiratory symptoms were consistent with E-RS content and structure. Quantitative: n=188; mean (SD) age 66 (10) years, FEV1 1.2(0.5) L; 50% male. Factor analysis (FA) showed 3 subscales: RS-Breathlessness, RS-Cough & Sputum, and RS-Chest Symptoms; second-order FA supported a general factor and total score. Reliability (total and subscales): 0.88, 0.86, 0.73, 0.81; 2-day test-retest ICC: 0.90, 0.86, 0.87, 0.82, respectively.

Validity

Total scores correlated significantly (p < 0.0001) with SGRQ Total (r=0.75), Symptoms (r=0.66), Activity (r=0.57), Impact (r=0.70) scores; subscale correlations were also significant (r=0.26, p?ConclusionsResults suggest the RS-Total is a reliable and valid instrument for evaluating respiratory symptom severity in stable COPD. Further study of sensitivity to change is warranted.

SUBMITTER: Leidy NK

PROVIDER: S-EPMC3995276 | biostudies-literature | 2014 May

REPOSITORIES: biostudies-literature

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Publications

Measuring respiratory symptoms in clinical trials of COPD: reliability and validity of a daily diary.

Leidy N K NK Sexton C C CC Jones P W PW Notte S M SM Monz B U BU Nelsen L L Goldman M M Murray L T LT Sethi S S

Thorax 20140304 5

<h4>Background</h4>Although respiratory symptoms are characteristic features of COPD, there is no standardised method for quantifying their severity in stable disease.<h4>Objective</h4>To evaluate the EXACT-Respiratory Symptom (E-RS) measure, a daily diary comprising 11 of the 14 items in the Exacerbations of Chronic Pulmonary Disease Tool (EXACT).<h4>Methods</h4>Qualitative: patient focus group and interviews to address content validity. Quantitative: secondary data analyses to test reliability ...[more]

PMID: 24595666

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Project description:Symptomatic relief is an important treatment goal for patients with COPD. To date, no diary for evaluating respiratory symptoms in clinical trials has been developed and scientifically-validated according to FDA and EMA guidelines. The EXACT - Respiratory Symptoms (E-RS) scale is a patient-reported outcome (PRO) measure designed to address this need. The E-RS utilizes 11 respiratory symptom items from the existing and validated 14-item EXACT, which measures symptoms of exacerbation. The E-RS total score quantifies respiratory symptom severity, and 3 domains assess breathlessness, cough and sputum, and chest symptoms.This study examined the performance of the E-RS in each of 3 controlled trials with common and unique validation variables: one 6-month (N = 235, US) and two 3-month (N = 749; N = 597; international). Subjects completed the E-RS as part of a daily eDiary. Tests of reliability, validity, and responsiveness were conducted in each dataset.In each study, RS-Total score was internally consistent (Cronbach ?) (0.88, 0.92, 0.92) and reproducible (intra-class correlation) in stable patients (2 days apart: 0.91; 7 days apart: 0.71, 0.74). RS-Total scores correlated significantly with the following criterion variables (Spearman's rho; p < 0.01, all comparisons listed here): FEV1% predicted (-0.19, -0.14, -0.15); St. George's Respiratory Questionnaire (SGRQ) (0.65, 0.52, 0.51); Breathlessness, Cough, and Sputum Scale (BCSS) (0.89, 0.89); modified Medical Research Council dyspnoea scale (mMRC) (0.40); rescue medication use (0.43, 0.42); Functional Performance Inventory Short-Form (FPI-SF) (0.43); 6-minute walk distance (6-MWT) (-0.30, -0.14) and incremental shuttle walk (ISWT) (-0.18) tests. Correlations between these variables and RS-Breathlessness, RS-Cough and Sputum, RS-Chest Symptoms scores supported subscale validity. RS-Total, RS-Breathlessness, and RS-Chest Symptoms differentiated mMRC levels of breathlessness severity (p < 0.0001). RS-Total and domain scores differentiated subjects with no rescue medication use and 3 or more puffs (p < 0.0001). Sensitivity to changes in health status (SGRQ), symptoms (BCSS), and exercise capacity (6MWT, ISWT) were also shown and responder definitions using criterion- and distribution-based methods are proposed.Results suggest the E-RS is a reliable, valid, and responsive measure of respiratory symptoms of COPD suitable for use in natural history studies and clinical trials.MPEX: NCT00739648 ; AZ1: NCT00949975 ; AZ 2: NCT01023516.

Project description:BACKGROUND:Reducing the severity of respiratory symptoms is a key goal in the treatment of chronic obstructive pulmonary disease (COPD). We evaluated the effect of aclidinium bromide 400 ?g twice daily (BID) on respiratory symptoms, assessed using the Evaluating Respiratory Symptoms in COPD (E-RS(™): COPD) scale (formerly EXACT-RS). METHODS:Data were pooled from the aclidinium 400 ?g BID and placebo arms of two 24-week, double-blind, randomized Phase III studies evaluating aclidinium monotherapy (ATTAIN) or combination therapy (AUGMENT COPD I) in patients with moderate to severe airflow obstruction. Patients were stratified by Global initiative for chronic Obstructive Lung Disease (GOLD) Groups A-D. Change from baseline in E-RS scores, proportion of responders (patients achieving pre-defined improvements in E-RS scores), and net benefit (patients who improved minus patients who worsened) were analyzed. RESULTS:Of 1210 patients, 1167 had data available for GOLD classification. Mean (standard deviation) age was 63.2 (8.6) years, 60.7 % were male, and mean post-bronchodilator forced expiratory volume in 1 s was 54.4 % predicted. Compared with placebo, aclidinium 400 ?g BID significantly improved RS-Total (2.38 units vs 0.79 units, p?<?0.001) and domain scores (all p?<?0.001) at Week 24, and doubled the likelihood of being an RS-Total score responder (p?<?0.05), irrespective of GOLD group. The net benefit for RS-Total (Overall: 56.9 % vs 19.4 %; A?+?C: 65.7 % vs 6.3 %; B?+?D: 56.0 % vs 20.8 %, for aclidinium 400 ?g BID and placebo respectively; all p?<?0.05) and domain scores (all p?<?0.05) was significantly greater with aclidinium compared with placebo, in both GOLD Groups A?+?C and B?+?D. CONCLUSIONS:Aclidinium 400 ?g BID significantly improved respiratory symptoms regardless of the patients' level of symptoms at baseline. Net treatment benefit was similar in patients with low or high levels of symptoms. TRIAL REGISTRATION:ATTAIN (ClinicalTrials.gov identifier: NCT01001494 ) and AUGMENT COPD I (ClinicalTrials.gov identifier: NCT01437397 ).

Project description:BackgroundDengue is the most prevalent arboviral infection causing an estimated 50-60 million cases of febrile illness globally per year, exacting considerable disease burden. Few instruments exist to assess the patient illness experience, with most based on healthcare provider assessment, lacking standardization in timepoints and symptom assessment. This study aimed to evaluate the content validity of the novel 'Dengue Virus Daily Diary (DENV-DD)', designed to measure symptom intensity and disease burden within outpatient infant to adult populations.MethodsThe Dengue Illness Index Report Card was used as a foundation to create the DENV-DD, consisting of patient- and observer-reported outcome (PRO/ObsRO) instruments. In two South American dengue-endemic communities, qualitative combined concept elicitation and cognitive debriefing interviews were conducted among individuals and caregivers of children with symptomatic laboratory-confirmed dengue. Interviews were conducted across two rounds allowing DENV-DD modifications. A small-scale quantitative assessment of the DENV-DD was also conducted with data from an independent Dengue Human Infection Model (DHIM) to generate early evidence of feasibility of DENV-DD completion, instrument performance and insight into the sign/symptom trajectory over the course of illness.ResultsForty-eight participants were interviewed (20 adults, 20 older children/adolescents with their caregivers, 8 caregivers of younger children). A wide spectrum of signs/symptoms lasting 3-15 days were reported with fever, headache, body ache/pain, loss of appetite, and body weakness each reported by > 70% participants. DENV-DD instructions, items and response scales were understood, and items were considered relevant across ages. DHIM data supported feasibility of DENV-DD completion.ConclusionsFindings demonstrate content validity of the DENV-DD (PRO/ObsRO instruments) in dengue-endemic populations. Psychometric and cultural validity studies are ongoing to support use of the DENV-DD in clinical studies.

Project description:Background:Asthma-chronic obstructive pulmonary disease overlap (ACO) differs from asthma and chronic obstructive pulmonary disease (COPD) in demographics, phenotypic characteristics and outcomes, yet the patient experience of ACO is poorly characterized. We aimed to understand and compare the patient experience of symptoms and domains of impact in ACO relative to COPD, and assess the content validity of existing patient-reported outcome (PRO) instruments in ACO. Methods:This US qualitative, interview study included patients who met American Thoracic Society/European Respiratory Society spirometric criteria for COPD. Additionally, patients with ACO demonstrated reversibility (forced expiratory volume in 1 s [FEV1] increase ≥ 12% and ≥ 200 mL) to albuterol/salbutamol and an FEV1/forced vital capacity ratio < 0.7. Patients took part in concept elicitation (CE) to explore symptoms and impacts of obstructive lung disease. The Exacerbations of Chronic Pulmonary Disease Tool (EXACT), St George's Respiratory Questionnaire (SGRQ) and a daily wheeze assessment were cognitively debriefed to assess relevance and comprehensiveness. Interviews were analyzed using Atlas.Ti. Concept saturation was evaluated at the symptom level. Results:Twenty patients with ACO and 10 patients with COPD were recruited. Patients from both groups indicated that shortness of breath was their most frequent and bothersome symptom. The most frequently reported symptoms in both groups were shortness of breath, cough, wheezing, difficulty breathing, mucus/phlegm, chest tightness, and tiredness, weakness or fatigue. The onset, severity, frequency and duration of symptoms were consistently described across both groups, although a higher proportion of patients with ACO experienced exacerbations versus those with COPD. Impacts on daily living, physical impacts and emotional impacts were commonly described (ACO: 90-100%, COPD: 80-100%). Concept saturation was achieved in both groups. Overall, the EXACT, SGRQ and daily wheeze assessment were well understood and relevant to most patients with ACO or COPD (50-100%) and patients generally found the assessments easy to complete. The PRO instruments adequately captured symptoms described during CE, demonstrating high content validity in ACO and COPD. Conclusions:Patients with ACO and COPD experienced similar symptoms and impacts. The EXACT, SGRQ and assessment of wheeze were well understood and captured concepts relevant to patients with ACO.

Dataset Information

Measuring respiratory symptoms in clinical trials of COPD: reliability and validity of a daily diary.

Background

Objective

Methods

Results

Validity

Publications

Measuring respiratory symptoms in clinical trials of COPD: reliability and validity of a daily diary.

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