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Shortness of Breath with Daily Activities questionnaire: validation and responder thresholds in patients with chronic obstructive pulmonary disease.

ABSTRACT: To test the reliability, validity and responsiveness of the 13-item Shortness of Breath with Daily Activities (SOBDA) questionnaire, and determine the threshold for response and minimal important difference (MID).6 week, randomised, double-blind, placebo-controlled study.40 centres in the USA between 29 October 2009 and 1 July 2010.547 patients with chronic obstructive pulmonary disease (COPD) were enrolled and 418 entered the 2-week run-in period. Data from the run-in period were collected to test internal consistency, test-retest reliability, convergent validity and known-groups validity of the SOBDA. Three hundred and sixty six patients were randomised 2:2:1 to fluticasone propionate/salmeterol 250/50 µg, salmeterol 50 µg or placebo, twice daily. Results from the SOBDA questionnaire, Patient Global Assessment of Change Question, modified Medical Research Council Dyspnoea Scale (mMRC), Clinician Global Impression of Dysponea Severity (CGI-S), Clinician Global Impression of Change Question and Chronic Respiratory Disease Questionnaire self-administered standardised version (CRQ-SAS) were evaluated; spirometry and safety parameters were measured. Study endpoints were selected to investigate the cross-sectional and longitudinal validity of the SOBDA questionnaire in relation to the clinical criteria.Internal consistency of the SOBDA questionnaire (Cronbach ?) was 0.89. Test-retest reliability (intraclass correlation) was 0.94. The SOBDA weekly scores correlated with the patient-reported and clinician-reported mMRC, CGI-S and CRQ-SAS dyspnoea domain scores (0.29, 0.24, 0.24 and -0.68, respectively). The SOBDA weekly scores differentiated between the responders and the non-responders as rated by the patients and the clinicians. Anchor-based and supportive distribution-based analyses produced a range of the potential values for the threshold for the responders and MID.The 13-item SOBDA questionnaire is reliable, valid and responsive to change in patients with COPD. On using anchor-based methods, the proposed responder threshold shows a -0.1 to -0.2 score change. A specific threshold value will be identified as more data are generated from future clinical trials.NCT00984659; GlaxoSmithKline study number: ASQ112989.

SUBMITTER: Watkins ML

PROVIDER: S-EPMC3808824 | biostudies-literature | 2013 Oct

REPOSITORIES: biostudies-literature

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Shortness of Breath with Daily Activities questionnaire: validation and responder thresholds in patients with chronic obstructive pulmonary disease.

Watkins Michael L ML Wilcox Teresa K TK Tabberer Maggie M Brooks Jean M JM Donohue James F JF Anzueto Antonio A Chen Wen-Hung WH Crim Courtney C

BMJ open 20131022 10

<h4>Objectives</h4>To test the reliability, validity and responsiveness of the 13-item Shortness of Breath with Daily Activities (SOBDA) questionnaire, and determine the threshold for response and minimal important difference (MID).<h4>Design</h4>6 week, randomised, double-blind, placebo-controlled study.<h4>Setting</h4>40 centres in the USA between 29 October 2009 and 1 July 2010.<h4>Primary and secondary outcome measures</h4>547 patients with chronic obstructive pulmonary disease (COPD) were e ...[more]

PMID: 24154513

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Project description:BackgroundChronic obstructive pulmonary disease (COPD) is characterized by non-reversible airflow limitation. A common symptom of COPD is dyspnea or shortness of breath. Dyspnea may vary daily, with a large impact on patients' lives. Previous clinical trials used patient-reported outcome (PRO) measures that quantified dyspnea at discrete intervals and hence did not reflect this variability. Recently the Shortness of Breath with Daily Activities (SOBDA) questionnaire was developed as a PRO measure of dyspnea utilizing a daily diary. This confirmatory post hoc meta-analysis of SOBDA data from a large clinical study program further supports the questionnaire and clarifies the minimum threshold of SOBDA response.MethodsData from four clinical trials (DB2113361, NCT01313637; DB2113373, NCT01313650; DB2113360, NCT01316900; DB2113374, NCT01316913) were analyzed. These 24-week trials were randomized, blinded studies investigating the efficacy and safety of several COPD treatments. These post hoc analyses focused on the SOBDA questionnaire properties. This electronic-diary consists of 13 items completed daily, in which patients rate their breathlessness level during common daily activities. Resultant SOBDA scores were compared with related, commonly used assessments: modified Medical Research Council Research Dyspnea Scale (mMRC), Baseline Dyspnea Index (BDI), Transition Dyspnea Index (TDI), St George's Respiratory Questionnaire (SGRQ), COPD Assessment Test (CAT), and trough forced expiratory volume in 1 s (FEV1). The consistency, reliability, validity (convergent, known groups), and responsiveness of the SOBDA questionnaire was assessed.ResultsIn total, 4967 patients with COPD provided data for these analyses. The SOBDA questionnaire had high internal consistency (Cronbach's alpha = 0.936), high test-retest reliability (Pearson's correlation coefficient = 0.86) and convergent validity with related measures (SGRQ total score, Pearson's correlation coefficient = 0.59; CAT, Spearman rank-order correlation coefficient = 0.50). SOBDA scores were statistically significantly lower in responders (as defined by TDI, SGRQ, CAT, and trough FEV1 levels) versus non-responders (p < 0.001 for all assessments and all time points). Using an anchor-based method, the threshold of a minimum response was calculated as a SOBDA score change of -0.2 (SOBDA score range = 1-4).ConclusionsThe reliability, validity, and responsiveness of the SOBDA questionnaire as a PRO measure to quantify dyspnea was supported in a large clinical trial population of patients with moderate-very severe COPD.

Project description:Abstract Background Patient perspectives on their disease are undoubtedly important in clinical trials and in practice. To date, no patient-reported outcome (PRO) questionnaire assessing symptoms of Clostridium difficile infection (CDI) has been developed following FDA guidance. The CDI-DaySyms™ is a new comprehensive measure of a broad range of local and systemic CDI symptoms (not only diarrhea) that patients report as meaningful. Objectives were to: finalize the CDI-DaySyms™ items (questions); assign them to domains (concepts); evaluate the questionnaire’s measurement properties; and define responder thresholds. Methods Blinded data from a sub-study of two Phase III trials (NCT01987895 and NCT01983683) in CDI patients were analyzed following FDA guidance for validating PRO questionnaires. Patients completed the CDI-DaySyms™ daily from Day 1 until end of treatment. Items were selected for inclusion in the final questionnaire based on a range of validation analyses, input from expert clinicians, and findings from prior qualitative patient research. Responder-threshold analyses used Day-3 data to align with the rapid symptom improvement generally seen in response to CDI therapy. Results Data were analyzed for 168 CDI patients (median age 60 years; 67.9% female; 81.5% mild/moderate, 11.3% severe, 7.1% unknown disease severity; 80.4% first CDI occurrence, 19.6% first recurrence). Three of the 13 items in the draft CDI-DaySyms™ were deleted; the remaining 10 were statistically assigned to three domains measuring different symptom concepts (Figure). Individual items in each domain correlated strongly with one another and their domain. Domain scores demonstrated acceptable consistency over time in stable patients, were sensitive to change, and correlated in expected directions with scores of other relevant symptom and disease-severity measures. Responder thresholds were defined as score changes of -1.00, -0.80, and -0.70 for Diarrhea, Abdominal, and Systemic/Other Symptoms domain scores, respectively. Conclusion The CDI-DaySyms™ is a valid measure of diarrhea and other CDI symptoms useful in assessing response to therapy. It has good measurement properties, and with only 10 items can be easily administered in clinical trials and in practice. Disclosures G. H. Talbot, Actelion: Board Member, Consultant and Scientific Advisor, Consulting fee; L. Kleinman, Actelion: Consultant, Research support; ? E. W. Davies, Actelion: Employee, Salary; E. Hunsche, Actelion: Employee and Shareholder, Salary; L. Roberts, Actelion: Consultant, Research support; C. E. Nord, Actelion: Scientific Advisor, Consulting fee

Project description:BackgroundIdiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease that often causes disabling dyspnea. In IPF and other lung diseases, patient-reported outcomes (PROs)-questionnaires designed to gather information from the patient's perspective-can determine whether therapies affect dyspnea or other outcomes meaningful to patients. Before a PRO can be used confidently as an outcome measure in a longitudinal trial, studies must demonstrate the PRO's ability to capture change over time in the target population. Our goal in this study was to examine whether the UCSD Shortness of Breath Questionnaire does so in patients with IPF.MethodsWe used data from the Sildenafil Trial of Exercise Performance in Idiopathic Pulmonary Fibrosis (STEP-IPF) to perform analyses that examined associations between UCSD scores and five external measures (anchors) at baseline and over time. Anchors included the Activity domain from St. George's Respiratory Questionnaire (SGRQ-A), the Physical Functioning domain from the SF-36 (SF36-PF), forced vital capacity (FVC), diffusing capacity of the lung for carbon monoxide (DLCO), and distance walked during a timed walk test (6MWD). Linear regression models were used to examine relationships between UCSD scores and anchors over time.ResultsAt baseline, UCSD scores were weakly correlated with percent predicted FVC (-0.21, p = 0.005) and percent predicted DLCO (-0.20, p = 0.008), moderately correlated with 6MWD (-0.39, p < 0.0001) and strongly correlated with SGRQ-A (0.79, p < 0.0001) and SF36-PF (-0.72, p < 0.0001). Change over time in UCSD scores was associated with change in FVC (estimate = 2.54, standard error [SE] = 1.23, p = 0.04), SGRQ-A (estimate = 7.94, SE = 1.11, p < 0.0001), SF36-PF (estimate = 6.00, SE = 1.13, p < 0.0001), and 6MWD (estimate = 4.23, SE = 1.18, p = 0.0004) but not DLCO (estimate = 0.33, SE = 1.33, p = 0.80).ConclusionsThese results support the validity of the UCSD to assess change in dyspnea over time in patients with IPF.

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Shortness of Breath with Daily Activities questionnaire: validation and responder thresholds in patients with chronic obstructive pulmonary disease.

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Shortness of Breath with Daily Activities questionnaire: validation and responder thresholds in patients with chronic obstructive pulmonary disease.

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