Pamela Berry

Development and first validation of the COPD Assessment Test.

There is need for a validated short, simple instrument to quantify chronic obstructive pulmonary disease (COPD) impact in routine practice to aid health status assessment and communication between patient and physician. Current health-related quality of life questionnaires provide valid assessment of COPD, but are complex, which limits routine use. The aim of the present study was to develop a short validated patient-completed questionnaire, the COPD Assessment Test (CAT), assessing the impact of COPD on health status. 21 candidate items identified through qualitative research with COPD patients were used in three prospective international studies (Europe and the USA, n = 1,503). Psychometric and Rasch analyses identified eight items fitting a unidimensional model to form the CAT. Items were tested for differential functioning between countries. Internal consistency was excellent: Cronbachs α = 0.88. Test re-test in stable patients (n = 53) was very good (intra-class correlation coefficient 0.8). In the sample from the USA, the correlation with the COPD-specific version of the St George’s Respiratory Questionnaire was r = 0.80. The difference between stable (n = 229) and exacerbation patients (n = 67) was five units of the 40-point scale (12%; p<0.0001). The CAT is a short, simple questionnaire for assessing and monitoring COPD. It has good measurement properties, is sensitive to differences in state and should provide a valid, reliable and standardised measure of COPD health status with worldwide relevance.

Tests of the Responsiveness of the COPD Assessment Test Following Acute Exacerbation and Pulmonary Rehabilitation

BACKGROUND The COPD Assessment Test (CAT) is an eight-item questionnaire suitable for routine clinical use that shows reliability and validity in stable and exacerbating COPD. METHODS Study 1 assessed CAT responsiveness to changes in health status in 67 patients during an exacerbation (days 1-14). Study 2 assessed CAT responsiveness in 64 patients undergoing pulmonary rehabilitation (days 1-42). Correlations between CAT and other outcome measures were examined. RESULTS In study 1, mean 14-day improvement in CAT score was -1.4 ± 5.3 units (P = .03). In patients judged to be responders (clinician defined) change in score was -2.6 ± 4.4; in nonresponders it was -0.2 ± 5.9. In study 2, the mean improvement in CAT score was -2.2 ± 5.3 (P = .002); the effect size for the change was -0.33. Effect size for changes in the Chronic Respiratory Questionnaire-Self Administered Standardized (CRQ-SAS) form domain scores ranged from -0.02 to 0.34. Change in 6-min walk distance (6MWD) was 41 ± 55 m. CAT and CRQ-SAS domain scores correlated at baseline (r = -0.54 to -0.69, P < .0001) and in terms of change following pulmonary rehabilitation (r = -0.39 to -0.63, P < .01). Correlations were less strong between change in the CAT and St. George Respiratory Questionnaire for COPD in study 1 (r < 0.24) and for 6MWD (r < 0.11) in study 2. CONCLUSIONS These studies indicate that the CAT is sensitive to changes in health status following exacerbations and is as responsive to pulmonary rehabilitation as more complex COPD health status measures.

Improving the process and outcome of care in COPD: development of a standardised assessment tool.

INTRODUCTION A major goal of COPD treatment is to reduce symptom burden and ensure that the patients health is as good as possible. This goal requires regular systematic assessment of the patients COPD with clear and efficient communication between the patient and clinician. AIM To explore patient and physician descriptions of COPD attributes, in order to inform content development of a patient-reported clinical assessment tool. METHODS Qualitative research methods (one-to-one interviews and patient focus groups) were used to elicit key characteristics to evaluate COPD health status and explore how patients with COPD experience their condition. ATLAS.ti version 5.0 was used to identify major themes and generate an item pool. RESULTS Fifty-eight patients with COPD (GOLD stages 1-4; MRC grades 2-5) and 10 clinicians participated in this research. Twenty-one items were generated, capturing patient assessment of breathlessness, wheeze, cough, sleep, activity limitation, energy/fatigue, social function, and anxiety. CONCLUSIONS This qualitative study identified a broad range of items that are potentially suitable for inclusion in a short, simple COPD assessment tool for use in routine clinical practice.

Development, dimensions, reliability and validity of the novel Manchester COPD fatigue scale

Introduction: Fatigue is a prominent symptom in chronic obstructive pulmonary disease (COPD) and it has distinctive features; however, there is a need for a robust scale to measure fatigue in COPD. Methods: At baseline, 122 patients with COPD (forced expiratory volume in 1 s (FEV1) 52%, women 38%, mean age 66 years) completed a pilot fatigue scale covering a pool of 57 items and underwent a range of tests, including indicators of mood and a short general fatigue questionnaire. All patients responded to the 57-item scale and it was readministered to a subset of 30 patients. The pilot scale was first subjected to constructive validated shortening steps and then to a principal components analysis. Results: The Manchester COPD fatigue scale (MCFS) consists of 27 items, loading into three dimensions: physical, cognitive and psychosocial fatigue. Internal consistency (Cronbach’s α = 0.97) and test–retest repeatability (r = 0.97, p<0.001) were tested. It had significant convergent validity, correlating with the FACIT (Functional Assessment of Chronic Illness Therapy) fatigue scale and the fatigue in Borg scale at baseline and after a 6 minute walk distance (6MWD) test (r = −0.81, 0.53 and 0.63, respectively, p<0.001). Its scores were associated with BODE, SGRQ (St George’s Respiratory Questionnaire) and MRC (Medical Research Council) dyspnoea scores (r = 0.46, 0.8 and 0.51, respectively, p<0.001). The scale demonstrated meaningful discriminating ability; patients who walked <350 m in a 6MWD test as well as depressed patients (⩾16 scores in the Center for Epidemiologic Study on Depression (CES-D) scale) had nearly twice as high fatigue scores as those who walked ⩾350 m or were not depressed (p<0.001). Conclusion: The MCFS provides a simple, reliable and valid measurement of total and dimensional fatigue in moderate stable COPD.

Treatment Satisfaction in Systemic Lupus Erythematosus: Development of a Patient-Reported Outcome Measure

ObjectiveThe aim of this study was to develop a patient-reported outcome measure specific for systemic lupus erythematosus (SLE) to assess patient satisfaction with treatment, treatment options, and medical care. MethodsPatients with SLE were recruited from four US rheumatology practices. Concept elicitation interviews identified aspects that patients considered important and relevant regarding satisfaction with treatment and medical care. Concept elicitation interviews and clinical input were used to draft the Lupus Satisfaction Questionnaire (LSQ). A second cohort of patients with SLE participated in combined concept elicitation/cognitive debriefing interviews, after which the LSQ was revised. ResultsFourteen patients completed concept elicitation interviews: 93% were female, 57% were white, and 85% had moderate/severe SLE. Current treatments included hydroxychloroquine (93%), steroids (79%), and belimumab (57%), and 43% were biologic naive. Patients were generally satisfied with their treatment and medical care; however, they were dissatisfied with treatment adverse effects and the number of available treatment options. Cognitive debriefing interviews (n = 8) demonstrated that the LSQ was comprehensive, clear, and relevant; therefore, only minor revisions were made to the questionnaire. The LSQ assesses satisfaction with current SLE treatments (25 items), medical care (11 items), and insurance coverage (3 items). The draft LSQ was evaluated in 195 adults with SLE. Fifty-eight percent of patients reported that they were “somewhat satisfied” with their SLE treatment. ConclusionsThe LSQ has been developed to assess treatment satisfaction among patients with SLE. Following further testing to support its validity and reliability, it will provide a useful tool to facilitate assessment of satisfaction with treatments for SLE and help inform treatment decisions.

Patient experience in systemic lupus erythematosus: development of novel patient-reported symptom and patient-reported impact measures

BackgroundComprehensive assessment of systemic lupus erythematosus (SLE) and its treatment requires patient-reported outcome (PRO) measures to capture impacts and fluctuating symptoms. The objective of this study was to develop PROs, in accordance with the Food and Drug Administration (FDA) PRO Guidance, to assess fluctuations in SLE symptoms and its impact.MethodsFollowing independent review board approval, six US rheumatology practices recruited patients with SLE to participate in concept elicitation (CE) interviews, in order to identify important SLE symptoms and their impacts. The SLE Symptom Severity Diary (SSD) and SLE Impact Questionnaire (SIQ) were drafted based on CE interview results and clinician input. The PROs were revised based on patient feedback from cognitive debriefing (CD) interviews, clinician feedback, and a translatability assessment.ResultsForty-one patients completed CE interviews. Commonly-reported symptoms included fatigue (98%), joint pain (93%), and rash (88%). The most frequently reported impact was difficulty with chores/housework (61%). Eighteen patients completed CD interviews. The PROs were considered comprehensive, clear, and relevant.The SSD contains 17 items assessing energy/vitality, joint and muscle pain/stiffness/swelling, flu-like symptoms, cognition, numbness/tingling, skin symptoms and hair loss using an 11-point numeric response scale and a 24-h recall period (with the exception of hair loss). It also evaluates steroid status and dose. The SIQ contains 50 items, uses a 5-point Likert scale and a 7-day recall period, to assess disease impacts including patients’ ability to make plans, work, and physical/social/emotional functioning.ConclusionThe SSD and SIQ are comprehensive SLE-specific PROs developed in accordance with the FDA PRO Guidance. Following assessment of their measurement properties, they may be useful in clinical studies and clinical practice to measure fluctuations in, and the impact of, symptoms in patients with SLE.

FRI0339 Development of a Novel Patient Reported Outcome (PRO) Measure: The Systemic Lupus Erythematosus (SLE) Steroid Questionnaire (SSQ)

Background Many SLE patients receive oral glucocorticoids (steroids) as part of their treatment regimen. While providing symptom relief, most patients experience short- and long-term side effects associated with steroid use. Objectives To develop a PRO to assess benefits, side effects, and impact associated with steroid use in SLE patients. Methods Following IRB approval, 6 US rheumatology practices enrolled SLE patients with current or recent (past year) steroid use to participate in concept elicitation (CE) interviews about their steroid experience. A draft SSQ was developed, cognitively debriefed (CD) with SLE subjects, and finalized based on patient feedback, clinical input and a translatability assessment (an evaluation to assure words can be easily translated into other languages). Results 33 subjects (95% female, 55% Caucasian, 25% African American, mean age 47 years, 52% moderate/severe SLE) completed CE interviews. Subjects reported symptom improvement and increased energy. Common side effects included weight gain (67%), swelling/moon face (36%), and mood swings/feelings of rage (21%). Most subjects (67%) would be happier if steroids were discontinued. During CD interviews (n=13), the SSQ was found to be comprehensive, clear, and relevant. Minor revisions were made to improve clarity. Key results from CE interviews and a summary of the final SSQ content are provided below. Concepts mentioned during CE interview % Reporting (n=33) Potential side effects discussed prior to receiving steroids 71% Expectations about side effects match experience once starting steroids? 50% Side effects: weight gain, swelling/moon face, mood swings/feelings of rage 67%/36%/21% Stopped or changing dose without consulting physician 30% Overall satisfied with steroids 75% Concepts in Final Questionnaire† Number of Items (50) Steroid dose/duration 4 Impact of steroids (in general) 19 Benefits of steroids 7 Work/productivity 3 Side effects 10 Emotions 6 Overall satisfaction 1 †Most subjects reported side effects were worse than expected. ‡Most items use a 7-day recall period. Conclusions The SSQ is the first comprehensive PRO, developed with significant patient and clinical input, to assess SLE patient experience of oral steroid use. Its measurement properties will be evaluated in the future. Acknowledgements The research presented in this abstract was funded by GlaxoSmithKline, USA. Disclosure of Interest S. D. Mathias Consultant for: GlaxoSmithKline, P. Berry Shareholder of: GlaxoSmithKline, Employee of: GlaxoSmithKline, J. deVries Shareholder of: GlaxoSmithKline, A. Askanase Consultant for: GlaxoSmithKline, K. Pascoe Shareholder of: GlaxoSmithKline, Employee of: GlaxoSmithKline, H. Colwell Consultant for: GlaxoSmithKline, D. Chang Shareholder of: GlaxoSmithKline, Employee of: GlaxoSmithKline