Patient reported outcome measures in chronic obstructive pulmonary disease: Which to use?

Expert Review of Respiratory Medicine

Volumn 10, Issue 3, 2016, Pages 351-362

  Ekström, Magnus ^a   Sundh, Josefin ^b   Larsson, Kjell ^c  

^a LUND UNIVERSITY   (Sweden)

^b ÖREBRO UNIVERSITY   (Sweden)

^c KAROLINSKA INSTITUTE   (Sweden)

Author keywords

COPD; Dyspnea; Exacerbation; Health status; Measurement; PROM; Quality of Life

Indexed keywords

ANXIETY; BASELINE DYSPNEA INDEX; BORG SCALE; CHRONIC OBSTRUCTIVE LUNG DISEASE; CHRONIC RESPIRATORY QUESTIONNAIRE; CLINICAL ASSESSMENT; CLINICAL COPD QUESTIONNAIRE; CLINICAL RESEARCH; COPD ASSESSMENT TEST; COUGHING; DISEASE EXACERBATION; DYSPNEA; DYSPNEA 12; EUROQOL FIVE DIMENSION; FATIGUE; HEALTH STATUS; HUMAN; MORTALITY; MULTIDIMENSIONAL DYSPNEA PROFILE; NUMERICAL RATING SCALE; OUTCOME ASSESSMENT; PAIN; PATIENT CARE; PATIENT CODING; PATIENT REPORTED OUTCOME; PHLEGMON; PRACTICE GUIDELINE; PREVALENCE; RATING SCALE; RESPIRATORY TRACT DISEASE ASSESSMENT; REVIEW; SELF REPORT; SHORT FORM 12; SHORT FORM 36; SHORTNESS OF BREATH QUESTIONNAIRE; SHORTNESS OF BREATH WITH DAILY ACTIVITIES QUESTIONNAIRE; ST. GEORGE RESPIRATORY QUESTIONNAIRE; TRANSITION DYSPNEA INDEX; VISUAL ANALOG SCALE; COST OF ILLNESS; DAILY LIFE ACTIVITY; DISEASE COURSE; HEALTH STATUS INDICATOR; LUNG; PATHOPHYSIOLOGY; PATIENT-REPORTED OUTCOME; PREDICTIVE VALUE; PSYCHOLOGY; PULMONARY DISEASE, CHRONIC OBSTRUCTIVE; QUALITY OF LIFE; REPRODUCIBILITY; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

ACTIVITIES OF DAILY LIVING; COST OF ILLNESS; DISEASE PROGRESSION; DYSPNEA; HEALTH STATUS; HEALTH STATUS INDICATORS; HUMANS; LUNG; PATIENT REPORTED OUTCOME MEASURES; PREDICTIVE VALUE OF TESTS; PULMONARY DISEASE, CHRONIC OBSTRUCTIVE; QUALITY OF LIFE; REPRODUCIBILITY OF RESULTS; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

EID: 84960428375     PISSN: 17476348     EISSN: 17476356     Source Type: Journal    
DOI: 10.1586/17476348.2016.1146595     Document Type: Review

References (93)

1. From the global strategy for the diagnosis, management and prevention of COPD, Global Initiative for Chronic Obstructive Lung Disease (GOLD). 2014 [cited 2015 Dec 1]. Available from: http://www.Goldcopd. Org/.
2. Guidance for Industry. Patient-reported Outcome Measures: Use in Medical Product Delvelopment to Support Labelling Claims. US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH); 2009. [cited 2015 Sept 23]. Available from: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf.
3. Guidance for Industry and FDA Staff. Qualification Process for Drug Development Tools. US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER); 2014. cited 2015 Sept 23]. Available from: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM230597.pdf.
4. Cazzola M, Hanania NA, MacNee W, et al. A review of the most common patient-reported outcomes in COPD-revisiting current knowledge and estimating future challenges. Int J Chron Obstruct Pulmon Dis. 2015;10:725-738.
5. Ekstrom M, Currow DC, Johnson MJ. Outcome measurement of refractory breathlessness: endpoints and important differences. Curr Opin Support Palliat Care. 2015;9(3):238-243.
6. Blinderman CD, Homel P, Billings JA, et al. Symptom distress and quality of life in patients with advanced chronic obstructive pulmonary disease. J Pain Symptom Manage. 2009;38(1):115-123.
7. Kessler R, Partridge MR, Miravitlles M, et al. Symptom variability in patients with severe COPD: a pan-European cross-sectional study. Eur Respir J. 2011;37(2):264-272.
8. Patrick DL, Guyatt GH, Acquadro C, et al. Patient reported outcomes. In: Higgins JPT, Green S, editors. Cochrane handbook for systematic reviews of interventions version 5.1.0. The Cochrane Collaboration; 2011. [updated March]. Available from: www.cochrane-handbook.org.)
9. Hodson M, Andrew S, Michael Roberts C. Towards an understanding of PREMS and PROMS in COPD. Breathe. 2013;9(5):358-364.
10. Jones PW. Health status measurement in chronic obstructive pulmonary disease. Thorax. 2001;56(11):880-887.
11. 1 in COPD: a review of patient-reported outcomes and their measurement. Int J Chron Obstruct Pulmon Dis. 2012;7:697-709.
12. Parshall MB, Schwartzstein RM, Adams L, et al. An official American Thoracic Society statement: update on the mechanisms, assessment, and management of dyspnea. Am J Respir Crit Care Med. 2012;185 (4):435-452.
13. Celli BR, Cote CG, Marin JM, et al. The body-mass index, airflow obstruction, dyspnea, and exercise capacity index in chronic obstructive pulmonary disease. N Engl J Med. 2004;350(10):1005-1012.
14. Nishimura K, Izumi T, Tsukino M, et al. Dyspnea is a better predictor of 5-year survival than airway obstruction in patients with COPD. Chest. 2002;121(5):1434-1440.
15. Casanova C, Marin JM, Martinez-Gonzalez C, et al. Differential effect of modified medical research council dyspnea, COPD assessment test, and clinical COPD questionnaire for symptoms evaluation within the new GOLD staging and mortality in COPD. Chest. 2015;148(1):159-168.
16. Gudmundsson G, Gislason T, Janson C, et al. Risk factors for rehospitalisation in COPD: role of health status, anxiety and depression. Eur Respir J. 2005;26(3):414-419.
17. Casanova C, Cote C, De Torres JP, et al. Inspiratory-to-total lung capacity ratio predicts mortality in patients with chronic obstructive pulmonary disease. Am J Respir Crit Care Med. 2005;171(6):591-597.
18. Jones PW, Donohue JF, Nedelman J, et al. Correlating changes in lung function with patient outcomes in chronic obstructive pulmonary disease: a pooled analysis. Respir Res. 2011;12:161.
19. Banzett RB, Adams L, O'Donnell CR, et al. Using laboratory models to test treatment: morphine reduces dyspnea and hypercapnic ventilatory response. Am J Respir Crit Care Med. 2011;184(8):920-927.
20. Wadell K, Webb KA, Preston ME, et al. Impact of pulmonary rehabilitation on the major dimensions of dyspnea in COPD. Copd. 2013.
21. Miravitlles M, Ferrer J, Baro E, et al. Differences between physician and patient in the perception of symptoms and their severity in COPD. Respir Med. 2013;107(12):1977-1985.
22. Montes De Oca M, Tálamo C, Halbert RJ, et al. Health status perception and airflow obstruction in five Latin American cities: the PLATINO study. Respir Med. 2009;103(9):1376-1382.
23. Simon ST, Altfelder N, Alt-Epping B, et al. Is breathlessness what the professional says it is? Analysis of patient and professionals' assessments from a German nationwide register. Support Care Cancer. 2014;22(7):1825-1832.
24. Santiveri C, Espinalt M, Díaz Carrasco FX, et al. Evaluation of male COPD patients' health status by proxies. Respir Med. 2007;101 (3):439-445.
25. Puhan MA, Behnke M, Devereaux PJ, et al. Measurement of agreement on health-related quality of life changes in response to respiratory rehabilitation by patients and physicians-a prospective study. Respir Med. 2004;98(12):1195-1202.
26. Ahmadi Z, Lundström S, Janson C, et al. End-of-life care in oxygendependent COPD and cancer: a national population-based study. Eur Respir J. 2015.
27. Campbell ML, Templin T, Walch J. A respiratory distress observation scale for patients unable to self-report dyspnea. J Palliat Med. 2010;13(3):285-290.
28. Yorke J, Horton M, Jones PW. A critique of Rasch analysis using the dyspnoea-12 as an illustrative example. J Adv Nurs. 2012;68(1):191-198.
29. Mokkink LB, Terwee CB, Patrick DL, et al. The COSMIN study reached international consensus on taxonomy, terminology, and definitions of measurement properties for health-related patient-reported outcomes. J Clin Epidemiol. 2010;63(7):737-745.
30. Wild D, Grove A, Martin M, et al. Principles of good practice for the translation and cultural adaptation process for patient-reported outcomes (PRO) measures: report of the ISPOR task force for translation and cultural adaptation. Value Health. 2005;8(2):94-104.
31. Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10(4):407-415.
32. Jones P, Beeh K, Chapman K, et al. Minimal clinically important differences in pharmacological trials. Am J Respir Crit Care Med. 2014;189(3):250-255.
33. Guyatt GH, Osoba D, Wu AW, et al. Methods to explain the clinical significance of health status measures. Mayo Clin Proc. 2002;77 (4):371-383.
34. Johnson MJ, Currow DC, Booth S. Prevalence and assessment of breathlessness in the clinical setting. Expert Rev Respir Med. 2014;8 (2):151-161.
35. Laviolette L, Laveneziana P. Dyspnoea: a multidimensional and multidisciplinary approach. Eur Respir J. 2014;43(6):1750-1762.
36. Abernethy AP, Currow DC, Frith P, et al. Randomised, double blind, placebo controlled crossover trial of sustained release morphine for the management of refractory dyspnoea. BMJ. 2003;327(7414):523-528.
37. Moens K, Higginson IJ, Harding R. Are there differences in the prevalence of palliative care-related problems in people living with advanced cancer and eight non-cancer conditions? A systematic review. J Pain Symptom Manage. 2014;48(4):660-677.
38. Higginson IJ, Bausewein C, Reilly CC, et al. An integrated palliative and respiratory care service for patients with advanced disease and refractory breathlessness: a randomised controlled trial. Lancet Respir Med. 2014;2(12):979-987.
39. Mahler DA, Weinberg DH, Wells CK, et al. The measurement of dyspnea. Contents, interobserver agreement, and physiologic correlates of two new clinical indexes. Chest. 1984;85(6):751-758.
40. Borg G. A category scale with ratio properties for intermodal and interindividual comparisons. In: Geissler HG, Petzol P, editors. Psychophysical judgement and the process of perception. Proceedings of the 22nd international congress of psychology. Amsterdam (The Netherlands): North Holland Publishing Co; 1980. p. 25-34.
41. Leidy N, Sexton C, Jones P, et al. Measuring respiratory symptoms in clinical trials of COPD: reliability and validity of a daily diary. Thorax. 2014;69:424-430.
42. Bestall JC, Paul EA, Garrod R, et al. Usefulness of the Medical Research Council (MRC) dyspnoea scale as a measure of disability in patients with chronic obstructive pulmonary disease. Thorax. 1999;54(7):581-586.
43. Mahler DA, Horowitz MB. Perception of breathlessness during exercise in patients with respiratory disease. Med Sci Sports Exerc. 1994;26(9):1078-1081.
44. Watkins ML, Tk W, Tabberer M, et al. Shortness of breath with daily activities questionnaire: validation and responder thresholds in patients with chronic obstructive pulmonary disease. BMJ Open. 2013;3(10):e003048.
45. Eakin EG, Resnikoff PM, Prewitt LM, et al. Validation of a new dyspnea measure: the UCSD shortness of breath questionnaire. University of California, San Diego. Chest. 1998;113(3):619-624.
46. Yorke J, Moosavi SH, Shuldham C, et al. Quantification of dyspnoea using descriptors: development and initial testing of the Dyspnoea-12. Thorax. 2010;65(1):21-26.
47. Banzett RB, O'Donnell CR, Guilfoyle TE, et al. Multidimensional dyspnea profile: an instrument for clinical and laboratory research. Eur Respir J. 2015;45(6):1681-1691.
48. Clark W, Yang JC, Tsui S-L, et al. Unidimensional pain rating scales: a multidimensional affect and pain survey (MAPS) analysis of what they really measure. Pain. 2002;98(3):241-247.
49. Hareendran A, Leidy NK, Bu M, et al. Proposing a standardized method for evaluating patient report of the intensity of dyspnea during exercise testing in COPD. Int J Chron Obstruct Pulmon Dis. 2012;7:345-355.
50. Wilcock A, Crosby V, Clarke D, et al. Repeatability of breathlessness measurements in cancer patients. Thorax. 1999;54(4):374.
51. Johnson MJ, Bland JM, Oxberry SG, et al. Measuring improvement in dyspnoea: should absolute or relative values be used? Eur Respir J. 2014.
52. Fletcher CM. Standardized questionaries on respiratory symptoms: a statement prepared and approved by the MRC committee on the aetiology of chronic bronchitis. Bmj. 1960;2(5213):1665-1665.
53. Mahler DA, Witek TJ Jr. The MCID of the transition dyspnea index is a total score of one unit. COPD. 2005;2(1):99-103.
54. Horita N, Miyazawa N, Morita S, et al. Small, moderate, and large changes, and the minimum clinically important difference in the University of California, San Diego shortness of breath questionnaire. COPD. 2014;11(1):26-32.
55. Leidy N, Murray L, Monz B, et al. Measuring respiratory symptoms of COPD: performance of the EXACT-respiratory symptoms tool (E-RS) in three clinical trials. Respir Res. 2014;15(1):124.
56. Yorke J, Swigris J, Russell AM, et al. Dyspnea-12 is a valid and reliable measure of breathlessness in patients with interstitial lung disease. Chest. 2011;139(1):159-164.
57. Lee AL, Harrison SL, Goldstein RS, et al. Pain and its clinical associations in individuals with COPD: A systematic review. Chest. 2015;147 (5):1246-1258.
58. Partridge MR, Miravitlles M, Ståhl E, et al. Development and validation of the capacity of daily living during the morning questionnaire and the global chest symptoms questionnaire in COPD. Eur Respir J. 2010;36(1):96-104.
59. Leidy NK, Rennard SI, Schmier J, et al. The breathlessness, cough, and sputum scale: the development of empirically based guidelines for interpretation. Chest. 2003;124(6):2182-2191.
60. Al-shair K, Muellerova H, Yorke J, et al. Examining fatigue in COPD: development, validity and reliability of a modified version of FACIT-F scale. Health Qual Life Outcomes. 2012;10:100.
61. Miravitlles M, Iriberri M, Barrueco M, et al. Usefulness of the LCOPD, CAFS and CASIS scales in understanding the impact of COPD on patients. Respiration. 2013;86(3):190-200.
62. Curtis JR, Martin DP, Martin TR. Patient-assessed health outcomes in chronic lung disease: what are they, how do they help us, and where do we go from here? Am J Respir Crit Care Med. 1997;156(4 Pt 1):1032-1039.
63. Domingo-Salvany A, Lamarca R, Ferrer M, et al. Health-related quality of life and mortality in male patients with chronic obstructive pulmonary disease. Am J Respir Crit Care Med. 2002;166(5):680-685.
64. Ware JE Jr., Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992;30(6):473-483.
65. Gandek B, Ware JE, Aaronson NK, et al. Cross-validation of item selection and scoring for the SF-12 health survey in nine countries: results from the IQOLA project. International quality of life assessment. J Clin Epidemiol. 1998;51(11):1171-1178.
66. Euro Qol-a new facility for the measurement of health-related quality of life. Health Policy. 1990;16(3):199-208.
67. Guyatt GH, Berman LB, Townsend M, et al. A measure of quality of life for clinical trials in chronic lung disease. Thorax. 1987;42(10):773-778.
68. Jones PW, Quirk FH, Baveystock CM, et al. A self-complete measure of health status for chronic airflow limitation. The St. George's Respiratory Questionnaire. Am Rev Respir Dis. 1992;145(6):1321-1327.
69. Meguro M, Barley EA, Spencer S, et al. Development and Validation of an Improved, COPD-Specific Version of the St. George Respiratory Questionnaire. Chest. 2007;132(2):456-463.
70. Jones PW, Harding G, Berry P, et al. Development and first validation of the COPD assessment test. Eur Respir J. 2009;34(3):648-654.
71. van der Molen T, Willemse BW, Schokker S, et al. Development, validity and responsiveness of the clinical COPD questionnaire. Health Qual Life Outcomes. 2003;1:13.
72. Mahler DA, Mackowiak JI. Evaluation of the short-form 36-item questionnaire to measure health-related quality of life in patients with COPD. Chest. 1995;107(6):1585-1589.
73. McHorney CA, Ware JE Jr., Raczek AE. The MOS 36-item short-form health survey (SF-36): II. Psychometric and clinical tests of validity in measuring physical and mental health constructs. Med Care. 1993;31 (3):247-263.
74. Boueri FM, Bucher-Bartelson BL, Glenn KA, et al. Quality of life measured with a generic instrument (short form-36) improves following pulmonary rehabilitation in patients with COPD. Chest. 2001;119(1):77-84.
75. Dolan P. Modeling valuations for Euro Qol health states. Med Care. 1997;35(11):1095-1108.
76. Rutten-van Mölken M, Roos B, Van Noord JA. An empirical comparison of the St George's Respiratory Questionnaire (SGRQ) and the Chronic Respiratory Disease Questionnaire (CRQ) in a clinical trial setting. Thorax. 1999;54(11):995-1003.
77. Agustí A, Soler JJ, Molina J, et al. Is the CAT questionnaire sensitive to changes in health status in patients with severe COPD exacerbations? COPD. 2012;9(5):492-498.
78. Dodd JW, Marns PL, Clark AL, et al. The COPD assessment test (CAT): short- and medium-term response to pulmonary rehabilitation. COPD. 2012;9(4):390-394.
79. Soler-Cataluña JJ, Martínez-García MA, Román Sánchez P, et al. Severe acute exacerbations and mortality in patients with chronic obstructive pulmonary disease. Thorax. 2005;60(11):925-931.
80. Donaldson GC, Seemungal TA, Bhowmik A, et al. Relationship between exacerbation frequency and lung function decline in chronic obstructive pulmonary disease. Thorax. 2002;57(10):847-852.
81. Donaldson GC, Hurst JR, Smith CJ, et al. Increased risk of myocardial infarction and stroke following exacerbation of COPD. Chest. 2010;137(5):1091-1097.
82. Osman IM, Godden DJ, Friend JA, et al. Quality of life and hospital readmission in patients with chronic obstructive pulmonary disease. Thorax. 1997;52(1):67-71.
83. Anthonisen NR, Manfreda J, Warren CP, et al. Antibiotic therapy in exacerbations of chronic obstructive pulmonary disease. Ann Intern Med. 1987;106(2):196-204.
84. Jones PW, Chen W-H, Wilcox TK, et al. Characterizing and quantifying the symptomatic features of COPD exacerbations. Chest. 2011;139 (6):1388-1394.
85. Leidy NK, Wilcox TK, Jones PW, et al. Development of the EXAcerbations of Chronic Obstructive Pulmonary Disease Tool (EXACT): a patient-reported outcome (PRO) measure. Value Health. 2010;13(8):965-975.
86. Leidy NK, Wilcox TK, Jones PW, et al. Standardizing measurement of chronic obstructive pulmonary disease exacerbations. Reliability and validity of a patient-reported diary. Am J Respir Crit Care Med. 2011;183(3):323-329.
87. Mackay AJ, Donaldson GC, Patel AR, et al. Detection and severity grading of COPD exacerbations using the exacerbations of chronic pulmonary disease tool (EXACT). Eur Respir J. 2014;43 (3):735-744.
88. Make BJ, Eriksson G, Calverley PM, et al. A score to predict short-term risk of COPD exacerbations (SCOPEX). Int J Chron Obstruct Pulmon Dis. 2015;10:201-209.
89. Troosters T, Sciurba F, Battaglia S, et al. Physical inactivity in patients with COPD, a controlled multi-center pilot-study. Respir Med. 2010;104(7):1005-1011.
90. Vorrink SN, Kort HS, Troosters T, et al. Level of daily physical activity in individuals with COPD compared with healthy controls. Respir Res. 2011;12:33.
91. Garcia-Aymerich J, Lange P, Benet M, et al. Regular physical activity reduces hospital admission and mortality in chronic obstructive pulmonary disease: a population based cohort study. Thorax. 2006;61(9):772-778.
92. Gimeno-Santos E, Frei A, Dobbels F, et al. Validity of instruments to measure physical activity may be questionable due to a lack of conceptual frameworks: a systematic review. Health Qual Life Outcomes. 2011;9:86.
93. Gimeno-Santos E, Raste Y, Demeyer H, et al. The PROactive instruments to measure physical activity in patients with chronic obstructive pulmonary disease. Eur Respir J. 2015;46 (4):988-1000.