Patient-reported outcomes in cystic fibrosis

Proceedings of the American Thoracic Society

Volumn 4, Issue 4, 2007, Pages 378-386

  Goss, Christopher H ^a   Quittner, Alexandra L ^b  

^a UNIVERSITY OF WASHINGTON MEDICAL CENTER   (United States)

^b UNIVERSITY OF MIAMI   (United States)

Author keywords

Instruments; Patient reported outcomes; Quality of life; Symptoms; Validity

Indexed keywords

AMINOGLYCOSIDE ANTIBIOTIC AGENT; BETA LACTAM ANTIBIOTIC; DORNASE ALFA; MACROLIDE; SODIUM CHLORIDE; TOBRAMYCIN;

BREATHING EXERCISE; CHRONIC OBSTRUCTIVE LUNG DISEASE; CLINICAL PROTOCOL; CLINICAL TRIAL; COMBINATION CHEMOTHERAPY; CONFERENCE PAPER; CONSTRUCT VALIDITY; CYSTIC FIBROSIS; DISEASE EXACERBATION; FOOD AND DRUG ADMINISTRATION; HUMAN; MONOTHERAPY; OUTCOME ASSESSMENT; PAIN; PRACTICE GUIDELINE; PSYCHOMETRY; QUALITY OF LIFE; RELIABILITY; SENSITIVITY AND SPECIFICITY; STANDARDIZATION; SYMPTOMATOLOGY;

CYSTIC FIBROSIS; HUMANS; OUTCOME ASSESSMENT (HEALTH CARE); QUALITY OF LIFE; REPRODUCIBILITY OF RESULTS; SELF DISCLOSURE;

EID: 34548255391     PISSN: 15463222     EISSN: None     Source Type: Journal    
DOI: 10.1513/pats.200703-039BR     Document Type: Conference Paper

References (103)

1. Fayer P, Hays R, editors. Assessing quality of life in clinical trials, 2nd ed. Oxford, UK: Oxford University Press; 2005.
2. Quittner AL, Davis M, Modi A. Health-related quality of life in pediatric populations. In: Roberts M, editor. Handbook of pediatric psychology. New York: Guilford Publications; 2003. pp. 696-709.
3. U.S. Food and Drug Administration. Guidance for industry patient-reported outcome measures: Use in medical product development to support labeling claims [internet]. Draft guidance; 2006. Available at: http://www.fda.gov/cder/ guidance/5460dft.pdf (accessed September 14, 2006).
4. Revicki DA, Osoba D, Fairclough D, Barofsky I, Berzon R, Leidy NK, Rothman M. Recommendations on health-related quality of life research to support labeling and promotional claims in the United States. Qual Life Res 2000;9:887-900.
5. Kaplan RM. Health outcome models for policy analysis. Health Psychol 1989;8:723-735.
6. Ware JE Jr, Sherbourne CD. The MOS 36-Item Short-Form Health Survey (SF-36). I. Conceptual framework and item selection. Med Care 1992;30:473-483.
7. Hays R. Developing and evaluating questionnaires. In: Fayers P, Hays R, editors. Assessing quality of life in clinical trials. Oxford, UK: Oxford University Press; 2005. pp. 3-8.
8. Quittner AL. Measurement of quality of life in cystic fibrosis. Curr Opin Pulm Med 1998;4:326-331.
9. Quittner AL, Sweeny S, Watrous M, Munzenberger P, Bearss K, Gibson NA, Fisher LA, Henry B. Translation and linguistic validation of a disease-specific quality of life measure for cystic fibrosis. J Pediatr Psychol 2000;25:403-414.
10. Cystic Fibrosis Foundation. Patient registry 2005: annual data report. Bethesda, MD; 2006.
11. World Health Organization. Preamble to the constitution of the World Health Organization. In: Basic Documents, 26th ed. Geneva, Switzerland: World Health Organization; 1976. p. 1.
12. Schor EL, Lerner DJ, Malspeis S. Physicians' assessment of functional health status and well-being: the patient's perspective. Arch Intern Med 1995;155:309-314.
13. Turk D, Melzack R. Handbook of pain assessment. New York: Guilford Press; 2001.
14. Modi AC, Lim CS, Yu N, Geller D, Wagner MH, Quittner ALA. Multimethod assessment of treatment adherence for children with cystic fibrosis. J Cyst Fibros 2006;5:177-185.
15. Szende A, Leidy NK, Revicki D. Health-related quality of life and other patient-reported outcomes in the European centralized drug regulatory process: a review of guidance documents and performed authorizations of medicinal products 1995 to 2003. Value Health 2005;8:534-548.
16. Detmar SB, Muller MJ, Schornagel JH, Wever LD, Aaronson NK. Health-related quality-of-life assessments and patient-physician communication: a randomized controlled trial. JAMA 2002;288:3027-3034.
17. Detmar SB, Weaver LD, Aaronson NK. Role of health-related quality of life in palliative chemotherapy treatment decisions. J Clin Oncol 2002;20:1056-1062.
18. Feeny D. Preference-based measures: utility and quality-adjusted life years. In: Fayers P, Hays R, editors. Assessing quality of life in clinical trials. Oxford, UK: Oxford University Press; 2005. pp. 405-430.
19. Leidy NK, Revicki DA, Geneste B. Recommendations for evaluating the validity of quality of life claims for labeling and promotion. Value Health 1999;2:113-127.
20. Quittner AL, Buu A, Messer MA, Modi AC, Watrous M. Development and validation of the Cystic Fibrosis Questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis. Chest 2005;128:2347-2354.
21. Guyatt GH, Osoba D, Wu AW, Wyrwich KW, Norman GR. Methods to explain the clinical significance of health status measures. Mayo Clin Proc 2002;77:371-383.
22. Varni JW, Seid M, Kurtin PS. PedsQL 4.0: reliability and validity of the Pediatric Quality of Life Inventory version 4.0 generic core scales in healthy and patient populations. Med Care 2001;39:800-812.
23. Osoba D, Bezjak A, Brundage M, Zee B, Tu D, Pater J. Analysis and interpretation of health-related quality-of-life data from clinical trials: basic approach of the National Cancer Institute of Canada Clinical Trials Group. Eur J Cancer 2005;41:280-287.
24. Redelmeier DA, Guyatt GH, Goldstein RS. Assessing the minimal important difference in symptoms: a comparison of two techniques. J Clin Epidemiol 1996;49:1215-1219.
25. Sloan JA. Assessing the minimally clinically significant difference: scientific considerations, challenges and solutions. COPD 2005;2:57-62.
26. Cohen J. Statistical power for the behavioral sciences, 2nd ed. Hillsdale, NJ: Lawrence Erlbaum Associates; 1988.
27. Wyrwich KW, Nienaber NA, Tierney WM, Wolinsky FD. Linking clinical relevance and statistical significance in evaluating intra-individual changes in health-related quality of life. Med Care 1999;37:469-478.
28. Cella D, Bullinger M, Scott C, Barofsky I. Group vs individual approaches to understanding the clinical significance of differences or changes in quality of life. Mayo Clin Proc 2002;77:384-392.
29. Fuchs HJ, Borowitz DS, Christiansen DH, Morris EM, Nash ML, Ramsey BW, Rosenstein BJ, Smith AL, Wohl ME. Effect of aerosolized recombinant human dnase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group. N Engl J Med 1994;331:637-642.
30. Britto MT, Kotagal UR, Hornung RW, Atherton HD, Tsevat J, Wilmott RW. Impact of recent pulmonary exacerbations on quality of life in patients with cystic fibrosis. Chest 2002;121:64-72.
31. Dobbin CJ, Bartlett D, Melehan K, Grunstein RR, Bye PT. The effect of infective exacerbations on sleep and neurobehavioral function in cystic fibrosis. Am J Respir Crit Care Med 2005;172:99-104.
32. Gee L, Abbott J, Conway SP, Etherington C, Webb AK. Development of a disease specific health related quality of life measure for adults and adolescents with cystic fibrosis. Thorax 2000;55:946-954.
33. Caine N, Sharples LD, Smyth R, Scott J, Hathaway T, Higenbottam TW, Wallwork J. Survival and quality of life of cystic fibrosis patients before and after heart-lung transplantation. Transplant Proc 1991;23:1203-1204.
34. Busschbach JJ, Horikx PE, van den Bosch JM, Brutel DLR, de Charro FT. Measuring the quality of life before and after bilateral lung transplantation in patients with cystic fibrosis. Chest 1994;105:911-917.
35. Burker EJ, Carels RA, Thompson LF, Rodgers L, Egan T. Quality of life in patients awaiting lung transplant: cystic fibrosis versus other end-stage lung diseases. Pediatr Pulmonol 2000;30:453-460.
36. Vermeulen KM, van der Bij W, Erasmus ME, Duiverman EJ, Koeter GH, TenVergert EM. Improved quality of life after lung transplantation in individuals with cystic fibrosis. Pediatr Pulmonol 2004;37:419-426.
37. Smeritschnig B, Jaksch P, Kocher A, Seebacher G, Aigner C, Mazhar S, Klepetko W. Quality of life after lung transplantation: a crosssectional study. J Heart Lung Transplant 2005;24:474-480.
38. Arrington-Sanders R, Yi MS, Tsevat J, Wilmott RW, Mrus JM, Britto MT. Gender differences in health-related quality of life of adolescents with cystic fibrosis. Health Qual Life Outcomes 2006;4:5.
39. Gee L, Abbott J, Conway SP, Etherington C, Webb AK. Quality of life in cystic fibrosis: the impact of gender, general health perceptions and disease severity. J Cyst Fibros 2003;2:206-213.
40. Gee L, Abbott J, Hart A, Conway SP, Etherington C, Webb AK. Associations between clinical variables and quality of life in adults with cystic fibrosis. J Cyst Fibros 2005;4:59-66.
41. Britto MT, Kotagal UR, Chenier T, Tsevat J, Atherton HD, Wilmott RW. Differences between adolescents' and parents' reports of health-related quality of life in cystic fibrosis. Pediatr Pulmonol 2004;37:165-171.
42. DeLambo KE, Ievers-Landis CE, Drotar D, Quittner AL. Association of observed family relationship quality and problem-solving skills with treatment adherence in older children and adolescents with cystic fibrosis. J Pediatr Psychol 2004;29:343-353.
43. Ziaian T, Sawyer MG, Reynolds KE, Carbone JA, Clark JJ, Baghurst PA, Couper JJ, Kennedy D, Martin AJ, Staugas RE, et al. Treatment burden and health-related quality of life of children with diabetes, cystic fibrosis and asthma. J Paediatr Child Health 2006;42:596-600.
44. Abbott J, Morton AM, Musson H, Conway SP, Etherington C, Gee L, Fitzjohn J, Kevin WA. Nutritional status, perceived body image and eating behaviours in adults with cystic fibrosis. Clin Nutr 2006;26:91-99.
45. Yi MS, Tsevat J, Wilmott RW, Kotagal UR, Britto MT. The impact of treatment of pulmonary exacerbations on the health-related quality of life of patients with cystic fibrosis: does hospitalization make a difference? J Pediatr 2004;144:711-718.
46. Wolter JM, Bowler SD, Nolan PJ, McCormack JG. Home intravenous therapy in cystic fibrosis: a prospective randomized trial examining clinical, quality of life and cost aspects. Eur Respir J 1997;10:896-900.
47. Selvadurai HC, Blimkie CJ, Meyers N, Mellis CM, Cooper PJ, Van Asperen PP. Randomized controlled study of in-hospital exercise training programs in children with cystic fibrosis. Pediatr Pulmonol 2002;33:194-200.
48. Klijn PH, van Stel HF, Quittner AL, van der Net J, Doeleman W, van der Schans CP, van der Ent CK. Validation of the Dutch Cystic Fibrosis Questionnaire (CFQ) in adolescents and adults. J Cyst Fibros 2004;3:29-36.
49. Saiman L, Marshall BC, Mayer-Hamblett N, Burns JL, Quittner AL, Cibene DA, Coquillette S, Fieberg AY, Accurso FJ, Campbell PW III. Azithromycin in patients with cystic fibrosis chronically infected with pseudomonas aeruginosa: a randomized controlled trial. JAMA 2003;290:1749-1756.
50. Donaldson SH, Bennett WD, Zeman KL, Knowles MR, Tarran R, Boucher RC. Mucus clearance and lung function in cystic fibrosis with hypertonic saline. N Engl J Med 2006;354:241-250.
51. Elkins MR, Robinson M, Rose BR, Harbour C, Moriarty CP, Marks GB, Belousova EG, Xuan W, Bye PT. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med 2006;354:229-240.
52. Clement A, Tamalet A, Leroux E, Ravilly S, Fauroux B, Jais JP. Long term effects of azithromycin in patients with cystic fibrosis: a double blind, placebo controlled trial. Thorax 2006;61:895-902.
53. Hardin DS, Adams-Huet B, Brown D, Chatfield B, Dyson M, Ferkol T, Howenstine M, Prestidge C, Royce F, Rice J, et al. Growth hormone treatment improves growth and clinical status in prepubertal children with cystic fibrosis: results of a multicenter randomized controlled trial. J Clin Endocrinol Metab 2006;91:4925-4929.
54. Borowitz D, Goss CH, Stevens C, Hayes D, Newman L, O'Rourke A, Konstan MW, Wagener J, Moss R, Hendeles L, et al. Safety and preliminary clinical activity of a novel pancreatic enzyme preparation in pancreatic insufficient cystic fibrosis patients. Pancreas 2006;32:258-263.
55. Borowitz D, Goss CH, Limauro S, Konstan MW, Blake K, Casey S, Quittner AL, Murray FT. Study of a novel pancreatic enzyme replacement therapy in pancreatic insufficient subjects with cystic fibrosis. J Pediatr 2006;149:658-662.
56. Suri R, Grieve R, Normand C, Metcalfe C, Thompson S, Wallis C, Bush A. Effects of hypertonic saline, alternate day and daily rhDNase on healthcare use, costs and outcomes in children with cystic fibrosis. Thorax 2002;57:841-846.
57. Suri R, Metcalfe C, Lees B, Grieve R, Flather M, Normand C, Thompson S, Bush A, Wallis C. Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial. Lancet 2001;358:1316-1321.
58. Fitzgerald DA, Hilton J, Jepson B, Smith LA. Crossover, randomized, controlled trial of dornase alfa before versus after physiotherapy in cystic fibrosis. Pediatrics 2005;116:e549-e554.
59. Ramsey BW, Pepe MS, Quan JM, Otto KL, Montgomery AB, Williams-Warren J, Vasiljev K, Borowitz D, Bowman CM, Marshall BC, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. N Engl J Med 1999;340:23-30.
60. Quittner AL, Buu A. Effects of tobramycin solution for inhalation on global ratings of quality of life in patients with cystic fibrosis and Pseudomonas aeruginosa infection. Pediatr Pulmonol 2002;33:269-276.
61. Abbott J, Hart A. Measuring and reporting quality of life outcomes in clinical trials in cystic fibrosis: a critical review. Health Qual Life Outcomes 2005;3:19.
62. Smith AL, Redding G, Doershuk C, Goldmann D, Gore E, Hilman B, Marks M, Moss R, Ramsey B, Rubio T, et al. Sputum changes associated with therapy for endobronchial exacerbation in cystic fibrosis. J Pediatr 1988;112:547-554.
63. Smith AL, Doershuk C, Goldmann D, Gore E, Hilman B, Marks M, Moss R, Ramsey B, Redding G, Rubio T, et al. Comparison of a beta-lactam alone versus beta-lactam and an aminoglycoside for pulmonary exacerbation in cystic fibrosis. J Pediatr 1999;134:413-421.
64. Jones PW, Quirk FH, Baveystock CM, Littlejohns PA. Self-complete measure of health status for chronic airflow limitation: the St. George's Respiratory Questionnaire. Am Rev Respir Dis 1992;145:1321-1327.
65. Jones PW, Bosh TK. Quality of life changes in COPD patients treated with salmeterol. Am J Respir Crit Care Med 1997;155:1283-1289.
66. Wilson CB, Jones PW, O'Leary CJ, Cole PJ, Wilson R. Validation of the St. George's Respiratory Questionnaire in bronchiectasis. Am J Respir Crit Care Med 1997;156:536-541.
67. Lahdensuo A, Haahtela T, Herrala J, Kava T, Kiviranta K, Kuusisto P, Peramaki E, Poussa T, Saarelainen S, Svahn T. Randomised comparison of guided self management and traditional treatment of asthma over one year. BMJ 1996;312:748-752.
68. Wijkstra PJ, TenVergert EM, Van Altena R, Otten V, Postma DS, Kraan J, Koeter GH. Reliability and validity of the Chronic Respiratory Questionnaire (CRQ). Thorax 1994;49:465-467.
69. Bradley J, Dempster M, Wallace E, Elborn S. The Adaptations of a quality of life questionnaire for routine use in clinical practice: the Chronic Respiratory Disease Questionnaire in Cystic Fibrosis. Qual Life Res 1999;8:65-71.
70. Quittner AL, Sweeny S, Watrous M, Munzenberger P, Bearss K, Gibson NA, Fisher LA, Henry B. Translation and linguistic validation of a disease-specific quality of life measure for cystic fibrosis. J Pediatr Psychol 2000;25:403-414.
71. Modi AC, Quittner AL. Validation of a disease-specific measure of health-related quality of life for children with cystic fibrosis. J Pediatr Psychol 2003;28:535-545.
72. van der Schans C, Prasad A, Main E. Chest physiotherapy compared to no chest physiotherapy for cystic fibrosis. Cochrane Database Syst Rev 2000;2:CD001401.
73. Prasad SA, Randall SD, Balfour-Lynn IM. Fifteen-count breathlessness score: an objective measure for children. Pediatr Pulmonol 2000;30:56-62.
74. Eakin EG, Resnikoff PM, Prewitt LM, Ries AL, Kaplan RM. Validation of a new dyspnea measure: the UCSD Shortness of Breath Questionnaire. Chest 1998;113:619-624.
75. Orenstein DM, Holt LS, Rebovich P, Campbell T, Nixon P. Measuring ease of breathing in young patients with cystic fibrosis. Pediatr Pulmonol 2002;34:473-477.
76. Fletcher CM, Elmes PC, Fairbairn AS, Wood CH. The significance of respiratory symptoms and the diagnosis of chronic bronchitis in a working population. Br Med J 1959;2:257-266.
77. Bestall JC, Paul EA, Garrod R, Garnham R, Jones PW, Wedzicha JA. Usefulness of the Medical Research Council (MRC) dyspnoea scale as ameasure of disability in patients with chronic obstructive pulmonary disease. Thorax 1999;54:581-586.
78. de Jong W, van der Schans CP, Mannes GP, van Aalderen WM, Grevink RG, Koeter GH. Relationship between dyspnoea, pulmonary function and exercise capacity in patients with cystic fibrosis. Respir Med 1997;91:41-46.
79. Aitken RC. Measurement of feelings using visual analogue scales. Proc R Soc Med 1969;62:989-993.
80. Leidy NK, Schmier JK, Jones MK, Lloyd J, Rocchiccioli K. Evaluating symptoms in chronic obstructive pulmonary disease: validation of the Breathlessness, Cough, and Sputum Scale. Respir Med 2003;97(Suppl A):S59-S70.
81. Leidy NK, Rennard SI, Schmier J, Jones MK, Goldman M. The Breathlessness, Cough, and Sputum Scale: the development of empirically based guidelines for interpretation. Chest 2003;124:2182-2191.
82. Van Der Molen T, Willemse BW, Schokker S, Ten Hacken NH, Postma DS, Juniper EF. Development, validity and responsiveness of the clinical COPD questionnaire. Health Qual Life Outcomes 2003;1:13.
83. Au DH, Blough DK, Kirchdoerfer L, Weiss KB, Udris EM, Sullivan SD. Development of a quantifiable symptom assessment tool for patients with chronic bronchitis: the Chronic Bronchitis Symptoms Assessment Scale. COPD 2005;2:209-216.
84. Koh JL, Harrison D, Palermo TM, Turner H, McGraw T. Assessment of acute and chronic pain symptoms in children with cystic fibrosis. Pediatr Pulmonol 2005;40:330-335.
85. Palermo TM, Harrison D, Koh JL. Effect of disease-related pain on the health-related quality of life of children and adolescents with cystic fibrosis. Clin J Pain 2006;22:532-537.
86. Ravilly S, Robinson W, Suresh S, Wohl ME, Berde CB. Chronic pain in cystic fibrosis. Pediatrics 1996;98:741-747.
87. Robinson WM, Ravilly S, Berde C, Wohl ME. End-of-life care in cystic fibrosis. Pediatrics 1997;100:205-209.
88. Massie RJ, Towns SJ, Bernard E, Chaitow J, Howman-Giles R, Van Asperen PP. The musculoskeletal complications of cystic fibrosis. J Paediatr Child Health 1998;34:467-470.
89. Festini F, Ballarin S, Codamo T, Doro R, Loganes C. Prevalence of pain in adults with cystic fibrosis. J Cyst Fibros 2004;3:51-57.
90. Bregnballe V, Thastum M, Schiotz P. Psychosocial problems in children with cystic fibrosis. Acta Paediatr 2007;96:58-61.
91. Wray J, Radley-Smith R. Depression in pediatric patients before and 1 year after heart or heart-lung transplantation. J Heart Lung Transplant 2004;23:1103-1110.
92. Anderson DL, Flume PA, Hardy KK. Psychological functioning of adults with cystic fibrosis. Chest 2001;119:1079-1084.
93. Burker EJ, Sedway J, Carone S. Psychological and educational factors: better predictors of work status than FEV1 in adults with cystic fibrosis. Pediatr Pulmonol 2004;38:413-418.
94. Goss CH, Ramsey BW, Aitken ML, Genatossio A, McNamara S, Curtis JR. Health related quality of life in adults and adolescents with severe cystic fibrosis. Pediatr Pulmonol 2006;S29:358.
95. Rabin HR, Butler SM, Wohl ME, Geller DE, Colin AA, Schidlow DV, Johnson CA, Konstan MW, Regelmann WE. Pulmonary exacerbations in cystic fibrosis. Pediatr Pulmonol 2004;37:400-406.
96. Mayer-Hamblett N, Rosenfeld M, Emerson J, Goss CH, Aitken ML. Developing cystic fibrosis lung transplant referral criteria using predictors of 2-year mortality. Am J Respir Crit Care Med 2002;166:1550-1555.
97. Liou TG, Adler FR, Fitzsimmons SC, Cahill BC, Hibbs JR, Marshall BC. Predictive 5-year survivorship model of cystic fibrosis. Am J Epidemiol 2001;153:345-352.
98. Ramsey BW, Boat TF. Outcome measures for clinical trials in cystic fibrosis: summary of a Cystic Fibrosis Foundation consensus conference. J Pediatr 1994;124:177-192.
99. Marshall BC. Pulmonary exacerbations in cystic fibrosis: it's time to be explicit! Am J Respir Crit Care Med 2004;169:781-782.
100. Cystic Fibrosis Foundation. Microbiology and infectious disease in cystic fibrosis: V (section 1). Bethesda, MD: Cystic Fibrosis Foundation; 1994.
101. Rosenfeld M, Emerson J, Williams-Warren J, Pepe M, Smith A, Montgomery AB, Ramsey B. Defining a pulmonary exacerbation in cystic fibrosis. J Pediatr 2001;139:359-365.
102. Schechter MS, Margolis P. Improving subspecialty healthcare: lessons from cystic fibrosis. J Pediatr 2005;147:295-301.
103. Cystic Fibrosis Foundation. CF Foundation Patient Registry, 2004 Annual Data Report to the Center Directors. Bethesda, MD: Cystic Fibrosis Foundation.