Disregarding clinical trial-based patient-reported outcomes is unwarranted: Five advances to substantiate the scientific stringency of quality-of-life measurement

Acta Oncologica

Volumn 49, Issue 2, 2010, Pages 155-163

  Sprangers, Mirjam A G ^a  

^a UNIVERSITY OF AMSTERDAM   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

DATA ANALYSIS; DISEASE COURSE; GENETIC PREDISPOSITION; HUMAN; MEASUREMENT; MEDICAL RESEARCH; PRIORITY JOURNAL; PROGNOSIS; QUALITY OF LIFE; RELIABILITY; REVIEW; SELF REPORT; SURVIVAL;

CLINICAL TRIALS AS TOPIC; GUIDELINES AS TOPIC; HUMANS; NEOPLASMS; PROGNOSIS; QUALITY OF LIFE; TREATMENT OUTCOME;

EID: 76649143747     PISSN: 0284186X     EISSN: 1651226X     Source Type: Journal    
DOI: 10.3109/02841860903440288     Document Type: Review

References (76)

1. Osoba D. The quality of life committee of the clinical trials group of the National Cancer Institute of Canada:Organization and functions. Qual Life res 1992;1:203-11.
2. Ganz PA, Bernhard J, Hurny C. Quality of life and psychosocial oncology research in Europe:State of the art. J Psychosoc Oncol 1991;9:1-22.
3. Nayfeld SG, Hailey BJ eds. Quality of life assessment in cancer clinical trials. Bethesda, MD: Division of Cancer Prevention and Control, National Cancer Institute, US. Department of Health and Human Services, 1991.
4. American Cancer Society. American Cancer Society's second workshop on methodo logy in behavioral and psychosocial cancer research. Cancer 1991;67(suppl).
5. Cella DF, Tulsky DS: Measuring quality of life today: Methodological aspects. Oncology 1990;4:29-38.
6. Siegrist J, Junge A. Conceptual and methodological problems in research on the quality of life in clinical medicine. Soc Sci Med 1989;29:463-8.
7. Patrick DL, Burke LB, Powers JH, Scott JA, Rock EP, Dawisha S, et al. Patient-reported outcomes to support medical product labeling claims:FDA perspective. Value Health 2007;10:S125-S137.
8. Rothman ML, Beltran P, Cappelleri JC, Lipscomb J, Teschendorf B, the Mayo/FDA Patient-reported Outcomes Consensus Meeting Group. Patient-reported outcomes: Conceptual issues. Value Health 2007;10:S66-S75.
9. Wagner LI, Wenzel L, Shaw E, Cella D. Patient-reported outcomes in phase II cancer clinical trials: Lessons learned and future directions. J Clin Oncol 2007;25:5058-62.
10. Goodwin PJ, Black JT, Bordeleau LJ, Ganz PA. Health-related quality-of-life measurement in randomized clinical trials in breast cancer-taking stock. J Natl Canc Inst 2003;95:263-81.
11. Blazeby JM, Kerry A, Sprangers M, Pikhart H, Fayers P, Donovan J. Health-related quality of life measurement in randomized clinical trials in surgical oncology. J Clin Oncol 2006;24:3178-86.
12. Contopoulos-Ioannidis DG, Karvouni A, Kouri I, Ioannidis JPA. Reporting and interpretation of SF-36 outcomes in randomized trials: Systematic review. Br Med J 2009;338: online.
13. Ware JE, Sherbourne CD. The MOS 36-item health survey (SF-36). Conceptual framework and item selection. Med Care 1992;30:473-83.
14. EuroQol Group. EuroQol: A new facility for the measurement of health-related quality of life. Health Policy 1990;16:199-208.
15. Cella DF, Tulsky DS, Gray G, Sarafan B, Linn E, Bonomi A, et al. The Functional Asses sment of Cancer Therapy Scale:Development and validation of the general measure. J Clin Oncol 1993;11:570-9.
16. Aaronson NK, Ahmedzai S, Bergman B, Bullinger M, Cull A, Duez Nj, et al. F for the European Organizati on for research and Treatment of Cancer Study Group on Quality of Life:The EOrTC QLQ-C30:A quality of life instrument for use in international clini cal trials in oncology. J Natl Cancer Inst 1993;85:365-7.
17. Melzack P. Pain measurement and assessment. New York:raven Press;1983.
18. Zigmund AS, Snaith R P. The hospital anxiety and depression scale. Acta Psyciatr Scand 1983;67:361-70.
19. Hahn EA, Cella D, Chassany O, Fairclough DL, Wong GY, Hays RD and the Clinical Signifcance Consensus Meeting Group. Precision of health-related quality-of-life data compared with other clinical measures. Mayo Clin Proceed 2007;82:1244-54.
20. Bjorner JB, Kosinski M, Ware Jr JE. The feasibility of applying item response theory to measures of migraine impact: A re-analysis of three clinical studies. Qual Life Res 2003;12:887-902.
21. Cella D, Wagner L, Cashy J, Hensing TA, Yount S, Lilenbaum RC. Should health-related quality of life be measured in cancer symptom management clinical trials? Lessons learned using the functional assessment of cancer therapy. J Natl Canc Inst Monogr 2007;37:53-60.
22. Fayers PM, Hopwood P, Harvey A, Girling DJ, Machin D, Stephens R. Quality of life assessment in clinical trials-guidelines and a checklist for protocol writers:The UK Medical research Council experience. MRC Cancer Trials Office. Eur J Canc 1997;33:20-8.
23. Haes de J, Curran D, Young T, Bottomley A, Flechtner H, Aaronson N, et al. for the EORTC Quality of Life Study Group. Quality of life evaluation in oncological clinical trials-the EORTC model. Eur J Canc 2000;36:821-5.
24. Sprangers MAG, Moinpour C, Moynihan T, Patrick D, Revicki D, and the Clinical Significance Consensus Meeting Group. Assessing meaningful change in quality of life over time: A users' guide for clinicians. Mayo Clin Proceed 2002;77:561-71.
25. Efficace F, Bottomley A, Osoba D, Gotay C, Flechtner H, D'haese S, et al. Beyond the development of health-related quality of life (HRQOL) measures. A checklist for evaluating HRQOL outcomes in cancer clinical trials-does HRQOL evaluation in prostate cancer research inform clinical decision-making? J Clin Oncol 2003;21:3502-11.
26. Efficace F, Osoba D, Gotay C, Sprangers M, Coens C, Bottomley A. Has the health-related quality of life reporting in clinical cancer trials improved over time? Towards bridging the gap with clinical decision making. Ann Oncol 2007;18:775-81.
27. Bottomley A, Jones D, Claassens L. Patient-reported outcomes:Assessment and current perspectives of the guidelines of the Food and Drug Administration and the refection paper of the European Medicines Agency. Eur j Cancer 2009;45:347-53.
28. Idler EL, Benyamini Y. Self-rated health and mortality: A review of 27 community studies. J Health Soc Behav 1997;38:21-37.
29. Singh JA, Nelson DB, Fink HA, Nichol KL. Health-related quality of life predicts future health-care utilization and mortality in veterans with self-reported physician-diagnosed arthritis: The veterans arthritis quality of life study. Semin Arthritis Rheum 2005;34:755-65.
30. Cunningham WE, Crystal S, Bozzette S, Hays RD. The association of health-related quality of life with survival among persons with HIV infection in the United States. J Gen Int Med 2005;20:21-7.
31. Mapes DL, Lopes AA, Satayathum S, Mccullough KP, Goodkin DA, Locatelli F, et al. Health-related quality of life as a predictor of mortality and hospitalization: The Dialysis Outcomes and Practice Patterns Study (DOPPS). Kidney Int 2003;64:339-49.
32. Gotay CC, Kawamoto CT, Bottomley A, Efficace F. The prognostic significance of patient-reported outcomes in cancer clinical trials. J Clin Oncol 2008;26:355-63.
33. Benyamini Y. Self-ratings of health and longevity: A health psychologist's viewpoint on epidemiological findings. Eur Health Psychol 2008;10:10-13.
34. Pressman SD, Cohen S. Does positive affect influence health? Psych Bull 2005;131:925-71.
35. Lydick E, Epstein RS. Interpretation of quality of life changes. Qual Life Res 1993;2:221-6.
36. Cohen J. Statistical power analysis for the behavio ral sciences. Hillsdale, New Jersey:Lawrence Erlbaum Associates; 1988.
37. jaeschke R, Singer J, Guyatt GH. Measurement of health status: Ascertaining the minimal clinically important difference. Contr Clin Trials 1989;10:407-15.
38. Junniper EF, Guyatt GH, Willan A, GriffIth LE. Determining a minimal important change in a disease-specific quality of life questionnaire. J Clin Epidem 1994;47:81-7.
39. Sloan JA, Cella D, Frost M, Guyatt G, Sprangers MAG, Symonds T and the Clinical Significance Consensus Meeting Group. Assessing clinical significance in measuring oncology patient quality of life: Introduction to the symposium, content overview, and defnition of terms. Mayo Clinic Proceed 2002;77:367-70.
40. Norman GR, Sloan JA, Wyrwich KW. Interpretation of changes in health-related quality of life. The remarkable universality of half a standard deviation. Med Care 2003;41:582-92.
41. Beaton DE. Simple as possible? Or too simple? Possible limits to the universality of the one half standard deviation. Med Care 2003;41:593-6.
42. Wright JG. Interpreting health-related quality of life scores. The simple rule of seven may not be so simple. Med Care 2003;41:597-8.
43. Farivar SS, Liu H, Hays RD. Half standard deviation estimates of the minimally important difference in HRQL scores? Exp Rev Pharmacoecon Outcom Res 2004;4:89-97.
44. Revicki D, Hays RD, Cella D, Sloan J. Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidem 2008;61:102-9.
45. Norman GR, Wyrwich KW, Patrick DW. The mathematical relationship among different forms of responsiveness coefficients. Qual Life Res 2007;16:815-22.
46. Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidem 2003;56:395-407.
47. Schwartz CE, Sprangers MAG. Methodological approaches for assessing response shift in longitudinal health-related quality-of-life research. Soc Sci Med 1999;48:1531-48.
48. Sprangers MAG, Schwartz CE. Integrating response shift into health-related quality-of-life research: A theoretical model. Soc Sci Med 1999;48:1507-15.
49. Bernhard J, Hürny C, Maibach R, Herrmann R, Laffer U. For the Swiss Group for Clinical Cancer research (SAKK). Quality-of-life as subjective experience: Reframing of perception in patients with colon cancer undergoing radical resection with or without adjuvant chemotherapy. Ann Oncol 1999;10:775-82.
50. Sprangers MAG, Schwartz CE. The challenge of response shift for quality-of-life based clinical oncology research. Ann Oncol 1999;10:747-9.
51. Barclay-Goddard R, Epstein JD, Mayo NE. response shift: A brief overview and proposed research priorities. Qual Life res 2009;18:335-46.
52. Sprangers MA, Van Dam FS, Broersen J, Lodder L, Wever L, Visser MR, et al. revealing response shift in longitudinal research on fatigue-the use of the thentest approach. Acta Oncol 1999;38:709-18.
53. Andrykowski MA, Donovan KA, Jacobsen PB. Magnitude and correlates of response shift in fatigue ratings in women undergoing adjuvant therapy for breast cancer. J Pain Sympt Manage 2009;37:341-51.
54. Broberger E, Sprangers MAG, Tishelman C. Do internal standards of quality of life change in lung cancer patients? Nurs res 2006;55:274-82.
55. Jansen SJT, Stiggelbout AM, Nooij MA, Noordijk EM, Kievit J. Response shift in quality of life measurement in early-stage breast cancer patients undergoing radiotherapy. Qual Life res 2000;9:603-15.
56. Korfage IJ, de Koning HJ, Essink-Bot ML. response shift due to diagnosis and primary treatment of localized prostate cancer: A then-test and a vignette study. Qual Life res 2007;16:1627-34.
57. rees J, Waldron D, O'Boyle C, Ewings P, MacDonagh R. The measurement of response shift in patients with advanced prostate cancer and their partners. Health Qual Life Outcom 2005;3:1-8.
58. Visser MRM, Oort FJ, Sprangers MAG. Methods to detect response shift in quality of life data:A convergent validity study. Qual Life Res 2005;14:629-39.
59. Schwartz ES, Bode R, repucci N, Becker J, Sprangers MAG, Fayers PM. The clinical significance of adaptation to changing health: A meta-analysis of response shift. Qual Life res 2006;15:1533-50.
60. Lykken D, Tellegen A. Happiness is a stochastic phenomenon. Psychol Sci 1996;7:186-9.
61. Stubbe JH, Posthuma D, Boomsma DI, De Geus EJ. Heritability of life satisfaction in adults:A twin-family study. Psychol Med 2005;35:1581-8.
62. Nes RB, røysamb E, Tambs K, Harris JR, Reichborn-Kjennerud T. Subjective well-being:Genetic and environmental contributions to stability and change. Psychol Med 2006;36:1033-42.
63. Sullivan PF, Neale MC, Kendler KS. Genetic epidemiology of major depression:review and meta-analysis. Am J Psychiat 2000;157:1552-62.
64. Hettema JM, Neale MC, Kendler KS. A review and meta-analysis of the genetic epidemiology of anxiety disorders. Am J Psychiat 2001;158:1568-78.
65. Leinonen R, Kaprio J, JylhÄ M, Tolvanen A, Koskenvuo M, Heikkinen E, et al. Genetic influences underlying self-rated health in older female twins. J Am Geriatr Soc 2005;53:1002-7.
66. Svedberg P, Gatz M, Lichtenstein P, Sandin S, Pedersen NL. Self-rated health in a longitudinal perspective: A 9-year follow-up twin study. J Gerontol B Psychol Sci Soc Sci 2005;60:S331-S340.
67. Svedberg P, Bardage C, Sandin S, Pedersen NL. A prospective study of health, life-style and psychosocial predictors of self-rated health. Eur J Epidemiol 2006;21:767-76.
68. romeis JC, Heath AC, Xian H, Eisen SA, Scherrer JF, Pedersen NL, et al. Heritability of SF-36 among middle-age, middle-class, male-male twins. Med Care 2005;43:1147-54.
69. Bao A, Meynen G, Swaab DF. The stress system in depression and neurodegeneration: Focus on the human hypothalamus. Brain Res Rev 2008;57:531-53.
70. Sprangers MAG, Sloan JA, Veenhoven R, Cleeland CS, Halyard MY, Abertnethy AP, et al. The establishment of the GENEQOL Consortium to investigate the genetic disposition of patient-reported quality-of-life outcomes. Twin res Hum Gen 2009;12:301-11.
71. Sloan J, Zhao X. Genetics and quality of life. Curr Probl Cancer 2006;30:255-60.
72. Yang P, Mandrekar SJ, Hillman SH, Allen Ziegler KL, Sun Z, Wampfer JA, et al. Evaluation of glutathione metabolic genes on outcomes in advanced non-small cell lung cancer patients after initial treatment with platinum-based chemotherapy: An NCCTG-97-24-51 based study. J Thorac Oncol 2009;4:479-85.
73. Wolff JL, Starfeld B, Anderson G. Prevalence, expenditures, and complications of multiple chronic conditions in the elderly. Arch Intern Med 2002;162:2269-76.
74. De Gargino JP. Children's health and the environment: A global perspective. Geneva: World Health Organization;2004.
75. Howell RT, Kern ML, Lyubomirsky S. Health benefits: Meta-analytically determining the impact of well-being on objective health outcomes. Health Psych Rev 2007;1:83-136.
76. Fowler JH, Christakis NA. Dynamic spread of happiness in a large social network: Longitudinal analysis over 20 years in the Framingham Heart Study. Br Med J 2008;337:a2338 (doi:10.1136/bmj.a2338).