Minimal important differences and response shift in health-related quality of life; a longitudinal study in patients with multiple myeloma

Ann K Kvam, Finn Wisløff, Peter M Fayers, Ann K Kvam, Finn Wisløff, Peter M Fayers

Abstract

Background: We previously reported that changes of 6-17 percent in the EORTC QLQ-C30 scores are regarded important by patients with multiple myeloma and thus may be considered as Minimal Important Differences (MIDs). However, patients' internal standard of measurement may have changed over time (response shift, RS). In the present work, we evaluated whether myeloma patients experience RS and if this could affect the MID-estimates.

Methods: Between 2006 and 2008, 239 patients with multiple myeloma completed the EORTC QLQ-C30 at baseline (T1) and after three months (T2). At T2, patients were asked if they had noticed any change in the domains pain, fatigue, physical function and global quality of life. They were also asked to give a retrospective judgment of their baseline values on all the four domains.

Results: We found clear evidence of RS in myeloma patients. However, there were differences in both magnitude and direction between patients who stated that they improved and those who deteriorated. Deteriorating patients retrospectively reported better health-related quality of life at T1 for the domains pain, fatigue and physical function. In these patients, MIDs adjusted for RS were observed to increase up to 12 percentage points. In contrast, for patients stating that they improved, we only found evidence of statistically significant RS in the domain global quality of life.

Conclusions: MIDs estimated from pre-test/post-test data appeared to be robust against RS in patients reporting improvement over 3-months. This could indicate that RS has a minimal impact on the results in patients who respond to treatment, and that RS may not have an important impact on interpretation of changes reported in clinical trials where an improvement occurs.Although the effect sizes of the RSs were small, RS in deteriorating patients may have an important impact on the interpretation of changes reported in clinical trials.

Trial registration: ClinicalTrials.gov NCT00290095.

Figures

Figure 1
Figure 1
Observed and adjusted scores of the EORTC QLQ-C30 for MM patients deteriorated in fatigue (n = 58). The patients evaluated their fatigue in retrospect as better (less fatigue) than they did at T1. The difference between the pre-test and then-test score is the response shift effect.

References

    1. Howard GS, Ralph KM, Gulanick NA, Maxwell SE, Nance DW, Gerber SK. Internal Invalidity in Pretest-Posttest Self-Report Evaluations and a Re-evaluation of Retrospective Pretests. Applied Psychological Measurement. 1979;3:1–23. doi: 10.1177/014662167900300101.
    1. Sprangers MA, Schwartz CE. Integrating response shift into health-related quality of life research: a theoretical model. Soc Sci Med. 1999;48:1507–15. doi: 10.1016/S0277-9536(99)00045-3.
    1. Schwartz CE, Sprangers MA. Methodological approaches for assessing response shift in longitudinal health-related quality-of-life research. Soc Sci Med. 1999;48:1531–48. doi: 10.1016/S0277-9536(99)00047-7.
    1. Visser MR, Smets EM, Sprangers MA, de Haes HJ. How response shift may affect the measurement of change in fatigue. J Pain Symptom Manage. 2000;20:12–8. doi: 10.1016/S0885-3924(00)00148-2.
    1. Jansen SJ, 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. doi: 10.1023/A:1008928617014.
    1. Sharpe L, Butow P, Smith C, McConnell D, Clarke S. Changes in quality of life in patients with advanced cancer: evidence of response shift and response restriction. J Psychosom Res. 2005;58:497–504. doi: 10.1016/j.jpsychores.2005.02.017.
    1. Hagedoorn M, Sneeuw KC, Aaronson NK. Changes in physical functioning and quality of life in patients with cancer: response shift and relative evaluation of one's condition. J Clin Epidemiol. 2002;55:176–83. doi: 10.1016/S0895-4356(01)00438-3.
    1. Bernhard J, Hurny C, Maibach R, Herrmann R, Laffer U. Quality of life as subjective experience: reframing of perception in patients with colon cancer undergoing radical resection with or without adjuvant chemotherapy. Swiss Group for Clinical Cancer Research (SAKK) Ann Oncol. 1999;10:775–82. doi: 10.1023/A:1008311918967.
    1. Rees J, Waldron D, O'Boyle C, Ewings P, MacDonagh R. Prospective vs retrospective assessment of lower urinary tract symptoms in patients with advanced prostate cancer: the effect of 'response shift'. BJU Int. 2003;92:703–6. doi: 10.1046/j.1464-410X.2003.04462.x.
    1. 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 Symptom Manage. 2009;37:341–51. doi: 10.1016/j.jpainsymman.2008.03.015.
    1. Schwartz CE, Bode R, Repucci N, Becker J, Sprangers MA, Fayers PM. The clinical significance of adaptation to changing health: a meta-analysis of response shift. Qual Life Res. 2006;15:1533–50. doi: 10.1007/s11136-006-0025-9.
    1. Kvam AK, Fayers P, Wisloff F. What changes in health-related quality of life matter to multiple myeloma patients? A prospective study. Eur J Haemato. 2010;84:345–53. doi: 10.1111/j.1600-0609.2009.01404.x.
    1. Sirohi B, Powles R. Epidemiology and outcomes research for MGUS, myeloma and amyloidosis. Eur J Cancer. 2006;42:1671–83. doi: 10.1016/j.ejca.2006.01.065.
    1. Gulbrandsen N, Hjermstad MJ, Wisloff F. Interpretation of quality of life scores in multiple myeloma by comparison with a reference population and assessment of the clinical importance of score differences. Eur J Haematol. 2004;72:172–80. doi: 10.1046/j.0902-4441.2003.00195.x.
    1. Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10:407–15. doi: 10.1016/0197-2456(89)90005-6.
    1. Aaronson NK, Ahmedzai S, Bergman B, Bullinger M, Cull A, Duez NJ, Filiberti A, Flechtner H, Fleishman SB, de Haes JC. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst. 1993;85:365–76. doi: 10.1093/jnci/85.5.365.
    1. Fayers PM, Aaronson NK, Bjordal K, Groenvold M, Curran D, Bottomley A on behalf of the EORTC Quality of life group. The EORTC QLQ-C30 Scoring Manual. 3. European Organisation for Research and Treatment of Cancer, Brussels; 2001.
    1. Wisloff F, Eika S, Hippe E, Hjorth M, Holmberg E, Kaasa S, Palva I, Westin J. Measurement of health-related quality of life in multiple myeloma. Nordic Myeloma Study Group. Br J Haematol. 1996;92:604–13. doi: 10.1046/j.1365-2141.1996.352889.x.
    1. Cohen J. Statistical power analysis for the behavioral sciences. Hillsdale, N. J.: Laurence Erlbaum; 1988.
    1. Fayers PM, Machin D. Quality of life: the assessment, analysis, and interpretation of patient-reported outcomes. Chichester: J. Wiley; 2007.
    1. Sprangers MA, Van Dam FS, Broersen J, Lodder L, Wever L, Visser MR, Oosterveld P, Smets EM. Revealing response shift in longitudinal research on fatigue--the use of the thentest approach. Acta Oncol. 1999;38:709–18. doi: 10.1080/028418699432824.
    1. Gulbrandsen N, Wisloff F, Brinch L, Carlson K, Dahl IM, Gimsing P, Hippe E, Hjorth M, Knudsen LM, Lamvik J, Lenhoff S, Lofvenberg E, Nesthus I, Nielsen JL, Turesson I, Westin J. Health-related quality of life in multiple myeloma patients receiving high-dose chemotherapy with autologous blood stem-cell support. Med Oncol. 2001;18:65–77. doi: 10.1385/MO:18:1:65.
    1. Dubois D, Dhawan R, van d V, Esseltine D, Gupta S, Viala M, de la Loge C. Descriptive and prognostic value of patient-reported outcomes: the bortezomib experience in relapsed and refractory multiple myeloma. J Clin Oncol. 2006;24:976–82. doi: 10.1200/JCO.2005.04.0824.
    1. Ring L, Hofer S, Heuston F, Harris D, O'Boyle CA. Response shift masks the treatment impact on patient reported outcomes (PROs): the example of individual quality of life in edentulous patients. Health Qual Life Outcomes. 2005;3:55–62. doi: 10.1186/1477-7525-3-55.
    1. Razmjou H, Schwartz CE, Yee A, Finkelstein JA. Traditional assessment of health outcome following total knee arthroplasty was confounded by response shift phenomenon. J Clin Epidemiol. 2009;62:91–6. doi: 10.1016/j.jclinepi.2008.08.004.
    1. Vogelzang NJ, Breitbart W, Cella D, Curt GA, Groopman JE, Horning SJ, Itri LM, Johnson DH, Scherr SL, Portenoy RK. Patient, caregiver, and oncologist perceptions of cancer-related fatigue: results of a tripart assessment survey. The Fatigue Coalition. Seminars in Hematology. 1997;34(3 Suppl 2):4–12.
    1. Norman G. Hi! How are you? Response shift, implicit theories and differing epistemologies. Qual Life Res. 2003;12:239–49. doi: 10.1023/A:1023211129926.
    1. Fayers PM, Hays RD. Assessing quality of life in clinical trials: methods and practice. Oxford: Oxford University Press; 2005.
    1. Visser MR, Oort FJ, Sprangers MA. Methods to detect response shift in quality of life data: a convergent validity study. Qual Life Res. 2005;14:629–39. doi: 10.1007/s11136-004-2577-x.
    1. Johnsen AT, Tholstrup D, Petersen MA, Pedersen L, Groenvold M. Health related quality of life in a nationally representative sample of haematological patients. Eur J Haematol. 2009;83:139–48. doi: 10.1111/j.1600-0609.2009.01250.x.
    1. Bottomley A, Therasse P, Piccart M, Efficace F, Coens C, Gotay C, Welnicka-Jaskiewicz M, Mauriac L, Dyczka J, Cufer T, Lichinitser MR, Schornagel JH, Bonnefoi H, Shepherd L. Health-related quality of life in survivors of locally advanced breast cancer: an international randomised controlled phase III trial. Lancet Oncol. 2005;6:287–94. doi: 10.1016/S1470-2045(05)70100-5.
    1. Zenger M, Hinz A, Stolzenburg JU, Rabenalt R, Schwalenberg T, Schwarz R. Health-related quality of life of prostate cancer patients compared to the general German population: age-specific results. Urol Int. 2009;83:166–70. doi: 10.1159/000230018.
    1. Turesson I, Velez R, Kristinsson SY, Landgren O. Patterns of Improved Survival in Patients With Multiple Myeloma in the Twenty-First Century: A Population-Based Study. J Clin Oncol. 2010;28:830–4. doi: 10.1200/JCO.2009.25.4177.

Source: PubMed

Patrocinadores y Colaboradores

Condiciones médicas

Intervenciones de drogas

3
Suscribir