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Methodological issues in designing and reporting health-related quality of life in cancer clinical trials: the challenge of brain cancer studies

  • Methods and Clinical Tools for Outcome Assessments
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Abstract

Health-related quality of life (HRQOL) and other types of patient-reported outcomes (PROs) are now important outcome measures in cancer clinical trials. A number of potentially less toxic drugs are available, and newer treatments can potentially offer cancer patients the possibility to be treated with less aggressive approaches, making PROs more critical in evaluating treatment effectiveness. However, assessing PROs in clinical trials requires careful consideration of a number of methodological issues. Robust methodology and accurate reporting of results are crucial to provide the scientific community and health care providers with a transparent message about the impact of a given drug or a new medical approach on patients’ health status. This paper provides basic guidance on methodological issues to be addressed when designing and reporting HRQOL in clinical trials and presents examples of relevant brain cancer studies.

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References

  1. Moher D, Jones A, Lepage L (2001) Use of the CONSORT statement and quality of reports of randomized trials: a comparative before-and-after evaluation. JAMA 285:1992–1995

    Article  PubMed  CAS  Google Scholar 

  2. Portney LG, Watkins MP (eds) (2000) Foundations of clinical research: applications to practice. Prentice Hall, Upper Saddle River

    Google Scholar 

  3. Osoba D (1994) Lessons learned from measuring health-related quality of life in oncology. J Clin Oncol 12:608–616

    PubMed  CAS  Google Scholar 

  4. Schumacher M, Olschewski M, Schulgen G (1991) Assessment of quality of life in clinical trials. Stat Med 10:1915–1930

    Article  PubMed  CAS  Google Scholar 

  5. Osoba D (2007) Translating the science of patient-reported outcomes assessment into clinical practice. J Natl Cancer Inst Monogr 37:5–11

    Article  PubMed  Google Scholar 

  6. US Food and Drug Administration (2009) Guidance for industry. patient-reported outcome measures: use in medical product development to support labeling claims. U.S. Department of Health and Human Services Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Accessed 13 Feb 2012

  7. Bottomley A, Therasse P (2002) Quality of life in patients undergoing systemic therapy for advanced breast cancer. Lancet Oncol 3:620–628

    Article  PubMed  Google Scholar 

  8. Bottomley A, Efficace F, Thomas R, Vanvoorden V, Ahmedzai S (2003) Health-related quality of life in non small-cell lung cancer: methodologic issues in randomized controlled trials. J Clin Oncol 21:2982–2992

    Article  PubMed  Google Scholar 

  9. Efficace F, Bottomley A, Vanvoorden V, Blazeby JM (2004) Methodological issues in assessing health-related quality of life of colorectal cancer patients in randomized controlled trials. Eur J Cancer 40:187–197

    Article  PubMed  CAS  Google Scholar 

  10. Efficace F, Bottomley A, van Andel G (2003) Health-related quality of life in prostate carcinoma patients: a systematic review of randomized controlled trials. Cancer 97:377–388

    Article  PubMed  Google Scholar 

  11. Fitzpatrick R, Davey C, Buxton MJ et al (1998) Evaluating patient-based outcome measures for use in clinical trials. Health Technol Assess 2:1–74

    PubMed  CAS  Google Scholar 

  12. Fayers P, Hays R (2005) Assessing quality of life in clinical trials. Oxford University Press, New York

    Google Scholar 

  13. de Haes J, Curran D, Young T, Bottomley A, Flechtner H, Aaronson N, Blazeby J, EORTC Quality of Life Study Group (2000) Quality of life evaluation in oncological clinical trials–the EORTC model. Eur J Cancer 36:821–825

    Article  PubMed  Google Scholar 

  14. Fayers PM, Hopwood P, Harvey A et al (1997) Quality of life assessment in clinical trials–guidelines and a checklist for protocol writers: the U.K. Medical Research Council experience. MRC Cancer Trials Office. Eur J Cancer 33:20–28

    Article  PubMed  CAS  Google Scholar 

  15. Sloan J, Symonds T, Vargas-Chanes D et al (2003) Practical guidelines for assessing the clinical significance of health-related quality of life changes within clinical trials. Drug Inf J 37:23–32

    Article  Google Scholar 

  16. Osoba D, Rodrigues G, Myles J et al (1998) Interpreting the significance of changes in health-related quality-of-life scores. J Clin Oncol 16:139–144

    PubMed  CAS  Google Scholar 

  17. Gotay CC, Korn EL, McCabe MS et al (1992) Quality-of-life assessment in cancer treatment protocols: research issues in protocol development. J Natl Cancer Inst 84:575–579

    Article  PubMed  CAS  Google Scholar 

  18. Chassany O, Sagnier P, Marquis P et al (2002) Patient-reported outcomes: the example of health related quality of life-a European guidance document for the improved integration of health related quality of life assessment in the drug regulatory process. Drug Inf J 36:209–238

    Article  Google Scholar 

  19. Efficace F, Bottomley A, Osoba D et al (2003) 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 21:3502–3511

    Article  PubMed  Google Scholar 

  20. Efficace F, Osoba D, Gotay C, Sprangers M, Coens C, Bottomley A (2007) Has the quality of health-related quality of life reporting in cancer clinical trials improved over time? Towards bridging the gap with clinical decision making. Ann Oncol 18:775–781

    Article  PubMed  CAS  Google Scholar 

  21. Cocks K, King MT, Velikova G et al (2008) Quality, interpretation and presentation of European Organisation for Research and Treatment of Cancer quality of life questionnaire core 30 data in randomised controlled trials. Eur J Cancer 44:1793–1798

    Article  PubMed  Google Scholar 

  22. van Meerbeeck JP, Gaafar R, Manegold C et al (2005) Randomized phase III study of cisplatin with or without raltitrexed in patients with malignant pleural mesothelioma: an intergroup study of the European Organisation for Research and Treatment of Cancer Lung Cancer Group and the National Cancer Institute of Canada. J Clin Oncol 23:6881–6889

    Article  PubMed  Google Scholar 

  23. Blazeby JM, Avery K, Sprangers M et al (2006) Health-related quality of life measurement in randomized clinical trials in surgical oncology. J Clin Oncol 24:3178–3186

    Article  PubMed  Google Scholar 

  24. Taphoorn MJ, Sizoo EM, Bottomley A (2010) Review on quality of life issues in patients with primary brain tumors. Oncologist 15:618–626

    Article  PubMed  Google Scholar 

  25. Henriksson R, Asklund T, Poulsen HS (2011) Impact of therapy on quality of life, neurocognitive function and their correlates in glioblastoma multiforme: a review. J Neurooncol 104:639–646

    Article  PubMed  CAS  Google Scholar 

  26. Stupp R, Mason WP, van den Bent MJ et al (2005) Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med 352:987–996

    Article  PubMed  CAS  Google Scholar 

  27. Brandsma D, van den Bent MJ (2007) Molecular targeted therapies and chemotherapy in malignant gliomas. Curr Opin Oncol 19:598–605

    Article  PubMed  CAS  Google Scholar 

  28. Taphoorn MJ, Stupp R, Coens C et al (2005) Health-related quality of life in patients with glioblastoma: a randomised controlled trial. Lancet Oncol 6:937–944

    Article  PubMed  Google Scholar 

  29. Aaronson NK, Ahmedzai S, Bergman B et al (1993) 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 85:365–376

    Article  PubMed  CAS  Google Scholar 

  30. Taphoorn MJ, Claassens L, Aaronson NK et al (2010) An international validation study of the EORTC brain cancer module (EORTC QLQ BN-20) for assessing health-related quality of life and symptoms in brain cancer patients. Eur J Cancer 46:1033–1040

    Article  PubMed  Google Scholar 

  31. Weitzner MA, Meyers CA, Gelke CK et al (1995) The functional assessment of cancer therapy (FACT) scale. Development of a brain subscale and revalidation of the general version (FACT-G) in patients with primary brain tumors. Cancer 75:1151–1161

    Article  PubMed  CAS  Google Scholar 

  32. Armstrong TS, Mendoza T, Gring I et al (2006) Validation of the M.D. Anderson symptom inventory brain tumor module (MDASI-BT). J Neurooncol 80:27–35

    Article  PubMed  CAS  Google Scholar 

  33. Armstrong TS, Gring I, Mendoza T et al (2009) Clinical utility of the MDASI-BT in patients with brain metastases. J Pain Symptom Manag 37:331–340

    Article  Google Scholar 

  34. Sneeuw KC, Sprangers MA, Aaronson NK (2002) The role of health care providers and significant others in evaluating the quality of life of patients with chronic disease. J Clin Epidemiol 55:1130–1143

    Article  PubMed  Google Scholar 

  35. Brown PD, Decker PA, Rummans TA et al (2008) A prospective study of quality of life in adults with newly diagnosed high-grade gliomas: comparison of patient and caregiver ratings of quality of life. Am J Clin Oncol 31:163–168

    Article  PubMed  Google Scholar 

  36. Taphoorn MJ, van den Bent MJ, Mauer ME et al (2007) Health-related quality of life in patients treated for anaplastic oligodendroglioma with adjuvant chemotherapy: results of a European Organisation for Research and Treatment of Cancer randomized clinical trial. J Clin Oncol 25:5723–5730

    Article  PubMed  Google Scholar 

  37. Keime-Guibert F, Chinot O, Taillandier L et al (2007) Radiotherapy for glioblastoma in the elderly. N Engl J Med 356:1527–1535

    Article  PubMed  CAS  Google Scholar 

  38. van den Bent MJ, Carpentier AF, Brandes AA et al (2006) Adjuvant procarbazine, lomustine, and vincristine improves progression-free survival but not overall survival in newly diagnosed anaplastic oligodendrogliomas and oligoastrocytomas: a randomized European Organisation for Research and Treatment of Cancer phase III trial. J Clin Oncol 24:2715–2722

    Article  PubMed  Google Scholar 

  39. Velikova G, Awad N, Coles-Gale R et al (2008) The clinical value of quality of life assessment in oncology practice-a qualitative study of patient and physician views. Psychooncology 17:690–698

    Article  PubMed  Google Scholar 

  40. Mauer M, Taphoorn MJB, Bottomley A et al (2007) The prognostic value of health-related quality of life data in predicting survival in anaplastic oligodendrogliomas cancer patients: results from an international randomized phase III EORTC Brain Cancer Group study. J Clin Oncol 25:5731–5737

    Article  PubMed  Google Scholar 

  41. Mauer M, Stupp R, Taphoorn MJ et al (2007) The prognostic value of health-related quality-of-life data in predicting survival in glioblastoma cancer patients: results from an international randomised phase III EORTC Brain Tumor and Radiation Oncology Groups, and NCIC Clinical Trials Group study. Br J Cancer 97:302–307

    Article  PubMed  CAS  Google Scholar 

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Conflict of interest

The authors declare that they have no conflict of interest.

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Authors and Affiliations

  1. Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), GIMEMA Data Center, Via Benevento, 600161, Rome, Italy

    Fabio Efficace

  2. Department of Neurology, VU University Medical Center Amsterdam, Amsterdam, The Netherlands

    Martin Taphoorn

  3. Department of Neurology, Medical Center Haaglanden, The Hague, The Netherlands

    Martin Taphoorn

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  1. Fabio Efficace
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  2. Martin Taphoorn
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Correspondence to Fabio Efficace.

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Efficace, F., Taphoorn, M. Methodological issues in designing and reporting health-related quality of life in cancer clinical trials: the challenge of brain cancer studies. J Neurooncol 108, 221–226 (2012). https://doi.org/10.1007/s11060-012-0819-2

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  • DOI: https://doi.org/10.1007/s11060-012-0819-2

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