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Measuring Clinical Benefit: Use of Patient-Reported Outcomes (PRO) in Primary Brain Tumor Clinical Trials

  • Neuro-oncology (MR Gilbert, Section Editor)
  • Published:
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Abstract

Primary brain tumors and their treatment are associated with a significant impact on function and quality of life (QOL). Patient-reported outcomes (PROs) are measures that allow report of the impact directly from the patient. Instruments to measure both QOL and symptom burden have been developed for use in the primary brain tumor patient population. Use of these instruments coupled with tumor response assessment and other objective measures will allow for evaluation of the net clinical benefit for the patient.

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References

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

  1. Armstrong TS. Head's up on the treatment of malignant glioma patients. Oncol Nurs Forum. 2009;36:E232–40.

    Article  PubMed  Google Scholar 

  2. Cahill JE, Armstrong TS. Caring for an adult with a malignant primary brain tumor. Nursing. 2011;41:28–33. quiz 33-4.

    PubMed  Google Scholar 

  3. Armstrong TS, Vera-Bolanos E, Gilbert MR. Clinical course of adult patients with ependymoma: results of the Adult Ependymoma Outcomes Project. Cancer. 2011;117:5133–41.

    Article  PubMed  Google Scholar 

  4. Bradley SE, Sherwood PR, Kuo J, et al. Perceptions of economic hardship and emotional health in a pilot sample of family caregivers. J Neurooncol. 2009;93:333–42.

    Article  PubMed  Google Scholar 

  5. Mackworth N, Fobair P, Prados MD. Quality of life self-reports from 200 brain tumor patients: comparisons with Karnofsky performance scores. J Neurooncol. 1992;14:243–53.

    Article  PubMed  CAS  Google Scholar 

  6. Salander P, Bergenheim AT, Henriksson R. How was life after treatment of a malignant brain tumour? Soc Sci Med. 2000;51:589–98.

    Article  PubMed  CAS  Google Scholar 

  7. Strang S, Strang P. Spiritual thoughts, coping and 'sense of coherence' in brain tumour patients and their spouses. Palliat Med. 2001;15:127–34.

    Article  PubMed  CAS  Google Scholar 

  8. Strang S, Strang P, Ternestedt BM. Existential support in brain tumour patients and their spouses. Support Care Cancer. 2001;9:625–33.

    Article  PubMed  CAS  Google Scholar 

  9. Bradley S, Sherwood PR, Donovan HS, et al. I could lose everything: understanding the cost of a brain tumor. J Neurooncol. 2007;85:329–38.

    Article  PubMed  Google Scholar 

  10. • Stupp R, Mason WP, van den Bent MJ, et al. Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med. 2005;352:987–96. Important paper establishing level 1 evidence of the impact of chemo-radiation on outcome in GBM.

    Article  PubMed  CAS  Google Scholar 

  11. Stupp R, Hegi ME, Mason WP, et al. Effects of radiotherapy with concomitant and adjuvant temozolomide versus radiotherapy alone on survival in glioblastoma in a randomised phase III study: 5-year analysis of the EORTC-NCIC trial. Lancet Oncol. 2009;10:459–66.

    Article  PubMed  CAS  Google Scholar 

  12. Mauer ME, Bottomley A, Taphoorn MJ. Evaluating health-related quality of life and symptom burden in brain tumour patients: instruments for use in experimental trials and clinical practice. Curr Opin Neurol. 2008;21:745–53.

    Article  PubMed  Google Scholar 

  13. Lamborn KR, Yung WK, Chang SM, et al. Progression-free survival: an important end point in evaluating therapy for recurrent high-grade gliomas. Neuro Oncol. 2008;10:162–70.

    Article  PubMed  Google Scholar 

  14. Polley MY, Lamborn KR, Chang SM, et al. Six-month progression-free survival as an alternative primary efficacy endpoint to overall survival in newly diagnosed glioblastoma patients receiving temozolomide. Neuro Oncol. 2010;12:274–82.

    Article  PubMed  CAS  Google Scholar 

  15. Quant EC, Wen PY. Response assessment in neuro-oncology. Curr Oncol Rep. 2011;13:50–6.

    Article  PubMed  Google Scholar 

  16. Meyers CA, Rock EP, Fine HA. Refining endpoints in brain tumor clinical trials. J Neurooncol. 2012;108:227–30.

    Article  PubMed  Google Scholar 

  17. Wen PY, Macdonald DR, Reardon DA, et al. Updated response assessment criteria for high-grade gliomas: response assessment in neuro-oncology working group. J Clin Oncol. 2010;28:1963–72.

    Article  PubMed  Google Scholar 

  18. Chamberlain MC. Pseudoprogression in glioblastoma. J Clin Oncol. 2008;26:4359. author reply 4359-60.

    Article  PubMed  Google Scholar 

  19. Fink J, Born D, Chamberlain MC. Pseudoprogression: relevance with respect to treatment of high-grade gliomas. Curr Treat Options Oncol. 2011;12:240–52.

    Article  PubMed  Google Scholar 

  20. Norden AD, Young GS, Setayesh K, et al. Bevacizumab for recurrent malignant gliomas: efficacy, toxicity, and patterns of recurrence. Neurology. 2008;70:779–87.

    Article  PubMed  CAS  Google Scholar 

  21. Chamberlain MC. MRI in patients with high-grade gliomas treated with bevacizumab and chemotherapy. Neurology. 2006;67:2089. author reply 2089.

    Article  PubMed  Google Scholar 

  22. Lucas J, Zada G. Radiology: criteria for determining response to treatment and recurrence of high-grade gliomas. Neurosurg Clin N Am. 2012;23:269–76. viii.

    Article  PubMed  Google Scholar 

  23. Lassman AB, Holland EC. Incorporating molecular tools into clinical trials and treatment for gliomas? Curr Opin Neurol. 2007;20:708–11.

    Article  PubMed  CAS  Google Scholar 

  24. Nelson SJ. Assessment of therapeutic response and treatment planning for brain tumors using metabolic and physiological MRI. NMR Biomed. 2011;24:734–49.

    PubMed  Google Scholar 

  25. •• Arpinelli F, Bamfi F. The FDA guidance for industry on PROs: the point of view of a pharmaceutical company. Health Qual Life Outcomes. 2006;4:85. Paper outlining FDA position on the use of PRO instruments.

    Article  PubMed  Google Scholar 

  26. DeMuro C, Clark M, Mordin M, et al. Reasons for rejection of patient-reported outcome label claims: a compilation based on a review of patient-reported outcome use among new molecular entities and biologic license applications, 2006-2010. Value Health. 2012;15:443–8.

    Article  PubMed  Google Scholar 

  27. Gnanasakthy A, Mordin M, Clark M, et al. A review of patient-reported outcome labels in the United States: 2006 to 2010. Value Health. 2012;15:437–42.

    Article  PubMed  Google Scholar 

  28. Rock EP, Kennedy DL, Furness MH, et al. Patient-reported outcomes supporting anticancer product approvals. J Clin Oncol. 2007;25:5094–9.

    Article  PubMed  CAS  Google Scholar 

  29. Patrick DL, Burke LB, Powers JH, et al. Patient-reported outcomes to support medical product labeling claims: FDA perspective. Value Health. 2007;10 Suppl 2:S125–37.

    Article  PubMed  Google Scholar 

  30. Galanis E, Wu W, Cloughesy T, et al. Phase 2 trial design in neuro-oncology revisited: a report from the RANO group. Lancet Oncol. 2012;13:e196–204.

    Article  PubMed  Google Scholar 

  31. • Armstrong TS, Mendoza T, Gning I, et al. Validation of the M.D. Anderson Symptom Inventory Brain Tumor Module (MDASI-BT). J Neurooncol. 2006;80:27–35. PRO instrument developed for report of symtpom burden in brain tumor patients.

    Article  PubMed  CAS  Google Scholar 

  32. Armstrong TS, Cohen MZ, Eriksen L, et al. Content validity of self-report measurement instruments: an illustration from the development of the Brain Tumor Module of the M.D. Anderson Symptom Inventory. Oncol Nurs Forum. 2005;32:669–76.

    Article  PubMed  Google Scholar 

  33. Armstrong TS, Gning I, Mendoza TR, et al. Clinical utility of the MDASI-BT in patients with brain metastases. J Pain Symptom Manag. 2009;37:331–40.

    Article  Google Scholar 

  34. Armstrong TS, Vera-Bolanos E, Gning I, et al. The impact of symptom interference using the MD Anderson Symptom Inventory-Brain Tumor Module (MDASI-BT) on prediction of recurrence in primary brain tumor patients. Cancer. 2011;117:3222–8.

    Article  PubMed  Google Scholar 

  35. Lien K, Zeng L, Nguyen J, et al. FACT-Br for assessment of quality of life in patients receiving treatment for brain metastases: a literature review. Expert Rev Pharmacoecon Outcomes Res. 2011;11:701–8.

    Article  PubMed  Google Scholar 

  36. • Weitzner MA, Meyers CA, Gelke CK, et al. 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. 1995;75:1151–61. Validation of Quality of life instrument for primary brain tumor patients.

    Article  PubMed  CAS  Google Scholar 

  37. • Taphoorn MJ, Claassens L, Aaronson NK, et al. An international validation study of the EORTC brain cancer module (EORTC QLQ-BN20) for assessing health-related quality of life and symptoms in brain cancer patients. Eur J Cancer. 2010;46:1033–40. Validation of Quality of life instrument for primary brain tumor patients.

    Article  PubMed  Google Scholar 

  38. Mauer M, Stupp R, Taphoorn MJ, et al. 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 Tumour and Radiation Oncology Groups, and NCIC Clinical Trials Group study. Br J Cancer. 2007;97:302–7.

    Article  PubMed  CAS  Google Scholar 

  39. Mauer ME, Taphoorn MJ, Bottomley A, et al. Prognostic value of health-related quality-of-life data in predicting survival in patients with anaplastic oligodendrogliomas, from a phase III EORTC brain cancer group study. J Clin Oncol. 2007;25:5731–7.

    Article  PubMed  Google Scholar 

  40. • Cleeland CS, Sloan JA. Assessing the Symptoms of Cancer Using Patient-Reported Outcomes (ASCPRO): searching for standards. J Pain Symptom Manag. 2010;39:1077–85. Paper reviewing position of ASCPRO committee on issues related to standards for use of PROs.

    Article  Google Scholar 

  41. Reyes-Gibby CC, Aday L, Cleeland C. Impact of pain on self-rated health in the community-dwelling older adults. Pain. 2002;95:75–82.

    Article  PubMed  Google Scholar 

  42. Schwartz CE, Bode R, Repucci N, et al. The clinical significance of adaptation to changing health: a meta-analysis of response shift. Qual Life Res. 2006;15:1533–50.

    Article  PubMed  Google Scholar 

  43. Tierney DK, Facione N, Padilla G, et al. Response shift: a theoretical exploration of quality of life following hematopoietic cell transplantation. Cancer Nurs. 2007;30:125–38.

    Article  PubMed  Google Scholar 

  44. Armstrong TS, Cron SG, Bolanos EV, et al. Risk factors for fatigue severity in primary brain tumor patients. Cancer. 2010;116:2707–15.

    PubMed  Google Scholar 

  45. Fox SW, Lyon D, Farace E. Symptom clusters in patients with high-grade glioma. J Nurs Scholarsh. 2007;39:61–7.

    Article  PubMed  Google Scholar 

  46. Gleason Jr JF, Case D, Rapp SR, et al. Symptom clusters in patients with newly-diagnosed brain tumors. J Support Oncol. 2007;5:427–33. 436.

    PubMed  Google Scholar 

  47. Cleeland CS: Symptom burden: multiple symptoms and their impact as patient-reported outcomes. J Natl Cancer Inst Monogr:16-21, 2007

  48. Armstrong TS, Wefel JS, Won M, et al: Abstract 2016: Clinical utility of neurocognitive function (NCF), quality of life (QOL), and symptom assessment as prognostic factors for survival and measures of treatment effects on RTOG 0525. Journal of Clinical Oncology 29 2011

  49. Witgert ME, Meyers CA. Neurocognitive and quality of life measures in patients with metastatic brain disease. Neurosurg Clin N Am. 2011;22:79–85. vii.

    Article  PubMed  Google Scholar 

  50. Nekolaichuk CL, Maguire TO, Suarez-Almazor M, et al. Assessing the reliability of patient, nurse, and family caregiver symptom ratings in hospitalized advanced cancer patients. J Clin Oncol. 1999;17:3621–30.

    PubMed  CAS  Google Scholar 

  51. Nekolaichuk CL, Bruera E, Spachynski K, et al. A comparison of patient and proxy symptom assessments in advanced cancer patients. Palliat Med. 1999;13:311–23.

    Article  PubMed  CAS  Google Scholar 

  52. Teske K, Daut RL, Cleeland CS. Relationships between nurses' observations and patients' self-reports of pain. Pain. 1983;16:289–96.

    Article  PubMed  CAS  Google Scholar 

  53. Parsaie FA, Golchin M, Asvadi I. A comparison of nurse and patient perceptions of chemotherapy treatment stressors. Cancer Nurs. 2000;23:371–4.

    Article  PubMed  CAS  Google Scholar 

  54. Puntillo K, Neighbor M, O'Neil N, et al. Accuracy of emergency nurses in assessment of patients' pain. Pain Manag Nurs. 2003;4:171–5.

    Article  PubMed  Google Scholar 

  55. Barsevick AM, Cleeland CS, Manning DC, et al. ASCPRO recommendations for the assessment of fatigue as an outcome in clinical trials. J Pain Symptom Manag. 2010;39:1086–99.

    Article  Google Scholar 

  56. Cella D, Bullinger M, Scott C, et al. Group vs individual approaches to understanding the clinical significance of differences or changes in quality of life. Mayo Clin Proc. 2002;77:384–92.

    Article  PubMed  Google Scholar 

  57. Tait RC, Chibnall JT, Kalauokalani D. Provider judgments of patients in pain: seeking symptom certainty. Pain Med. 2009;10:11–34.

    Article  PubMed  Google Scholar 

  58. Sprangers MA, Aaronson NK. The role of health care providers and significant others in evaluating the quality of life of patients with chronic disease: a review. J Clin Epidemiol. 1992;45:743–60.

    Article  PubMed  CAS  Google Scholar 

  59. Davies E, Clarke C. Views of bereaved relatives about quality of survival after radiotherapy for malignant cerebral glioma. J Neurol Neurosurg Psychiatry. 2005;76:555–61.

    Article  PubMed  CAS  Google Scholar 

  60. Armstrong TS, Wefel JS, Gning I, et al: Congruence of primary brain tumor patient and caregiver symptom report. Cancer, 2012

  61. Brown PD, Decker PA, Rummans TA, et al. 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. 2008;31:163–8.

    Article  PubMed  Google Scholar 

  62. Cleeland CS. Cancer-related symptoms. Semin Radiat Oncol. 2000;10:175–90.

    Article  PubMed  CAS  Google Scholar 

  63. Corkrey R, Parkinson L. Interactive voice response: review of studies 1989-2000. Behav Res Methods Instrum Comput. 2002;34:342–53.

    Article  PubMed  Google Scholar 

  64. Corkrey R, Parkinson L. A comparison of four computer-based telephone interviewing methods: getting answers to sensitive questions. Behav Res Methods Instrum Comput. 2002;34:354–63.

    Article  PubMed  Google Scholar 

  65. Lustria ML, Cortese J, Noar SM, et al. Computer-tailored health interventions delivered over the Web: review and analysis of key components. Patient Educ Couns. 2009;74:156–73.

    Article  PubMed  Google Scholar 

  66. Dumrongpakapakorn P, Hopkins K, Sherwood P, et al. Computer-mediated patient education: opportunities and challenges for supporting women with ovarian cancer. Nurs Clin North Am. 2009;44:339–54.

    Article  PubMed  Google Scholar 

  67. Alvina AA, Gilbert MR, Armstrong TS: Feasibility of Use of Electronic Data Capture in Primary Brain Tumor Patients Neuro-Oncology, In Press

  68. Snyder CF, Watson ME, Jackson JD, et al. Patient-reported outcome instrument selection: designing a measurement strategy. Value Health. 2007;10 Suppl 2:S76–85.

    Article  PubMed  Google Scholar 

  69. Burris H, Storniolo AM. Assessing clinical benefit in the treatment of pancreas cancer: gemcitabine compared to 5-fluorouracil. Eur J Cancer. 1997;33 Suppl 1:S18–22.

    Article  PubMed  CAS  Google Scholar 

  70. Burris 3rd HA, Moore MJ, Andersen J, et al. Improvements in survival and clinical benefit with gemcitabine as first-line therapy for patients with advanced pancreas cancer: a randomized trial. J Clin Oncol. 1997;15:2403–13.

    PubMed  CAS  Google Scholar 

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Armstrong, T.S. Measuring Clinical Benefit: Use of Patient-Reported Outcomes (PRO) in Primary Brain Tumor Clinical Trials. Curr Oncol Rep 15, 27–32 (2013). https://doi.org/10.1007/s11912-012-0276-2

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