Development and Validation of 11 Symptom Indexes to Evaluate Response to Chemotherapy for Advanced Cancer

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Recent guidance from the FDA discusses patient-reported outcomes as end points in clinical trials. Using methods consistent with this guidance, the authors developed symptom indexes for patients with advanced cancer. Input on the most important symptoms was obtained from 533 patients recruited from NCCN Member Institutions and 4 nonprofit social service organizations. Diagnoses included bladder, brain, breast, colorectal, head and neck, hepatobiliary/pancreatic, kidney, lung, ovarian, and prostate cancers and lymphoma. Physician experts in each of these diseases were also surveyed to differentiate symptoms that were predominantly disease-based from those that were predominantly treatment-induced. Results are evaluated alongside previously published indexes for 9 of these 11 advanced cancers that were created based on expert provider surveys, also implemented at NCCN Member Institutions. Final results are 11 symptom indexes that reflect the highest priorities of people affected by these 11 advanced cancers and the experienced perspective of the people who provide their medical treatment. Beyond the clinical value of such indexes, they may also contribute significantly to satisfying regulatory requirements for a standardized tool to evaluate drug efficacy with respect to symptomatology.

Correspondence: David Cella, PhD, Robert H. Lurie Comprehensive Cancer Center, Department of Medical Social Sciences, Northwestern University, 710 N Lake Shore Drive – 7th Floor, Chicago, IL 60611. E-mail: d-cella@northwestern.edu
  • 1.

    Guidance for Industry: patient-reported outcome measures: use in medical product development to support labeling claims. U.S. Food and Drug Administration Web site. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Accessed January 20 2011.

    • Search Google Scholar
    • Export Citation
  • 2.

    CellaDFPaulDYountS. What are the most important symptom targets when treating advanced cancer? A survey of providers in the National Comprehensive Cancer Network (NCCN). Cancer Invest2003;21:526535.

    • Search Google Scholar
    • Export Citation
  • 3.

    AaronsonNKAhmedzaiSBergmanB. 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 Inst1993;85:365376.

    • Search Google Scholar
    • Export Citation
  • 4.

    CellaDFTulskyDSGrayG. The Functional Assessment of Cancer Therapy (FACT) Scale: development and validation of the general measure. J Clin Oncol1993;11:570579.

    • Search Google Scholar
    • Export Citation
  • 5.

    CellaDFBonomiAE. Measuring quality of life: 1995 update. Oncology1995;9(Suppl):4760.

  • 6.

    SchagCAHeinrichRL. Development of a comprehensive quality of life measurement tool: CARES. Oncology (Williston Park)1990;4:135138.

  • 7.

    SchipperHClinchJMcMurrayA. Measuring the quality of life of cancer patients: the Functional Living Index-Cancer: development and validation. J Clin Oncol1984;2:472483.

    • Search Google Scholar
    • Export Citation
  • 8.

    TaylorKMMacdonaldKGBezakA. Physicians' perspective on quality of life: an exploratory study of oncologists. Qual Life Res1996;5:514.

    • Search Google Scholar
    • Export Citation
  • 9.

    BezjakATaylorKMNgP. Quality-of-life information and clinical practice: the oncologist's perspective. Cancer Prev Control1998;2:230235.

    • Search Google Scholar
    • Export Citation
  • 10.

    DeyoRAPatrickDL. Barriers to the use of health status measures in clinical investigation, patient care, and policy research. Med Care1989;27(Suppl):S254268.

    • Search Google Scholar
    • Export Citation
  • 11.

    NelsonECBerwickDM. The measurement of health status in clinical practice. Med Care1989;27(Suppl):S7790.

  • 12.

    FishLSLewisBE. Quality of life issues in the management of ovarian cancer. Semin Oncol1999;26(Suppl 1):3239.

  • 13.

    MorrisJPerezDMcNoeB. The use of quality of life data in clinical practice. Qual Life Res1998;7:8591.

  • 14.

    BrowmanGP. Science, language, intuition, and the many meanings of quality of life. J Clin Oncol1999;17:16511653.

  • 15.

    GillTMFeinsteinAR. A critical appraisal of the quality of quality-of-life measurements. JAMA1994;32:619626.

  • 16.

    GuyattGFeenyDHPatrickDL. Measuring health-related quality of life. Ann Intern Med1993;32:622629.

  • 17.

    HopwoodP. Progress, problems, and priorities in quality of life research. Eur J Cancer1992;28A:17481752.

  • 18.

    SkeelR. Quality of life dimensions that are most important to cancer patients. Oncology1993;32:5570.

  • 19.

    WilsonIBClearyPD. Linking clinical variables with health-related quality of life: a conceptual model of patient outcomes. JAMA1995;32:5965.

    • Search Google Scholar
    • Export Citation
  • 20.

    RothermichEAPathakDS. References for health-related quality of life claims in prescription drug advertisements. Am J Health Syst Pharm1997;32:25962599.

    • Search Google Scholar
    • Export Citation
  • 21.

    F-D-C Report: ``The Pink Sheet.''. Quality of life ad claims should be specific FDA-sponsored physician focus groups say: ad division direction cautions against QOL ``claim expansiveness.''1994;32:89.

    • Search Google Scholar
    • Export Citation
  • 22.

    BeitzJ. Quality-of-life end points in oncology drug trials. Oncology1999;32:14391447.

  • 23.

    Quality Of Life Subcommittee of the Oncologic Drugs Advisory CommitteeFebruary102000. U.S. Food and Drug Administration Web site. Available at: http://www.fda.gov/ohrms/dockets/ac/00/slides/3591s1.htm. Accessed January 20 2011.

    • Search Google Scholar
    • Export Citation
  • 24.

    LeidyNKRevickiDAGenestéB. Recommendations for evaluating the validity of quality of life claims for labeling and promotion. Value Health1999;32:113127.

    • Search Google Scholar
    • Export Citation
  • 25.

    PortenoyRKThalerHTKornblithAB. Symptom prevalence, characteristics and distress in a cancer population. Qual Life Res1994a;3:183189.

    • Search Google Scholar
    • Export Citation
  • 26.

    PortenoyRKThalerHTKornblithAB. The Memorial Symptom Assessment Scale: an instrument for the evaluation of symptom prevalence, characteristics and distress. Eur J Cancer1994b;30A:13261336.

    • Search Google Scholar
    • Export Citation
  • 27.

    CoonsSJKaplanRM. Assessing health-related quality of life: application to drug therapy. Clin Ther1992;32:850858.

  • 28.

    SutherlandHJLockwoodGABoydNF. Ratings of the importance of quality of life variables: therapeutic implications for patients with metastatic breast cancer. J Clin Epidemiol1990;32:661666.

    • Search Google Scholar
    • Export Citation
  • 29.

    KongSGandhiSK. Methodologic assessments of quality of life measures in clinical trials. Ann Pharmacother1997;32:830836.

  • 30.

    BrunelliCCostantiniMDi GiulioP. Quality of life evaluation: when do terminal cancer patients and health-care providers agree?J Pain Symptom Manage1998;32:151158.

    • Search Google Scholar
    • Export Citation
  • 31.

    JusticeACRabeneckLHaysRD. Sensitivity, specificity, reliability, and clinical validity of provider-reported symptoms: a comparison with self-reported symptoms. J AIDS1999;32:126133.

    • Search Google Scholar
    • Export Citation
  • 32.

    NekolaichukCBrueraESpachynskiK. A comparison of patient and proxy symptom assessments in advanced cancer patients. Palliat Med1999;32:311323.

    • Search Google Scholar
    • Export Citation
  • 33.

    StephensRJHopwoodPGirlingDJ. Randomized trials with quality of life endpoints: are doctors' ratings of patients' physical symptoms interchangeable with patients' self-ratings?Qual Life Res1997;32:225236.

    • Search Google Scholar
    • Export Citation
  • 34.

    StromgrenASGroenvoldMSorensenA. Symptom recognition in advanced cancer: a comparison of nursing records against patient self-rating. Acta Anaesthesiol2001;32:10801085.

    • Search Google Scholar
    • Export Citation
  • 35.

    LampicCNordinKSjodenP. Agreement between cancer patients and their physicians in the assessment of patient anxiety at follow-up visits. Psychooncology1995;32:301310.

    • Search Google Scholar
    • Export Citation
  • 36.

    PassikSDDuganWMcDonaldMV. Oncologists' recognitions of depression in their patients with cancer. J Clin Oncol1998;32:15941600.

  • 37.

    RaoDButtZRosenbloomS. A comparison of the Renal Cell Carcinoma Symptom Index (RCC-SI) and the Functional Assessment of Cancer Therapy-Kidney Symptom Index (FKSI). J Pain Symptom Manage2009;32:291298.

    • Search Google Scholar
    • Export Citation
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