29
Views
0
CrossRef citations to date
0
Altmetric
Review

Cancer symptom response as an oncology clinical trial end point

, , &
Pages 35-46 | Received 18 Apr 2018, Accepted 29 May 2018, Published online: 07 Jun 2018

References

  • Driscoll JJ, Rixe O. Overall survival: still the gold standard: why overall survival remains the definitive end point in cancer clinical trials. Cancer J. 2009;15(5):401–405.
  • Lebwhol D, Kay A, Berg W, et al. Progression-free survival: gaining on overall survival as a gold standard and accelerating drug development. Cancer J. 2009;15(5):386–394.
  • Korn RL, Crowley JJ. Overview: progression-free survival as an end point in clinical trials with solid tumors. Clin Cancer Res. 2013;19(10):2607–2612.
  • Booth CM, Eisenhauer EA. Progression-free survival: meaningful or simply measurable? J Clin Oncol. 2012;20(10):1030–1033.
  • Leonardi MC, Zampino MG, Luca F, et al. Pre-operative radiochemotherapy with raltitrexed for resectable locally-advanced rectal cancer: a phase II study. Anticancer Res. 2006;26(3B):2419–2423.
  • Kobayashi M, Tsuburaya A, Nagata N, et al. A feasibility study of sequential paclitaxel and S-1 (PTX/S-1) chemotherapy as postoperative adjuvant chemotherapy for advanced gastric cancer. Gastric Cancer. 2006;9(2):114–119.
  • Saif MW, Mehra R. Incidence and management of bevacizumab-related toxicities in colorectal cancer. Expert Opin Drug Saf. 2006;5(4):553–566.
  • United States Food and Drug Administration (FDA). Guidance for Industry. Clinical Trial End points for the Approved of Cancer Drugs and Biologics. Washington DC: US Department of Health and Human Services; 2005.
  • ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US); [2000 Feb 29 2018 Mar 02].
  • Cella D. Quality of life considerations in patients with advanced lung cancer. Semin Oncol. 2004;31(6 Suppl 11):16–20.
  • Brown DJ, McMillan DC, Milroy R. The correlation between fatigue, physical function, the systemic inflammatory response, and psychological distress in patients with advanced lung cancer. Cancer. 2005;103(2):377–382.
  • Victorson D, Soni M, Cella D. Metaanalysis of the correlation between radiographic tumor response and patient-reported outcomes. Cancer. 2006;106(3):494–504.
  • Secord AA, Coleman RL, Havrilesky LJ, et al. Patient-reported outcomes as end points and outcome indicators in solid tumors. Nat Rev Clin Oncol. 2015;12(6):358–370.
  • Wilson MK, Karakasis K, Oza AM. Outcomes and end points in trials of cancer treatment: the past, present, and future. Lancet Oncol. 2015;16(1):e32–e42.
  • Temel JS, Pirl WF, Lynch TJ. Comprehensive symptom management in patients with advanced-stage non-small-cell lung cancer. Clin Lung Cancer. 2006;7(4):241–249.
  • Chio A, Logroscino G, Hardiman O, et al. Prognostic factors in ALS: a critical review. Amyotrophic Lateral Sclerosis. 2009;10(5–6):310–323.
  • Ren XS, Kazis LE, Lee A, et al. The role of generic and disease-specific measures of physical and role functioning in assessing patient outcomes: a longitudinal study. J Ambul Care Manage. 2005;28(2):157–166.
  • Yamada K, Ito H, Nakamura H, et al. Stroke patients’ evolving symptoms assessed by tractography. J Magn Reson Imaging. 2004;20(6):923–929.
  • Webb A, Norton M. Clinical assessment of symptom-focused health-related quality of life in HIV/AIDS. J Assoc Nurses AIDS Care. 2004;15(2): 67–78 quiz 79–81.
  • Astoro NW, Samsuridjal D, Djoerban Z, et al. Quality of life of HIV patients and influential factors. Acta Medica Indonesiana. 2007;37(1):2–7.
  • Richards JM Jr., Hemstreet MP. Measures of life quality, role performance, and functional status in asthma research. Am J Resp Crit Care. 2012;149(2 Pt 2):S31–S39.
  • Habraken JM, Van Der Wal WM, Ter Riet G, et al. Health-related quality of life and functional status in end-stage COPD: a longitudinal study. Eur Respir J. 2011;37(2):280–288.
  • Calvert M, Kyte D, Mercieca-Bebber R, et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: the SPIRIT-PRO extension. JAMA. 2018;319(5):483–494.
  • Hollen PJ, Gralla RJ, Rittenberg CN. Quality of life as a clinical trial end point: determining the appropriate interval for repeated assessments in patients with advanced lung cancer. Support Care Cancer. 2004;12(11):767–773.
  • Llovet J, Ricci S, Mazzaferro V, et al. Randomized phase III trial of soragenib versus placebo in patients with advanced hepatocellular carcinoma (HCC). J Clin Oncol. 2007;25: LBA1-LBA1.
  • Yount S, Cella D, Webster K, et al. Assessment of patient-reported clinical outcome in pancreatic and other hepatobiliary cancers: the FACT Hepatobiliary Symptom Index. J Pain Symptom Manage. 2002;24(1):32–44.
  • Steel JL, Eton DT, Cella D, et al. Clinically meaningful changes in health-related quality of life in patients diagnosed with hepatobiliary carcinoma. Ann Oncol. 2006;17(2):304–312.
  • Choueiri TK, Escudier B, Powles T, et al. Cabozantinib versus everolimus in advanced renal cell carcinoma. N Engl J Med. 2015;373:1814–1823.
  • Choueiri TK, Escudier B, Powles T, et al. Cabozantinib versus everolimus in advanced renal cell carcinoma (METEOR): final results from a randomised, open-label, phase 3 trial. Lancet Oncol. 2016;17:917–927.
  • Rao D, Butt Z, Rosenbloom S, et al. 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 Manage. 2009;38:291–298.
  • Herdman M, Gudex C, Lloyd A, et al. Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Qual Life Res. 2011;20:1727–1736.
  • Cella D, Escudier B, Tannir NM, et al. Quality of life outcomes for cabosantinib versus everolimus in patients with metastatic renal cell carcinoma: METEOR phase III randomized trial. J Clin Oncol. 2018;36:1–11.
  • Pavel ME, Singh S, Strosberg JR, et al. Health-related quality of life for everolimus versus placebo in patients with advanced, non-functional, well-differentiated gastrointestinal or lung neuroendocrine tumours (RADIANT-4): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Oncol. 2017;18:1411–1422.
  • Yao JC, Fazio N, Singh S, et al. Everolimus for the treatment of advanced, non-functional neuroendocrine tumours of the lung or gastrointestinal tract (RADIANT-4): a ransomised, placebo-controlled, phase 3 study. Lancet. 2016;387:968–997.
  • Webster K, Cella D, Yost K. The Functional Assessment of Chronic Illness Therapy (FACIT) measurement system: properties, applications, and interpretation. Health Qual Life Outcomes. 2003;1:79.
  • Pardo Y, Guedea F, Aguilo F, et al. Quality-of-life impact on primary treatments for localized prostate cancer in patients without hormonal treatment. J Clin Oncol. 2010;28(31):4687–4696.
  • Brazier JE, Harper R, Jones NM, et al. Validating the SF-36 health survey questionnaire: new outcome measure for primary care. BMJ. 1992;305:160–164.
  • Wei JT, Dunn RL, Litwin MS, et al. Development and validation of the expanded prostate cancer index composite (EPIC) for comprehensive assessment of health-related quality of life in men with prostate cancer. Urology. 2000;56:899–905.
  • Fizazi K, Scher HI, Miller K, et al. Effect of enzalutamide on time to first skeletal-related event, pain, and quality of life in men with castration-resistant prostate cancer: results from the randomised phase 3 AFFIRM trial. Lancet Oncol. 2014;15:1147–1156.
  • Scher HI, Fizazi K, Saad F, et al. Increased survival with enzalutamide in prostate cancer after chemotherapy. New Engl J Med. 2012;367(13):1187–1197.
  • Esper P, Mo F, Chodak G, et al. Measuring quality of life in men with prostate cancer using the functional assessment of cancer therapy-prostate instrument. Adult Urology. 1997;50(6):920–928.
  • Victorson DE, Beaumont JL, Rosenbloom SK, et al. Efficient assessment of the most important symptoms in advanced prostate cancer: the NCCN/FACT-P symptom index. Psycho-Oncol. 2011;20(9):977–983.
  • Cleeland CS, Ryan KM. Pain assessment: global use of the Brief Pain Inventory. Ann Acad Med Singap. 1994;23:129–138.
  • Heidenreich A, Chowdhury S, Klotz L, et al. Impact of enzalutamide compared with bicalutamide on quality of life in men with metastatic castration-resistant prostate cancer: additional analyses from the TERRAIN randomized clinical trial. Eur Urol. 2017;71(4):534–542.
  • Pickard AS, Neary MP, Cella D. Estimation of minimally important differences in EQ-5D utility and VAS scores in cancer. Health Qual Life Outcomes. 2007;5:70–78.
  • Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors. JNCI-J Natl Cancer I. 2000;92(3):205–216.
  • Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009;45:228–247.
  • Beer TM, Armstrong AJ, Rathkopf DE, et al. Enzalutamide in metastatic prostate cancer before chemotherapy. New Engl J Med. 2014;371(5):424–433.
  • Beer TM, Miller K, Tombal B, et al. The association between health-related quality-of-life scores and clinical outcomes in metastatic castration-resistant prostate cancer patients: exploratory analyses of AFFIRM and PREVAIL studies. Eur J Cancer. 2017;87:21–29.
  • Cella D, Nichol MB, Eton D, et al. Estimating clinically meaningful changes for the functional assessment of cancer therapy-prostate: results from a clinical trial of patients with metastatic hormone-refractory prostate cancer. Value Health. 2009;12(1):124–129.
  • Cella D, Grunwald V, Nathan P, et al. Quality of life in patients with advanced renal cell carcinoma given nivolumab versus everolimus in CheckMate 025: a randomised, open-label, phase 3 trial. Lancet Oncol. 2016;17:994–1003.
  • Cella D, Young S, Brucker PS, et al. Development and validation of a scale to measure disease-related symptoms of kidney cancer. Value Health. 2007;10:285–293.
  • Smith EML, Pang H, Cirrincione C, et al. Effect of duloxetine on pain, function, and quality of life among patients with chemotherapy-induced painful peripheral neuropathy. JAMA-J Am Med Assoc. 2013;309(13):1359–1367.
  • Gotay CC, Kawamoto CT, Bottomley A, et al. The prognostic significance of patient-reported outcomes in cancer clinical trials. J Clin Oncol. 2008;26(8):1355–1636.
  • Quinten C, Coens C, Mauer M, et al. Baseline quality of life as a prognostic indicator of survival: a meta-analysis of individual patient data from EORTC clinical trials. Lancet Oncol. 2009;10(9):865–871.
  • Zwinderman AH. Statistical analysis of longitudinal quality of life data with missing measurements. Qual Life Res. 1992;1(3):219–224.
  • Hollen PJ, Gralla RJ, Cox C, et al. A dilemma in analysis: issues in the serial measurement of quality of life in patients with advanced lung cancer. Lung Cancer. 1997;18(2):119–136.
  • Morita S, Kobayaski K, Eguchi K, et al. Analysis of incomplete quality of life data in advanced stage cancer: a practical application of multiple imputation. Qual Life Res. 2005;14(6):1533–1544.
  • Fairclough D. Design and analysis of quality of life studies in clinical trials. Boca Raton, FL: Chapman & Hall; 2002.
  • Fielding S, Fayers P, Ramsay C. Predicting missing quality of life data that were later recovered: an empirical comparison of approaches. Clin Trials. 2010;7(4):333–342.
  • Vasey PA, Jayson GC, Gordon A, et al. Phase III randomized trial of docetaxel-carboplatin versus paclitaxel-carboplatin as first-line chemotherapy for ovarian cancer. JNCI-J Natl Cancer I. 2004;96(22):1682–1691.
  • Aaronson KN, Ahmedzai S, Bergman B, et al. The European Organization for Research and Treatment of Cancer QOQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. JNCI. 1993;85(5):365–376.
  • Smith I. Goals of treatment for patients with metastatic breast cancer. Semin Oncol. 2006;33(Suppl 2):2–5.
  • Wilson D, Hiller L, Gray L, et al. The effect of biological effective dose on time to symptom progression in metastatic renal cell carcinoma. Clin Oncol. 2003;15(7):400–407.
  • Verma S, Miles D, Gianni L, et al. Trastuzumab emtansine for HER2-positive advanced breast cancer. New Engl J Med. 2012;367(19):1783–1791.
  • Janne PA, Van Den Heuvel MM, Barlesi F, et al. Selumetinib plus docetaxel compared with docetaxel alone and progression-free survival in patients with KRAS-mutant advanced non-small cell lung cancer. JAMA. 2017;317(18):1844–1853.
  • Vansteenkiste J, Vandebroek J, Nackaerts K, et al. Influence of cisplatin-use, age, performance status, and duration of chemptherapy on symptom control in advanced non-small cell lung cancer: detailed symptom analysis of a randomised study comparing cisplatin-vindesine to gemcitabine. Lung Cancer. 2003;40(2):191–199.
  • Hollen PJ, Gralla RJ, Kris MG, et al. Quality of life assessment in individuals with lung cancer: testing the lung cancer symptom scale (LCSS). Eur J Cancer. 1993;29(Suppl 1):S51–S58.
  • Heyes A. Clinical trial experience with functional assessment of cancer therapy-lung in conventional and targeted non-small cell lung cancer therapy. Semin Oncol. 2004;31(Suppl 9):16–22.
  • Cella D, Eton D, Hensing TA, et al. Relationship between symptom change, objective tumor measurements, and performance status during chemptherapy for advanced lung cancer. Clin Lung Cancer. 2008;9(1):51–58.
  • Abendstein H, Nordgren M, Boysen M, et al. Quality of life and head and neck cancer: a 5 year prospective study. Laryngoscope. 2005;115(12):2183–2192.
  • Popov I, Jelic S, Radosavljevic D, et al. The role of stable disease in objective response assessment and its impact on survival in advanced colorectal cancer: is “stable disease” a homogenous response category? Neoplasma. 1999;46(2):132–139.
  • Cella D, Traina S, Li T, et al. Relationship between patient-reported outcomes and clinical outcomes in metastatic castration-resistant prostate cancer: post hoc analysis of COU-AA-301 and COU-AA-302. Ann Oncol. 2018;39:392–397.
  • Bonomi P, Kim I, Fairclough D, et al. Comparison of survival and quality of life in advanced non-small-cell lung cancer patients treated with two dose levels of paclitaxel combined with cisplatin versus etoposide with cisplatin: results of an Eastern cooperative Oncology Group Trial. J Clin Oncol. 2000;18(3):623–631.
  • Davis K, Cella D. Assessing quality of life in oncology clinical practice: a review of barriers and critical success factors. J Clin Outcomes Manag. 2002;9:327–332.
  • Sloan JA, Frost MH, Berzon R, et al. The clinical significance of quality of life assessments in oncology: a summary for clinicians. Support Care Cancer. 2006;14(10):988–998.
  • Varricchio CG, Ferrans CE. Quality of life assessments in clinical practice. Semin Oncol Nurs. 2010;26(1):12–17.
  • Detmar SB, Aaronson NK, Wever LD, et al. How are you feeling? Who wants to know? Patients’ and oncologists’ preferences for discussing health-related quality-of-life issues. J Clin Oncol. 2000;18(18):3295–3301.
  • Kotronoulas G, Kearney N, Maguire R, et al. What is the value of the routine use of patient-reported outcome measures toward improvement of patient outcomes, processes of care, and health service oucomes in cancer care? A systematic review of controlled trials. J Clin Oncol. 2014;32(14):1480–1501.
  • Basch E, Deal AM, Kris MG, et al. Symptom monitoring with patient-reported outcomes during routine cancer treatment: a randomized controlled trial. J Clin Oncol. 2016;34(6):557–565.
  • Basch E, Deal AM, Dueck AC, et al. Overall survival results of a trial assessing patient-reported outcomes for symptom monitoring during routine cancer treatment. JAMA. 2017;318(2):197–198.
  • Snyder CF, Aaronson NK, Choucair AK, et al. Implementing patient-reported outcomes assessment in clinical practice: a review of the options and considerations. Qual Life Res. 2012;21(8):1305–1314.
  • Jensen RE, Snyder CF, Abernethy AP, et al. Review of electronic patient-reported outcomes systems used in cancer clinical care. J Oncol Pract. 2014;10(4):e215–e222.
  • PCORI. Users’ Guide to Integrating Patient-Reported Outcomes in Electronic Health Records. 2017. Available from https://www.pcori.org/document/users-guide-integrating-patient-reported-outcomes-electronic-health-records. Accessed 23 March 2018.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.