Recommendations for including multiple symptoms as endpoints in cancer clinical trials: A report from the ASCPRO (Assessing the Symptoms of Cancer Using Patient-Reported Outcomes) Multisymptom Task Force

Cancer

Volumn 119, Issue 2, 2013, Pages 411-420

  Cleeland, Charles S ^a   Sloan, Jeff A ^b   Cella, David ^c   Chen, Connie ^d   Dueck, Amylou C ^e   Janjan, Nora A ^f   Liepa, Astra M ^g   Mallick, Rajiv ^h   O'Mara, Ann ⁱ   Pearson, Jay D ^j   Torigoe, Yasuhiro ^k   Wang, Xin Shelley ^a   Williams, Loretta A ^a   Woodruff, Jeanie F ^a  

^a UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^b Mayo Clinic College of Medicine   (United States)

^c NORTHWESTERN UNIVERSITY   (United States)

^d PFIZER INC   (United States)

^e MAYO CLINIC   (United States)

^f National Center for Policy Analysis   (United States)

^g ELI LILLY AND COMPANY   (United States)

^h DAIICHI SANKYO INC   (United States)

ⁱ NATIONAL CANCER INSTITUTE   (United States)

^j MERCK RESEARCH LABORATORIES   (United States)

^k GENENTECH INC   (United States)

more..

Author keywords

cancer; clinical trial; composite endpoint; labeling claim; outcome; patient report; recommendation; symptom; toxicity; treatment

Indexed keywords

ANTINEOPLASTIC AGENT;

ARTICLE; CANCER PATIENT; CANCER RESEARCH; CLINICAL STUDY; CONCEPTUAL FRAMEWORK; DISEASE ASSOCIATION; DISEASE SEVERITY; DRUG APPROVAL; HEALTH CARE POLICY; HUMAN; HYPOTHESIS; ONCOLOGY; OUTCOME ASSESSMENT; PATIENT ASSESSMENT; PATIENT REPORTED OUTCOME; PRIORITY JOURNAL; PROCESS OPTIMIZATION; QUALITY OF LIFE; SCORING SYSTEM; SELF REPORT; SYMPTOMATOLOGY; VALIDATION PROCESS;

CLINICAL TRIALS AS TOPIC; HUMANS; NEOPLASMS; OUTCOME ASSESSMENT (HEALTH CARE); SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

EID: 84872058479     PISSN: 0008543X     EISSN: 10970142     Source Type: Journal    
DOI: 10.1002/cncr.27744     Document Type: Article

References (60)

1. Temel JS, Greer JA, Muzikansky A, et al. Early palliative care for patients with metastatic non-small-cell lung cancer. N Engl J Med. 2010; 363: 733-742.
2. Williams G, Pazdur R, Temple R,. Assessing tumor-related signs and symptoms to support cancer drug approval. J Biopharm Stat. 2004; 14: 5-21.
3. Bruner DW, Bryan CJ, Aaronson N, et al. Issues and challenges with integrating patient-reported outcomes in clinical trials supported by the National Cancer Institute-sponsored clinical trials networks. J Clin Oncol. 2007; 25: 5051-5057.
4. Ganz PA, Gotay CC,. Use of patient-reported outcomes in phase III cancer treatment trials: lessons learned and future directions. J Clin Oncol. 2007; 25: 5063-5069.
5. Cleeland CS, Sloan JA,. Assessing the Symptoms of Cancer Using Patient-Reported Outcomes (ASCPRO): searching for standards. J Pain Symptom Manage. 2010; 39: 1077-1085.
6. Barsevick AM, Cleeland CS, Manning DC, et al. ASCPRO recommendations for the assessment of fatigue as an outcome in clinical trials. J Pain Symptom Manage. 2010; 39: 1086-1099.
7. Merriam-Webster Inc. Webster's Third New International Dictionary of the English Language, Unabridged. Springfield, MA: Merriam-Webster, Inc.; 2002.
8. Fayers PM, Hand DJ,. Causal variables, indicator variables and measurement scales: an example from quality of life. J R Stat Soc A. 2002; 165: 233-253.
9. Sloan JA,. Applying QOL assessments: solutions for oncology clinical practice and research, part 1-overview and introduction. Curr Probl Cancer. 2005; 29: 274-277.
10. Bezjak A, Tu D, Seymour L, et al. Symptom improvement in lung cancer patients treated with erlotinib: quality of life analysis of the National Cancer Institute of Canada Clinical Trials Group Study BR. 21. J Clin Oncol. 2006; 24: 3831-3837.
11. Cella D, Li JZ, Cappelleri JC, et al. Quality of life in patients with metastatic renal cell carcinoma treated with sunitinib or interferon alfa: Results from a phase III randomized trial. J Clin Oncol. 2008; 26: 3763-3769.
12. Cella D,. Beyond traditional outcomes: improving quality of life in patients with renal cell carcinoma. Oncologist. 2011; 16 (suppl 2): 23-31.
13. Cianfrocca M, Lee S, Von Roenn J, et al. Randomized trial of paclitaxel versus pegylated liposomal doxorubicin for advanced human immunodeficiency virus-associated Kaposi sarcoma: evidence of symptom palliation from chemotherapy. Cancer. 2010; 116: 3969-3977.
14. Kim YW, Baik YH, Yun YH, et al. Improved quality of life outcomes after laparoscopy-assisted distal gastrectomy for early gastric cancer: Results of a prospective randomized clinical trial. Ann Surg. 2008; 248: 721-727.
15. Dooms CA, Pat KE, Vansteenkiste JF,. The effect of chemotherapy on symptom control and quality of life in patients with advanced non-small cell lung cancer. Expert Rev Anticancer Ther. 2006; 6: 531-544.
16. US Food and Drug Administration. Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Available at: http://www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/UCM071975.pdf. Accessed November 16, 2011.
17. US Food and Drug Administration. Guidance for Industry. Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/ Guidances/UCM071590.pdf. Accessed November 11, 2011.
18. Basch E,. The missing voice of patients in drug-safety reporting. N Engl J Med. 2010; 362: 865-869.
19. Basch E, Minasian L, Burke L, et al. Symptom measurement in clinical trials. Paper presented at: 2011 Conference on Clinical Cancer Research; Washington, DC; November 10, 2011. Available at: http://www.focr.org/images/ stories/pdf/panel3final110411.pdf Accessed May 18, 2012.
20. Doward LC, Gnanasakthy A, Baker MG,. Patient reported outcomes: looking beyond the label claim. Health Qual Life Outcomes. 2010; 8: 89.
21. Janjan N, Goodman J,. Health-care reform and the treatment of metastatic cancer [serial online]. ASCO Post. 2011; 2: 1-2.
22. Hillman SL, Mandrekar SJ, Bot B, et al. Evaluation of the value of attribution in the interpretation of adverse event data: a North Central Cancer Treatment Group and American College of Surgeons Oncology Group investigation. J Clin Oncol. 2010; 28: 3002-3007.
23. Kirkova J, Davis MP, Walsh D, et al. Cancer symptom assessment instruments: a systematic review. J Clin Oncol. 2006; 24: 1459-1473.
24. Aaronson NK, Ahmedzai S, Bergman B, et al. 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: 365-376.
25. Cella DF, Tulsky DS, Gray G, et al. The Functional Assessment of Cancer Therapy scale: development and validation of the general measure. J Clin Oncol. 1993; 11: 570-579.
26. Portenoy RK, Thaler HT, Kornblith AB, et al. The Memorial Symptom Assessment Scale: an instrument for the evaluation of symptom prevalence, characteristics and distress. Eur J Cancer. 1994; 30A: 1326-1336.
27. Cleeland CS, Mendoza TR, Wang XS, et al. Assessing symptom distress in cancer patients: the M. D. Anderson Symptom Inventory. Cancer. 2000; 89: 1634-1646.
28. Bruera E, Kuehn N, Miller MJ, Selmser P, Macmillan K,. The Edmonton Symptom Assessment System (ESAS): a simple method for the assessment of palliative care patients. J Palliat Care. 1991; 7: 6-9.
29. Mendoza TR, Wang XS, Lu C, et al. Measuring the symptom burden of lung cancer: the validity and utility of the lung cancer module of the M. D. Anderson Symptom Inventory. Oncologist. 2011; 16: 217-227.
30. Wang XS, Williams LA, Eng C, et al. Validation and application of a module of the M. D. Anderson Symptom Inventory for measuring multiple symptoms in patients with gastrointestinal cancer (the MDASI-GI). Cancer. 2010; 116: 2053-2063.
31. Garcia SF, Rosenbloom SK, Beaumont JL, et al. Priority symptoms in advanced breast cancer: development and initial validation of the National Comprehensive Cancer Network-Functional Assessment of Cancer Therapy-Breast Cancer Symptom Index (NFBSI-16). Value Health. 2012; 15: 183-190.
32. Cella D, Rosenbloom SK, Beaumont JL, et al. Development and validation of 11 symptom indexes to evaluate response to chemotherapy for advanced cancer. J Natl Compr Canc Netw. 2011; 9: 268-278.
33. Rothman M, Burke L, Erickson P, Leidy NK, Patrick DL, Petrie CD,. Use of existing patient-reported outcome (PRO) instruments and their modification: the ISPOR Good Research Practices for Evaluating and Documenting Content Validity for the Use of Existing Instruments and Their Modification PRO Task Force Report. Value Health. 2009; 12: 1075-1083.
34. Cella D, Paul D, Yount S, et al. What are the most important symptom targets when treating advanced cancer? A survey of providers in the National Comprehensive Cancer Network (NCCN). Cancer Invest. 2003; 21: 526-535.
35. Lorenz KA, Dy SM, Naeim A, et al. Quality measures for supportive cancer care: the Cancer Quality-ASSIST Project. J Pain Symptom Manage. 2009; 37: 943-964.
36. Patrick DL, Ferketich SL, Frame PS, et al. National Institutes of Health State-of-the-Science Conference Statement: symptom management in cancer: pain, depression, and fatigue, July 15-17, 2002. J Natl Cancer Inst Monogr. 2004;(32): 9-16.
37. Barbera L, Seow H, Howell D, et al. Symptom burden and performance status in a population-based cohort of ambulatory cancer patients. Cancer. 2010; 116: 5767-5776.
38. Henry DH, Viswanathan HN, Elkin EP, Traina S, Wade S, Cella D,. Symptoms and treatment burden associated with cancer treatment: Results from a cross-sectional national survey in the US. Support Care Cancer. 2008; 16: 791-801.
39. Bennett MI, Rayment C, Hjermstad M, Aass N, Caraceni A, Kaasa S,. Prevalence and aetiology of neuropathic pain in cancer patients: a systematic review. Pain. 2012; 153: 359-365.
40. Fiorentino L, Rissling M, Liu L, Ancoli-Israel S,. The symptom cluster of sleep, fatigue and depressive symptoms in breast cancer patients: severity of the problem and treatment options. Drug Discov Today Dis Models. 2011; 8: 167-173.
41. Martinelli F, Quinten C, Maringwa JT, et al. Examining the relationships among health-related quality-of-life indicators in cancer patients participating in clinical trials: a pooled study of baseline EORTC QLQ-C30 data. Expert Rev Pharmacoecon Outcomes Res. 2011; 11: 587-599.
42. Wang XS, Cleeland CS, Mendoza TR, et al. Impact of cultural and linguistic factors on symptom reporting by patients with cancer. J Natl Cancer Inst. 2010; 102: 732-738.
43. Wang XS, Fairclough DL, Liao Z, et al. Longitudinal study of the relationship between chemoradiation therapy for non-small-cell lung cancer and patient symptoms. J Clin Oncol. 2006; 24: 4485-4491.
44. Cleeland CS, Mendoza TR, Wang XS, et al. Levels of symptom burden during chemotherapy for advanced lung cancer: differences between public hospitals and a tertiary cancer center. J Clin Oncol. 2011; 29: 2859-2865.
45. Cheville AL, Novotny PJ, Sloan JA, et al. Fatigue, dyspnea, and cough comprise a persistent symptom cluster up to 5 years after diagnosis with lung cancer. J Pain Symptom Manage. 2011; 42: 202-212.
46. Velikova G, Stark D, Selby P,. Quality of life instruments in oncology. Eur J Cancer. 1999; 35: 1571-1580.
47. Skerman HM, Yates PM, Battistutta D,. Multivariate Methods to identify cancer-related symptom clusters. Res Nurs Health. 2009; 32: 345-360.
48. Cheung WY, Le LW, Gagliese L, Zimmermann C,. Age and gender differences in symptom intensity and symptom clusters among patients with metastatic cancer. Support Care Cancer. 2011; 19: 417-423.
49. Kim E, Jahan T, Aouizerat BE, et al. Changes in symptom clusters in patients undergoing radiation therapy. Support Care Cancer. 2009; 17: 1383-1391.
50. Cleeland CS, Bennett GJ, Dantzer R, et al. Are the symptoms of cancer and cancer treatment due to a shared biologic mechanism? Cancer. 2003; 97: 2919-2925.
51. Cheville AL, Novotny PJ, Sloan JA, et al. The value of a symptom cluster of fatigue, dyspnea, and cough in predicting clinical outcomes in lung cancer survivors. J Pain Symptom Manage. 2011; 42: 213-221.
52. Mesa RA, Kantarjian H, Tefferi A, et al. Evaluating the serial use of the Myelofibrosis Symptom Assessment Form for measuring symptomatic improvement: performance in 87 myelofibrosis patients on a JAK1 and JAK2 inhibitor (INCB018424) clinical trial. Cancer. 2011; 117: 4869-4877.
53. Mesa RA, Gotlib J, Gupta V, et al. Associations between improvements in myelofibrosis (MF) symptoms and quality of life measures with splenomegaly reduction in COMFORT-I: a randomized, double-blind, phase III trial of the JAK1 and JAK2 inhibitor ruxolitinib versus placebo in patients with MF [abstract]. Blood (ASH Annual Meeting Abstracts). 2011; 118. Abstract 3842.
54. Corey-Lisle PK, Peck R, Mukhopadhyay P, et al. Q-TWiST analysis of ixabepilone in combination with capecitabine on quality of life in patients with metastatic breast cancer. Cancer. 2012; 118: 461-468.
55. Wang XS, Shi Q, Lu C, et al. Prognostic value of symptom burden for overall survival in patients receiving chemotherapy for advanced nonsmall cell lung cancer. Cancer. 2010; 116: 137-145.
56. Cella D, Eton D, Hensing TA, Masters GA, Parasuraman B,. Relationship between symptom change, objective tumor measurements, and performance status during chemotherapy for advanced lung cancer. Clin Lung Cancer. 2008; 9: 51-58.
57. Hollen PJ, Gralla RJ, Kris MG, Eberly SW, Cox C,. Normative data and trends in quality of life from the Lung Cancer Symptom Scale (LCSS). Support Care Cancer. 1999; 7: 140-148.
58. Gralla RJ,. Quality-of-life considerations in patients with advanced lung cancer: effect of topotecan on symptom palliation and quality of life. Oncologist. 2004; 9 (suppl 6): 14-24.
59. Sloan JA, Loprinzi CL, Laurine JA, et al. A simple stratification factor prognostic for survival in advanced cancer: the good/bad/uncertain index. J Clin Oncol. 2001; 19: 3539-3546.
60. Turk DC, Dworkin RH, McDermott MP, et al. Analyzing multiple endpoints in clinical trials of pain treatments: IMMPACT recommendations. Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials. Pain. 2008; 139: 485-493.