Progression-free survival is simply a measure of a drug's effect while administered and is not a surrogate for overall survival

Cancer Journal

Volumn 15, Issue 5, 2009, Pages 379-385

  Wilkerson, Julia ^a   Fojo, Tito ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

Author keywords

Clinical endpoints; Overall survival; Progressive free survival

Indexed keywords

ALTERNATIVE MEDICINE; CANCER RESEARCH; CANCER SURVIVAL; CELL DEATH; CELL GROWTH; CLINICAL RESEARCH; DRUG APPROVAL; DRUG EFFECT; FOOD AND DRUG ADMINISTRATION; HUMAN; METASTASIS; OUTCOME ASSESSMENT; OVERALL SURVIVAL; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; REVIEW; TUMOR GROWTH;

ANTINEOPLASTIC AGENTS; DISEASE PROGRESSION; DISEASE-FREE SURVIVAL; HUMANS; NEOPLASM METASTASIS; NEOPLASMS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; REGRESSION ANALYSIS; TREATMENT OUTCOME;

EID: 70350135938     PISSN: 15289117     EISSN: 1540336X     Source Type: Journal    
DOI: 10.1097/PPO.0b013e3181bef8cd     Document Type: Review

References (61)

1. Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther. 2001;69:89-95.
2. Buyse M, ed. Is PFS a "Valid" Surrogate for OS in Advanced Ovarian Cancer? A Meta-Analysis. Presented in FDA/ASCO/AACR Public Workshop on Endpoints for Ovarian Cancer 2006; North Bethesda, MD; 2006.
3. Cleophas TJ, Zwinderman AH, Chaib AH. Novel procedures for validating surrogate endpoints in clinical trials. Curr Clin Pharmacol. 2007;2:123-128.
4. Beckman M. More clinical cancer treatments judged by progression-free rather than overall survival. J Natl Cancer Inst. 2007;99:1068-1069.
5. Bast RC, Thigpen JT, Arbuck SG, et al. Clinical trial endpoints in ovarian cancer: report of an FDA/ASCO/AACR Public Workshop. Gynecol Oncol. 2007;107:173-176.
6. Chakravarty A, Sridhara R. Use of progression-free survival as a surrogate marker in oncology trials: some regulatory issues. Stat Methods Med Res. 2008;17:515-518.
7. Lassere MN, Johnson KR, Boers M, et al. Definitions and validation criteria for biomarkers and surrogate endpoints: development and testing of a quantitative hierarchical levels of evidence schema. J Rheumatol. 2007;34:607-615.
8. Johnson JR, Williams G, Pazdur R. End points and United States Food and Drug Administration approval of oncology drugs. J Clin Oncol. 2003;21: 1404-1411.
9. Allegra C, Blanke C, Buyse M, et al. End points in advanced colon cancer clinical trials: a review and proposal. J Clin Oncol. 2007;25:3572-3575.
10. Christensen E. Choosing the best endpoint. J Hepatol. 2008;49:672-673.
11. Dhani N, Tu D, Sargent DJ, et al. Alternate endpoints for screening phase II studies. Clin Cancer Res. 2009;15:1873-1882.
12. Grothey A. Surrogate endpoints for overall survival in early colorectal cancer from the clinician's perspective. Stat Methods Med Res. 2008;17:529-535.
13. Hughes MD. Practical issues arising in an exploratory analysis evaluating progression-free survival as a surrogate endpoint for overall survival in advanced colorectal cancer. Stat Methods Med Res. 2008;17:487-495.
14. Piedbois P, Buyse M. Endpoints and surrogate endpoints in colorectal cancer: a review of recent developments. Curr Opin Oncol. 2008;20:466-471.
15. Sargent DJ, Hayes DF. Assessing the measure of a new drug: is survival the only thing that matters? J Clin Oncol. 2008;26:1922-1923.
16. Shi Q, Sargent DJ. Meta-analysis for the evaluation of surrogate endpoints in cancer clinical trials. Int J Clin Oncol. 2009;14:102-111.
17. Buyse M, Burzykowski T, Carroll K, et al. Progression-free survival is a surrogate for survival in advanced colorectal cancer. J Clin Oncol. 2007;25: 5218-5224.
18. Burzykowski T, Buyse M, Piccart-Gebhart MJ, et al. Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer. J Clin Oncol. 2008;26:1987-1992.
19. Buyse M, Burzykowski T, Michiels S, et al. Individual- and trial-level surrogacy in colorectal cancer. Stat Methods Med Res. 2008;17:467-475.
20. Di Leo A, Buyse M, Bleiberg H. Is overall survival a realistic primary end point in advanced colorectal cancer studies? A critical assessment based on four clinical trials comparing fluorouracil plus leucovorin with the same treatment combined either with oxaliplatin or with CPT-11. Ann Oncol. 2004;15:545-549.
21. Golfinopoulos V, Salanti G, Pavlidis N, et al. Survival and disease-progression benefits with treatment regimens for advanced colorectal cancer: a meta-analysis. Lancet Oncol. 2007;8:898-911.
22. Hackshaw A, Knight A, Barrett-Lee P, et al. Surrogate markers and survival in women receiving first-line combination anthracycline chemotherapy for advanced breast cancer. Br J Cancer. 2005;93:1215-1221.
23. Korn EL, Liu PY, Lee SJ, et al. Meta-analysis of phase II cooperative group trials in metastatic stage IV melanoma to determine progression-free and overall survival benchmarks for future phase II trials. J Clin Oncol. 2008;26:527-534.
24. Michiels S, Le Maitre A, Buyse M, et al. Surrogate endpoints for overall survival in locally advanced head and neck cancer: meta-analyses of individual patient data. Lancet Oncol. 2009;10:341-350.
25. Miksad RA, Zietemann V, Gothe R, et al. Progression-free survival as a surrogate endpoint in advanced breast cancer. Int J Technol Assess Health Care. 2008;24:371-383.
26. Sargent DJ, Patiyil S, Yothers G, et al. End points for colon cancer adjuvant trials: observations and recommendations based on individual patient data from 20,898 patients enrolled onto 18 randomized trials from the ACCENT Group. J Clin Oncol. 2007;25:4569-4574.
27. Sargent DJ, Wieand HS, Haller DG, et al. Disease-free survival versus overall survival as a primary end point for adjuvant colon cancer studies: individual patient data from 20,898 patients on 18 randomized trials. J Clin Oncol. 2005;23:8664-8670.
28. Sertdemir Y, Burgut R. Does the decision in a validation process of a surrogate endpoint change with level of significance of treatment effect? A proposal on validation of surrogate endpoints. Contemp Clin Trials. 2009;30:8-12.
29. Tang PA, Bentzen SM, Chen EX, et al. Surrogate end points for median overall survival in metastatic colorectal cancer: literature-based analysis from 39 randomized controlled trials of first-line chemotherapy. J Clin Oncol. 2007;25:4562-4568.
30. Fleming TR, Rothmann MD, Lu HL. Issues in Using Progression-Free Survival When Evaluating Oncology Products. J Clin Oncol. 2009;27:2874-2880.
31. Saad ED, Katz A. Progression-free survival and time to progression as primary end points in advanced breast cancer: often used, sometimes loosely defined. Ann Oncol. 2009;20:460-464.
32. Panageas KS, Ben-Porat L, Dickler MN, et al. When you look matters: the effect of assessment schedule on progression-free survival. J Natl Cancer Inst. 2007;99:428-432.
33. Burzykowski T. Surrogate endpoints: wishful thinking or reality?. Stat Methods Med Res. 2008;17:463-466.
34. Ballman KV, Buckner JC, Brown PD, et al. The relationship between six-month progression-free survival and 12-month overall survival end points for phase II trials in patients with glioblastoma multiforme. Neuro Oncol. 2007;9:29-38.
35. Johnson KR, Ringland C, Stokes BJ, et al. Response rate or time to progression as predictors of survival in trials of metastatic colorectal cancer or non-small-cell lung cancer: a meta-analysis. Lancet Oncol. 2006;7:741-746.
36. Milella M, Bria E, Carlini P, et al. Surrogate endpoints for overall survival (OS) in advanced pancreatic cancer (APC): Analysis of randomized clinical trials (RCTs) exploring gemcitabine (G)-based combinations. J Clin Oncol [meeting abstracts]. 2007;25(18 suppl):4575.
37. Sargent D. General and statistical hierarchy of appropriate biologic endpoints. Oncology (Williston Park). 2006;20(6 suppl 5):5-9.
38. Dodd LE, Korn EL, Freidlin B, et al. Blinded independent central review of progression-free survival in phase III clinical trials: important design element or unnecessary expense? J Clin Oncol. 2008;26:3791-3796.
39. Pazdur R. Endpoints for assessing drug activity in clinical trials. Oncologist. 2008;13(suppl 2):19-21.
40. Yothers G. Toward progression-free survival as a primary end point in advanced colorectal cancer. J Clin Oncol. 2007;25:5153-5154.
41. SAS version 9.1 software. SAS Institute, Inc. Cary, NC. Copyright 2002- 2003, SAS Institute Inc.
42. Ashburner M, Ball CA, Blake JA, et al. Gene Ontology: tool for the unification of biology. Nat Genet. 2000;25:25-29.
43. Nagarajan V. Gene List Venn Diagram. Available at: http://www. bioinformatics. org/gvenn/index.htm.
44. Stordal B, Davey R. A systematic review of genes involved in the inverse resistance relationship between cisplatin and paclitaxel chemotherapy: role of BRCA1. Curr Cancer Drug Targets. 2009;9:354-365.
45. Wilson TR, Johnston PG, Longley DB. Anti-apoptotic mechanisms of drug resistance in cancer. Curr Cancer Drug Targets. 2009;9:307-319.
46. Hector S, Prehn JH. Apoptosis signaling proteins as prognostic biomarkers in colorectal cancer: a review. Biochim Biophys Acta. 2009;1795:117-129.
47. Savage P, Stebbing J, Bower M, Crook T, et al. Why does cytotoxic chemotherapy cure only some cancers? Nat Clin Pract Oncol. 2009;6:43-52.
48. Broxterman HJ, Gotink KJ, Verheul HM. Understanding the causes of multidrug resistance in cancer: a comparison of doxorubicin and sunitinib. Drug Resist Updat. In press.
49. El Maalouf G, Le Tourneau C, Batty GN, et al. Markers involved in resistance to cytotoxics and targeted therapeutics in pancreatic cancer. Cancer Treat Rev. 2009;35:167-174.
50. Muggia F. Platinum compounds 30 years after the introduction of cisplatin: implications for the treatment of ovarian cancer. Gynecol Oncol. 2009;112:275-281.
51. Borst P, Rottenberg S, Jonkers J. How do real tumors become resistant to cisplatin? Cell Cycle. 2008;7:1353-1359.
52. Stein WD, Yang J, Bates SE, et al. Bevacizumab reduces the growth rate constants of renal carcinomas: a novel algorithm suggests early discontinuation of bevacizumab resulted in a lack of survival advantage. Oncologist. 2008;13:1055-1062.
53. Rixe O, Fojo T. Is cell death a critical end point for anticancer therapies or is cytostasis sufficient? Clin Cancer Res. 2007;13:7280-7287.
54. National Cancer Institute. FDA Approval for Pemetrexed Disodium. 2009. Available at: http://www.cancer.gov/cancertopics/druginfo/fda- pemetrexeddisodium. Accessed September 2, 2009.
55. Belani CP, Brodowicz T, Ciuleanu T, et al. Maintenance pemetrexed (Pem) plus best supportive care (BSC) versus placebo (Plac) plus BSC: A randomized phase III study in advanced non-small cell lung cancer (NSCLC) [meeting abstracts]. J Clin Oncol. 2009;27(18 suppl):CRA8000.
56. Patel JD, Bonomi P, Socinski MA, et al. Treatment rationale and study design for the pointbreak study: a randomized, open-label phase III study of pemetrexed/ carboplatin/bevacizumab followed by maintenance pemetrexed/ bevacizumab versus paclitaxel/carboplatin/bevacizumab followed by maintenance bevacizumab in patients with stage IIIB or IV nonsquamous non-smallcell lung cancer. Clin Lung Cancer. 2009;10:252-256.
57. Miller K, Wang M, Gralow J, et al. Paclitaxel plus bevacizumab versus paclitaxel alone for metastatic breast cancer. N Engl J Med. 2007;357:2666-2676.
58. Schneider BP, Wang M, Radovich M, et al. Association of vascular endothelial growth factor and vascular endothelial growth factor receptor-2 genetic polymorphisms with outcome in a trial of paclitaxel compared with paclitaxel plus bevacizumab in advanced breast cancer: ECOG 2100. J Clin Oncol. 2008;26:4672-4678.
59. Paez-Ribes M, Allen E, Hudock J, et al. Antiangiogenic therapy elicits malignant progression of tumors to increased local invasion and distant metastasis. Cancer Cell. 2009;15:220-231.
60. Ebos JM, Lee CR, Cruz-Munoz W, et al. Accelerated metastasis after short-term treatment with a potent inhibitor of tumor angiogenesis. Cancer Cell. 2009;15: 232-239.
61. Loges S, Mazzone M, Hohensinner P, et al. Silencing or fueling metastasis with VEGF inhibitors: antiangiogenesis revisited. Cancer Cell. 2009;15:167-170.