Surrogate end points for median overall survival in metastatic colorectal cancer: Literature-based analysis from 39 randomized controlled trials of first-line chemotherapy

Journal of Clinical Oncology

Volumn 25, Issue 29, 2007, Pages 4562-4568

  Tang, Patricia A ^a   Bentzen, Søren M ^a   Chen, Eric X ^a   Siu, Lillian L ^a  

^a UNIVERSITY OF TORONTO   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

5 ETHYNYLURACIL; BEVACIZUMAB; CAPECITABINE; CISPLATIN; FLUOROURACIL; FOLINIC ACID; INTERFERON; IRINOTECAN; OXALIPLATIN; PLACEBO; RALTITREXED; SPARFOSIC ACID; TAUROMUSTINE; THYMOSTIMULIN; TRIMETREXATE; UFT;

ARTICLE; CANCER CHEMOTHERAPY; CANCER GROWTH; CANCER RISK; CANCER SURVIVAL; CLINICAL TRIAL; COLORECTAL CANCER; COMBINATION CHEMOTHERAPY; CONTINUOUS INFUSION; CORRELATION ANALYSIS; CORRELATION COEFFICIENT; DRUG DOSE COMPARISON; DRUG MEGADOSE; EVIDENCE BASED MEDICINE; HAZARD RATIO; HUMAN; LINEAR REGRESSION ANALYSIS; LOW DRUG DOSE; META ANALYSIS; METASTASIS; MONOTHERAPY; MULTIPLE CYCLE TREATMENT; PRIORITY JOURNAL; REGRESSION ANALYSIS; RISK REDUCTION; STATISTICAL ANALYSIS; SYSTEMATIC REVIEW; TREATMENT OUTCOME; TREATMENT RESPONSE;

ANTINEOPLASTIC AGENTS; BIOLOGICAL MARKERS; CLINICAL TRIALS; COLORECTAL NEOPLASMS; DISEASE PROGRESSION; DISEASE-FREE SURVIVAL; ENDPOINT DETERMINATION; HUMANS; NEOPLASM METASTASIS; RANDOMIZED CONTROLLED TRIALS; REGRESSION ANALYSIS; RESEARCH DESIGN; TREATMENT OUTCOME;

EID: 35649022759     PISSN: 0732183X     EISSN: None     Source Type: Journal    
DOI: 10.1200/JCO.2006.08.1935     Document Type: Article

References (63)

1. Tournigand C, Cervantes A, Figer A, et al: OPTIMOX1: A randomized study of FOLFOX4 or FOLFOX7 with oxaliplatin in a stop-and-go fashion in advanced colorectal cancer - A GERCOR study. J Clin Oncol 24:394-400, 2006
2. Tournigand C, Andre T, Achille E, et al: FOLFIRI followed by FOLFOX6 or the reverse sequence in advanced colorectal cancer: A randomized GERCOR study. J Clin Oncol 22:229-237, 2004
3. Souglakos J, Androulakis N, Syrigos K, et al: FOLFOXIRI (folinic acid, 5-fluorouracil, oxaliplatin and irinotecan) vs FOLFIRI (folinic acid, 5-fluorouracil and irinotecan) as first-line treatment in metastatic colorectal cancer (MCC): A multicentre randomised phase III trial from the Hellenic Oncology Research Group (HORG). Br J Cancer 94:798-805, 2006
4. Köhne CH, van Cutsem E, Wils J, et al: Phase III study of weekly high-dose infusional fluorouracil plus folinic acid with or without irinotecan in patients with metastatic colorectal cancer: European Organisation for Research and Treatment of Cancer Gastrointestinal Group Study 40986. J Clin Oncol 23:4856-4865, 2005
5. Goldberg RM, Sargent DJ, Morton RF, et al: A randomized controlled trial of fluorouracil plus leucovorin, irinotecan, and oxaliplatin combinations in patients with previously untreated metastatic colorectal cancer. J Clin Oncol 22:23-30, 2004
6. Giacchetti S, Bjarnson G, Garufi C, et al: First line infusion of 5-fluorouracil, leucovorin and oxaliplatin for metastatic colorectal cancer: 4-day chronomodulated (FFL4-10) versus 2-day FOLFOX2 - A multicenter randomized phase III trial of the Chronotherapy Group of the European Organization for Research and Treatment of Cancer (EORTC 05963). J Clin Oncol 22:251s, 2004 (suppl; abstr 3526)
7. Colucci G, Gebbia V, Paoletti G, et al: Phase III randomized trial of FOLFIRI versus FOLFOX4 in the treatment of advanced colorectal cancer: A multicenter study of the Gruppo Oncologico Dell'Italia Meridionale. J Clin Oncol 23:4866-4875, 2005
8. Roberts TG Jr, Lynch TJ Jr, Chabner BA: The phase III trial in the era of targeted therapy: Unraveling the "go or no go" decision. J Clin Oncol 21:3683-3695, 2003
9. de Gramont A, Figer A, Seymour M, et al: Leucovorin and fluorouracil with or without oxaliplatin as first-line treatment in advanced colorectal cancer. J Clin Oncol 18:2938-2947, 2000
10. Douillard JY, Cunningham D, Roth AD, et al: Irinotecan combined with fluorouracil compared with fluorouracil alone as first-line treatment for metastatic colorectal cancer: A multicentre randomised trial. Lancet 355:1041-1047, 2000
11. Saltz LB, Cox JV, Blanke C, et al: Irinotecan plus fluorouracil and leucovorin for metastatic colorectal cancer: Irinotecan Study Group. N Engl J Med 343:905-914, 2000
12. Hurwitz H, Fehrenbacher L, Novotny W, et al: Bevacizumab plus irinotecan, fluorouracil, and leucovorin for metastatic colorectal cancer. N Engl J Med 350:2335-2342, 2004
13. Miller AB, Hoogstraten B, Staquet M, et al: Reporting results of cancer treatment. Cancer 47: 207-214, 1981
14. Therasse P, Arbuck SG, Eisenhauer EA, et al: New guidelines to evaluate the response to treatment in solid tumors: European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. J Natl Cancer Inst 92:205-216, 2000
15. Kelly H, Goldberg RM: Systemic therapy for metastatic colorectal cancer: Current options, current evidence. J Clin Oncol 23:4553-4560, 2005
16. Folprecht G, Cunningham D, Ross P, et al: Efficacy of 5-fluorouracil-based chemotherapy in elderly patients with metastatic colorectal cancer: A pooled analysis of clinical trials. Ann Oncol 15:1330-1338, 2004
17. Meta-analysis of randomized trials testing the biochemical modulation of fluorouracil by methotrexate in metastatic colorectal cancer: Advanced Colorectal Cancer Meta-Analysis Project. J Clin Oncol 12:960-969, 1994
18. Modulation of fluorouracil by leucovorin in patients with advanced colorectal cancer: Evidence in terms of response rate - Advanced Colorectal Cancer Meta-Analysis Project. J Clin Oncol 10:896-903, 1992
19. Pazdur R, Vincent M: Raltitrexed (Tomudex) versus 5-fluorouracil and leucovorin (5FU -+ LV) in patients with advanced colorectal cancer (ACC): Results of randomized, multicenter, North American trial. Proc Am Soc Clin Oncol 16:228a, 1997 (abstr 801)
20. Bajetta E, Colleoni M, Rosso R, et al: Prospective randomised trial comparing fluorouracil versus doxifluridine for the treatment of advanced colorectal cancer. Eur J Cancer 29A:1658-1663, 1993
21. Blijham G, Wagener T, Wils J, et al: Modulation of high-dose infusional fluorouracil by low-dose methotrexate in patients with advanced or metastatic colorectal cancer: Final results of a randomized European Organization for Research and Treatment of Cancer Study. J Clin Oncol 14:2266-2273, 1996
22. Price TJ, Ross PJ, Hickish T, et al: Phase III study of mitomycin-C with protracted venous infusion or circadian-timed infusion of 5-fluorouracil in advanced colorectal carcinoma. Clin Colorectal Cancer 3:235-242, 2004
23. Seymour MT, Slevin ML, Kerr DJ, et al: Randomized trial assessing the addition of interferon alpha-2a to fluorouracil and leucovorin in advanced colorectal cancer: Colorectal Cancer Working Party of the United Kingdom Medical Research Council. J Clin Oncol 14:2280-2288, 1996
24. Maughan TS, James RD, Kerr DJ, et al: Comparison of intermittent and continuous palliative chemotherapy for advanced colorectal cancer: A multicentre randomised trial. Lancet 361:457-464, 2003
25. Laufman LR, Bukowski RM, Collier MA, et al: A randomized, double-blind trial of fluorouracil plus placebo versus fluorouracil plus oral leucovorin in patients with metastatic colorectal cancer. J Clin Oncol 11:1888-1893, 1993
26. Buroker TR, O'Connell MJ, Wieand HS, et al: Randomized comparison of two schedules of fluorouracil and leucovorin in the treatment of advanced colorectal cancer. J Clin Oncol 12:14-20, 1994
27. Mustacchi G, Pavesi L, Milani S, et al: High-dose folinic acid (FA) and fluorouracil (FU) plus or minus thymostimulin (TS) for treatment of metastatic colorectal cancer: Results of a randomized multicenter clinical trial. Anticancer Res 14:617-619, 1994
28. Smyth JF, Hardcastle JD, Denton G, et al: Two phase III trials of tauromustine (TCNU) in advanced colorectal cancer. Ann Oncol 6:948-949, 1995
29. Cunningham D, Zalcberg JR, Rath U, et al: Final results of a randomised trial comparing 'Tomudex' (raltitrexed) with 5-fluorouracil plus leucovorin in advanced colorectal cancer: "Tomudex" Colorectal Cancer Study Group. Ann Oncol 7:961-965, 1996
30. Greco FA, Figlin R, York M, et al: Phase III randomized study to compare interferon alfa-2a in combination with fluorouracil versus fluorouracil alone in patients with advanced colorectal cancer. J Clin Oncol 14:2674-2681, 1996
31. Hansen RM, Ryan L, Anderson T, et al: Phase III study of bolus versus infusion fluorouracil with or without cisplatin in advanced colorectal cancer. J Natl Cancer Inst 88:668-674, 1996
32. Jäger E, Heike M, Bernhard H, et al: Weekly high-dose leucovorin versus low-dose leucovorin combined with fluorouracil in advanced colorectal cancer: Results of a randomized multicenter trial - Study Group for Palliative Treatment of Metastatic Colorectal Cancer Study Protocol 1. J Clin Oncol 14:2274-2279, 1996
33. de Gramont A, Bosset JF, Milan C, et al: Randomized trial comparing monthly low-dose leucovorin and fluorouracil bolus with bimonthly high-dose leucovorin and fluorouracil bolus plus continuous infusion for advanced colorectal cancer: A French intergroup study. J Clin Oncol 15:808-815, 1997
34. Goldberg RM, Hatfield AK, Kahn M, et al: Prospectively randomized North Central Cancer Treatment Group trial of intensive-course fluorouracil combined with the l-isomer of intravenous leucovorin, oral leucovorin, or intravenous leucovorin for the treatment of advanced colorectal cancer. J Clin Oncol 15:3320-3329, 1997
35. Labianca R, Cascinu S, Frontini L, et al: Highversus low-dose levo-leucovorin as a modulator of 5-fluorouracil in advanced colorectal cancer: A 'GISCAD' phase III study - Italian Group for the Study of Digestive Tract Cancer. Ann Oncol 8:169-174, 1997
36. Scheithauer W, Kornek G, Marczell A, et al: Fluorouracil plus racemic leucovorin versus fluorouracil combined with the pure l-isomer of leucovorin for the treatment of advanced colorectal cancer: A randomized phase III study. J Clin Oncol 15:908-914, 1997
37. Aranda E, Diaz-Rubio E, Cervantes A, et al: Randomized trial comparing monthly low-dose leucovorin and fluorouracil bolus with weekly high-dose 48-hour continuous-infusion fluorouracil for advanced colorectal cancer: A Spanish Cooperative Group for Gastrointestinal Tumor Therapy (TTD) study. Ann Oncol 9:727-731, 1998
38. Borner MM, Castiglione M, Bacchi M, et al: The impact of adding low-dose leucovorin to monthly 5-fluorouracil in advanced colorectal carcinoma: Results of a phase III trial - Swiss Group for Clinical Cancer Res (SAKK). Ann Oncol 9:535-541, 1998
39. Cocconi G, Cunningham D, Van Cutsem E, et al: Open, randomized, multicenter trial of raltitrexed versus fluorouracil plus high-dose leucovorin in patients with advanced colorectal cancer: Tomudex Colorectal Cancer Study Group. J Clin Oncol 16:2943-2952, 1998
40. Giacchetti S, Perpoint B, Zidani R, et al: Phase III multicenter randomized trial of oxaliplatin added to chronomodulated fluorouracil-leucovorin as first-line treatment of metastatic colorectal cancer. J Clin Oncol 18:136-147, 2000
41. Hoff PM, Ansari R, Batist G, et al: Comparison of oral capecitabine versus intravenous fluorouracil plus leucovorin as first-line treatment in 605 patients with metastatic colorectal cancer: Results of a randomized phase III study. J Clin Oncol 19:2282-2292, 2001
42. O'Dwyer PJ, Manola J, Valone FH, et al: Fluorouracil modulation in colorectal cancer: Lack of improvement with N-phosphonoacetyl-l-aspartic acid or oral leucovorin or interferon, but enhanced therapeutic index with weekly 24-hour infusion schedule - An Eastern Cooperative Oncology Group/Cancer and Leukemia Group B Study. J Clin Oncol 19:2413-2421, 2001
43. Van Cutsem E, Twelves C, Cassidy J, et al: Oral capecitabine compared with intravenous fluorouracil plus leucovorin in patients with metastatic colorectal cancer: Results of a large phase III study. J Clin Oncol 19:4097-4106, 2001
44. Blanke CD, Shultz J, Cox J, et al: A doubleblind placebo-controlled randomized phase III trial of 5-fluorouracil and leucovorin, plus or minus trimetrexate, in previously untreated patients with advanced colorectal cancer. Ann Oncol 13:87-91, 2002
45. Carmichael J, Popiela T, Radstone D, et al: Randomized comparative study of tegafur/uracil and oral leucovorin versus parenteral fluorouracil and leucovorin in patients with previously untreated metastatic colorectal cancer. J Clin Oncol 20:3617-3627, 2002
46. Douillard JY, Hoff PM, Skillings JR, et al: Multicenter phase III study of uracil/tegafur and oral leucovorin versus fluorouracil and leucovorin in patients with previously untreated metastatic colorectal cancer. J Clin Oncol 20:3605-3616, 2002
47. Grothey A, Deschler B, Kroening H, et al: Phase III study of bolus 5-fluorouracil (5-FU)/ folinic acid (FA) (Mayo) vs weekly high-dose 24h 5-FU infusion/ FA +- oxaliplatin (OXA) (FUFOX) in advanced colorectal cancer (ACRC). Proc Am Soc Clin Oncol 20:129a, 2002 (abstr 512)
48. Maughan TS, James RD, Kerr DJ, et al: Comparison of survival, palliation, and quality of life with three chemotherapy regimens in metastatic colorectal cancer: A multicentre randomised trial. Lancet 359:1555-1563, 2002
49. Punt CJ, Keizer HJ, Douma J, et al: Trimetrexate as biochemical modulator of 5-fluorouracil/leucovorin in advanced colorectal cancer: Final results of a randomised European study. Ann Oncol 13:81-86, 2002
50. Schilsky RL, Levin J, West WH, et al: Randomized, open-label, phase III study of a 28-day oral regimen of eniluracil plus fluorouracil versus intravenous fluorouracil plus leucovorin as first-line therapy in patients with metastatic/advanced colorectal cancer. J Clin Oncol 20:1519-1526, 2002
51. Köhne CH, Wils J, Lorenz M, et al: Randomized phase III study of high-dose fluorouracil given as a weekly 24-hour infusion with or without leucovorin versus bolus fluorouracil plus leucovorin in advanced colorectal cancer: European Organization of Research and Treatment of Cancer Gastrointestinal Group Study 40952. J Clin Oncol 21:3721-3728, 2003
52. Fischer von Weikersthal L, Schalhorn A, Quietzsch D, et al: Randomised comparison of 5-FU/ folinic acid plus irinotecan (FOLFIRI) and irinotecan plus oxaliplatin (IROX) in first-line therapy of metastatic colorectal cancer (CRC): The fire-trial. Eur J Cancer 3:167, 2005 (suppl 2; abstr 598)
53. Hurwitz HI, Fehrenbacher L, Hainsworth JD, et al: Bevacizumab in combination with fluorouracil and leucovorin: An active regimen for first-line metastatic colorectal cancer. J Clin Oncol 23:3502-3508, 2005
54. Pluzanska A, Mainwaring P, Cassidy J, et al: Final results of a randomized phase III study evaluating the addition of oxaliplatin first line to 5-FU followed by irinotecan at progression in advanced colorectal cancer (LIFE study). J Clin Oncol 23:250s, 2005 (suppl; abstr 3517)
55. 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 23:8664-8670, 2005
56. Baker SG: Surrogate endpoints: Wishful thinking or reality? J Natl Cancer Inst 98:502-503, 2006
57. Prentice RL: Surrogate endpoints in clinical trials: Definition and operational criteria. Stat Med 8:431-440, 1989
58. Rustin GJ, Nelstrop AE, Bentzen SM, et al: Selection of active drugs for ovarian cancer based on CA-125 and standard response rates in phase II trials. J Clin Oncol 18:1733-1739, 2000
59. Buyse M, Molenberghs G, Burzykowski T, et al: The validation of surrogate endpoints in metaanalyses of randomized experiments. Biostatistics 1:49-67, 2000
60. Begg CB, Leung DHY: On the use of surrogate endpoints in randomized trials. J R Stat Soc (Ser A) 163:15-28, 2000
61. Louvet C, de Gramont A, Tournigand C, et al: Correlation between progression free survival and response rate in patients with metastatic colorectal carcinoma. Cancer 91:2033-2038, 2001
62. Buyse M, Thirion P, Carlson RW, et al: Relation between tumour response to first-line chemotherapy and survival in advanced colorectal cancer: A meta-analysis - Meta-Analysis Group in Cancer. Lancet 356:373-378, 2000
63. Buyse M, Burzykowski T, Carroll K, et al: Progression-free survival (PFS) as a surrogate for overall survival (OS) in patients with advanced colorectal cancer: An analysis of 3159 patients randomized in 11 trials. J Clin Oncol 23:249s, 2005 (suppl; abstr 3513)