Progression-free survival is a surrogate for survival in advanced colorectal cancer

Marc Buyse, Tomasz Burzykowski, Kevin Carroll, Stefan Michiels, Daniel J. Sargent, Langdon L. Miller, Gary L. Elfring, Jean Pierre Pignon, Pascal Piedbois

Research output: Contribution to journalArticle

250 Citations (Scopus)

Abstract

Purpose: The traditional end point for assessing efficacy of first-line chemotherapies for advanced cancer is overall survival (OS), but this end point requires prolonged follow-up and is potentially confounded by the effects of second-line therapies. We investigated whether progression-free survival (PFS) could be considered a valid surrogate for OS in advanced colorectal cancer. Patients and Methods: Individual patient data were available from 10 historical trials comparing fluouracil (FU) + leucovorin with either FU alone (1,744 patients) or with raltitrexed (1,345 patients) and from three validation trials comparing FU + leucovorin with or without irinotecan or oxaliplatin (1,263 patients). Correlation coefficients were estimated in historical trials between the end points of PFS and OS, and between the treatment effects on these end points. Treatment effects on OS were predicted in validation trials, and compared with the observed effects. Results: In historical trials, 1,760 patients (57%) had progressed or died at 6 months, and 1,622 (52%) had died at 12 months. The rank correlation coefficient between PFS and OS was equal to 0.82 (95% CI, 0.82 to 0.83). The correlation coefficient between treatment effects on PFS and on OS ranged from 0.99 (95% CI, 0.94 to 1.04) when all trials were considered to 0.74 (95% CI, 0.44 to 1.04) after exclusion of one highly influential trial. In the validation trials, the observed OS hazard ratios were within the 95% prediction intervals. A hazard ratio of 0.77 or lower in terms of PFS would predict a benefit in terms of OS. Conclusion: PFS is an acceptable surrogate for OS in advanced colorectal cancer.

Original languageEnglish (US)
Pages (from-to)5218-5224
Number of pages7
JournalJournal of Clinical Oncology
Volume25
Issue number33
DOIs
StatePublished - Nov 20 2007

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Disease-Free Survival
Colorectal Neoplasms
Survival
oxaliplatin
irinotecan
Leucovorin
Therapeutics
Drug Therapy
Neoplasms

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

Cite this

Buyse, M., Burzykowski, T., Carroll, K., Michiels, S., Sargent, D. J., Miller, L. L., ... Piedbois, P. (2007). Progression-free survival is a surrogate for survival in advanced colorectal cancer. Journal of Clinical Oncology, 25(33), 5218-5224. https://doi.org/10.1200/JCO.2007.11.8836

Progression-free survival is a surrogate for survival in advanced colorectal cancer. / Buyse, Marc; Burzykowski, Tomasz; Carroll, Kevin; Michiels, Stefan; Sargent, Daniel J.; Miller, Langdon L.; Elfring, Gary L.; Pignon, Jean Pierre; Piedbois, Pascal.

In: Journal of Clinical Oncology, Vol. 25, No. 33, 20.11.2007, p. 5218-5224.

Research output: Contribution to journalArticle

Buyse, M, Burzykowski, T, Carroll, K, Michiels, S, Sargent, DJ, Miller, LL, Elfring, GL, Pignon, JP & Piedbois, P 2007, 'Progression-free survival is a surrogate for survival in advanced colorectal cancer', Journal of Clinical Oncology, vol. 25, no. 33, pp. 5218-5224. https://doi.org/10.1200/JCO.2007.11.8836
Buyse M, Burzykowski T, Carroll K, Michiels S, Sargent DJ, Miller LL et al. Progression-free survival is a surrogate for survival in advanced colorectal cancer. Journal of Clinical Oncology. 2007 Nov 20;25(33):5218-5224. https://doi.org/10.1200/JCO.2007.11.8836
Buyse, Marc ; Burzykowski, Tomasz ; Carroll, Kevin ; Michiels, Stefan ; Sargent, Daniel J. ; Miller, Langdon L. ; Elfring, Gary L. ; Pignon, Jean Pierre ; Piedbois, Pascal. / Progression-free survival is a surrogate for survival in advanced colorectal cancer. In: Journal of Clinical Oncology. 2007 ; Vol. 25, No. 33. pp. 5218-5224.
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abstract = "Purpose: The traditional end point for assessing efficacy of first-line chemotherapies for advanced cancer is overall survival (OS), but this end point requires prolonged follow-up and is potentially confounded by the effects of second-line therapies. We investigated whether progression-free survival (PFS) could be considered a valid surrogate for OS in advanced colorectal cancer. Patients and Methods: Individual patient data were available from 10 historical trials comparing fluouracil (FU) + leucovorin with either FU alone (1,744 patients) or with raltitrexed (1,345 patients) and from three validation trials comparing FU + leucovorin with or without irinotecan or oxaliplatin (1,263 patients). Correlation coefficients were estimated in historical trials between the end points of PFS and OS, and between the treatment effects on these end points. Treatment effects on OS were predicted in validation trials, and compared with the observed effects. Results: In historical trials, 1,760 patients (57{\%}) had progressed or died at 6 months, and 1,622 (52{\%}) had died at 12 months. The rank correlation coefficient between PFS and OS was equal to 0.82 (95{\%} CI, 0.82 to 0.83). The correlation coefficient between treatment effects on PFS and on OS ranged from 0.99 (95{\%} CI, 0.94 to 1.04) when all trials were considered to 0.74 (95{\%} CI, 0.44 to 1.04) after exclusion of one highly influential trial. In the validation trials, the observed OS hazard ratios were within the 95{\%} prediction intervals. A hazard ratio of 0.77 or lower in terms of PFS would predict a benefit in terms of OS. Conclusion: PFS is an acceptable surrogate for OS in advanced colorectal cancer.",
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AU - Pignon, Jean Pierre

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N2 - Purpose: The traditional end point for assessing efficacy of first-line chemotherapies for advanced cancer is overall survival (OS), but this end point requires prolonged follow-up and is potentially confounded by the effects of second-line therapies. We investigated whether progression-free survival (PFS) could be considered a valid surrogate for OS in advanced colorectal cancer. Patients and Methods: Individual patient data were available from 10 historical trials comparing fluouracil (FU) + leucovorin with either FU alone (1,744 patients) or with raltitrexed (1,345 patients) and from three validation trials comparing FU + leucovorin with or without irinotecan or oxaliplatin (1,263 patients). Correlation coefficients were estimated in historical trials between the end points of PFS and OS, and between the treatment effects on these end points. Treatment effects on OS were predicted in validation trials, and compared with the observed effects. Results: In historical trials, 1,760 patients (57%) had progressed or died at 6 months, and 1,622 (52%) had died at 12 months. The rank correlation coefficient between PFS and OS was equal to 0.82 (95% CI, 0.82 to 0.83). The correlation coefficient between treatment effects on PFS and on OS ranged from 0.99 (95% CI, 0.94 to 1.04) when all trials were considered to 0.74 (95% CI, 0.44 to 1.04) after exclusion of one highly influential trial. In the validation trials, the observed OS hazard ratios were within the 95% prediction intervals. A hazard ratio of 0.77 or lower in terms of PFS would predict a benefit in terms of OS. Conclusion: PFS is an acceptable surrogate for OS in advanced colorectal cancer.

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