Cited by 713Statistical Methods in Clinical TrialsColorectal Cancer Treatments and StudiesColorectal Cancer Surgical Treatments

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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

Sargent, Daniel J., Wieand, Harry S., Haller, Daniel G., Gray, Richard, Benedetti, Jacqueline K., Buyse, Marc, Labianca, Roberto, Seitz, Jean Francois, O'Callaghan, Christopher J., Francini, Guido, Grothey, Axel, O'Connell, Michael, Catalano, Paul J., Blanke, Charles D., Kerr, David, Green, Erin, Wolmark, Norman, Andre, Thierry, Goldberg, Richard M., De Gramont, Aimery·2005·Journal of Clinical Oncology

DOI 10.1200/jco.2005.01.6071

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Abstract

PURPOSE: A traditional end point for colon adjuvant clinical trials is overall survival (OS), with 5 years demonstrating adequate follow-up. A shorter-term end point providing convincing evidence to allow treatment comparisons could significantly speed the translation of advances into practice. METHODS: Individual patient data were pooled from 18 randomized phase III colon cancer adjuvant clinical trials. Trials included 43 arms, with a pooled sample size of 20,898 patients. The primary hypothesis was that disease-free survival (DFS), with 3 years of follow-up, is an appropriate primary end point to replace OS with 5 years of follow-up. RESULTS: The recurrence rates for years 1 through 5 were 12%, 14%, 8%, 5%, and 3%, respectively. Median time from recurrence to death was 12 months. Eighty percent of recurrences were in the first 3 years; 91% of patients with recurrence by 3 years died before 5 years. Correlation between 3-year DFS and 5-year OS was 0.89. Comparing control versus experimental arms within each trial, the correlation between hazard ratios for DFS and OS was 0.92. Within-trial log-rank testing using both DFS and OS provided the same conclusion in 23 (92%) of 25 cases. Formal measures of surrogacy were satisfied. CONCLUSION: In patients treated on phase III adjuvant colon clinical trials, DFS and OS are highly correlated, both within patients and across trials. These results suggest that DFS after 3 years of median follow-up is an appropriate end point for adjuvant colon cancer clinical trials of fluorouracil-based regimens, although marginally significant DFS improvements may not translate into significant OS benefits.

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Regression Models and Life-Tables
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BackgroundInfluential
Single centre guidelines for radiological follow-up based on 775 patients treated by cytoreductive surgery and HIPEC for appendiceal pseudomyxoma peritonei
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Background
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Background
The Multidisciplinary Management of Rectal Cancer
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BackgroundMethod
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Li, Y., Li, S., Wu, Z., Hu, F., Zhu, L., Zhao, X., Cui, B., Dong, X., Tian, S., Wang, F., et al. (2013) · Medical Oncology · doi:10.1007/s12032-013-0505-z · Cited by 40
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Leukemia-free survival as a surrogate end point for overall survival in the evaluation of maintenance therapy for patients with acute myeloid leukemia in complete remission
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“Other criteria have been proposed in the literature for the validation of surrogate end points, but those adopted here have been used extensively in solid tumors both in the adjuvant treatment and advanced disease settings.(3-5,7,13,15,16,23,31-34) The trial on which this analysis was based did not achieve significanc…”
Method
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“However, the response rate is usually less than 50% [2] and the five-year survival rate of mCRC patients remains below 10% [3].”
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“[5] in the analysis of the adjuvant colorectal cancer studies and therefore exists as a potential for possible future exploration.”
BackgroundMethod
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Background
Conditional copula models for correlated survival endpoints: Individual patient data meta-analysis of randomized controlled trials
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“For instance, Buyse et al.4 applied DFS (PFS) for early (advanced) colorectal cancer; see also Sargent et al.11 for the application of DFS for adjuvant colon cancer studies.”
BackgroundResult
Physical Activity in Stage III Colon Cancer: CALGB/SWOG 80702 (Alliance)
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“Absolute effects are presented as risk differences (RDs) at 3 years for DFS and 5 years for OS, as these time horizons are clinically meaningful.(26) CIs for the RD were calculated using the bootstrap method with 1,000 replicates.”
Method
Cinnamaldehyde/chemotherapeutic agents interaction and drug-metabolizing genes in colorectal cancer
YU, C., Liu, S., QI, M.-H., & Zou, X. (2013) · Molecular Medicine Reports · doi:10.3892/mmr.2013.1830 · Cited by 38
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FOXE1 represses cell proliferation and Warburg effect by inhibiting HK2 in colorectal cancer
Dai, W., Meng, X., Mo, S., Xiang, W., Xu, Y., Zhang, L., Wang, R., Li, Q., & Cai, G. (2020) · Cell Communication and Signaling · doi:10.1186/s12964-019-0502-8 · Cited by 38
Pitfalls in outcome prediction of breast cancer
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Determinants of Early Mortality Among 37,568 Patients With Colon Cancer Who Participated in 25 Clinical Trials From the Adjuvant Colon Cancer Endpoints Database
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“(%) 1970s-1980s 5,669 (15) 1990s 17,940 (48) 2000s 13,955 (37) Missing 4 (0) Treatment, No.”
An Update on Colorectal Cancer
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Response Rate, Event-Free Survival, and Overall Survival in Newly Diagnosed Acute Myeloid Leukemia: US Food and Drug Administration Trial-Level and Patient-Level Analyses
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Vera-Badillo, F. E., Al-Mubarak, M., Templeton, A. J., & Amir, E. (2013) · Current Oncology Reports · doi:10.1007/s11912-013-0303-y · Cited by 35
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Wind, J., Duineveld, L. A. M., van der Heijden, R. P., van Asselt, K. M., Bemelman, W. A., & van Weert, H. C. P. M. (2013) · European Journal of Surgical Oncology · doi:10.1016/j.ejso.2013.04.001 · Cited by 36
Effect of BRAF V600E mutation on tumor response of anti-EGFR monoclonal antibodies for first-line metastatic colorectal cancer treatment: a meta-analysis of randomized studies
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“However, the median survival of mCRC patients is only about 20 months [4].”
Background
Linear quantification of lymphoid infiltration of the tumor margin: a reproducible method, developed with colorectal cancer tissues, for assessing a highly variable prognostic factor
Allard, M., Bachet, J., Beauchet, A., Julié, C., Malafosse, R., Penna, C., Nordlinger, B., & Emile, J. (2012) · Diagnostic Pathology · doi:10.1186/1746-1596-7-156 · Cited by 39
Prognostic significance of neoadjuvant rectal score in locally advanced rectal cancer after neoadjuvant chemoradiotherapy and construction of a prediction model
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New Approaches to Assessing and Treating Early-Stage Colon and Rectal Cancers: Cooperative Group Strategies for Assessing Optimal Approaches in Early-Stage Disease
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What Constitutes Reasonable Evidence of Efficacy and Effectiveness to Guide Oncology Treatment Decisions?
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ctDNA guided adjuvant chemotherapy versus standard of care adjuvant chemotherapy after curative surgery in patients with high risk stage II or stage III colorectal cancer: a multi-centre, prospective, randomised control trial (TRACC Part C)
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“0% respectively [6], with the risk of recurrence being highest in the first 2 years following surgery [7].”
Background
From Chemotherapy to Targeted Therapy in Adjuvant Treatment for Stage III Colon Cancer
de Gramont, A., Gramont, A. D. E., Chibaudel, B., Bachet, J.-B., Larsen, A. K., Tournigand, C., Louvet, C., & André, T. (2011) · Seminars in Oncology · doi:10.1053/j.seminoncol.2011.05.006 · Cited by 35
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“[1] Many CRC patients will die of their recurrent disease unless detected early enough to receive curative treatment.[2-4]”
Background
Comparative assessment of trial-level surrogacy measures for candidate time-to-event surrogate endpoints in clinical trials
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Statistical Assessment of Biosimilar Products
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“Two-step copula models [3] are the state-of-the art statistical methodology, commonly used in numerous clinical publications over the last 15 years [53, 54, 55, 56, 31, 5, 14, 32, 57, 33, 58, 59], including a surrogate evaluation conducted by the US FDA [60].”
Method
Update on the Colon Health and Life-Long Exercise Change Trial: A Phase III Study of the Impact of an Exercise Program on Disease-Free Survival in Colon Cancer Survivors
Courneya, K. S., Vardy, J. L., Gill, S., Jonker, D. J., O’Brien, P., Friedenreich, C. M., Dhillon, H. M., Wong, R. K. S., Meyer, R. M., Crawford, J., et al. (2014) · Current Colorectal Cancer Reports · doi:10.1007/s11888-014-0231-8 · Cited by 34
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“demonstrated in a pooled analysis of patients with colon cancer that 80% of patients who experienced a recurrence were within the first 3 years [14].”
Background
Rectal cancer staging: focus on the prognostic significance of the findings described by high-resolution magnetic resonance imaging
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“[134] Sargent DJ, Wieand HS, Haller DG, et al.”
Background
Impact of frailty on oncological outcomes in patients undergoing surgery for colorectal cancer – A systematic review and meta-analysis
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Prognostic Value of Radiologic Extranodal Extension in Human Papillomavirus-Related Oropharyngeal Squamous Cell Carcinoma
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“To avoid extended follow-up and ensure efficient assessment, progression-free survival (PFS) was chosen as the primary outcome (19).”
Method
Integration of RNA-Seq and proteomics data identifies glioblastoma multiforme surfaceome signature
Syafruddin, S. E., Nazarie, W. F. W. M., Moidu, N. A., Soon, B. H., & Mohtar, M. A. (2021) · BMC Cancer · doi:10.1186/s12885-021-08591-0 · Cited by 33
Definition of disease-free survival: this is my truth–show me yours
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Cinnamaldehyde: Pharmacokinetics, anticancer properties and therapeutic potential (Review)
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“In addition, Sargent et al (44) demonstrated that patients with colon cancer still have relatively low 5‑year survival rates in chemotherapy, with high recurrence rates.”
Background
Surrogate Endpoint Analysis: An Exercise in Extrapolation
Baker, S. G., & Kramer, B. S. (2012) · JNCI Journal of the National Cancer Institute · doi:10.1093/jnci/djs527 · Cited by 32
Efficacy and safety of extended duration to perioperative thromboprophylaxis with low molecular weight heparin on disease-free survival after surgical resection of colorectal cancer (PERIOP-01): multicentre, open label, randomised controlled trial
Auer, R. C., Ott, M., Karanicolas, P. J., Brackstone, M. R., Ashamalla, S., Weaver, J., Tagalakis, V., Boutros, M., Stotland, P., Marulanda, A. C., et al. (2022) · BMJ · doi:10.1136/bmj-2022-071375 · Cited by 32
Adjuvant Chemotherapy of Stage III Colon Cancer
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Authors: Sargent, D. J., Wieand, H. S., Haller, D. G., Gray, R., Benedetti, J. K., Buyse, M., Labianca, R., Seitz, J. F., O'Callaghan, C. J., Francini, G., Grothey, A., O'Connell, M., Catalano, P. J., Blanke, C. D., Kerr, D., Green, E., Wolmark, N., Andre, T., Goldberg, R. M., & De Gramont, A.
Affiliations: University of Siena (IT)
From the North Central Cancer Treatment Group, Mayo Clinic, Rochester, MN; National Surgical Adjuvant Breast and Bowel Project Statistical Center and Operations Office, Pittsburgh; Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA; QUASAR Collaborative Group, Birmingham and Oxford, United Kingdom; Southwest Oncology Group Statistical Center, Seattle, WA; International Drug Development Institute, Brussels, Belgium; Ospedali Riuniti, Bergamo; University of Siena, Siena, Italy; University...
ORCID: Sargent, Daniel J.
Haller, Daniel G.
Gray, Richard
Buyse, Marc
Labianca, Roberto
Grothey, Axel
Catalano, Paul J.
Blanke, Charles D.
Kerr, David
Green, Erin
Wolmark, Norman
Goldberg, Richard M.
Venue: Journal of Clinical Oncology
Publisher: American Society of Clinical Oncology (ASCO)
Published: 2005-12-01
Volume: 23
Issue: 34
Pages: 8664-8670
ISSN: 0732-183X (ISSN-L)
1527-7755
DOI: 10.1200/jco.2005.01.6071
OpenAlex: W2172116130
Cited by: 713
References: 34
Citations by year: 2026: 19 · 2025: 14 · 2024: 19 · 2023: 20 · 2022: 34 · 2021: 32 · 2020: 27 · 2019: 20 · 2018: 24 · 2017: 30 · 2016: 26 · 2015: 40 · 2014: 36 · 2013: 40 · 2012: 44
FWCI: 48.43
Type: journal article
Language: en
Open access: closed
Locations: Journal of Clinical Oncology (publishedVersion)
PubMed (publishedVersion)
Document Server@UHasselt (UHasselt) (submittedVersion)
University of Birmingham Research Portal (University of Birmingham) (submittedVersion)
Use Siena air (University of Siena) (submittedVersion)
Funders: National Cancer Institute ((CA-25224, U10CA025224)
Topics: Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
Health Sciences › Medicine › Oncology › Colorectal Cancer Treatments and Studies
Health Sciences › Medicine › Oncology › Colorectal Cancer Surgical Treatments
Keywords: colon cancer, adjuvant chemotherapy, disease-free survival, overall survival, primary endpoint, surrogate endpoints, hazard ratio correlation, recurrence, individual patient data pooling
MeSH terms: Aged, Antineoplastic Combined Chemotherapy Protocols, Colonic Neoplasms, Combined Modality Therapy, Female, Follow-Up Studies, Humans, Male, Middle Aged, Neoplasm Recurrence, Local, Regression Analysis, Randomized Controlled Trials as Topic, Treatment Outcome, Chemotherapy, Adjuvant, Disease-Free Survival

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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

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