A statistical model for the dependence between progression-free survival and overall survival

Statistics in Medicine

Volumn 28, Issue 21, 2009, Pages 2669-2686

  Fleischer, Frank ^a   Gaschler Markefski, Birgit ^a   Bluhmki, Erich ^a  

^a BOEHRINGER INGELHEIM PHARMA GMBH AND CO KG   (Germany)

Author keywords

Dependence; Oncology; Overall survival; Progression free survival; Statistical modelling; Surrogate endpoint

Indexed keywords

ARTICLE; DISEASE COURSE; HUMAN; LUNG NON SMALL CELL CANCER; MAXIMUM LIKELIHOOD METHOD; ONCOLOGICAL PARAMETERS; OVERALL SURVIVAL; PROGRESSION FREE SURVIVAL; STATISTICAL MODEL; STATISTICAL SIGNIFICANCE; THERAPY EFFECT; TIME TO PROGRESSION; TREATMENT DURATION;

BIOLOGICAL MARKERS; CARCINOMA, NON-SMALL-CELL LUNG; DISEASE-FREE SURVIVAL; HUMANS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC;

EID: 70449397174     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.3637     Document Type: Article

References (23)

1. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER). Guidance for Industry: Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics, U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), 2007.
2. Therasse P, Arbuck SG, Eisenhauer EA, Wanders J, Kaplan RS, Rubinstein L, Verweij J, van Glabbeke M, van Oosterom AT, Christian MC, Gwyther SG. New guidelines to evaluate the response to treatment in solid tumors. Journal of the National Cancer Institute 2000; 92:205-216. DOI: 10.1093/jnci/92.3.205. (Pubitemid 30099779)
3. Eisenhauer EA, Therasse P, Bogaerts J, Schwartz LH, Sargent D, Ford R, Dancey J, Arbuck S, Gwyther S, Mooney M, Rubinstein L, Shankar L, Dodd L, Kaplan R, Lacombe D, Verweij J. New response evaluation criteria in solid tumours: revised RECIST guidelines (version 1.1). European Journal of Cancer 2009; 45:228-247.
4. Eskens FA, Verweij J. Clinical studies in the development of new anticancer agents exhibiting growth inhibition in models: facing the challenge of a proper study design. Critical Reviews in Oncology/Haematology 2000; 34:83-88. DOI: 10.1016/S1040-8428(00)00055-X. (Pubitemid 30229918)
5. Stone A, Wheeler C, Barge A. Improving the design of phase II trials of cytostatic anticancer agents. Contemporary Clinical Trials 2007; 28:138-145. DOI: 10.1016/j.cct.2006.05.009. (Pubitemid 44937880)
6. Buyse M, Burzykowski T, Carroll K, Michiels S, Sargent DJ, Miller LL, Elfring GL, Pignon JP, Piedbois P. Progression-free survival is a surrogate for survival in advanced colorectal cancer. Journal of Clinical Oncology 2007; 25:5218-5224. DOI: 10.1200/JCO.2007.11.8836. (Pubitemid 350232253)
7. Gordan LN, Sugrue MW, Lynch JW, Williams KD, Khan SA, Moreb JS. Correlation of early lymphocyte recovery and progression-free survival after autologous stem-cell transplant in patients with Hodgkin's and non-Hodgkin's Lymphoma. Bone Marrow Transplantation 2003; 31:1003-1019. DOI: 10.1038/sj.bmt.1704050.
8. Louvet C, de Gramont A, Tournigand A, Artru P, Maindrault-Goebel F, Krulik M. Correlation between progression free survival and response rate in patients with metastatic colorectal carcinoma. Cancer 2001; 91:2033-2038. DOI: 10.1002/1097-0142(20010601)91:11(2033::AID-CNCR1229)3.0.CO;2-J. (Pubitemid 32479191)
9. Tang AP, Bentzen SM, Chen EX, Siu LL. 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 2007; 25:4562-4568. DOI: 10.1200/JCO.2006.08.1935.
10. Sherrill BH, Hirst C, Amonkar MM, Stein SH. Correlation between time to progression and overall survival in patients with metastatic breast cancer. ISPOR 12th Annual International Meeting, Arlington, VA, 2007.
11. Anscombe FJ. Graphs in statistical analysis. American Statistician 1973; 27:17-21.
12. Ellis S, Carroll KJ, Pemberton K. Analysis of duration of response in oncology trials. Contemporary Clinical Trials 2008; 29:456-465. DOI: 10.1016/j.cct.2007.10.008.
13. Begg BC, Larson M. A study of the use of the probability-of-being-in- response function as a summary of tumour response data. Biometrics 1982; 38:59-66.
14. Collett D. Modelling Survival Data in Medical Research (2nd edn). Chapman & Hall/CRC: Boca Raton, 2003.
15. Hainsworth J, Herbst R. A phase III multicenter, placebo-controlled double-blind randomized clinical trial to evaluate the efficacy of Bevacizumab in combination with Erlotinib compared with Erlotinib alone for treatment of advanced non-small cell lung cancer after failure of standard first-line chemotherapy. Multidisciplinary Symposium in Thoracic Oncology, Chicago, IL, 2008.
16. Meira-Machado L, de Una-Alvarez J, Cadarso-Suarez C, Andersen PK. Multi-state models for the analysis of time-to-event data. Statistical Methods in Medical Research 2008. DOI: 10.1177/0962280208092301.
17. Wu GH-D, Chang SH, Chen TH-H. A Bayesian random effects Markov model for tumour progression in women with a family history of breast cancer. Biometrics 2008; 64:1231-1237. DOI: 10.1111/j.1541-0420.2007.000979.x.
18. Commenges D. Inference for multi-state models from interval-censored data. Statistical Methods in Medical Research 2002; 11:167-182. DOI: 10.1191/0962280202sm279ra. (Pubitemid 34516545)
19. Oakes D. Model based and or marginal analysis of multiple event time data. In Proceedings of the First Seattle Symposium in Biostatistics: Survival Analysis, Lin DY, Fleming TR (eds). Springer: New York, 1997; 85-98.
20. Miller KD, Chap LI, Holmes FA, Cobleigh MA, Marcom PK, Fehrenbacher L, Dickler M, Overmoyer BA, Reimann JD, Sing AP, Langmuir V, Rugo HS. Randomized phase III trial of capecitabine compared with bevacizumab plus capecitabine in patients with previously treated metastatic breast cancer. Journal of Clinical Oncology 2005; 23:792-799. DOI: 10.1200/JCO.2005.05.098.
21. Yu RX, Holmgren E. Endpoints for agents that slow tumor growth. Contemporary Clinical Trials 2007; 28:18-24. DOI: 10.1016/j.cct.2006.05.011. (Pubitemid 44780204)
22. Carroll KJ. Analysis of progression-free survival in oncology trials: some common statistical issues. Pharmaceutical Statistics 2007; 6:99-113. DOI: 10.1002/pst.251.
23. Freidlin B, Korn LE, Hunsberger S, Gray R, Saxman S, Zujewski JA. Proposal for the use of progression-free survival in unblinded randomized trials. Journal of Clinical Oncology 2007; 25:2122-2126. DOI:10.1200/JCO.2006. 09.6198. (Pubitemid 46972800)