Biomarker-adaptive threshold design: A procedure for evaluating treatment with possible biomarker-defined subset effect

Journal of the National Cancer Institute

Volumn 99, Issue 13, 2007, Pages 1036-1043

  Jiang, Wenyu ^a   Freidlin, Boris ^a   Simon, Richard ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; BIOLOGICAL MARKER; DIETHYLSTILBESTROL; PLACEBO; TUMOR MARKER;

ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG SENSITIVITY; EXPERIMENTAL DESIGN; HUMAN; MAJOR CLINICAL STUDY; MALE; MEDICAL DECISION MAKING; OVERALL SURVIVAL; PRIORITY JOURNAL; PROSTATE CANCER; RANDOMIZED CONTROLLED TRIAL; RISK BENEFIT ANALYSIS; SAMPLE SIZE; SIMULATION; VALIDATION PROCESS; ALGORITHM; BIOASSAY; BLOOD; COMPUTER SIMULATION; DRUG SCREENING; METABOLISM; METHODOLOGY; NEOPLASM; PHASE 3 CLINICAL TRIAL; PROSTATE TUMOR; STATISTICAL ANALYSIS;

ALGORITHMS; ANTICARCINOGENIC AGENTS; CLINICAL TRIALS; COMPUTER SIMULATION; DATA INTERPRETATION, STATISTICAL; DRUG EVALUATION; DRUG EVALUATION, PRECLINICAL; ENDPOINT DETERMINATION; HUMANS; MALE; NEOPLASMS; PROSTATIC NEOPLASMS; RESEARCH DESIGN; SAMPLE SIZE; TUMOR MARKERS, BIOLOGICAL;

EID: 34447264769     PISSN: 00278874     EISSN: 14602105     Source Type: Journal    
DOI: 10.1093/jnci/djm022     Document Type: Article

References (17)

1. Simon R, Maitournam A. Evaluating the efficiency of targeted designs for randomized clinical trails. Clin Cancer Res 2004;10:6759-63.
2. Simon R, Wang SJ. Use of genomic signatures in therapeutics development in oncology and other diseases. Pharmacogenomics J 2006;6:166-73.
3. Freidlin B, Simon R. Adaptive signature design: An adaptive clinical trial design for generating and prospectively testing a gene expression signature for sensitive patients. Clin Cancer Res 2005;11:7872-8.
4. Dziadziuszko R, Hirsch FR, Varella-Garcia M, Bunn PA Jr. Selecting lung cancer patients for treatment with epidermal growth factor receptor tyrosine kinase inhibitors by immunohistochemistry and fluorescence in situ hybridization - why, when, and how? Clin Cancer Res 2006;12: 4409s-15s.
5. Cox DR. Regression models and life tables with discussion. J Royal Stat Soc B 1972;34:187-220.
6. Altman DG, Lausen B, Sauerbrei W, Schumacher M. Dangers of using "optimal" cutpoints in the evaluation of prognostic factors. J Natl Cancer Inst 1994;86:829-35.
7. Miller R, Siegmund D. Maximally selected chi-square statistics. Biometrics 1982;38:1011-6.
8. Simon R, Altman DG. Statistical aspects of prognostic factor studies in oncology. Br J Cancer 1994;69:979-85.
9. Simon RM, Korn EL, McShane LM, Radmacher MD, Wright GW, Zhao Y. Design and analysis of DNA microarray investigations. New York NY:SPringer;2004.
10. Byar DP, Corle DK. Selecting optimal treatment in clinical trials using covariate information. J Chronic Dis 1977;30:445-59.
11. Andrews DF, Herzberg AM. Data. Chapter 46. New York NY:SPringer; 1985. p. 261-74.
12. Tukey JW. Some thoughts on clinical-trials, especially problems of multiplicity. Science 1977;198:679-84.
13. Simon R. Problems of multiplicity in clinical trials. Journal of Statistical Planning and Inference 1994;42:209-221.
14. Ravaud A, Gardner J, Hawkins R, Von der Maase H, Zantl N, Harper P, et al.; Tykerb Renal Cell Cancer Study Group and GSK Core T. Efficacy of lapatinib in patients with high tumor EGFR expression: Results of a phase III trial in advanced renal cell carcinoma RCC 2006 ASCO Annual Meeting Proceedings 2006;24 Abst4502.
15. Rubinstein LV, Gail MH, Santner TJ. Planning the duration of a comparative clinical trial with loss to follow-up and a period of continued observation. J Chronic Dis 1981;34:469-79.
16. Tsao MS, Sakurada A, Cutz JC, Zhu CQ, Kamel-Reid S, Squire J, et al. Erlotinib in lung cancer - molecular and clinical predictors of outcome. N Engl J Med 2005;353:133-44.
17. Simon R. Size of phase III cancer clinical trials. Cancer Treat Rep 1985;69:1087-93.