Two-stage dose finding for cytostatic agents in phase I oncology trials

Statistics in Medicine

Volumn 32, Issue 4, 2013, Pages 644-660

  Yin, Guosheng ^a,b   Zheng, Shurong ^c   Xu, Jiajing ^a  

^a UNIVERSITY OF HONG KONG   (Hong Kong)

^b UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^c NORTHEAST NORMAL UNIVERSITY   (China)

Author keywords

Adaptive design; Censored data; Delayed response; Dose de escalation; Dose escalation; Efficacy; Kaplan Meier estimator; Time to event; Toxicity

Indexed keywords

CYTOSTATIC AGENT; CYTOTOXIC AGENT;

ARTICLE; DOSE CALCULATION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG TOLERABILITY; HUMAN; MULTICENTER STUDY; PHASE 1 CLINICAL TRIAL; SIMULATION; TOXICITY;

ALGORITHMS; BIOSTATISTICS; CLINICAL TRIALS, PHASE I AS TOPIC; CYTOSTATIC AGENTS; DOSE-RESPONSE RELATIONSHIP, DRUG; HUMANS; MAXIMUM TOLERATED DOSE; MODELS, BIOLOGICAL; MODELS, STATISTICAL; MOLECULAR TARGETED THERAPY; NEOPLASMS; TREATMENT OUTCOME;

EID: 84872822809     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.5546     Document Type: Article

References (28)

1. Chevret S. Statistical Methods for Dose-Finding Experiments. John Wiley & Sons: England, 2006.
2. Ting N. Dose Finding in Drug Development. Springer: Cambridge, MA, 2006.
3. Storer BE. Design and analysis of phase I clinical trials. Biometrics 1989; 45:925-937.
4. Durham SD, Flournoy N, Rosenberger WF. A random walk rule for phase I clinical trials. Biometrics 1997; 53:745-760.
5. O'Quigley J, Pepe M, Fisher L. Continual reassessment method: a practical design for phase I clinical trials in cancer. Biometrics 1990; 46:33-48.
6. Goodman SN, Zahurak ML, Piantadosi S. Some practical improvements in the continual reassessment method for phase I studies. Statistics in Medicine 1995; 14:1149-1161.
7. Heyd JM, Carlin BP. Adaptive design improvements in the continual reassessment method for phase I studies. Statistics in Medicine 1999; 18:1307-1321.
8. Ishizuka N, Ohashi Y. The continual reassessment method and its applications: a Bayesian methodology for phase I cancer clinical trials. Statistics in Medicine 2001; 20:2661-2681.
9. Yin G. Clinical Trial Design: Bayesian and Frequentist Adaptive Methods. John Wiley & Sons: Hoboken, New Jersey, 2012.
10. Korn EL. Nontoxicity endpoints in phase I trial designs for targeted, non-cytotoxic agents. Journal of the National Cancer Institute 2004; 96:977-978.
11. Hunsberger S, Rubinstein VL, Dancey J, Korn LE. Dose escalation trial designs based on a molecularly targeted endpoint. Statistics in Medicine 2005; 24:2171-2181.
12. Green SJ, Dahlberg S. Planned versus attained design in phase II clinical trials. Statistics in Medicine 1992; 11:853-862.
13. Gooley TA, Martin PJ, Fisher LD, Pettinger M. Simulation as a design tool for phase I/II clinical trial: an example from bone marrow transplantation. Controlled Clinical Trial 1994; 15:450-462.
14. Thall PF, Russell KE. A strategy for dose-finding and safety monitoring based on efficacy and adverse outcomes in phase I/II clinical trials. Biometrics 1998; 54:251-264.
15. O'Quigley J, Hughes MD, Fenton T. Dose-finding designs for HIV studies. Biometrics 2001; 57:1018-1029.
16. Thall PF, Cook J. Dose-finding based on toxicity-efficacy trade-offs. Biometrics 2004; 60:684-693.
17. Yin G, Li Y, Ji Y. Bayesian dose-finding in phase I/II trials using toxicity and efficacy odds ratio. Biometrics 2006; 62:777-784.
18. Korn EL, Arbuck SG, Pluda JM, Simon R, Kaplan RS, Christian MC. Clinical trial designs for cytostatic agents: are new approaches needed? Journal of Clinical Oncology 2001; 19:265-272.
19. Ratain MJ, Sargent D. Optimising the design of phase II oncology trials: the importance of randomisation. European Journal of Cancer 2009; 45:275-280.
20. Kaplan EL, Meier P. Non-parametric estimation from incomplete observations. Journal of the American Statistical Association 1958; 53:457-481.
21. Efron B. The two-sample problem with censored data. In Proceeding of Fifth Berkeley Symposium in Mathematical Statistics, IV, Le Cam L, Neyman J (eds). Prentice-Hall: New York, 1967.
22. Portnoy S. Censored regression quantiles. Journal of the American Statistical Association 2003; 98:1001-1012.
23. Dabrowska DM. Uniform consistency of kernel conditional Kaplan-Meier estimate. The Annals of Statistics 1989; 17:1157-1167.
24. Leconte E, Poiraud-Casanova S, Thomas-Agnan C. Smooth conditional distribution function and quantiles under random censorship. Lifetime Data Analysis 2002; 8:229-246.
25. Wang H, Wang L. Locally weighted censored quantile regression. Journal of the American Statistical Association 2009; 104:1117-1128.
26. Clayton DG. A model for association in bivariate life tables and its application in epidemiological studies of familial tendency in chronic disease incidence. Biometrika 1978; 65:141-152.
27. Simon R, Freidlin B, Rubinstein L, Arbuck SG, Collins J, Christian MC. Accelerated titration designs for phase I clinical trials in oncology. Journal of the National Cancer Institute 1997; 89:1138-47.
28. Cox DR. Regression models and life-tables (with discussion). Journal of the Royal Statistical Society, Series B 1972; 34:187-220.