Bayesian decision theoretic two-stage design in phase II clinical trials with survival endpoint

Statistics in Medicine

Volumn 31, Issue 17, 2012, Pages 1804-1820

  Zhao, Lili ^a   Taylor, Jeremy M G ^b   Schuetze, Scott M ^c  

^a UNIVERSITY OF MICHIGAN COMPREHENSIVE CANCER CENTER   (United States)

^b UNIVERSITY OF MICHIGAN   (United States)

^c UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

Bayesian; Decision theory; Phase II clinical trial; Time to event; Two stage design

Indexed keywords

CYCLOPHOSPHAMIDE; RAPAMYCIN;

ANALYTIC METHOD; ANALYTICAL ERROR; ARTICLE; BAYES THEOREM; CALIBRATION; CLINICAL RESEARCH; COST CONTROL; EXPONENTIAL DISTRIBUTION; H EXPECTED TOTAL STUDY LENGTH DESIGN; HUMAN; MINIMAX DESIGN; MULTIPLE CYCLE TREATMENT; OPTIMAL DESIGN; SARCOMA; STATISTICAL ANALYSIS; STATISTICAL DISTRIBUTION; STIMULATION; SURVIVAL; WEIBULL DISTRIBUTION;

BAYES THEOREM; CLINICAL TRIALS, PHASE II AS TOPIC; COMPUTER SIMULATION; CYCLOPHOSPHAMIDE; DISEASE-FREE SURVIVAL; HUMANS; RESEARCH DESIGN; SARCOMA; SIROLIMUS;

EID: 84863994177     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.4511     Document Type: Article

References (31)

1. Chan JK, Ueda SM, Sugiyama VE, et al. 578 Analysis of phase II studies on targeted agents and subsequent phase III trials: what are the 579 predictors for success? J Clin Oncol 2008; 26:1511-1518.
2. Korn EL, Arbuck SG, Pluda JM, et al. Clinical trial designs for cytostatic agents: are new approaches needed? J Clin Oncol 2001; 19:265-72.
3. Stone A, Wheeler C, Barge A. Improving the design of phase II trials of cytostatic anticancer agents. Contemporary Clinical Trials 2007; 28:138-145.
4. Panageas KS, Ben-Porat L, Dickler MN, et al. When you look matters: the effect of assessment schedule on progression-free survival. J Natl Cancer Inst 2007; 99:428-32.
5. Gehan EA. The determination of the number of patients required in a preliminary and a follow up 555 trial of a new chemotherapeutic agent. J Chronic Dis 1961; 13:346-353.
6. Fleming TR. One-sample multiple testing procedure for phase II clinical trials. Biometrics 1982; 38(558):143-151.
7. Simon R. Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 1989; 10:1-10.
8. Herndon JE. A design alternative for two-stage, phase II, multicenter cancer clinical. Contemporary Clinical Trials 1998; 19(5):440-450.
9. Case LD, Morgan TM. Design of Phase II cancer trials evaluating survival probabilities. BMC Medical Research Methodology 2003; 3:6. DOI: 10.1186/1471-2288-3-6.
10. Lin DY, Shen L, Ying Z, Breslow NE. Group sequential designs for monitoring survival probabilities. Biometrics 1996; 52:1033-1041. DOI: 10.1200/JCO.2009.22.4329.
11. Huang B, Talukder E, Thomas N. Optimal two-stage phase II designs with long-term endpoints. American Statistical Association, Statsitics in Biopharmaceutical Research 2010; 2:51-60. DOI: 10.1198/sbr.2010.09001.
12. Follman DA, Albert PS. Bayesian monitoring of event rates with censored data. Biometrics 1999; 55(2):603-607.
13. Rosner GL. Bayesian monitoring of clinical trials with failure-time endpoint. Biometrics 2005; 61:239-245.
14. Cheung YK, Thall PF. Monitoring the rates of composite events with censored data in phase II clinical trials. Biometrics 2002; 58:89-97.
15. Thall PF, Wooten LH, Tannir NM. Monitoring event times in early phase clinical trials: some practical issues. Clin Trials 2005; 2(6):467-78.
16. Thall PF, Simon R, Estey EH. Bayesian sequential monitoring designs for single-arm clinical trials with multiple outcomes. Statistics in Medicine 1995; 14:357-379.
17. Lachin J. A review of methods for futility stopping based on conditional power. Statistics in Medicine 2005; 24:2747-2764. DOI: 10.1002/sim.2151.
18. Pepe MS, Anderson GL. Two-stage experimental designs: early stopping with a negative result. Applied Statistics 1992; 41:181-190.
19. Berry DA. Bayesian clinical trials. Nature Reviews Drug Discovery 2006; 5:27-36. DOI: 10.1038/nrd1927.
20. Staquet MJ, Sylvester RJ. A decision theory approach to phase II clinical trials. Biomedicine 1977; 26(4):262-266.
21. Sylvester RJ, Staquet MJ. Design of phase II clinical trials in cancer using decision theory. Cancer Treatment Report 1980; 64(2-3):519-524.
22. Sylvester RJ. A Bayesian approach to the design of phase II clinical trials. Biometrics 1988; 44(3):823-836.
23. Brunier HC, Whitehead J. Sample sizes for phase II clinical trials derived from Bayesian decision theory. Statistics in Medicine 1994; 13(23-24):2493-2502.
24. Stallard N. Sample size determination for phase II clinical trials based on Bayesian decision theory. Biometrics 1998; 54(1):279-294.
25. Zhao L, Woodworth G. Bayesian decision sequential analysis with survival endpoint in phase II clinical trials. Statistics in Medicine 2009; 28:1339-1352. DOI: 10.1002/sim.3544.
26. Berry SM, Carlin BP, Lee J, Muller P. Bayesian Adaptive Methods for Clinical Trials, Chapman & Hall/CRC Biostatistics Series, Boca Raton: CRC Press, 2011.
27. Albert J. Bayesian Computation with R. Springer: New York, 2009.
28. Kalbfleisch J. Nonparametric Bayesian analysis of survival time data. Journal of the Royal Statistical Society 1978; 40:214-221.
29. Ibrahim JG, Chen MH, Sinha D. Bayesian Survival Analysis. Springer: New York, 2001. 50-53.
30. Muller P, Berry DA, Grieve AP, et al. Simulation-based sequential Bayesian design. Journal of Statistical Planning and Inference 2007; 137(10):3140-3150. DOI: 10.1016/j.jspi.2006.05.021.
31. Brockwell AE, Kadane JB. Sequential analysis by griding sufficient statistics. J. Comput. Graph. Statist 2003; 12:566-584.