Hierarchical Bayesian approaches to phase II trials in diseases with multiple subtypes

Statistics in Medicine

Volumn 22, Issue 5, 2003, Pages 763-780

  Thall, Peter F ^a   Wathen, J Kyle ^a   Bekele, B Nebiyou ^a   Champlin, Richard E ^a   Baker, Laurence H ^b   Benjamin, Robert S ^a  

^a UNIVERSITY OF TEXAS   (United States)

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

Author keywords

Activity trial; Bone marrow transplantation; Heterogeneity; Phase II clinical trial; Sarcoma

Indexed keywords

ANTINEOPLASTIC AGENT; BORTEZOMIB; BUSULFAN; CYCLOPHOSPHAMIDE; FLUDARABINE; IMATINIB; PROTEIN TYROSINE KINASE;

ACUTE GRAFT REJECTION; ACUTE GRANULOCYTIC LEUKEMIA; ALLOGENIC BONE MARROW TRANSPLANTATION; ANTINEOPLASTIC ACTIVITY; BAYES THEOREM; CANCER THERAPY; CLINICAL STUDY; CLINICAL TRIAL; CORRELATION ANALYSIS; DEATH; DISEASE CLASSIFICATION; DISEASE COURSE; EXPERIMENTAL DESIGN; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; MYELODYSPLASTIC SYNDROME; PHASE 2 CLINICAL TRIAL; PROSTATE CANCER; REVIEW; SARCOMA; SIDE EFFECT; STATISTICAL MODEL; TREATMENT OUTCOME;

ANTINEOPLASTIC AGENTS; BAYES THEOREM; BONE MARROW TRANSPLANTATION; BUSULFAN; CLINICAL TRIALS, PHASE II; DISEASE PROGRESSION; HUMANS; IMMUNOSUPPRESSIVE AGENTS; LEUKEMIA, MYELOCYTIC, ACUTE; MYELODYSPLASTIC SYNDROMES; PIPERAZINES; PYRIMIDINES; RESEARCH DESIGN; SARCOMA; TREATMENT OUTCOME; VIDARABINE;

EID: 0037445360     PISSN: 02776715     EISSN: None     Source Type: Journal    
DOI: 10.1002/sim.1399     Document Type: Review

References (24)

1. Gehan EA. The determination of the number of patients required in a follow-up trial of a new chemotherapeutic agent. Journal of Chronic Diseases 1961; 13:346-353.
2. Thall PF, Sung H-G. Some extensions and applications of a Bayesian strategy for monitoring multiple outcomes in clinical trials. Statistics in Medicine 1998; 17:1563-1580.
3. Fleming TR. One sample multiple testing procedure for phase II clinical trials. Biometrics 1982; 38:143-151.
4. Simon R. Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 1989; 10:1-10.
5. Chang M, Therneau T, Wieand HS. Designs for group sequential phase II clinical trials. Biometrics 1987; 43:865-874.
6. Thall PF, Simon R. Practical Bayesian guidelines for phase IIB clinical trials. Biometrics 1994; 50:337-349.
7. Heitjan DF. Bayesian interim analysis of phase II cancer clinical trials. Statistics in Medicine 1995; 16:1791-1802.
8. Stallard N. Sample size determination for phase II clinical trials based on Bayesian decision theory. Biometrics 1998; 54:279-294.
9. Bryant J, Day R. Incorporating toxicity considerations into the design of two-stage phase II clinical trials. Biometrics 1995; 51:1372-1383.
10. Conaway MR, Petroni GR. Bivariate sequential designs for phase II trials. Biometrics 1995; 51:656-664.
11. Zee B, Melnychuk D, Dancey J, Eisenhauer E. Multinomial phase II cancer trials incorporating response and early progression. Journal of Biopharmaceutical Statistics 1999; 9:351-363.
12. Thall PF, Simon R, Estey E. Bayesian sequential monitoring designs for single-arm clinical trials with multiple outcomes. Statistics in Medicine 1995; 14:357-379.
13. Stallard N, Thall PF, Whitehead J. Decision theoretic designs for phase II clinical trials with multiple outcomes. Biometrics 1999; 55:971-977.
14. Lindley DV, Smith AFM. Bayesian estimates for the linear model (with discussion). Journal of the Royal Statistical Society, Series B 1972; 34:1-41.
15. Kass RE, Steffey D. Approximate Bayesian inference in conditionally independent hierarchical models (parametric empirical Bayes models). Journal of the American Statistical Association 1989; 84:717-726.
16. Skene AM, Wakefield JC. Hierarchical models for multicentre binary response studies. Statistics in Medicine 1990; 9:919-929.
17. Wingo PA, Tong T, Bolden S. Cancer statistics, 1995. CA J Clin. 1995; 45:8-30.
18. Enzinger FM, Weiss SW. General Considerations, Soft Tissue Tumors. Mosby: St. Louis, 1995; 1-16.
19. Patel SR, Benjamin RS. Sarcomas of soft tissue and bone. In Harrison's Principles of Internal Medicine, 14th edn, Fauci AS, Braunwald E, Isselbacher KJ et al. (eds). McGraw Hill: New York, 1997; 611-614.
20. Blanke CD, von Mehren M, Joensuu H et al. Evaluation of the safety and efficacy of an oral molecularly targeted therapy, STI571, in patients with unresectable or metastatic gastrointestinal stromal tumours expressing C-KIT. Proceedings of the American Society of Clinical Oncology 2001; 20:1a.
21. Thomas ED. Marrow transplantation for malignant diseases. Journal of Clinical Oncology 1983; 1:517-531.
22. Gilks W, Richardson S, Spiegelhalter D. Markov Chain Monte Carlo in Practice. Chapman and Hall: London, 1996.
23. Gilks WR, Wild P. Adaptive rejection sampling for Gibbs sampling. Applied Statistics 1992; 41:337-348.
24. Best N, Cowles MK, Vines K. CODA-Convergence Diagnosis and Output Analysis Software for Gibbs Sampling Output. Medical Research Council, Biostatistics Unit, Cambridge, 1995.