Bayesian models and decision algorithms for complex early phase clinical trials

Statistical Science

Volumn 25, Issue 2, 2010, Pages 227-244

  Thall, Peter F ^a  

^a UNIVERSITY OF TEXAS   (United States)

Author keywords

Adaptive design; Bayesian design; Clinical trial; Dose finding; Phase I trial; Phase I II trial

Indexed keywords

EID: 78650242755     PISSN: 08834237     EISSN: None     Source Type: Journal    
DOI: 10.1214/09-STS315     Document Type: Article

References (33)

1. ALBERT, J. H. and CHIB, S. (1993). Bayesian analysis of binary and polytomous response data. J. Amer. Statist. Assoc. 88 669- 679.
2. ANDERSSON, B. S., THALL, P. F., MADDEN, T., COURIEL, D., WANG, X., TRAN, H. T., ANDERLINI, P., DE LIMA, M., GAJEWSKI, J. and CHAMPLIN, R. E. (2002). Busulfan systemic exposure relative to regimen-related toxicity and acute graft vs. host disease; defining a therapeutic window for IVBuCy2 in chronic myelogenous leukemia. Biology of Blood and Marrow Transplantation 8 477-485.
3. BABB, J. S. and ROGATKO, A. (2001). Patient specific dosing in a phase I cancer trial. Stat. Med. 20 2079-2090.
4. BEKELE, B. N. and THALL, P. F. (2004). Dose-finding based on multiple toxicities in a soft tissue sarcoma trial. J. Amer. Statist. Assoc. 99 26-35.
5. BEKELE, B. N., JI, Y., SHEN, Y. and THALL, P. F. (2008). Monitoring late onset toxicities in phase I trials using predicted risks. Biostatistics 9 442-457.
6. BRAUN, T. (2002). The bivariate continual reassessment method: Extending the CRM to phase I trials of two competing outcomes. Controlled Clinical Trials 23 240-256.
7. BRAUN, T. M., YUAN, Z. and THALL, P. F. (2005). Determining a maximum tolerated schedule of a cytotoxic agent. Biometrics 61 335-343.
8. BRAUN, T. M., THALL, P. F., NGUYEN, H. and DE LIMA, M. (2007). Simultaneously optimizing dose and schedule of a new cytotoxic agent. Clinical Trials 4 113-124.
9. CHEUNG, Y. K. (2005). Coherence principles in dose-finding studies. Biometrika 92 863-873.
10. CHEUNG, Y. and CHAPPELL, R. (2000). Sequential designs for phase I clinical trials with late-onset toxicities. Biometrics 56 1177-1182.
11. DETTE, H., BRETZ, F., PEPELYSHEV, A. and PINHIERO, J. (2008). Optimal designs for dose-finding studies. J. Amer. Statist. Assoc. 103 1225-1237.
12. GOODMAN, S. G., ZAHURAK, M. L. and PIANTADOSI, S. (1995). Some practical improvements in the continual reassessment method. Stat. Med. 14 1149-1161.
13. GOOLEY, T. A., MARTIN, P. J., FISHER, L. D. and PETTINGER, M. (1994). Simulation as a design tool for phase I/II clinical trials: An example from bone marrow transplantation. Controlled Clinical Trials 15 450-462.
14. HAINES, L. M., PEREVOZSKAYA, I. and ROSENBERGER, W. F. (2003). Bayesian optimal designs for phase I clinical trials. Biometrics 59 591-600.
15. HORSTMANN, E., MCCABE, M. S., GROCHOW, L., YAMAMOTO, S., RUBINSTEIN, L., BUDD, T., SHOEMAKER, D., EMANUEL, E. J. and GRADY, C. (2005). Risks and benefits of phase 1 oncology trials, 1991 through 2002. New England J. Medicine 352 895-904.
16. HOUEDE, N., THALL, P. F., NGUYEN, H., PAOLETTI, X. and KRAMAR, A. (2010). Utility-based optimization of combination therapy using ordinal toxicity and efficacy in phase I/II trials. Biometrics. In press.
17. IVANOVA, A. (2003). A new dose-finding design for bivariate outcomes. Biometrics 59 1001-1007.
18. LIU, C. A. and BRAUN, T. M. (2009). Parametric non-mixture cure models for schedule-finding of therapeutic agents. Appl. Statist. 58 225-236.
19. MORITA, S., THALL, P. F. and MÜLLER, P. (2008). Determining the effective sample size of a parametric prior. Biometrics 64 595-602.
20. O'QUIGLEY, J. (1990). Sequential design and analysis of dosefinding studies in patients with life threatening disease. Fund. Clin. Pharmacology 4 (Suppl. 2) 81s-91s.
21. O'QUIGLEY, J., HUGHES, M. D. and FENTON, T. (2001). Dosefinding designs for HIV studies. Biometrics 57 1018-1029.
22. O'QUIGLEY, J., PEPE, M. and FISHER, L. (1990). Continual reassessment method: A practical design for phase I clinical trials in cancer. Biometrics 46 33-48.
23. PALMER, C. R. (2002). Ethics, data-dependent designs, and the strategy of clinical trials: Time to start learning-as-we-go? Stat. Methods Med. Res. 5 381-402.
24. RATAIN, M. J., MICK, R., JANISCH, L., BEREZIN, F., SCHILSKY, R. L., VOGELZANG, N. J. and KUT, M. (1996). Individualized dosing of amonafide based on a pharmacodynamic model incorporating acetylator phenotype and gender. Pharmacogenetics 6 93-101.
25. THALL, P. F. and COOK, J. D. (2004). Dose-finding based on efficacy-toxicity trade-offs. Biometrics 60 684-693.
26. THALL, P. F. and RUSSELL, K. T. (1998). A strategy for dose finding and safety monitoring based on efficacy and adverse outcomes in phase I/II clinical trials. Biometrics 54 251- 264.
27. THALL, P. F., COOK, J. D. and ESTEY, E. H. (2006). Adaptive dose selection using efficacy-toxicity trade-offs: Illustrations and practical considerations. J. Biopharm. Statist. 16 623-638.
28. THALL, P. F., NGUYEN, H. and ESTEY, E. H. (2008). Patientspecific dose-finding based on bivariate outcomes and covariates. Biometrics 64 1126-1136.
29. THALL, P. F., LEE, J. J., TSENG, C.-H. and ESTEY, E. H. (1999). Accrual strategies for phase I trials with delayed patient outcome. Stat. Med. 18 1155-1169.
30. THALL, P. F., MILLIKAN, R. E., MÜLLER, P. and LEE, S.-J. (2003). Dose-finding with two agents in phase I oncology trials. Biometrics 59 487-496.
31. THALL, P. F., SIMON, R. and ESTEY, E. H. (1995). Bayesian sequential monitoring designs for single-arm clinical trials with multiple outcomes. Stat. Med. 14 357-379.
32. TUKEY, J. W. (1949). One degree of freedom for non-additivity. Biometrics 5 232-242.
33. WHELAN, H. T., COOK, J. D., AMLIE-LEFOND, C. M., HOVINGA, C. A., CHAN, A. K., ICHORD, R. N., DEVEBER, G. A. and THALL, P. F. (2008). Practical model-based dose-finding in early phase clinical trials: Optimizing tissue plasminogen activator dose for treatment of ischemic stroke in children. Stroke 39 2627-2636.