Multi-stage dose expansion cohort (MSDEC) design with Bayesian stopping rule

References

• Braun, T. M. 2002. The bivariate continual reassessment method: Extending the CRM to phase I trials of two competing outcomes. Controlled Clinical Trials 23 (3):240–256. doi:10.1016/S0197-2456(01)00205-7.
   PubMedGoogle Scholar
• Chen, M. H., J. G. Ibrahim, and Q. M. Shao. 2000. Power prior distributions for generalized linear models. Journal of Statistical Planning and Inference 84 (1–2):121–137. doi:10.1016/S0378-3758(99)00140-8.
   Web of Science ®Google Scholar
• Dahlberg, S. E., G. I. Shapiro, J. W. Clark, and B. E. Johnson. 2014. Evaluation of statistical designs in phase I expansion cohorts: The Dana-Farber/Harvard Cancer Center experience. JNCI: Journal of the National Cancer Institute 106 (7). doi:10.1093/jnci/dju163.
   PubMedGoogle Scholar
• Geller, N. L., D. Follman, D. S. Leifer, and S. L. Carter. 2003. Design of early trials in stem cell transplantation: A hybrid frequentist-Bayesian approach. Advances in Clinical Trial Biostatistics 41.
   Google Scholar
• Gönen, M. 2005. A Bayesian evaluation of enrolling additional patients at the maximum tolerated dose in Phase I trials. Contemporary Clinical Trials 26 (2):131–140. doi:10.1016/j.cct.2004.12.007.
   PubMed Web of Science ®Google Scholar
• Iasonos, A., and J. O’quigley. 2015. Clinical trials: Early phase clinical trials—are dose expansion cohorts needed? Nature Reviews Clinical Oncology 12 (11):626. doi:10.1038/nrclinonc.2015.174.
   PubMed Web of Science ®Google Scholar
• Iasonos, A., and J. O’Quigley. 2013. Design considerations for dose-expansion cohorts in phase I trials. Journal of Clinical Oncology 31 (31):4014. doi:10.1200/JCO.2012.47.9949.
   PubMed Web of Science ®Google Scholar
• Iasonos, A., and J. O’Quigley. 2016a. Dose expansion cohorts in Phase I trials. Statistics in Biopharmaceutical Research 8 (2):161–170. doi:10.1080/19466315.2015.1135185.
   PubMed Web of Science ®Google Scholar
• Iasonos, A., and J. O’Quigley. 2016b. Integrating the escalation and dose expansion studies into a unified phase I clinical trial. Contemporary Clinical Trials 50:124–134. doi:10.1016/j.cct.2016.06.010.
   PubMed Web of Science ®Google Scholar
• Ibrahim, J. G., and M. H. Chen. 2000. Power prior distributions for regression models. Statistical Science 15 (1):46–60.
   Web of Science ®Google Scholar
• Le Tourneau, C., J. J. Lee, and L. L. Siu. 2009. Dose escalation methods in phase I cancer clinical trials. JNCI: Journal of the National Cancer Institute 101 (10):708–720. doi:10.1093/jnci/djp079.
   PubMedGoogle Scholar
• Little, R. J. 2006. Calibrated Bayes: A Bayes/frequentist roadmap. The American Statistician 60 (3):213–223. doi:10.1198/000313006X117837.
   Web of Science ®Google Scholar
• Liu, S., and Y. Yuan. 2015. Bayesian optimal interval designs for phase I clinical trials. Journal of the Royal Statistical Society: Series C (Applied Statistics) 64 (3):507–523.
   Web of Science ®Google Scholar
• Neuenschwander, B., M. Branson, and T. Gsponer. 2008. Critical aspects of the Bayesian approach to phase I cancer trials. Statistics in Medicine 27 (13):2420–2439. doi:10.1002/sim.3230.
   PubMed Web of Science ®Google Scholar
• Neuenschwander, B., M. Branson, and D. J. Spiegelhalter. 2009. A note on the power prior. Statistics in Medicine 28 (28):3562–3566. doi:10.1002/sim.3722.
   PubMed Web of Science ®Google Scholar
• O’Quigley, J. 2002. Curve‐free and model‐based continual reassessment method designs. Biometrics 58 (1):245–249. doi:10.1111/j.0006-341X.2002.00245.x.
   PubMed Web of Science ®Google Scholar
• O’Quigley, J., and M. Conaway. 2010. Continual reassessment and related dose-finding designs. Statistical Science: a Review Journal of the Institute of Mathematical Statistics 25 (2):202. doi:10.1214/10-STS332.
   PubMed Web of Science ®Google Scholar
• O’Quigley, J., M. Pepe, and L. Fisher. 1990. Continual reassessment method: A practical design for phase 1 clinical trials in cancer. Biometrics 46 (1):33–48. doi:10.2307/2531628.
   PubMed Web of Science ®Google Scholar
• Simon, R. 1989. Optimal two-stage designs for phase II clinical trials. Contemporary Clinical Trials 10 (1):1–10. doi:10.1016/0197-2456(89)90015-9.
   Google Scholar
• Tan, M. T., and X. Xiong. 2011. A flexible multi‐stage design for phase II oncology trials. Pharmaceutical Statistics 10 (4):369–373. doi:10.1002/pst.478.
   PubMed Web of Science ®Google Scholar
• Tan, S. B., and D. Machin. 2002. Bayesian two‐stage designs for phase II clinical trials. Statistics in Medicine 21 (14):1991–2012. doi:10.1002/sim.1176.
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration. 2010. Guidance for the use of Bayesian statistics in medical device clinical trials. Maryland: US Food and Drug Administration.
   Google Scholar
• Xiong, X. 1995. A class of sequential conditional probability ratio tests. Journal of the American Statistical Association 90 (432):1463–1473. doi:10.1080/01621459.1995.10476653.
   Web of Science ®Google Scholar
• Yuan, Y., K. R. Hess, S. G. Hilsenbeck, and M. R. Gilbert. 2016. Bayesian optimal interval design: A simple and well-performing design for phase I oncology trials. Clinical Cancer Research 22 (17):4291–4301. doi:10.1158/1078-0432.CCR-16-0592.
   PubMed Web of Science ®Google Scholar
• Zhang, W., D. J. Sargent, and S. Mandrekar. 2006. An adaptive dose‐finding design incorporating both toxicity and efficacy. Statistics in Medicine 25 (14):2365–2383. doi:10.1002/sim.2325.
   PubMed Web of Science ®Google Scholar