×

Patient-specific dose finding in seamless phase I/II clinical trials. (English) Zbl 07596762

Summary: This article incorporates a covariate to determine the optimum dose in a seamless phase I/II clinical trial. A binary covariate and its interaction effect are assumed to keep the method simple. Each patient’s outcome is assumed to be trinomial, and the continuation ratio model is utilized to model the dose-response data. The Bayesian approach estimates parameters of the dose-response model. Eight plausible dose-response scenarios are investigated to check the proposed methodology. A simulation study shows that covariate consideration can enhance the identification of the optimum dose when it is appropriate to do.

MSC:

62P10 Applications of statistics to biology and medical sciences; meta analysis
62K99 Design of statistical experiments
62L05 Sequential statistical design
PDFBibTeX XMLCite
Full Text: DOI

References:

[1] Alam, M. I.; Coad, D. S.; Bogacka, B., Combined Criteria for Dose Optimisation in Early Phase Clinical Trials, Statistics in Medicine, 38, 21, 4172-88 (2019) · doi:10.1002/sim.8292
[2] Bandyopadhyay, U.; Biswas, A.; Bhattacharya, R., A Covariate Adjusted Two-Stage Allocation Design for Binary Responses in Randomized Clinical Trials, Statistics in Medicine, 26, 24, 4386-99 (2007) · doi:10.1002/sim.2869
[3] Braun, T. M., The Bivariate Continual Reassessment Method: Extending the CRM to Phase I Trials of Two Competing Outcomes, Controlled Clinical Trials, 23, 3, 240-56 (2002) · doi:10.1016/S0197-2456(01)00205-7
[4] Cheung, Y. K., Simple Benchmark for Complex Dose Finding Studies, Biometrics, 70, 2, 389-97 (2014) · Zbl 1419.62325 · doi:10.1111/biom.12158
[5] Cotterill, A.; Jaki, T., Dose-Escalation Strategies Which Use Subgroup Information, Pharmaceutical Statistics, 17, 5, 414-36 (2018) · doi:10.1002/pst.1860
[6] Cunningham, D.; Humblet, Y.; Siena, S.; Khayat, D.; Bleiberg, H.; Santoro, A.; Bets, D.; Mueser, M.; Harstrick, A.; Verslype, C., Cetuximab Monotherapy and Cetuximab plus Irinotecan in Irinotecan-Refractory Metastatic Colorectal Cancer, New England Journal of Medicine, 351, 4, 337-45 (2004) · doi:10.1056/NEJMoa033025
[7] Das, I.; Mukhopadhyay, S.; Xu, H., Individualized Dosing for Multiple Ordered Groups of Patients, Journal of Statistical Theory and Practice, 7, 1, 95-106 (2013) · Zbl 1425.62142 · doi:10.1080/15598608.2013.756348
[8] Guo, B.; Yuan, Y., Bayesian Phase I/II Biomarker-Based Dose Finding for Precision Medicine with Molecularly Targeted Agents, Journal of the American Statistical Association, 112, 518, 508-20 (2017) · doi:10.1080/01621459.2016.1228534
[9] Mao, X.; Cheung, Y. K., Sequential Designs for Individualized Dosing in Phase I Cancer Clinical Trials, Contemporary Clinical Trials, 63, 51-8 (2017) · doi:10.1016/j.cct.2016.08.018
[10] McCullagh, P.; Nelder, J., Generalized Linear Models. Chapman and Hall/CRC Monographs on Statistics and Applied Probability Series (1989), London: Chapman & Hall/CRC · Zbl 0744.62098
[11] Morita, S.; Thall, P. F.; Takeda, K., A Simulation Study of Methods for Selecting Subgroup-Specific Doses in Phase I Trials, Pharmaceutical Statistics, 16, 2, 143-56 (2017) · doi:10.1002/pst.1797
[12] O’Quigley, J.; Hughes, M. D.; Fenton, T., Dose-Finding Designs for HIV Studies, Biometrics, 57, 4, 1018-29 (2001) · Zbl 1209.62319 · doi:10.1111/j.0006-341x.2001.01018.x
[13] (2013)
[14] (2021)
[15] Thall, P. F.; Nguyen, H. Q.; Estey, E. H., Patient-Specific Dose Finding Based on Bivariate Outcomes and Covariates, Biometrics, 64, 4, 1126-36 (2008) · Zbl 1151.62092 · doi:10.1111/j.1541-0420.2008.01009.x
[16] Wathen, J. K.; Thall, P. F.; Cook, J. D.; Estey, E. H., Accounting for Patient Heterogeneity in Phase II Clinical Trials, Statistics in Medicine, 27, 15, 2802-15 (2008) · doi:10.1002/sim.3109
[17] Wijesinha, M. C.; Piantadosi, S., Dose-Response Models with Covariates, Biometrics, 51, 3, 977-87 (1995) · Zbl 0867.62086 · doi:10.2307/2532998
[18] Zhang, W.; Sargent, D. J.; Mandrekar, S., An Adaptive Dose-Finding Design Incorporating Both Toxicity and Efficacy, Statistics in Medicine, 25, 14, 2365-83 (2006) · doi:10.1002/sim.2325
This reference list is based on information provided by the publisher or from digital mathematics libraries. Its items are heuristically matched to zbMATH identifiers and may contain data conversion errors. In some cases that data have been complemented/enhanced by data from zbMATH Open. This attempts to reflect the references listed in the original paper as accurately as possible without claiming completeness or a perfect matching.