261
Views
3
CrossRef citations to date
0
Altmetric
Research Article

Optimal biological dose selection in dose-finding trials with model-assisted designs based on efficacy and toxicity: a simulation study

ORCID Icon, ORCID Icon, & ORCID Icon
Pages 379-393 | Received 18 Nov 2022, Accepted 06 Apr 2023, Published online: 28 Apr 2023

References

  • Ambler, G., and A. Benner. Mfp: Multivariable fractional polynomials. Last Modified October 13, 2022. Accessed November 10, 2022. https://cran.r-project.org/web/packages/mfp/mfp.pdf.
  • Barlow, R. E., D. J. Bartholomew, J. M. Bremner, and H. D. Brunk. 1972. Statistical inference under order restrictions. London: Wiley.
  • Bril, G., R. Dykstra, C. Pillers, and T. Robertson. 1984. Isotonic regression in two independent variables. Journal of the Royal Statistical Society: Series C (Applied Statistics) 33 (3):352–357. doi:10.2307/2347723.
  • Calabrese, E. J., and L. A. Baldwin. 2001. U-shaped dose-responses in biology, toxicology, and public health. Annual Review of Public Health 22 (1):15–33. doi:10.1146/annurev.publhealth.22.1.15.
  • Corbaux, P., M. El-Madani, M. Tod, J. Peron, D. Maillet, J. Lopez, G. Freyer, and B. You. 2019. Clinical efficacy of the optimal biological dose in early phase trials of anti-cancer targeted therapies. European Journal of Cancer 120:40–46. doi:10.1016/j.ejca.2019.08.002.
  • Eisenhauer, E. A., P. J. O’Dwyer, M. Christian, and J. S. Humphrey. 2000. Phase I clinical trial design in cancer drug development. Journal of Clinical Oncology 18 (3):684–692. doi:10.1200/JCO.2000.18.3.684.
  • Feng, Y., X. Wang, G. Bajaj, S. Agrawal, A. Bello, B. Lestini, F. G. Finckenstein, J. -S. Park, and A. Roy. 2017. Nivolumab exposure–response analyses of efficacy and safety in previously treated squamous or nonsquamous non–small cell lung cancer. Clinical Cancer Research 23 (18):5394–5405. doi:10.1158/1078-0432.CCR-16-2842.
  • Food and Drug Administration (FDA). Expansion cohorts: Use in first-in-human clinical trials to expedite development of oncology drugs and biologics guidance for industry. Last Modified March, 2022. Accessed November 10, 2022. https://www.fda.gov/media/115172/download.
  • Fraisse, J., D. Dinart, D. Tosi, C. Bellera, and C. Mollevi. 2021. Optimal biological dose: A systematic review in cancer phase I clinical trials. BMC Cancer 21 (1):60. doi:10.1186/s12885-021-07782-z.
  • Hjort, N. L., and G. Claeskens. 2003. Frequentist model average estimators. Journal of the American Statistical Association 98 (464):879–899. doi:10.1198/016214503000000828.
  • Hurwitz, H. I., A. Dowlati, S. Saini, S. Savage, A. B. Suttle, D. M. Gibson, J. P. Hodge, E. M. Merkle, and L. Pandite. 2009. Phase I trial of pazopanib in patients with advanced cancer. Clinical Cancer Research 15 (12):4220–4227. doi:10.1158/1078-0432.CCR-08-2740.
  • Ji, Y., Y. Li, and B. Nebiyou. 2007. Dose-finding in phase I clinical trials based on toxicity probability intervals. Clinical Trials 4 (3):235–244. doi:10.1177/1740774507079442.
  • Le Tourneau, C., J. J. Lee, and L. L. Siu. 2009. Dose escalation methods in phase I cancer clinical trials. Journal of the National Cancer Institute 101 (10):708–720. doi:10.1093/jnci/djp079.
  • Li, P., R. Liu, J. Lin, and Y. Ji. 2020. TEPI-2 and UBI: Designs for optimal immuno-oncology and cell therapy dose finding with toxicity and efficacy. Journal of Biopharmaceutical Statistics 30 (6):979–992. doi:10.1080/10543406.2020.1814802.
  • Lin, X., and Y. Ji. 2020. The joint i3+3 (Ji3+3) design for phase I/II adoptive cell therapy clinical trials. Journal of Biopharmaceutical Statistics 30 (6):993–1005. doi:10.1080/10543406.2020.1818250.
  • Lin, X., and Y. Ji. 2021. Probability intervals of toxicity and efficacy design for dose-finding clinical trials in oncology. Statistical Methods in Medical 30 (3):843–856. doi:10.1177/0962280220977009.
  • Lin, R., and G. Yin. 2017. STEIN: A simple toxicity and efficacy interval design for seamless phase I/II clinical trials. Statistics in Medicine 36 (26):4106–4120. doi:10.1002/sim.7428.
  • Lin, R., Y. Zhou, F. Yan, D. H. Li, and Y. Yuan. 2020. BOIN12: Bayesian optimal interval phase I/II trial design for utility-based dose finding in immunotherapy and targeted therapies. JCO Precision Oncology 4 (4):1393–1402. doi:10.1200/PO.20.00257.
  • Liu, S., and V. E. Johnson. 2016. A robust Bayesian dose-finding design for phase I/II clinical trials. Biostatistics 17:249–263. doi:10.1093/biostatistics/kxv040.
  • 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) 64:507–523. doi:10.1111/rssc.12089.
  • Li, D. H., J. B. Whitmore, W. Guo, and Y. Ji. 2017. Toxicity and efficacy probability interval design for phase I adoptive cell therapy dose-finding clinical trials. Clinical Cancer Research 23 (1):13–20. doi:10.1158/1078-0432.CCR-16-1125.
  • LoRusso, P. M., S. A. Boerner, and L. Seymour. 2010. An overview of the optimal planning, design, and conduct of phase I studies of new therapeutics. Clinical Cancer Research 16:1710–1718. doi:10.1158/1078-0432.CCR-09-1993.
  • Murtaugh, P. A., and L. D. Fisher. 1990. Bivariate binary models of efficacy and toxicity in dose-ranging trials. Communications in Statistics, Part A - Theory and Methods 19:2003–2020. doi:10.1080/03610929008830305.
  • Penel, N., A. Adenis, S. Clisant, and J. Bonneterre. 2011. Nature and subjectivity of dose-limiting toxicities in contemporary phase 1 trials: Comparison of cytotoxic versus non-cytotoxic drugs. Investigational New Drugs 29:1414–1419. doi:10.1007/s10637-010-9490-7.
  • Postel-Vinay, S., H. -T. Arkenau, D. Olmos, J. Ang, J. Barriuso, S. Ashley, U. Banerji, J. De-Bono, I. Judson, and S. Kaye. 2009. Clinical benefit in Phase-I trials of novel molecularly targeted agents: Does dose matter? British Journal of Cancer 100:1373–1378. doi:10.1038/sj.bjc.6605030.
  • Reynolds, A. R. 2010. Potential relevance of bell-shaped and u-shaped dose-responses for the therapeutic targeting of angiogenesis in cancer. Dose-Response: A Publication of International Hormesis Society 8:253–284. doi:10.2203/dose-response.09-049.Reynolds.
  • Royston, P., and D. G. Altman. 1994. Regression using fractional polynomials of continuous covariates: Parsimonious parametric modelling. Journal of the Royal Statistical Society: Series C (Applied Statistics) 43:429–467. doi:10.2307/2986270.
  • Takeda, K., S. Morita, and M. Taguri. 2019. TITE-BOIN-ET: Time-to-event Bayesian optimal interval design to accelerate dose-finding based on both efficacy and toxicity outcomes. Pharmaceutical Statistics 19:335–349. doi:10.1002/pst.1995.
  • Takeda, K., M. Taguri, and S. Morita. 2018. BOIN-ET: Bayesian optimal interval design for dose finding based on both efficacy and toxicity outcomes. Pharmaceutical Statistics 17:383–395. doi:10.1002/pst.1864.
  • Thall, P. F., and J. D. Cook. 2004. Dose-finding based on efficacy-toxicity trade-offs. Biometrics 60:684–693. doi:10.1111/j.0006-341X.2004.00218.x.
  • Tosi, D., Y. Laghzali, M. Vinches, M. Alexandre, K. Homicsko, A. Fasolo, G. Del Conte, A. Durigova, N. Hayaoui, S. Gourgou, et al. 2015. Clinical development strategies and outcomes in first-in-human trials of monoclonal antibodies. Journal of Clinical Oncology 33:2158–2165. doi:10.1200/JCO.2014.58.1082.
  • Turner, R. Iso: Functions to Perform Isotonic Regression. Last Modified October 12, 2022. Accessed November 10, 2022. https://cran.r-project.org/web/packages/Iso/Iso.pdf.
  • Turner, T. R., and P. C. Wollan. 1997. Locating a maximum using isotonic regression. Computational Statistics & Data Analysis 25:305–320. doi:10.1016/S0167-9473(97)00009-1.
  • Yan, F., S. J. Mandrekar, and Y. Yuan. 2017. Keyboard: A novel Bayesian toxicity probability interval design for phase I clinical trials. Clinical Cancer Research 23:3994–4003. doi:10.1158/1078-0432.CCR-17-0220.
  • Yin, G., Y. Li, and Y. Ji. 2006. Bayesian dose-finding in phase I/II clinical trials using toxicity and efficacy odds ratios. Biometrics 62:777–787. doi:10.1111/j.1541-0420.2006.00534.x.
  • Yin, J., and Y. Yuan. 2020. Checkerboard: A Bayesian efficacy and toxicity interval design for phase I/II dose-finding trials. Journal of Biopharmaceutical Statistics 30:1006–1025. doi:10.1080/10543406.2020.1815033.
  • Yuan, Y., K. R. Hess, S. G. Hilsenbeck, and M. R. 2016. Gilbert. Bayesian optimal interval design: A simple and well-performing design for phase I oncology trials. Clinical Cancer Research 22:4291–4301. doi:10.1158/1078-0432.CCR-16-0592.
  • Yuan, Y., J. J. Lee, and S. G. Hilsenbeck. 2019. Model-assisted designs for early-phase clinical trials: Simplicity meets superiority. JCO Precision Oncology 3:O.19.00032. doi:10.1200/PO.19.00032.
  • Yuan, Y., H. Q. Nguyen, and P. F. Thall. 2016. Bayesian designs for phase I-II clinical trials. New York: Chapman and Hall/CRC.
  • Zhang, Z. 2016. Multivariable fractional polynomial method for regression model. Annals of Translational Medicine 4:174. doi:10.21037/atm.2016.05.01.
  • Zhou, Y., J. J. Lee, and Y. Yuan. 2019. A utility-based Bayesian optimal interval (U-BOIN) phase I/II design to identify the optimal biological dose for targeted and immune therapies. Statistics in Medicine 38:5299–5316. doi:10.1002/sim.8361.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.