On optimal subset designs for phase II clinical trials with both total response and disease control. (English) Zbl 1477.62323
Summary: Phase II clinical trials in oncology are used to initially evaluate the therapeutic efficacy of a new treatment. In the past, the total response was a frequently used endpoint to access the effectiveness of the treatment. When the total response is modest, clinicians may also be interested in disease control (defined as the total response or stable disease) since it may better predict clinical outcomes. Thus, formally testing both the total response and disease control in a phase II trial has an important clinical implication. In this paper, we propose a new method to construct optimal subset designs for one-stage and two-stage phase II clinical trials with two binary endpoints, i.e. the total response and disease control. A new set of hypotheses under the framework of intersection-union tests is provided in which the treatment is considered promising if both the total response and disease control are good. We show that the power function for each test in a large family of tests is nondecreasing in both the total response rate and disease control rate; identify the parameter configurations at which the maximum Type I error rate and minimum power are achieved and derive level-\( \alpha\) tests. We also provide optimal one-stage designs with the minimum sample size and optimal two-stage designs with the least expected total sample size.
MSC:
| 62P10 | Applications of statistics to biology and medical sciences; meta analysis |
| 62K05 | Optimal statistical designs |
Keywords:
least expected total sample size; maximum type I error rate; minimum power; multinomial distributionReferences:
| [1] | Abrey, L. E.; Olson, J. D.; Raizer, J. J.; Mack, M.; Rodavitch, A.; Boutros, D. Y.; Malkin, M. G., A phase II trial of temozolomide for patients with recurrent or progressive brain metastases, J. Neuro Oncol., 53, 259-265 (2001) |
| [2] | Berger, R. L., Multiparameter hypothesis testing and acceptance sampling, Technometrics, 24, 4, 295-300 (1982) · Zbl 0497.62091 |
| [3] | Casella, G.; Berger, R. L., Statistical Inference (1990), Duxbury: Duxbury Belmont, CA · Zbl 0699.62001 |
| [4] | Cohen, A.; Gatsonis, C.; Marden, J. I., Hypothesis testing for marginal probabilities in a \(2 \times 2 \times 2\) contingency table with conditional independence, J. Amer. Statist. Assoc., 78, 920-929 (1983) · Zbl 0528.62048 |
| [5] | Cohen, E. E.; Rosen, F.; Stadler, W. M.; Recant, W.; Stenson, K.; Huo, D.; Vokes, E. E., Phase II trial of zd1839 in recurrent or metastatic squamous cell carcinoma of the head and neck, J. Clin. Oncol. Off. J. Amer. Soc. Clin. Oncol., 21, 1980-1987 (2003) |
| [6] | Eisenhauer, E. A.; Therasse, P.; Bogaerts, J.; Schwartz, L. H.; Sargent, D.; Ford, R.; Rubinstein, L., New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1), Eur. J. Cancer, 45, 2, 228-247 (2009) |
| [7] | Ivanova, A.; Monaco, J.; Stinchcombe, T., Efficient designs for phase II oncology trials with ordinal outcome, Statist. Interface, 5, 4, 463-469 (2012) · Zbl 1383.62274 |
| [8] | Lara, P. N.; Redman, M. W.; Kelly, K.; Edelman, M. J.; Williamson, S. K.; Crowley, J. J.; Gandara, D. R., Disease control rate at 8 weeks predicts clinical benefit in advanced non-small-cell lung cancer: results from southwest oncology group randomized trials, J. Clin. Oncol., 26, 3, 463-467 (2008) |
| [9] | Lehmann, E. L., Testing multiparameter hypotheses, Ann. Math. Stat., 23, 4, 541-552 (1952) · Zbl 0048.11702 |
| [10] | Lin, S. P.; Chen, T. T., Optimal two-stage designs for phase II clinical trials with differentiation of complete and partial responses, Comm. Statist. Theory Methods, 29, 5-6, 923-940 (2000) · Zbl 1026.62121 |
| [11] | Lu, Y.; Jin, H.; Lamborn, K. R., A design of phase II cancer trials using total and complete response endpoints, Statist. Med., 24, 20, 3155-3170 (2005) |
| [12] | Mander, A. P.; Thompson, S. G., Two-stage designs optimal under the alternative hypothesis for phase II cancer clinical trials, Contemp. Clin. Trials, 31, 6, 572-578 (2010) |
| [13] | Panageas, K. S.; Smith, A.; Gonen, M.; Chapman, P. B., An optimal two-stage phase II design utilizing complete and partial response information separately experiments, Control. Clin. Trials, 23, 4, 367-369 (2002) |
| [14] | Pazdur, R., Endpoints for assessing drug activity in clinical trials, Oncologist, 13, 2, 19 (2008) |
| [15] | Pocock, S. J.; Geller, N. L.; Tsiatis, A. A., The analysis of multiple endpoints in clinical trials, Biometrics, 43, 3, 487-498 (1987) |
| [16] | Shan, G.; Zhang, H.; Jiang, T.; Peterson, H.; Young, D.; Ma, C., Exact p-values for simon’s two-stage designs in clinical trials, Stat. Biosci., 8, 2, 351-357 (2016) |
| [17] | Simon, R., Optimal two-stage designs for phase II clinical trials, Control. Clin. Trials, 10, 1, 1-10 (1989) |
| [18] | WHO Handbook for Reporting Results of Cancer Treatment (1979), World Health Organization Offset Publication No. 48: World Health Organization Offset Publication No. 48 Geneva (Switzerland) |
| [19] | Yang, G. J.; Yang, J. B.; Yang, X.; Wang, W. Z., Optimal two-stage phase II clinical trial with high-safety and effectiveness, J. Syst. Sci. Math. Sci., 40, 2, 318-326 (2020), (in Chinese) · Zbl 1463.62231 |
| [20] | Yin, H.; Wang, W.; Zhang, Z., On construction of single-arm two-stage designs with consideration of both response and toxicity, Biom. J., 61, 6, 1462-1476 (2019) · Zbl 1429.62637 |
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