Reducing sample sizes in two-stage phase II cancer trials by using continuous tumour shrinkage end-points

European Journal of Cancer

Volumn 47, Issue 7, 2011, Pages 983-989

  Wason, James M S ^a   Mander, Adrian P ^a   Eisen, Tim G ^b  

^a MRC BIOSTATISTICS UNIT   (United Kingdom)

^b Cambridge Cancer Trials Centre   (United Kingdom)

Author keywords

Phase II cancer clinical trials; RECIST; Sample sizes; Trial design

Indexed keywords

ARTICLE; CANCER RESEARCH; CLINICAL RESEARCH; CONTROLLED STUDY; INTERMETHOD COMPARISON; PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; SAMPLE SIZE; STUDY DESIGN; TUMOR GROWTH;

CLINICAL TRIALS, PHASE II AS TOPIC; FALSE POSITIVE REACTIONS; HUMANS; NEOPLASM STAGING; NEOPLASMS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SAMPLE SIZE; STATISTICS AS TOPIC;

EID: 79953717724     PISSN: 09598049     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ejca.2010.12.007     Document Type: Article

References (24)

1. Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: Revised RECIST guideline (version 1.1). Eur J Cancer 2009;45:228-47.
2. Farewell VT, Tom BDM, Royston P. The impact of dichotomization on the efficiency of testing for an interaction effect in exponential family models. JASA 2004;99:822-31. (Pubitemid 39332864)
3. DeCoster J, Iselin AR, Gallucci M. A conceptual and empirical examination of justifications for dichotomization. Psychol Methods 2009;14:349-66.
4. Lavin PT. An alternative model for the evaluation of antitumor activity. Cancer Clin Trials 1981;4:451-7.
5. Karrison TG, Maitland ML, Stadler WM, Ratain MJ. Design of phase II cancer trials using a continuous endpoint of change in tumour size: application to a study of sorafenib and erlotinib in non-small-cell lung cancer. JNCI 2007;99:1455-61.
6. Tang H, Foster NR, Grothey A, et al. Comparison of error rates in single-arm versus randomized phase II cancer clinical trials. J Clin Oncol 2010;28:1936-41.
7. Simon R. Optimal two-stage designs for phase II clinical trials. Controlled Clin Trials 1989;10:1-10. (Pubitemid 19099520)
8. Lee J, Feng L. Randomized phase II designs in cancer clinical trials: current status and future directions. J Clin Oncol 2005;23:4450-7. (Pubitemid 46209969)
9. Pocock SJ. Group sequential methods in the design and analysis of clinical trials. Biometrika 1977;64:191-9. (Pubitemid 8145484)
10. O'Brien PC, Flemming TR. A multiple-testing procedure for clinical trials. Biometrics 1979;35:549-56.
11. Emerson SS, Flemming TR. Symmetric group sequential designs. Biometrics 1989;45:905-23.
12. Eales JD, Jennison C. An improved method for deriving optimal one-sided group sequential tests. Biometrika 1992;79:13-24.
13. Whitehead J, Stratton I. Group sequential clinical trials with triangular continuation regions. Biometrics 1983;39:227-36.
14. Jennison C, Turnbull BW. Group sequential methods with applications to clinical trials. Chapman and Hall; 2000.
15. Whitehead J, Valdes-Marquez E, Lissmats A. A simple two-stage design for quantitative responses with application to a study in diabetic neuropathic pain. Pharm Stat 2009;8:125-35.
16. Supplementary material for "Reducing sample sizes in two-stage phase II cancer trials by using continuous tumour shrinkage endpoints". www.mrc-bsu.cam.ac.uk/Publications/ addmaterial.
17. Mander AP, Thompson SG. Two-stage designs optimal under the alternative hypothesis for phase II cancer clinical trials. Contemporary Clin Trials 2010;31:572-8.
18. Miller AB, Hoogstraten B, Staquet M, Winkler A. Reporting results of cancer treatment. Cancer 1981;47:207-14. (Pubitemid 11167383)
19. Cressie NAC, Sheffield LJ, Whitford HJ. Use of the one sample t-test in the real world. J Chron Dis 1984;37:107-14. (Pubitemid 14221382)
20. Azim HA, Ganti AK. Targeted therapy in advanced non-small cell lung cancer (NSCLC): where do we stand? Cancer Treat Rev 2006;32:630-6. (Pubitemid 44645025)
21. Lui P, Cashin R, Machado M, et al. Treatments for metastatic melanoma: synthesis of evidence from randomized trials. Cancer Treat Rev 2007;33:665-80. (Pubitemid 350082665)
22. Nelson EC, Evans CP, Lara PN. Renal cell carcinoma: current status and emerging therapies. Cancer Treat Rev 2007;33:299-313. (Pubitemid 46767941)
23. Box GEP, Cox DR. An analysis of transformations. J R Stat Soc Ser B 1964;26:211-52.
24. Erasmus JJ, Gladish GW, Broemeling L, et al. Interobserver and intraobserver variability in measurement of non-small-cell carcinoma lung lesions: implications for assessment of tumour response. J Clin Oncology 2003;21:2574-82. (Pubitemid 46606340)