Using continuous data on tumour measurements to improve inference in phase II cancer studies

Statistics in Medicine

Volumn 32, Issue 26, 2013, Pages 4639-4650

  Wason, James M S ^a   Seaman, Shaun R ^a  

^a MRC BIOSTATISTICS UNIT   (United Kingdom)

Author keywords

Continuous tumour shrinkage endpoints; Informative dropout; Longitudinal model; Phase II cancer trial

Indexed keywords

CAPECITABINE; PLACEBO; PYRIDOXINE; ANTINEOPLASTIC AGENT; DEOXYCYTIDINE; FLUOROURACIL;

ARTICLE; CANCER COMBINATION CHEMOTHERAPY; CLINICAL STUDY; CONTROLLED STUDY; DRUG DOSE REDUCTION; HAND FOOT SYNDROME; HUMAN; LOGISTIC REGRESSION ANALYSIS; MAJOR CLINICAL STUDY; MALIGNANT NEOPLASTIC DISEASE; MEDICAL INFORMATION; MONOTHERAPY; MONTE CARLO METHOD; MULTICENTER STUDY; NORMAL DISTRIBUTION; PALLIATIVE THERAPY; PHASE 2 CLINICAL TRIAL; PROBABILITY; RANDOMIZED CONTROLLED TRIAL; SENSITIVITY ANALYSIS; SIMULATION; TREATMENT DURATION; TREATMENT FAILURE; TREATMENT RESPONSE; TUMOR SHRINKAGE; TUMOR VOLUME; ANALOGS AND DERIVATIVES; COMPUTER SIMULATION; HAND-FOOT SYNDROME; NEOPLASMS; PATHOLOGY; PHASE 2 CLINICAL TRIAL (TOPIC); PROCEDURES; STATISTICAL ANALYSIS;

ANTINEOPLASTIC AGENTS; CLINICAL TRIALS, PHASE II AS TOPIC; COMPUTER SIMULATION; DATA INTERPRETATION, STATISTICAL; DEOXYCYTIDINE; FLUOROURACIL; HAND-FOOT SYNDROME; HUMANS; NEOPLASMS;

EID: 84885431341     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.5867     Document Type: Article

References (18)

1. Simon R.Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 1989; 10:1-10.
2. Rubinstein L, Crowley J, Ivy P, LeBlanc M, Sargent D.Randomized phase II designs. Clinical Cancer Research 2009; 15:1883-1890.
3. Eisenhauer EA, Therasse P, Bogaerts J, Schwartz LH, Sargent D, Ford R, Dancey J, Arbuck S, Gwyther S, Mooney M, Rubinstein L, Shankar L, Dodd L, Kaplan R, Lacombe D, Verweij J.New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). European Journal of Cancer 2009; 45:228-247.
4. Altman D, Royston P.The cost of dichotomising continuous variables. BMJ 2006; 332:1080.
5. Lavin P.An alternative model for the evaluation of antitumor activity. Cancer Clinical Trials 1981; 4:451-457.
6. Karrison T, Maitland M, Stadler W, Ratain M.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; 99:1455-1461.
7. Wason J, Mander A, Eisen T.Reducing sample sizes in two-stage phase II cancer trials by using continuous tumour shrinkage endpoints. European Journal of Cancer 2011; 47:983-989.
8. Bryant J, Day R.Incorporating toxicity considerations into the design of two-stage phase II clinical trials. Biometrics 1995; 51:1372-1383.
9. Suissa S.Binary methods for continuous outcomes: a parametric alternative. Journal of Clinical Epidemiology 1991; 44:241-248.
10. Wason J, Mander A.The choice of test in phase II cancer trials assessing continuous tumour shrinkage when complete responses are expected. Statistical Methods in Medical Research 2012; Epub. DOI: 10.1177/0962280211432192; epublished in 2011.
11. R Development Core Team.R: A language and environment for statistical computing, R Foundation for Statistical Computing, Vienna, Austria, 2011. http://www.R-project.org, ISBN 3-900051-07-0.
12. Pinheiro J, Bates D, DebRoy S, Sarkar D, R Development Core Team. nlme: Linear and nonlinear mixed effects models, 2011. R package version 3.1-101.
13. Wilson E.Probable inference, the law of succession, and statistical inference. Journal of the American Statistical Association 1927; 22:209-212.
14. Peduzzi P, Concato J, Kemper E, Holford T, Feinstein A.A simulation study of the number of events per variable in logistic regression analysis. Journal of Clinical Epidemiology 1996; 49:1373-1379.
15. Corrie PG, Bulusu R, Wilson CB, Armstrong G, Bond S, Hardy R, Lao-Sirieix S, Parashar D, Ahmad A, Daniel F, Hill M, Wilson G, Blesing C, Moody AM, McAdam K, Osborne M.A randomised study evaluating the use of pyridoxine to avoid capecitabine dose modifications. British Journal of Cancer 107:585-587.
16. Jaki T, Andre V, Su T-L, Whitehead J.Designing exploratory cancer trials using change in tumour size as primary endpoint. Statistics in Medicine 2013; 32(15):2544-2554.
17. Henderson R, Diggle P, Dobson A.Joint modelling of longitudinal measurements and event time data. Biostatistics 1:465-480.
18. An MW, Mandrekar SJ, Branda ME, Hillman SL, Adjei AA, Pitot HC, Goldberg RM, Sargent DJ.Comparison of continuous versus categorical tumor measurement-based metrics to predict overall survival in cancer treatment trials. Clinical Cancer Research 2011; 17:6592-6599.