Subgroup identification from randomized clinical trial data

Statistics in Medicine

Volumn 30, Issue 24, 2011, Pages 2867-2880

  Foster, Jared C ^a   Taylor, Jeremy M G ^a   Ruberg, Stephen J ^b  

^a UNIVERSITY OF MICHIGAN   (United States)

^b ELI LILLY AND COMPANY   (United States)

Author keywords

Random forests; Randomized clinical trials; Regression trees; Subgroups; Tailored therapeutics

Indexed keywords

AREA UNDER THE CURVE; ARTICLE; CROSS-SECTIONAL STUDY; HUMAN; LOGISTIC REGRESSION ANALYSIS; MATHEMATICAL ANALYSIS; METHODOLOGY; PATIENT IDENTIFICATION; PREDICTION; PREDICTIVE VALUE; PROBABILITY; SAMPLE SIZE; SENSITIVITY AND SPECIFICITY; SIMULATION; THERAPY EFFECT; VALIDATION STUDY; VIRTUAL TWINS METHOD;

BIAS (EPIDEMIOLOGY); BIOSTATISTICS; COMPUTER SIMULATION; DATA INTERPRETATION, STATISTICAL; DATA MINING; HUMANS; LOGISTIC MODELS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLE SIZE;

EID: 80053563163     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.4322     Document Type: Article

References (21)

1. Ruberg SJ, Chen L, Wang Y. The mean doesn't mean as much anymore: finding sub-groups for tailored therapeutics. Clinical Trials 2010; 7:574-583.
2. Assmann SF, Pocock SJ, Enos LE. Subgroup analysis and other (mis)uses of baseline data in clinical trials. The Lancet 2000; 355:1064-1069.
3. Brookes ST, Whitley E, Peters TJ, Mulheran PA, Egger M, Smith GD. Subgroup analyses in randomized controlled trials: quantifying the risks of false-positives and false-negatives. Health Technology Assessment 2001; 5(33):1-56.
4. Brookes ST, Whitely E, Egger M, Smith GD, Mulheran PA, Peters TJ. Subgroup analyses in randomized trials: risks of subgroup-specific analyses; power and sample size for the interaction test. Journal of Clinical Epidemiology 2004; 57:229-236.
5. Cui L, Hung HMJ, Wang SJ, Tsong Y. Issues related to subgroup analysis in clinical trials. Journal of Biopharmaceutical Statistics 2002; 12(3):347-358.
6. Lagakos SW. The challenge of subgroup analyses-reporting without distorting. New England Journal of Medicine 2006; 354(16):1667-1669.
7. Peto R, Collins R, Gray R. Large-scale randomized evidence: large simple trials and overviews of trials. Journal of Clinical Epidemiology 1995; 48:23-40.
8. Pocock SJ, Assmann SE, Enos LE, Kasten LE. Subgroup analysis, covariate adjustment and baseline comparisons in clinical trial reporting: current practice and problems. Statistics in Medicine 2002; 21:2917-2930.
9. Rothwell PM. Subgroup analysis in randomized controlled trials: importance, indications and interpretation. The Lancet 2005; 365:176-186.
10. Yusuf S, Wittes J, Probstfield J, Tyroler HA. Analysis and interpretation of treatment effects in subgroups of patients in randomized clinical trials. The Journal Of The American Medical Association 1991; 266(1):93-98.
11. Wang R, Lagakos SW, Ware JH, Hunter DJ, Drazen JM. Statistics in medicine-reporting of subgroup analyses in clinical trials. New England Journal of Medicine 2007; 357(21):2189-2194.
12. Feinstein AR. The problem of cogent subgroups: a clinicostatistical tragedy. Journal of Clinical Epidemiology 1998; 51(4):297-299.
13. Kehl V, Ulm K. Responder identification in clinical trials with censored data. Computational Statistics & Data Analysis 2006; 50(5):1338-1355. .
14. Dixon DO, Simon R. Bayesian subset analysis. Biometrics 1991; 47:871-881.
15. Gail M, Simon R. Testing for qualitative interactions between treatment effects and patient subsets. Biometrics 1985; 41:361-372.
16. Simon R. Bayesian subset analysis: application to studying treatment-by-gender interactions. Statistics in Medicine 2002; 21:2909-2916.
17. Negassa A, Ciampi A, Abrahamowicz M, Shapiro S, Boivin JR. Tree-structured subgroup analysis for censored survival data: Validation of computationally inexpensive model selection criteria. Statistics and Computing 2005; 15:231-239.
18. Su X, Tsai CL, Wang H, Nickerson DM, Bogong L. Subgroup analysis via recursive partitioning. The Journal of Machine Learning Research 2009; 10:141-158.
19. Su X, Zhou T, Yan X, Fan J, Yang S. Interaction trees with censored survival data. The International Journal of Biostatistics 2008; 4(1). Article 2.
20. Freidlin B, Jiang W, Simon R. The cross-validated adaptive signature design. Clinical Cancer Research 2010; 16(2):691-698.
21. Breiman L. Random forests. Machine Learning 2001; 45(1):5-32.