Asymptotic simultaneous confidence intervals for many-to-one comparisons of binary proportions in randomized clinical trials

Journal of Biopharmaceutical Statistics

Volumn 19, Issue 2, 2009, Pages 292-310

  Schaarschmidt, Frank ^a   Biesheuvel, Egbert ^b   Hothorn, Ludwig A ^a  

^a LEIBNIZ UNIVERSITY HANNOVER   (Germany)

^b Biometrics Department   (Netherlands)

Author keywords

Binomial; Comparison to control; Multiple comparisons; One sided confidence intervals; Risk difference

Indexed keywords

CARBOPLATIN; CISPLATIN; DOCETAXEL; GEMCITABINE; LIAROZOLE; PACLITAXEL;

ARTICLE; CLINICAL TRIAL; COMPARATIVE STUDY; CONFIDENCE INTERVAL; DRUG DOSE COMPARISON; HUMAN; LUNG NON SMALL CELL CANCER; MATHEMATICAL MODEL; PRIORITY JOURNAL; PROBABILITY; PROBABILITY SAMPLE; PSORIASIS; RANDOMIZED CONTROLLED TRIAL; SIMULATION;

ALGORITHMS; ANTINEOPLASTIC AGENTS; CARCINOMA, NON-SMALL-CELL LUNG; CONFIDENCE INTERVALS; DATA INTERPRETATION, STATISTICAL; LUNG NEOPLASMS; PROBABILITY; PSORIASIS; RANDOM ALLOCATION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SAMPLE SIZE; SURVIVAL ANALYSIS; TREATMENT OUTCOME;

EID: 60749117479     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543400802622501     Document Type: Article

References (36)

1. Agresti, A., Coull, B. A. (1998). Approximate is better than "exact" for interval estimation of binomial proportions. Am. Stat. 52:119-126.
2. Agresti, A., Caffo, B. (2000). Simple and effective confidence intervals for proportions and differences of proportions result from adding two successes and two failures. Am. Stat. 54:280-288.
3. Agresti, A, Min, Y. (2001). On small-sample confidence intervals for parameters in discrete distributions. Biometrics 57:963-971.
4. Andres, A. M., Tejedor, I. H. (2003). Unconditional confidence interval for the difference of two proportions. Biometrical J. 45:426-436.
5. Ballenger, J. C., Wheadon, D. E., Steiner, M., Bushnell, W., Gergel, I. P. (1998). Double-blind, fixed dose, placebo-controlled study of paroxetine in the treatment of panic disorder. Am. J. Psychiat. 155:36-42.
6. Berth-Jones, J., Todd, G., Hutchinson, P. E., Thestrup-Pedersen, K., Vanhoutte, F. P. (2000). Treatment of psoriasis with oral liarozole: a dose-ranging study. Brit. J. Dermatol. 143:1170-1176.
7. Bretz, F., Hothorn, L. A. (2002). Detecting dose-response using contrasts: asymptotic power and sample size determination for binomial data. Stat. Med. 21:3325-3335.
8. Brown, L. D., Li, X. (2005). Confidence intervals for two sample binomial distribution. J. Stat. Plan. Infer. 130:359-375.
9. Brown, L. D., Cai, T. T., DasGupta, A. (2001). Interval estimation for a binomial proportion. Stat. Sci. 16:101-128.
10. Cai, T. T. (2005). One-sided confidence intervals in discrete distributions. J. Stat. Plan. Infer. 131:63-88.
11. Chan, I. S. F., Zhang, Z. (1999). Test based exact confidence intervals for the difference of two binomial proportions. Biometrics 55:1202-1209.
12. Chen, J., Sarkar, S. K. (2004). Multiple testing of response rates with a control: a Bayesian stepwise approach. J. Stat. Plan. Infer. 125:3-16.
13. Chuang-Stein, C., Heft, S., Koury, K. (2005). Four case studies to highlight some opportunities and challenges in developing anti-bacterial and anti-fungal agents. Pharm. Stat. 4:253-265.
14. Dunnett, C. W. (1955). A multiple comparison procedure for comparing several treatments with a control. J. Am. Stat. Assoc. 50:1096-1121.
15. Eisenberg, P., MacKintosh, F. R., Ritch, P., Cornett, P. A., Macciocchi, A. (2004). Efficacy, safety and pharmacokinetics of palonosetron in patients receiving highly emetogenic cisplatin-based chemotherapy: a dose-ranging clinical study. Ann. Oncol. 15:330-337.
16. Genz, A., Bretz, F. (1999). Numerical computation of multivariate t-probabilities with application to power calculation of multiple contrasts. J. Stat. Comput. Sim. 63:361-378.
17. Genz, A., Bretz, F., Hothorn, T. (2007). Mvtnorm: multivariate normal and t distribution. R package version 0.8-1.
18. Hedenus, M., Hansen, S., Taylor, K., Arthur, C., Emmerich, B., Dewey, C., et al. (2002). Randomized, dose-finding study of darbepoetin alfa in anaemic patients with lymphoproliferative malignancies. Brit. J. Haematol. 119:79-86.
19. Holford, T. R., Walter, S. D., Dunnett, C. W. (1989). Simultaneous interval estimates of the odds ratio in studies with two or more comparisons. J. Clin. Epidemiol. 42:427-434.
20. Hothorn, T., Bretz, F., Genz, A. (2001). On multivariate t and Gauß probabilities in R. R News 1(2):27-29.
21. Koch, H. F., Hothorn, L. A. (1999). Exact unconditional distributions for dichotomous data in many-to-one comparisons. J. Stat. Plan. Infer. 82:83-99.
22. Marschner, I. C. (2007). Optimal design of clinical trials comparing several treatments with a control. Pharm. Stat. 6:23-33.
23. McCullagh, J., Nelder, J. A. (1989). Generalized Linear Models. London: Chapman and Hall.
24. Nadarajah, S., Kotz, S. (2007). Statistical distribution of the difference of two proportions. Stat. Med. 26:3518-3523.
25. Newcombe, R. G. (1998). Interval estimation for the difference between independent proportions: comparison of eleven methods. Stat. Med. 17:873-890.
26. Pan, W. (2002). Approximate confidence intervals for one proportion and difference of two proportions. Comp. Stat. Data An. 40:143-157.
27. Piegorsch, W. W. (1991). Multiple comparisons for analyzing dichotomous response. Biometrics 47:45-52.
28. Piegorsch, W. W. (1995). Many-to-one comparison procedures for dichotomous endpoints. Biometrie in der Chemisch-Pharmazeutischen Industrie 6:61-76.
29. R Development Core Team (2007). R: A Language and Environment for Statistical Computing. R Foundation for Statistical Computing, Vienna, Austria. URL http://www.Rproject.org
30. Röhmel, J. (2005). Problems with existing procedures to calculate exact unconditional p-values for non-inferiority/superiority and confidence intervals for two binomials and how to resolve them. Biometrical J. 47:37-47.
31. Santner, T. J., Pradhan, V., Senchaudhuri, P., Mehta, C.R., Tamhane, A. (2007). Smallsample comparisons of confidence intervals for the difference of two independent binomial proportions. Comput. Stat. Data An. 51:5791-5799.
32. SAS Institute Inc. (1999). SAS OnlineDoc®. Version 8. Cary, NC: SAS Institute, Inc.
33. Schiller, J. H., Harrington, D., Belani, C. P., Langer, C., Sandler, A., Krook, J., et al. (2002). Comparison of four chemotherapy regimens for advanced non-small-cell lung cancer. New Engl. J. Med. 346:92-98.
34. Wilson, E. B. (1927). Probable inference, the law of succession, and statistical inference. J. Am. Stat. Assoc. 22:209-212.
35. Zhou, X. H., Tsao, M., Qin, G. (2004). New intervals for the difference between two independent binomial proportions. J. Stat. Plan. Infer. 123:97-115.
36. Zou, G., Donner, A. (2004). A simple alternative confidence interval for the difference between two proportions. Control. Clin. Trial 25:3-12.