Continual reassessment method vs. traditional empirically based design: Modifications motivated by phase i trials in pediatric oncology by the pediatric brain tumor consortium

Journal of Biopharmaceutical Statistics

Volumn 19, Issue 3, 2009, Pages 437-455

  Onar, Arzu ^a   Kocak, Mehmet ^a   Boyett, James M ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

Continual reassessment method; Dose finding; Dose toxicity model; Simulation; Up and down studies

Indexed keywords

ACCURACY; BRAIN TUMOR; CLINICAL TRIAL; CONTINUAL REASSESSMENT METHOD; EMPIRICALLY BASED TRADITIONAL METHOD; EMPIRICISM; GROUPS BY AGE; HUMAN; METHODOLOGY; ONCOLOGY; POPULATION; PRIORITY JOURNAL; REVIEW; SIMULATION; ARTICLE; CHILD; MAXIMUM TOLERATED DOSE; PHASE 1 CLINICAL TRIAL; PROBABILITY; STATISTICS; THEORETICAL MODEL;

ANTINEOPLASTIC AGENT;

ANTINEOPLASTIC AGENTS; BRAIN NEOPLASMS; CHILD; CLINICAL TRIALS, PHASE I AS TOPIC; HUMANS; MAXIMUM TOLERATED DOSE; MODELS, THEORETICAL; PROBABILITY; RESEARCH DESIGN;

EID: 67651052151     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543400902800486     Document Type: Review

References (20)

1. Fitzpatrick, S., Scott, A. (1987). Quick simultaneous confidence intervals for multinomial proportions. JASA 82:875-878.
2. Goodman, S. N., Zahurak, M. L., Piantadosi, S. (1995). Some practical improvements in the continual reassessment method for Phase I studies. Stat. Med. 14:1149-1161.
3. Heyd, J., Carlin, B. (1999). Adaptive design improvements in the continual reassessment method for Phase I studies. Stat. Med. 18:1307-1321.
4. Kasahara, K., Myo, S., Iwasa, K., Kimura, H., Shirasaki, H., Yasuda, U., Shibata, K., Shintani, H., Nishi, K., Fujimura, M., Nakao, S. (2002). A Phase I study of carboplatin and docetaxel for advanced non-small cell lung cancer using the continual reassessment method. Japanese J. Clin. Oncol. 32:512-516.
5. Korn, E. L., Midthune, D., Chen, T. T., Rubinstein, L. V., Christian, M. C., Simon, R. M. (1994). A comparison of two Phase I trial designs. Stat. Med., Sept. 30, 13(18): 1799-1806.
6. Lee, D. P., Skolnik, J. M., Adamson, P. C. (2005). Pediatric Phase I trials in oncology: An analysis of study conduct efficiency. J. Clin. Oncol. 23:8431-8441.
7. Lin, Y., Shih, W. J. (2001). Statistical properties of the traditional algorithm-based designs for Phase I cancer clinical trials. Biostatistics 2:203-215.
8. Marsoni, S., Ungerleider, R. S., Hurson, S. B., Simon, R. M., Hammershaimb, L. D. (1985). Tolerance to antineoplastic agents in children and adults. Cancer Treat. Reports 69:1263-1269.
9. O'Quigley, J. (2002). Continual reassessment designs with early termination. Biostatistics 3:87-99.
10. O'Quigley, J., Shen, L. Z. (1996). Continual reassessment method: A likelihood approach. Biometrics 52:673-684.
11. O'Quigley, J., Reiner, E. (1998). A stopping rule for the continual reassessment method. Biometrika 85:741-748.
12. O'Quigley, J., Pepe, M., Fisher, L. (1990). Continual reassessment method: A practical design for Phase I clinical studies in cancer. Biometrics 46:33-48.
13. Piantadosi, S., Liu, G. (1996). Improved designs for dose escalation studies using pharmacokinetic measurements. Stat. Med. 15:1605-1618.
14. Piantadosi, S., Fisher, J. D., Grossman, S. (1998). Practical implementation of a modified continual reassessment method for dose finding trials. Cancer Chemother. Pharmacol. 41:429-436.
15. Royce, M. E., Hoff, P. M., Dumas, P., Lassere, Y., Lee, J. J., Coyle, J., Ducharme, M. P., De Jager, R., Pazdur, R. (2001). Phase I and pharmacokinetic study of exatecan mesylate (DX-8951f): a novel camptothecin analog. J. Clin. Oncol. 19:1493-1500.
16. Schoffski, P., Riggert, S., Fumoleau, P., Campone, M., Bolte, O., Marreaud, S., Lacombe, D., Baron, B., Herold, M., Zwierzina, H., Wilhelm-Ogunbiyi, K., Lentzen, H., Twelves, C. (2004). Phase I trial of intravenous aviscumine (rViscumin) in patients with solid tumors: a study of the European Organization for Research and Treatment of Cancer New Drug Development Group. Ann. Oncol. 15:1816-1824.
17. Shen, L. Z., O'Quigley, J. (1996). Consistency of continual reassessment method under model misspecification. Biometrika 83:395-405.
18. Smith, M., Bernstein, M., Bleyer, W. A., Borsi, J. D., Ho, P., Lewis, I. J., Pearson, A., Pein, F., Pratt, C., Reaman, G., Riccardi, R., Seibel, N., Trueworthy, R., Ungerleider, R., Vassal, G., Vietti, T. (1998). Conduct of Phase I trials in children with cancer. J. Clin. Oncol. 16:966-978. (Pubitemid 28108729)
19. Storer, B. (1989). Design and analysis of Phase I clinical trials. Biometrics 45:925-937.
20. Thall, P. F., Lee, J. J., Tseng, C., Etsey, E. H. (1999). Accrual strategies for Phase I trials with delayed patient outcome. Stat. Med. 18:1155-1169.