Clinical trial designs for prospective validation of biomarkers

American Journal of PharmacoGenomics

Volumn 5, Issue 5, 2005, Pages 317-325

  Mandrekar, Sumithra J ^a   Grothey, Axel ^a   Goetz, Matthew P ^a   Sargent, Daniel J ^a,b  

^a MAYO CLINIC   (United States)

^b Cancer Center Statistics Unit   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

7 ETHYL 10 HYDROXYCAMPTOTHECIN; BIOLOGICAL MARKER; IRINOTECAN;

ARTICLE; BREAST CANCER; CANCER RECURRENCE; CANCER STAGING; CANCER SURVIVAL; CLINICAL TRIAL; CORRELATION ANALYSIS; DIFFERENTIAL DIAGNOSIS; DOSE RESPONSE; GENETIC POLYMORPHISM; GENETICS; GENOTYPE; HUMAN; ONCOLOGY; OUTCOMES RESEARCH; PHARMACOGENETICS; PREDICTION; PREVALENCE; PRIORITY JOURNAL; QUALITY OF LIFE; RISK ASSESSMENT; TOXICITY; VALIDATION PROCESS;

CAMPTOTHECIN; CLINICAL TRIALS; GENOTYPE; HUMANS; METABOLIC DETOXICATION, DRUG; NEOPLASMS; OUTCOME ASSESSMENT (HEALTH CARE); PREDICTIVE VALUE OF TESTS; REPRODUCIBILITY OF RESULTS; RESEARCH DESIGN; TUMOR MARKERS, BIOLOGICAL;

EID: 26444543829     PISSN: 11752203     EISSN: 11752203     Source Type: Journal    
DOI: 10.2165/00129785-200505050-00004     Document Type: Article

References (37)

1. Goetz MP, Ames MM, Weinshilboum RM. Primer on medical genomics: part XII. Pharmacogenomics: general principles with cancer as a model. Mayo Clin Proc 2004; 79 (3): 376-84
2. Toi M, Matsumoto T, Bando H. Vascular endothelial growth factor: its prognostic, predictive, and therapeutic implications. Lancet Oncol 2001; 2: 667-73
3. Albain KS. Adjuvant chemotherapy for lymph node-negative, estrogen receptor-negative breast cancer: a tale of three trials. J Natl Cancer Inst 2004; 96 (24): 1801-2
4. Paik S. Clinical trial methods to discover and validate predictive markers for treatment response in cancer. Biotechnol Annu Rev 2003; 9: 259-67
5. Elizabeth M, Hammond H, Taube SE. Issues and barriers to development of clinically useful tumor markers: a development pathway proposal. Semin Oncol 2002; 29 (3): 213-21
6. Pajak TF, Clark GM, Sargent DJ, et al. Statistical issues in tumor marker studies. Arch Pathol Lab Med 2000; 124: 1011-5
7. Cordon-Cardo C. p53 and RB: simple interesting correlates or tumor markers of critical predictive nature? J Clin Oncol 2004; 22 (6): 975-7
8. Pepe MS, Etzioni R, Feng Z, et al. Phases of biomarker development for early detection of cancer. J Natl Cancer Inst 2001; 93 (14): 1054-61
9. Hayes DF, Bast RC, Desch CE, et al. Tumor marker utility grading system: a framework to evaluate clinical utility of tumor markers. J Natl Cancer Inst 1996; 88 (20): 1456-66
10. O'Leary J, Muggia FM. Camptothecins: a review of their development and schedules of administration. Eur J Cancer 1998; 34 (10): 1500-8
11. Pommier Y, Tanizawa A, Kohn KW. Mechanisms of topoisomerase I inhibition by anticancer drugs. Adv Pharmacol 1994; 29B: 73-92
12. Hsiang YH, Lihou MG, Liu LF. Arrest of replication forks by drug-stabilized topoisomerase I-DNA cleavable complexes as a mechanism of cell killing by camptothecin. Cancer Res 1989; 49: 5077-82
13. Holm C, Covey JM, Kerrigan D, et al. Differential requirement of DNA replication for the cytotoxicity of DNA topoisomerase I and II inhibitors in Chinese hamster DC3F cells. Cancer Res 1989; 49: 6365-8
14. Shin CG, Snapka RM. Exposure to camptothecin breaks leading and lagging strand simian virus 40 DNA replication forks. Biochem Biophys Res Commun 1990; 168: 135-40
15. Lampe JW, Bigler J, Horner NK, et al. UDP-glucouronosyltransferase (UGT1A1*28 and UGT1A6*2) polymorphisms in Caucasians and Asians: relationships to serum bilirubin concentrations. Pharmacogenetics 1999; 9: 341-9
16. Innocenti F, Iyer L, Ratain MJ. Pharmacogenetics of anticancer agents: lessons from amonafide and irinotecan. Drug Metab Dispos 2001; 29 (4): 596-600
17. Iyer L, Hall D, Das S, et al. Phenotype-genotype correlation of in vitro SN-38 (active metabolite of irinotecan) and bilirubin glucuronidation in human liver tissue with UGT1A1 promoter polymorphism. Clin Pharmacol Ther 1999; 65: 576-82
18. McLeod HL, Watters JW. Irinotecan pharmacogenetics: is it time to intervene? J Clin Oncol 2004; 22 (8): 1356-9
19. Goetz MP, Safgren S, Goldberg RM, et al. A phase I does escalation study of irinotecan (CPT-11), oxaliplatin (Oxal) capecitabine (Cap) within three UGT1A1 TA promoter cohorts (6/6, 6/7, and 7/7) [abstract no. 2014]. Proceedings of the 41st Annual Meeting of the American Society of Clinical Oncology; 2005 May 13-17; Orlando (FL). J Clin Oncol 2005; 28 (16S): 138s
20. Sargent DJ, Goldberg RM. A flexible design for multiple armed screening trials. Stat Med 2001; 20: 1051-60
21. Sargent DJ, Conley BA, Allegra C, et al. Clinical trial design for predictive marker validation in cancer treatment trials. J Clin Oncol 2005; 23 (9): 2020-7
22. Chung KY, Shia J, Kemeny NE, et al. Cetuximab shows activity in colorectal cancer patients with tumors that do not express the epidermal growth factor receptor by immunohistochemistry. J Clin Oncol 2005; 23 (9): 1803-10
23. Ma XJ, Wang Z, Ryan PD, et al. A Two-gene expression ratio predicts clinical outcome in breast cancer patients treated with tamoxifen. Cancer Cell 2004; 5: 607-16
24. Jansen MP, Foekens JA, van Staveren IL, et al. Molecular classification of tamoxifen-resistant breast carcinomas by gene expression profiling. J Clin Oncol 2005; 23 (4): 732-40
25. Loi S, Piccart M, Haibe-Kains B, et al. Prediction of early distant relapses on tamoxifen in early-stage breast cancer (BC): a potential tool for adjuvant aromatase inhibitor (AI) tailoring [abstract no. 509]. Proceedings of the 41st Annual Meeting of the American Society of Clinical Oncology; 2005 May 13-17; Orlando (FL). J Clin Oncol 2005; 28 (16S): 6s
26. Paik S, Shak S, Tang G, et al. A multigene assay to predict recurrence of tamoxifen-treated, node-negative breast cancer. N Engl J Med 2005; 351: 2817-26
27. Esteva FJ, Sahin AA, Cristofanilli M, et al. Prognostic role of a multigene reverse transcriptase-pcr assay in patients with node-negative breast cancer not receiving adjuvant systemic therapy. Clin Cancer Res 2005; 11 (9): 3315-9
28. Fiets WE, Nortier JWR. A multi-gene RT-PCR assay used to predict recurrence in early breast cancer: two presentations with contradictory results. Breast Cancer Res 2004; 6: 185-7
29. Paik S, Shak S, Tang G, et al. Expression of the 21 genes in the recurrence score assay and prediction of clinical benefit from tamoxifen in NSABP study B-14 and chemotherapy in NSABP study B-20 [abstract no. 24]. Breast Cancer Res Treat 2004; 88: S15
30. Sparano JA, Fazzari MJ, Childs G. Clinical application of molecular profiling in breast cancer. Fut Oncol 2005; 1 (4): 485-96
31. Winer EP, Hudis C, Burstein HJ, et al. American Society of Clinical Oncology technology assessment on the use of aromatase inhibitors as adjuvant therapy for postmenopausal women with hormone receptor-positive breast cancer: status report 2004. J Clin Oncol 2005; 23 (3): 619-29
32. Kurbacher CM, Grecu OM, Stier U, et al. ATP Chemosensitivity testing in ovarian and breast cancer: early clinical trials. Recent Results Cancer Res 2003; 161: 221-30
33. Song X, Pepe MS. Evaluating markers for selecting a patient's treatment. Biometrics 2004; 60: 874-83
34. Simon R, Maitournam A. Evaluating the efficiency of targeted designs for randomized clinical trials. Clin Cancer Res 2004; 10: 6759-63
35. Cappuzzo F, Hirsch FR, Rossi E, et al. Epidermal growth factor receptor gene and protein and gefitinib sensitivity in non-small-cell lung cancer. J Natl Cancer Inst 2005; 97 (9): 643-55
36. Romond J. Special session: modoclonal antibody therapy in breast cancer. Proceedings of the 41st Annual Meeting of the American Society of Clinical Oncology; 2005 May 13-17; Orlando (FL)
37. Perez E. Special session: monoclonal antibody therapy in breast cancer. Proceedings of the 41st Annual Meeting of the American Society of Clinical Oncology; 2005 May 13-17; Orlando (FL)