Meta-analysis of the relationship between dose and benefit in phase i targeted agent trials

Journal of the National Cancer Institute

Volumn 104, Issue 24, 2012, Pages 1860-1866

  Gupta, Sachin ^a   Hunsberger, Sally ^b   Boerner, Scott A ^a   Rubinstein, Larry ^b   Royds, Robert ^c,d   Ivy, Percy ^b   Lorusso, Patricia ^a  

^a WAYNE STATE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b NATIONAL INSTITUTES OF HEALTH   (United States)

^c NATIONAL CANCER INSTITUTE   (United States)

^d NONE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANGIOGENESIS INHIBITOR; ANTINEOPLASTIC AGENT; CYCLIN DEPENDENT KINASE INHIBITOR; CYTOTOXIC T LYMPHOCYTE ANTIGEN 4; EPIDERMAL GROWTH FACTOR RECEPTOR KINASE INHIBITOR; HISTONE DEACETYLASE INHIBITOR; INTEGRIN; MAMMALIAN TARGET OF RAPAMYCIN INHIBITOR; MONOCLONAL ANTIBODY CD20; MONOCLONAL ANTIBODY CD30; MONOCLONAL ANTIBODY HER 2; MONOCLONAL ANTIBODY TAG 72; PROTEASOME INHIBITOR; PROTEIN BCL 2 INHIBITOR; PROTEIN FARNESYLTRANSFERASE INHIBITOR; UNCLASSIFIED DRUG; VASCULOTROPIN INHIBITOR;

CANCER CHEMOTHERAPY; CANCER COMBINATION CHEMOTHERAPY; DOSE RESPONSE; HEMATOLOGIC MALIGNANCY; HUMAN; MAXIMUM TOLERATED DOSE; MONOTHERAPY; OVERALL SURVIVAL; PHASE 1 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; REVIEW; SOLID TUMOR; TREATMENT RESPONSE;

ANTINEOPLASTIC AGENTS; CLINICAL TRIALS, PHASE I AS TOPIC; CONFOUNDING FACTORS (EPIDEMIOLOGY); DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG ADMINISTRATION SCHEDULE; HEMATOLOGIC NEOPLASMS; HUMANS; KAPLAN-MEIER ESTIMATE; LOGISTIC MODELS; MAXIMUM TOLERATED DOSE; MOLECULAR TARGETED THERAPY; NEOPLASMS; ODDS RATIO; PATIENT SELECTION; PROPORTIONAL HAZARDS MODELS; REMISSION INDUCTION; RESEARCH DESIGN; RETROSPECTIVE STUDIES;

EID: 84871833722     PISSN: 00278874     EISSN: 14602105     Source Type: Journal    
DOI: 10.1093/jnci/djs439     Document Type: Review

References (21)

1. Korn EL. Nontoxicity endpoints in phase I trial designs for targeted, noncytotoxic agents. J Natl Cancer Inst. 2004;96(13):977-978.
2. Parulekar WR, Eisenhauer EA. Phase I trial design for solid tumor studies of targeted, non-cytotoxic agents: theory and practice. J Natl Cancer Inst. 2004;96(13):990-997.
3. Von Hoff DD, Turner J. Response rates, duration of response, and dose response effects in phase I studies of antineoplastics. Invest New Drugs. 1991;9(1):115-122.
4. Postel-Vinay S, Arkenau HT, Olmos D, et al. Clinical benefit in Phase-I trials of novel molecularly targeted agents: does dose matter? Br J Cancer. 2009;100(9):1373-1378.
5. Pratt WB, Ruddon RW, Ensminger WD, Maybaum J. The anticancer drugs. 2nd Edition ed. USA: Oxford University Press; 1994.
6. Jain RK, Lee JJ, Hong D, et al. Phase I oncology studies: evidence that in the era of targeted therapies patients on lower doses do not fare worse. Clin Cancer Res. 16(4):1289-1297.
7. Sleijfer S, Wiemer E. Dose selection in phase I studies: why we should always go for the top. J Clin Oncol. 2008;26(10):1576-1578.
8. Haines IE. Dose selection in phase I studies: why we should always go for the most effective. J Clin Oncol. 2008;26(21):3650-3652; author reply 3652-3653.
9. Cannistra SA. Challenges and pitfalls of combining targeted agents in phase I studies. J Clin Oncol. 2008;26(22):3665-3667.
10. Korn EL, Arbuck SG, Pluda JM, et al. Clinical trial designs for cytostatic agents: are new approaches needed? J Clin Oncol. 2001;19(1):265-272.
11. Alam SM, Fehrmann RS, Olmos D, et al. Defining the risk of toxicity in phase I oncology trials of novel molecularly targeted agents: A singlecenter experience. J Clin Oncol. 2010;28(15s):abstr 2519.
12. Booth CM, Calvert AH, Giaccone G, et al. Endpoints and other considerations in phase I studies of targeted anticancer therapy: recommendations from the task force on Methodology for the Development of Innovative Cancer Therapies (MDICT). Eur J Cancer. 2008;44(1):19-24.
13. Wolf M, Swaisland H, Averbuch S. Development of the novel biologically targeted anticancer agent gefitinib: determining the optimum dose for clinical efficacy. Clin Cancer Res. 2004;10(14):4607-4613.
14. Fox E, Curt GA, Balis FM. Clinical trial design for target-based therapy. Oncologist. 2002;7(5):401-409.
15. Sleijfer S, Wiemer E. In Reply. Journal of Clinical Oncology. 2008;26(21):3652-3653.
16. Blanke CD, Rankin C, Demetri GD, et al. Phase III randomized, intergroup trial assessing imatinib mesylate at two dose levels in patients with unresectable or metastatic gastrointestinal stromal tumors expressing the kit receptor tyrosine kinase: S0033. J Clin Oncol. 2008;26(4):626-632.
17. Verweij J, Casali PG, Zalcberg J, et al. Progression-free survival in gastrointestinal stromal tumours with high-dose imatinib: randomised trial. Lancet. 2004;364(9440):1127-1134.
18. Agrawal M, Emanuel EJ. Ethics of phase 1 oncology studies: reexamining the arguments and data. JAMA. 2003;290(8):1075-1082.
19. Miller AB, Hoogstraten B, Staquet M, Winkler A. Reporting results of cancer treatment. Cancer. 1981;47(1):207-214.
20. Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. J Natl Cancer Inst. 2000;92(3):205-216.
21. Kalbfleisch JD, Prentice RL. The statistical analysis of failure time data. New York, NY: John Wiley and Sons, Inc; 1980.