Dose-toxicity models in oncology

Expert Opinion on Drug Metabolism and Toxicology

Volumn 7, Issue 2, 2011, Pages 201-211

  Adamina, Michel ^a   Joerger, Markus ^a  

^a CANTONAL HOSPITAL ST GALLEN   (Switzerland)

Author keywords

Bayesian statistics; continual reassessment method; dose finding studies; dose toxicity model; estimation with overdose control; molecularly targeted agents; oncology; Phase I clinical trial; therapeutic drug monitoring

Indexed keywords

ANTINEOPLASTIC AGENT; CISPLATIN; ETOPOSIDE; GEMCITABINE; MELPHALAN; METHOTREXATE; NAVELBINE; NEW DRUG; PACLITAXEL; SURAMIN; TENIPOSIDE; TOPOTECAN;

AREA UNDER THE CURVE; BAYES THEOREM; BAYESIAN LEARNING; CANCER COMBINATION CHEMOTHERAPY; CANCER RADIOTHERAPY; CHILDHOOD CANCER; CLINICAL TRIAL (TOPIC); DRUG ABSORPTION; DRUG DISTRIBUTION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG ELIMINATION; DRUG MEGADOSE; DRUG METABOLISM; DRUG MONITORING; DRUG SAFETY; DRUG SURVEILLANCE PROGRAM; HUMAN; LUNG NON SMALL CELL CANCER; MAXIMUM TOLERATED DOSE; MOLECULARLY TARGETED THERAPY; MULTIPLE CYCLE TREATMENT; NEUTROPENIA; NONHUMAN; ONCOLOGY; PANCREAS CANCER; PATIENT SELECTION; PHARMACODYNAMICS; PROBABILITY; REVIEW;

ANTINEOPLASTIC AGENTS; BAYES THEOREM; CLINICAL TRIALS AS TOPIC; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUGS, INVESTIGATIONAL; HUMANS; MODELS, BIOLOGICAL; MODELS, STATISTICAL; NEOPLASMS; RESEARCH DESIGN;

EID: 79952172623     PISSN: 17425255     EISSN: None     Source Type: Journal    
DOI: 10.1517/17425255.2011.543674     Document Type: Review

References (104)

1. E8 harmonised tripartite guideline. General considerations for clinical trials. International Conference on Harmonisation of technical requirements for registration of pharmaceuticals for human use. Available from: http://www.ich.org/LOB/media/MEDIA484.pdf [Last accessed 5 November 2010]
2. S6 preclinical safety evaluation of biotechnology-derived pharmaceuticals. International Conference on Harmonisation of technical requirements for registration of pharmaceuticals for human use. Available from: http://www.ich.org/LOB/media/MEDIA503.pdf [Last accessed 5 November 2010]
3. S7A safety pharmacology studies for human pharmaceuticals. International Conference on Harmonisation of technical requirements for registration of pharmaceuticals for human use. Available from: http://www.ich.org/LOB/media/ MEDIA504.pdf [Last accessed 5 November 2010]
4. M3 (R2) Guidance on nonclinical safety studies for the conduct of human clinical trials and marketing authorization for pharmaceuticals. International Conference on Harmonisation of technical requirements for registration of pharmaceuticals for human use. Available from: http://www.ich.org/LOB/media/ MEDIA5544.pdf [Last accessed 5 November 2010]
5. O'Quigley J, Zohar S. Experimental designs for phase I and phase I/II dose-finding studies. Br J Cancer 2006;94:609-13 (Pubitemid 43361888)
6. Kola I, Landis J. Can the pharmaceutical industry reduce attrition rates? Nat Rev Drug Discov 2004;3:711-15
7. Laska DA, Houchins JO, Pratt SE, et al. Characterization and application of a vinblastine-selected CACO-2 cell line for evaluation of p-glycoprotein. In Vitro Cell Dev Biol Anim 2002;38:401-10 (Pubitemid 36150677)
8. Yuan R, Parmelee T, Balian JD, et al. In vitro metabolic interaction studies: experience of the Food and Drug Administration. Clin PharmacolTher 1999;66:9-15
9. Frank R, Hargreaves R. Clinical biomarkers in drug discovery and development. Nat Rev Drug Discov 2003;2:566-80 (Pubitemid 37361748)
10. Johnson JI, Decker S, Zaharevitz D, et al. Relationships between drug activity in NCI preclinical in vitro and in vivo models and early clinical trials. Br J Cancer 2001;84:1424-31 (Pubitemid 32522115)
11. Mandrekar SJ, Cui Y, Sargent DJ. An adaptive phase I design for identifying a biologically optimal dose for dual agent drug combinations. Stat Med 2007;26:2317-30 (Pubitemid 46730251)
12. Suntharalingam G, Perry MR, Ward S, et al. Cytokine storm in a phase 1 trial of the anti-CD28 monoclonal antibody TGN1412. N Engl J Med 2006;355:1018-28 (Pubitemid 44343545)
13. Kinders R, Parchment RE, Ji J, et al. Phase 0 clinical trials in cancer drug development: from FDA guidance to clinical practice. Mol Interv 2007;7:325-34
14. The Lancet. Phase 0 trials: a platform for drug development? Lancet 2009;374:176
15. Guidance for Industry, Investigators, and Reviewers: Exploratory IND Studies. U.S. Department of Health and Human Services and Center for Drug Evaluation and Research, Food and Drug Administration, Available from: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/ Guidances/ucm078933.pdf [Last accessed 5 November 2010]
16. Ivy SP, Siu LL, Garrett-Mayer E, Rubinstein L. Approaches to phase 1 clinical trial design focused on safety, efficiency, and selected patient populations: a report from the clinical trial design task force of the national cancer institute investigational drug steering committee. Clin Cancer Res 2010;16:1726-36
17. Rosenberger WF, Haines LM. Competing designs for phase I clinical trials: a review. Stat Med 2002;21:2757-70
18. Le TC, Lee JJ, Siu LL. Dose escalation methods in phase I cancer clinical trials. J Natl Cancer Inst 2009;101:708-20
19. 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;100:1373-8
20. Von Hoff DD, Turner J. Response rates, duration of response, and dose response effects in phase I studies of antineoplastics. Invest New Drugs 1991;9:115-22
21. Rogatko A, Schoeneck D, Jonas W, et al. Translation of innovative designs into phase I trials. J Clin Oncol 2007;25:4982-6 (Pubitemid 350220431)
22. O'Quigley J, Chevret S. Methods for dose finding studies in cancer clinical trials: a review and results of a Monte Carlo study. Stat Med 1991;10:1647-64
23. Storer BE. Design and analysis of phase I clinical trials. Biometrics 1989;45:925-37 (Pubitemid 19249331)
24. Kwak EL, Camidge DR, Clark J, et al. Clinical activity observed in a phase I dose escalation trial of an oral c-met and ALK inhibitor, PF-02341066. J Clin Oncol 2009;27:abstract 3509
25. Flaherty K, Puzanov I, Sosman JKK, et al. Phase I study of PLX4032: proof of concept for V600E BRAF mutation as a therapeutic target in human cancer. J Clin Oncol 2009;27:abstract 9000
26. Sheiner LB, Beal SL, Sambol NC. Study designs for dose-ranging. Clin Pharmacol Ther 1989;46:63-77 (Pubitemid 19195776)
27. Sheiner LB. Implications of an alternative approach to dose-response trials. J Acquir Immune Defic Syndr 1990;3(Suppl 2):S20-6
28. Simon R, Freidlin B, Rubinstein L, et al. Accelerated titration designs for phase I clinical trials in oncology. J Natl Cancer Inst 1997;89:1138-47 (Pubitemid 27337928)
29. Dancey J, Freidlin B, Rubinstein L. Accelerated titration designs. In: Statistical methods for dose-finding experiments. Wiley, Chichester; 2006. p. 91-114
30. Mross K, Stefanic M, Gmehling D, et al. Phase I study of the angiogenesis inhibitor BIBF 1120 in patients with advanced solid tumors. Clin Cancer Res 2010;16:311-19
31. Aghajanian C, Burris HA III, Jones S, et al. Phase I study of the novel epothilone analog ixabepilone (BMS-247550) in patients with advanced solid tumors and lymphomas. J Clin Oncol 2007;25:1082-8 (Pubitemid 46596760)
32. Horstmann E, McCabe MS, Grochow L, et al. Risks and benefits of phase 1 oncology trials, 1991 through 2002. N Engl J Med 2005;352:895-904
33. Kurzrock R, Benjamin RS. Risks and benefits of phase 1 oncology trials, revisited. N Engl J Med 2005;352:930-2 (Pubitemid 40300304)
34. Decoster G, Stein G, Holdener EE. Responses and toxic deaths in phase I clinical trials. Ann Oncol 1990;1:175-81 (Pubitemid 20218532)
35. Babb JS, Rogatko A. Bayesian methods for cancer phase I clinical trials. In: Advances in Clinical Trial Biostatistics. Marcel Dekker, New York; 2004
36. Collins JM, Grieshaber CK, Chabner BA. Pharmacologically guided phase I clinical trials based upon preclinical drug development. J Natl Cancer Inst 1990;82:1321-6 (Pubitemid 20238486)
37. Collins JM, Zaharko DS, Dedrick RL, Chabner BA. Potential roles for preclinical pharmacology in phase I clinical trials. Cancer Treat Rep 1986;70:73-80 (Pubitemid 16140371)
38. Biomarkers Definitions Working Group. Biomarkers and surrogate endpoints: Preferred definitions and conceptual framework. Clin Pharmacol Ther 2001;69:89-95
39. Goulart BH, Clark JW, Pien HH, et al. Trends in the use and role of biomarkers in phase I oncology trials. Clin Cancer Res 2007;13:6719-26 (Pubitemid 350206809)
40. Collins JM. Phase 0 clinical studies in oncology. Clin Pharmacol Ther 2009;85:204-7
41. Bayes T. An essay towards solving a problem in the doctrine of chances. Philos Trans R Soc Lond 1763;53:370-418
42. Berry DA. Introduction to Bayesian methods III: use and interpretation of Bayesian tools in design and analysis. Clin.Trials 2005;2:295-300 (Pubitemid 41306270)
43. Berry DA, Cronin KA, Plevritis SK, et al. Effect of screening and adjuvant therapy on mortality from breast cancer. N Engl J Med 2005;353:1784-92 (Pubitemid 41549890)
44. Cheng JD, Babb JS, Langer C, et al. Individualized patient dosing in phase I clinical trials: the role of escalation with overdose control in PNU-214936. J Clin Oncol 2004;22:602-9 (Pubitemid 41095062)
45. Gallo JM, Laub PB, Rowinsky EK, et al. Population pharmacokinetic model for topotecan derived from phase I clinical trials. J Clin Oncol 2000;18:2459-67 (Pubitemid 30415833)
46. Maki RG, Wathen JK, Patel SR, et al. Randomized phase II study of gemcitabine and docetaxel compared with gemcitabine alone in patients with metastatic soft tissue sarcomas: results of sarcoma alliance for research through collaboration study 002. J Clin Oncol 2007;25:2755-63 (Pubitemid 47123184)
47. Saunders M, Dische S, Barrett A, et al. Continuous hyperfractionated accelerated radiotherapy (CHART) versus conventional radiotherapy in non-small-cell lung cancer: a randomised multicentre trial. CHART Steering Committee. Lancet 1997;350:161-5
48. Dische S, Saunders M, Barrett A, et al. A randomised multicentre trial of CHART versus conventional radiotherapy in head and neck cancer. Radiother Oncol 1997;44:123-36
49. Saunders M, Dische S, Barrett A, et al. Continuous, hyperfractionated, accelerated radiotherapy (CHART) versus conventional radiotherapy in non-small cell lung cancer: mature data from the randomised multicentre trial. CHART Steering committee. Radiother Oncol 1999;52:137-48
50. Adamina M, Tomlinson G, Guller U. Bayesian statistics in oncology: a guide for the clinical investigator. Cancer 2009;115:5371-81
51. Spiegelhalter DJ, Abrams KR, Myles JP. Bayesian approaches to clinical trials and health care evaluation. Wiley, New York; 2004
52. Kadane JB. Prime time for Bayes. Control Clin Trials 1995;16:313-18
53. Goodman SN. Toward evidence-based medical statistics. 1: the P value fallacy. Ann Intern Med 1999;130:995-1004
54. Goodman SN. Introduction to Bayesian methods I: measuring the strength of evidence. Clin Trials 2005;2:282-90 (Pubitemid 41306268)
55. Sung L, Hayden J, Greenberg ML, et al. Seven items were identified for inclusion when reporting a Bayesian analysis of a clinical study. J Clin Epidemiol 2005;58:261-8 (Pubitemid 40249468)
56. Austin PC, Brunner LJ, Hux JE. Bayeswatch: an overview of Bayesian statistics. J Eval Clin Pract 2002;8:277-86 (Pubitemid 34685904)
57. Huang X, Biswas S, Oki Y, et al. A parallel phase I/II clinical trial design for combination therapies. Biometrics 2007;63:429-36
58. Yin G, Li Y, Ji Y. Bayesian dose-finding in phase I/II clinical trials using toxicity and efficacy odds ratios. Biometrics 2006;62:777-84
59. Vey N, Kantarjian H, Beran M, et al. Combination of topotecan with cytarabine or etoposide in patients with refractory or relapsed acute myeloid leukemia: results of a randomized phase I/II study. Invest New Drugs 1999;17:89-95 (Pubitemid 29452752)
60. Rogatko A, Babb JS, Tighiouart M, et al. New paradigm in dose-finding trials: patient-specific dosing and beyond phase I. Clin Cancer Res 2005;11:5342-6 (Pubitemid 41060806)
61. Whitehead J, Zhou Y, Stevens J, et al. Bayesian decision procedures for dose-escalation based on evidence of undesirable events and therapeutic benefit. Stat Med 2006;25:37-53 (Pubitemid 43136948)
62. Loke YC, Tan SB, Cai Y, Machin D. A Bayesian dose finding design for dual endpoint phase I trials. Stat Med 2006;25:3-22 (Pubitemid 43136946)
63. Hamberg P, Ratain MJ, Lesaffre E, Verweij J. Dose-escalation models for combination phase I trials in oncology. Eur J Cancer 2010;46:2870-8
64. DeMets DL. Practical aspects in data monitoring: a brief review. Stat Med 1987;6:753-60
65. O'Quigley J, Pepe M, Fisher L. Continual reassessment method: a practical design for phase 1 clinical trials in cancer. Biometrics 1990;46:33-48 (Pubitemid 20245604)
66. O'Quigley J. Estimating the probability of toxicity at the recommended dose following a phase I clinical trial in cancer. Biometrics 1992;48:853-62
67. O'Quigley J, Shen LZ. Continual reassessment method: a likelihood approach. Biometrics 1996;52:673-84 (Pubitemid 26187071)
68. Goodman SN, Zahurak ML, Piantadosi S. Some practical improvements in the continual reassessment method for phase I studies. Stat Med 1995;14:1149-61
69. O'Quigley J, Paoletti X. Continual reassessment method for ordered groups. Biometrics 2003;59:430-40 (Pubitemid 36750357)
70. Faries D. Practical modifications of the continual reassessment method for phase I cancer clinical trials. J Biopharm Stat 1994;4:147-64 (Pubitemid 24248939)
71. Potter DM. Adaptive dose finding for phase I clinical trials of drugs used for chemotherapy of cancer. Stat Med 2002;21:1805-23 (Pubitemid 34732642)
72. Siu LL, Rowinsky EK, Hammond LA, et al. A phase I and pharmacokinetic study of SAM486A, a novel polyamine biosynthesis inhibitor, administered on a daily-times-five every-three-week schedule in patients with Advanced solid malignancies. Clin Cancer Res 2002;8:2157-66 (Pubitemid 34753586)
73. Tighiouart M, Rogatko A, Babb JS. Flexible Bayesian methods for cancer phase I clinical trials. Dose escalation with overdose control. Stat Med 2005;24:2183-96
74. Babb J, Rogatko A, Zacks S. Cancer phase I clinical trials: efficient dose escalation with overdose control. Stat Med 1998;17:1103-20 (Pubitemid 28221028)
75. Babb JS, Rogatko A. Patient specific dosing in a cancer phase I clinical trial. Stat Med 2001;20:2079-90 (Pubitemid 32684630)
76. Normolle D, Lawrence T. Designing dose-escalation trials with late-onset toxicities using the time-to-event continual reassessment method. J Clin Oncol 2006;24:4426-33 (Pubitemid 46630980)
77. Cheung YK, Chappell R. Sequential designs for phase I clinical trials with late-onset toxicities. Biometrics 2000;56:1177-82
78. Muler JH, McGinn CJ, Normolle D, et al. Phase I trial using a time-to-event continual reassessment strategy for dose escalation of cisplatin combined with gemcitabine and radiation therapy in pancreatic cancer. J Clin Oncol 2004;22:238-43 (Pubitemid 41095087)
79. Masson E, Zamboni WC. Pharmacokinetic optimisation of cancer chemotherapy. Effect on outcomes. Clin Pharmacokinet 1997;32:324-43
80. Freyer G, Ligneau B, Tranchand B, et al. Pharmacokinetic studies in cancer chemotherapy: usefulness in clinical practice. Cancer Treat Rev 1997;23:153-69 (Pubitemid 27356925)
81. Calvert AH, Newell DR, Gumbrell LA, et al. Carboplatin dosage: prospective evaluation of a simple formula based on renal function. J Clin Oncol 1989;7:1748-56 (Pubitemid 19272848)
82. Chatelut E, Canal P, Brunner V, et al. Prediction of carboplatin clearance from standard morphological and biological patient characteristics. J Natl Cancer Inst 1995;87:573-80
83. Mercier C, Ciccolini J, Pourroy B, et al. Dose individualization of carboplatin after a 120-hour infusion schedule: higher dose intensity but fewer toxicities. Ther Drug Monit 2006;28:212-18 (Pubitemid 44314951)
84. Rousseau A, Marquet P, Debord J, et al. Adaptive control methods for the dose individualisation of anticancer agents. Clin Pharmacokinet 2000;38:315-53 (Pubitemid 30229470)
85. Johansen MJ, Madden T, Mehra RC, et al. Phase I pharmacokinetic study of multicycle high-dose carboplatin followed by peripheral-blood stem-cell infusion in patients with cancer. J Clin Oncol 1997;15:1481-91 (Pubitemid 27167384)
86. Salas S, Mercier C, Ciccolini J, et al. Therapeutic drug monitoring for dose individualization of Cisplatin in testicular cancer patients based upon total platinum measurement in plasma. Ther Drug Monit 2006;28:532-9 (Pubitemid 44181487)
87. Jelliffe RW, Schumitzky A, Bayard D, et al. Model-based, goal-oriented, individualised drug therapy. Linkage of population modelling, new 'multiple model' dosage design, bayesian feedback and individualised target goals. Clin Pharmacokinet 1998;34:57-77
88. Perdaems N, Bachaud JM, Rouzaud P, et al. Relation between unbound plasma concentrations and toxicity in a prolonged oral etoposide schedule. Eur J Clin Pharmacol 1998;54:677-83 (Pubitemid 29024197)
89. Rodman JH, Furman WL, Sunderland M, et al. Escalating teniposide systemic exposure to increase dose intensity for pediatric cancer patients. J Clin Oncol 1993;11:287-93 (Pubitemid 23046819)
90. Rodman JH, Sunderland M, Kavanagh RL, et al. Pharmacokinetics of continuous infusion of methotrexate and teniposide in pediatric cancer patients. Cancer Res. 1990;50:4267-71 (Pubitemid 20225662)
91. Wong M, Balleine RL, Blair EY, et al. Predictors of vinorelbine pharmacokinetics and pharmacodynamics in patients with cancer. J Clin Oncol 2006;24:2448-55 (Pubitemid 46630621)
92. Gauvin A, Pinguet F, Culine S, et al. Bayesian estimate of vinorelbine pharmacokinetic parameters in elderly patients with advanced metastatic cancer. Clin Cancer Res 2000;6:2690-5 (Pubitemid 30482105)
93. Tranchand B, Amsellem C, Chatelut E, et al. A limited-sampling strategy for estimation of etoposide pharmacokinetics in cancer patients. Cancer Chemother Pharmacol 1999;43:316-22 (Pubitemid 29075889)
94. Sonnichsen DS, Ribeiro RC, Luo X, et al. Pharmacokinetics and pharmacodynamics of 21-day continuous oral etoposide in pediatric patients with solid tumors. Clin Pharmacol Ther 1995;58:99-107
95. Rousseau A, Sabot C, Delepine N, et al. Bayesian estimation of methotrexate pharmacokinetic parameters and area under the curve in children and young adults with localised osteosarcoma. Clin Pharmacokinet 2002;41:1095-104 (Pubitemid 35246783)
96. Evans WE, Relling MV, Rodman JH, et al. Conventional compared with individualized chemotherapy for childhood acute lymphoblastic leukemia. N Engl J Med 1998;338:499-505 (Pubitemid 28103133)
97. Pignon T, Lacarelle B, Duffaud F, et al. Pharmacokinetics of high-dose methotrexate in adult osteogenic sarcoma. Cancer Chemother Pharmacol 1994;33:420-4 (Pubitemid 24057695)
98. Pignon T, Lacarelle B, Duffaud F, et al. Dosage adjustment of high-dose methotrexate using Bayesian estimation: a comparative study of two different concentrations at the end of 8-h infusions. Ther Drug Monit 1995;17:471-8
99. Furman WL, Baker SD, Pratt CB, et al. Escalating systemic exposure of continuous infusion topotecan in children with recurrent acute leukemia. J Clin Oncol 1996;14:1504-11 (Pubitemid 26134206)
100. Woo MH, Relling MV, Sonnichsen DS, et al. Phase I targeted systemic exposure study of paclitaxel in children with refractory acute leukemias. Clin Cancer Res 1999;5:543-9 (Pubitemid 29131963)
101. Pinguet F, Culine S, Bressolle F, et al. A phase I and pharmacokinetic study of melphalan using a 24-hour continuous infusion in patients with advanced malignancies. Clin Cancer Res 2000;6:57-63 (Pubitemid 30064976)
102. Eisenberger MA, Sinibaldi VJ, Reyno LM, et al. Phase I and clinical evaluation of a pharmacologically guided regimen of suramin in patients with hormone-refractory prostate cancer. J Clin Oncol 1995;13:2174-86
103. Falcone A, Antonuzzo A, Danesi R, et al. Suramin in combination with weekly epirubicin for patients with advanced hormone-refractory prostate carcinoma. Cancer 1999;86:470-6 (Pubitemid 29347409)
104. Rousseau A, Marquet P. Application of pharmacokinetic modelling to the routine therapeutic drug monitoring of anticancer drugs. Fundam Clin Pharmacol 2002;16:253-62 (Pubitemid 35205335)