Randomized reverse marker strategy design for prospective biomarker validation

Statistics in Medicine

Volumn 33, Issue 18, 2014, Pages 3089-3099

  Eng, Kevin H ^a  

^a ROSWELL PARK CANCER INSTITUTE   (United States)

Author keywords

Biomarker validation; Interaction; Ovarian cancer; Randomized trial; Trial design

Indexed keywords

BIOLOGICAL MARKER; PACLITAXEL; PLATINUM; TUMOR MARKER;

ARTICLE; CANCER RECURRENCE; CLINICAL DECISION MAKING; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; HUMAN; MAJOR CLINICAL STUDY; OVARY CANCER; PARAMETRIC TEST; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PROSPECTIVE STUDY; REVERSE MARKER STRATEGY DESIGN; SAMPLE SIZE; SINGLE BLIND PROCEDURE; STATISTICAL BIAS; STUDY DESIGN; VALIDATION STUDY; BIOSTATISTICS; FEMALE; OVARIAN NEOPLASMS; PROBABILITY THEORY; RANDOMIZED CONTROLLED TRIAL (TOPIC); STATISTICS AND NUMERICAL DATA; TUMOR RECURRENCE;

BIOMARKERS, TUMOR; BIOSTATISTICS; FEMALE; HUMANS; NEOPLASM RECURRENCE, LOCAL; OVARIAN NEOPLASMS; PROBABILITY THEORY; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLE SIZE; VALIDATION STUDIES AS TOPIC;

EID: 84903775882     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.6146     Document Type: Article

References (18)

1. Vaughan S, Coward J, Bast R, Berchuck A, Berek J, Brenton J, Coukos G, Crum C, Drapkin R, Etemadmoghadam D, Friedlander M, Gabra H, Kaye SB, Lord CJ, Lengyel E, Levine DA, McNeish IA, Menon U, Mills GB, Nephew KP, Oza AM, Sood AK, Stronach EA, Walczak H, Bowtell DD, Balkwill FR. Rethinking ovarian cancer: recommendations for improving outcomes. Nature Reviews Cancer 2011; 11(10):719-725.
2. Thatcher N, Chang A, Parikh P, RodriguesPereira J, Ciuleanu T, von Pawel J, Thongprasert S, Tan E, Pemberton K, Archer V, Carroll K. Gefitinib plus best supportive care in previously treated patients with refractory advanced non-small-cell lung cancer: results from a randomised, placebo-controlled, multicentre study (iressa survival evaluation in lung cancer). The Lancet 2005; 366(9496):1527-1537.
3. Ludwig JA, Weinstein JN. Biomarkers in cancer staging, prognosis and treatment selection. Nature Reviews Cancer 2005; 5(11):845-856.
4. Sargent D, Conley B, Allegra C, Collette L. Clinical trial designs for predictive marker validation in cancer treatment trials. Journal of Clinical Oncology 2005; 23(9):2020-2027.
5. Tang L, Zhou X. A general framework of marker design with optimal allocation to assess clinical utility. Statistics in Medicine 2013; 32(4):620-630.
6. Pepe M, Feng Z, Janes H, Bossuyt P, Potter J. Pivotal evaluation of the accuracy of a biomarker used for classification or prediction: standards for study design. Journal of the National Cancer Institute 2008; 100(20):1432-1438.
7. Mandrekar S, Sargent D. Clinical trial designs for predictive biomarker validation: theoretical considerations and practical challenges. Journal of Clinical Oncology 2009; 27(24):4027-4034.
8. Maitournam A, Simon R. On the efficiency of targeted clinical trials. Statistics in Medicine 2005; 24(3):329-339.
9. Freidlin B, McShane L, Korn E. Randomized clinical trials with biomarkers: design issues. Journal of the National Cancer Institute 2010; 102(3):152-160.
10. Mandrekar S, Sargent D. Clinical trial designs for predictive biomarker validation: one size does not fit all. Journal of Biopharmaceutical Statistics 2009; 19(3):530-542.
11. Schoenfeld D. Sample-size formula for the proportional-hazards regression model. Biometrics 1983; 39(2):499-503.
12. Mandrekar S, Grothey A, Goetz M, Sargent D. Clinical trial designs for prospective validation of biomarkers. American Journal of Pharmacogenomics 2005; 5(5):317-325.
13. Cooke S, Brenton J. Evolution of platinum resistance in high-grade serous ovarian cancer. The Lancet Oncology 2011; 12(12):1169-1174.
14. Bookman M. Standard treatment in advanced ovarian cancer in 2005: the state of the art. International Journal of Gynecological Cancer 2005; 15: 212-220.
15. Bhoola S, Hoskins W. Diagnosis and management of epithelial ovarian cancer. Obstetrics & Gynecology 2006; 107(6):1399-1410.
16. Na Y, Farley J, Zeh A, del Carmen M, Penson R, Birrer M. Ovarian cancer: markers of response. International Journal of Gynecological Cancer 2009; 19(11):S21-S29.
17. Ushijima K. Treatment for recurrent ovarian cancer - at first relapse. Journal of Oncology 2010; 2010: ID497429.
18. O'Malley D, Azodi M, Makkenchery A, Tangir J, McAlpine J, Kelly M, Schwartz P, Rutherford T. Weekly topotecan in heavily pretreated patients with recurrent epithelial ovarian carcinoma. Gynecologic Oncology 2005; 98(2):242-249.