Estimating the proportion of treatment effect explained by a surrogate marker

Statistics in Medicine

Volumn 16, Issue 13, 1997, Pages 1515-1527

  Lin, D Y ^a   Fleming, T R ^a   De Gruttola, V ^b  

^a UNIVERSITY OF WASHINGTON   (United States)

^b HARVARD SCHOOL OF PUBLIC HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL MARKER;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ARTICLE; CLINICAL TRIAL; DISEASE MARKER; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; REGRESSION ANALYSIS; STATISTICAL MODEL; TREATMENT INDICATION;

ACQUIRED IMMUNODEFICIENCY SYNDROME; ANTI-HIV AGENTS; BIOLOGICAL MARKERS; DOUBLE-BLIND METHOD; FOLLOW-UP STUDIES; HIV SEROPOSITIVITY; HUMANS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS; REGRESSION ANALYSIS; TREATMENT OUTCOME; ZIDOVUDINE;

EID: 0030771191     PISSN: 02776715     EISSN: None     Source Type: Journal    
DOI: 10.1002/(SICI)1097-0258(19970715)16:13<1515::AID-SIM572>3.0.CO;2-1     Document Type: Article

References (18)

1. Prentice, R. L. 'Surrogate endpoints in clinical trials: definition and operational criteria', Statistics in Medicine, 8, 431-440 (1989).
2. + lymphocytes are an incomplete surrogate marker for clinical progression in persons with asymptomatic HIV infection taking zidovudine', Annals of Internal Medicine, 118, 674-680 (1993).
3. O'Brien, W. A., Hartigan, P. M., Martin, D., Esinhart, J., Hill, A., Benoit, S., Rubin, M., Simberkoff, M. S., Hamilton, J. D., and the Veterans Affairs Cooperative Study Group on AIDS. 'Changes in plasma HIV-1 RNA and CD4+ lymphocyte counts and the risk of progression to AIDS', New England Journal of Medicine, 334, 426-431 (1996).
4. Freedman, L. S., Graubard, B. I. and Schatzkin, A. 'Statistical validation of intermediate endpoints for chronic diseases', Statistics in Medicine, 11, 167-178 (1992).
5. Struthers, C. A. and Kalbfleisch, J. D. 'Misspecified proportional hazard models', Biometrika, 73, 363-369 (1986).
6. Lin, D. Y. and Wei, L. J. 'The robust inference for the Cox proportional hazards model', Journal of the American Statistical Association, 84, 1074-1078 (1989).
7. Hjort, N. L. 'On inference in parametric survival data models', International Statistical Review, 60, 355-387 (1992).
8. Wei, L. J., Lin, D. Y. and Weissfeld, L. 'Regression analysis of multivariate incomplete failure time data by modeling marginal distributions', Journal of the American Statistical Association, 84, 1065-1073 (1989).
9. Lin, D. Y. 'MULCOX: a computer program for the Cox regression analysis of multiple failure time variables', Computer Methods and Programs in Biomedicine, 32, 125-135 (1990).
10. Lin, D. Y. 'MULCOX2: a general computer program for the Cox regression analysis of multivariate failure time data', Computer Methods and Programs in Biomedicine, 40, 279-293 (1993).
11. Fieller, E. C. 'The biological standardization of insulin', Journal of the Royal Statistical Society, 7, Supplement 1-15 (1940).
12. Volberding, P. A., Lagakos, S. W., Koch, M. A. Pettinelli, C., Myers, M. W., Booth, D. K., Balfour H. H., Reichman, R. C., Bartlett, J. A., Hirsch, M. S., Murphy, R. L., Hardy, W. D., Soeiro, R., Fischl, M. A., Bartlett, J. G., Merigan, T. C., Hyslop, N. E., Richman, D. D., Valentine, F. T., Corey, L. and the AIDS Clinical Trials Group of the National Institute of Allergy and Infectious Diseases. 'Zidovudine in asymptomatic human immunodeficiency virus infection: a controlled trial in persons with fewer than 500 CD4-positive cells per cubic millimeter', New England Journal of Medicine, 322, 941-949 (1990).
13. Fischl, M. A., Richman, D. D., Grieco, M. H., Gottlieb, M. S., Volberding, P. A., Laskin, O. L., Leedom, J. M., Groopman, J. E., Mildvan, D., Schooley, R. T., Jackson, G. G., Durack, D. T., King, D. and the AZT Collaborative Working Group. 'The efficacy of azidothymidine (AZT) in the treatment of patients with AIDS and AIDS-related complex', New England Journal of Medicine, 317, 185-191 (1987).
14. De Gruttola, V., Wulfsohn, M., Fischl, M. A. and Tsiatis, A. A. 'Modeling the relationship between survival and CD4-lymphocytes in patients with AIDS and AIDS-related complex', Journal of AIDS, 6, 359-365 (1993).
15. Lin, D. Y., Fischl, M. A. and Schoenfeld, D. A. 'Evaluating the role of CD4-lymphocyte counts as surrogate endpoints in human immunodeficiency virus clinical trials', Statistics in Medicine, 12, 835-842 (1993).
16. Tsiatis, A. A., De Gruttola, V. and Wulfsohn, M. 'Modeling the relationship of survival to longitudinal data measured with error. Applications to survival and CD4 counts in patients with AIDS', Journal of the American Statistical Association, 90, 27-37 (1995).
17. De Gruttola, V., Fleming, T. R., Lin, D. Y. and Coombs, R. 'Validating surrogate markers - are we being naive?', Journal of Infectious Diseases, 175, 237-246 (1997).
18. White, H. 'Maximum likelihood estimation of misspecified models', Econometrica, 50, 1-25 (1982).