Statistical challenges in the evaluation of surrogate endpoints in randomized trials

Controlled Clinical Trials

Volumn 23, Issue 6, 2002, Pages 607-625

  Molenberghs, Geert ^a   Buyse, Marc ^b   Geys, Helena ^a   Renard, Didier ^a   Burzykowski, Tomasz ^a   Alonso, Ariel ^a  

^a HASSELT UNIVERSITY   (Belgium)

^b INTERNATIONAL DRUG DEVELOPMENT INSTITUTE   (Belgium)

Author keywords

Adjusted association; Meta analysis; Proportion explained; Random effects model; Relative effect; Surrogate endpoint; Validation

Indexed keywords

ALPHA INTERFERON; BIOLOGICAL MARKER; CISPLATIN; CYCLOPHOSPHAMIDE; DOXORUBICIN; FLUOROURACIL; FOLINIC ACID; INTERFERON; RISPERIDONE;

ARTICLE; CASE STUDY; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; DATA ANALYSIS; EVALUATION; HUMAN; RANDOMIZED CONTROLLED TRIAL; STATISTICAL ANALYSIS; VALIDATION PROCESS;

ENDPOINT DETERMINATION; HUMANS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS; REPRODUCIBILITY OF RESULTS;

EID: 0036916785     PISSN: 01972456     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0197-2456(02)00236-2     Document Type: Article

References (30)

1. Ellenberg S.S., Hamilton J.M. Surrogate endpoints in clinical trials. cancer Stat Med. 8:1989;405-413.
2. Prentice R.L. Surrogate endpoints in clinical trials. definitions and operational criteria Stat Med. 8:1989;431-440.
3. Fleming T.R., Prentice R.L., Pepe M.S., Glidden D. Surrogate and auxiliary endpoints in clinical trials, with potential applications in cancer and AIDS research. Stat Med. 13:1994;955-968.
4. Buyse M., Molenberghs G. The validation of surrogate endpoints in randomized experiments. Biometrics. 54:1998;1014-1029.
5. Freedman L.S., Graubard B.I., Schatzkin A. Statistical validation of intermediate endpoints for chronic diseases. Stat Med. 11:1992;167-178.
6. Albert J.M., Ioannidis J.P.A., Reichelderfer P.et al. Statistical issues for HIV surrogate endpoints. point/counterpoint Stat Med. 17:1998;2435-2462.
7. Daniels M.J., Hughes M.D. Meta-analysis for the evaluation of potential surrogate markers. Stat Med. 16:1997;1515-1527.
8. Buyse M., Molenberghs G., Burzykowski T., Renard D., Geys H. The validation of surrogate endpoints in meta-analyses of randomized experiments. Biostatistics. 1:2000;49-67.
9. Gail M.H., Pfeiffer R., van Houwelingen H.C., Carroll R.J. On meta-analytic assessment of surrogate outcomes. Biostatistics. 1:2000;231-246.
10. Fleming T.R., DeMets D.L. Surrogate endpoints in clinical trials. are we being misled? Ann Intern Med. 125:1996;605-613.
11. Volberding P.A., Lagakos S.W., Koch M.A.et al. Zidovudine in asymptomatic human immunodeficiency virus infection. a controlled trial in persons with fewer than 500 CD4-positive cells per cubic millimeter N Engl J Med. 322:1990;941-949.
12. Choi S., Lagakos S., Schooley R.T., Volberding P.A. CD4+ lymphocytes are an incomplete surrogate marker for clinical progression in persons with asymptomatic HIV infection taking zidovudine. Ann Intern Med. 118:1993;674-680.
13. Lin D.Y., Fleming T.R., DeGruttola V. Estimating the proportion of treatment effect explained by a surrogate marker. Stat Med. 16:1997;1515-1527.
14. Flandre P., Saidi Y. Letters to the editor. estimating the proportion of treatment effect explained by a surrogate marker Stat Med. 18:1999;107-115.
15. Begg C.B., Leung D.H.Y. On the use of surrogate endpoints in randomized trials (with discussion). J R Stat Soc A. 163:2000;15-28.
16. Cook R.D., Weisberg S. Residuals and influence in regression. 1982;Chapman & Hall, London.
17. Chatterjee S., Hadi A.S. Sensitivity analysis in linear regression. 1988;John Wiley & Sons, New York.
18. Burzykowski T., Molenberghs G., Buyse M., Geys H., Renard D. Validation of surrogate endpoints in multiple randomized clinical trials with failure-time endpoints. Appl Stat. 50:2001;405-422.
19. Day N.E., Duffy S.W. Trial design based on surrogate end points-application to comparison of different breast screening frequencies. J R Stat Soc A. 159:1996;49-60.
20. Pharmacological Therapy for Macular Degeneration Study Group Interferon a-IIA is ineffective for patients with choroidal neovascularization secondary to age-related macular degeneration. Results of a prospective randomized placebo-controlled clinical trial. Arch Ophthalmol. 115:1997;865-872.
21. Ovarian Cancer Meta-Analysis Project Cyclophosphamide plus cisplatin versus cyclophosphamide, doxorubicin, and cisplatin chemotherapy of ovarian carcinoma. a meta-analysis J Clin Oncol. 9:1991;1668-1674.
22. Ovarian Cancer Meta-Analysis Project Cyclophosphamide plus cisplatin versus cyclophosphamide, doxorubicin, and cisplatin chemotherapy of ovarian carcinoma. a meta-analysis Classic Papers and Current Comments. 3:1998;237-243.
23. Corfu-A Study Group Phase III randomized study of two fluorouracil combinations with either interferon alfa-2a or leucovorin for advanced colorectal cancer. J Clin Oncol. 13:1995;921-928.
24. Greco F.A., Figlin R., York M.et al. Phase III randomized study to compare interferon alfa-2a in combination with fluorouracil versus fluorouracil alone in patients with advanced colorectal cancer. J Clin Oncol. 4:1996;2674-2681.
25. Kay S.R., Opler L.A., Lindenmayer J.P. Reliability and validity of the positive and negative syndrome scale for schizophrenics. Psychiatry Res. 23:1988;99-110.
26. Nair NPV and the Risperidone Study Group. Therapeutic equivalence of risperidone given once daily and twice daily in patients with schizophrenia. J Clin Psychopharmacol 1998;18:103-110.
27. Jacobson M.A., Bacchetti P., Kolokathis A. Surrogate markers for survival in patients with AIDS and AIDS related complex treated with zidovudine. BMJ. 302:1991;73-78.
28. O'Brien W.A., Hartigan P.M., Martin D.et al. Changes in plasma HIV-1 RNA and CD4+ lymphocyte counts and the risk of progression to AIDS. N Engl J Med. 334:1996;426-431.
29. Dale J.R. Global cross ratio models for bivariate, discrete, ordered responses. Biometrics. 42:1986;909-917.
30. Renard D., Geys H., Molenberghs G., Burzykowski T., Buyse M. Validation of surrogate endpoints in multiple randomized clinical trials with discrete outcomes. Biometrical J. 44:2002;1-15.