Evaluating a surrogate endpoint at three levels, with application to vaccine development

Statistics in Medicine

Volumn 27, Issue 23, 2008, Pages 4758-4778

  Gilbert, Peter B ^a   Qin, Li ^a   Self, Steven G ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

Author keywords

Clinical trial; Counterfactual; Immune correlate; Meta analysis; Potential outcomes; Principal surrogate; Statistical surrogate

Indexed keywords

BIOLOGICAL MARKER; HUMAN IMMUNODEFICIENCY VIRUS ANTIBODY; HUMAN IMMUNODEFICIENCY VIRUS VACCINE; INFLUENZA VACCINE; PLACEBO;

ANALYSIS OF COVARIANCE; ANTIBODY BLOOD LEVEL; ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CORRELATION ANALYSIS; DISEASE MARKER; DRUG EFFICACY; DRUG RESEARCH; DRUG RESPONSE; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IMMUNE RESPONSE; INFLUENZA; OUTCOME ASSESSMENT; PREDICTION; SIMULATION; STATISTICAL ANALYSIS; VALIDATION PROCESS;

AIDS VACCINES; ALGORITHMS; BIOLOGICAL MARKERS; CLINICAL TRIALS AS TOPIC; HUMANS; IMMUNOTHERAPY, ACTIVE; INFLUENZA VACCINES; META-ANALYSIS AS TOPIC; RESEARCH DESIGN; TREATMENT OUTCOME;

EID: 53849099349     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.3122     Document Type: Article

References (48)

1. Fauci AS, Haynes BF, Pantaleo G. Toward an understanding of the correlates of protective immunity to HIV infection. Science 1996; 271:324-328.
2. Clements-Mann ML. Lessons for AIDS vaccine development from non-AIDS vaccines. AIDS Research and Human Retroviruses 1998; 14(Suppl. 3):S197-S203.
3. Burton DR, Desrosiers RC, Doms RW, Koff WC, Kwong PD, Moore JP, Nabel GJ, Sodroski J, Wilson IA, Wyatt RT. HIV vaccine design and the neutralizing antibody problem. Nature Immunology 2004; 5:233-236.
4. Siber GR. Methods for estimating serological correlates of protection. Developments in Biological Standardization 1997; 89:283-296.
5. Chan ISF, Shu L, Matthews H, Chan C, Vessey R, Sadoff J, Heyse J. Use of statistical models for evaluating antibody response as a correlate of protection against varicella. Statistics in Medicine 2002; 21:3411-3430.
6. Dunning AJ. A model for immunological correlates of protection. Statistics in Medicine 2006; 25:1485-1497.
7. Burzykowski T, Molenberghs G, Buyse M. The Evaluation of Surrogate Endpoints. Springer: New York, 2005.
8. Qin L, Gilbert PB, Corey L, McElrath J, Self SG. A framework for assessing an immunological correlate of protection in vaccine trials. Journal of Infectious Diseases 2007; 196:1304-1312.
9. Szmuness W, Stevens CE, Zang EA, Harley EJ, Kellner A. A controlled clinical trial of the efficacy of the hepatitis B vaccine (Heptavax B): a final report. Hepatology 1981; 1:377-385.
10. Lanata CF, Black RE, del Aguila R et al. Protection of Peruvian children against rotavirus diarrhea of specific serotypes of one, two, or three doses of the RIT 4237 attenuated bovine rotavirus vaccine. Journal of Infectious Diseases 1989; 159:452-459.
11. Clemens JD, Sack DA, Harris JR et al. Field trial of oral cholera vaccines in Bangladesh: results from three-year follow-up. The Lancet 1990; 335:270-273.
12. Villa LL, Costa RL, Petta CA et al. Prophylactic quadrivalent human papillomavirus (types 6, 11, 16, and 18) L1 virus-like particle vaccine in young women: a randomised double-blind placebo-controlled multicentre phase II efficacy trial. The Lancet Oncology 2005; 6:271-278.
13. Mehrotra DV, Li X, Gilbert PB. A comparison of eight methods for the dual-endpoint evaluation of efficacy in a proof-of-concept HIV vaccine trial. Biometrics 2006; 62:893-900.
14. Buyse M, Molenberghs G. Criteria for the validation of surrogate endpoints in randomized experiments. Biometrics 1998; 54:1014-1029.
15. Huang Y, Pepe M, Feng Z. Evaluating the predictiveness of a continuous marker. 2007, DOI: 10.1111/j.1541-0420.2007.00878.x.
16. Fleming TR. Surrogate markers in AIDS and cancer trials. Statistics in Medicine 1994; 13:1423-1435.
17. DeMets DL, Fleming TR. Surrogate endpoints in clinical trials: are we being misled? Annals of Internal Medicine 1996; 125:605-613.
18. DeGruttola VG, Clax P, DeMets DL, Downing GJ, Ellenberg SS, Friedman L, Gail MH, Prentice R, Wittes J, Zeger SL. Considerations in the evaluation of surrogate endpoints in clinical trials. Controlled Clinical Trials 2002; 22:485-502.
19. Prentice RL. Surrogate endpoints in clinical trials: definition and operational criteria. Statistics in Medicine 1989; 8:431-440.
20. Holland P. Statistics and causal inference. Journal of the American Statistical Association 1986; 81:945-961.
21. Rubin DB. Causal inference using potential outcomes: design, modeling, decisions. Journal of the American Statistical Association 2005; 100:322-331.
22. Frangakis CE, Rubin DB. Principal stratification in causal inference. Biometrics 2002; 58:21-29.
23. Robins JM. An analytic method for randomized trials with informative censoring: Part I. Lifetime Data Analysis 1995; 1:241-254.
24. Rubin DB. Statistics and causal inference: which ifs have causal answers. Journal of the American Statistical Association 1986; 81:961-962.
25. Hudgens MG, Halloran ME. Causal vaccine effects on binary post-infection outcomes. Journal of the American Statistical Association 2006; 101:51-64.
26. Gilbert PB, Bosch RJ, Hudgens MG. Sensitivity analysis for the assessment of causal vaccine effects on viral load in HIV vaccine trials. Biometrics 2003; 59:531-541.
27. Gilbert PB, Hudgens MG. Evaluating causal effect predictiveness of candidate surrogate endpoints. 2006, submitted.
28. Kim HW, Canchola JG, Brandt CD, Pyles G, Chanock RM, Jensen K, Parrott RH. Respiratory synctial virus disease in infants despite prior administration of antigenic inactivated vaccine. American Journal of Epidemiology 1969; 89:405-421.
29. Kliks SC, Nisalak A, Brandt WE, Wahl L, Burke DS. Antibody-dependent enhancement of dengue virus growth in human monocytes as a risk factor for dengue hemorrhagic fever. American Journal of Tropical Medicine and Hygiene 1989; 40:444-451.
30. Hughes MD. Evaluating surrogate endpoints. Controlled Clinical Trials 2002; 23:703-707.
31. Molenberghs G, Buyse M, Geys H, Renard D, Burzykowski T, Alonso A. Statistical challenges in the evaluation of surrogate endpoints in randomized trials. Controlled Clinical Trials 2002; 23:607-625.
32. Robins JM, Hernan MA, Brumback B. Marginal structural models and causal inference in epidemiology. Epidemiology 2000; 11:550-560.
33. Follmann D. Augmented designs to assess immune response in vaccine trials. Biometrics 2006; 62:1161-1169.
34. Qin L, Gilbert PB, Follmann D, Li D. Assessing surrogate endpoints in vaccine trials with case-cohort sampling and the Cox model. Annals of Applied Statistics 2006, submitted.
35. Pearl J. Causality: Models, Reasoning, and Inference. Cambridge University Press: Cambridge, 2000.
36. Flynn NM, Forthal DN, Harro CD, Mayer KH. The rgp120 HIV Vaccine Study Group. Placebo-controlled trial of a recombinant glycoprotein 120 vaccine to prevent HIV infection. Journal of Infectious Diseases 2005; 191:654-665.
37. Prentice RL. A case-cohort design for epidemiologic cohort studies and disease prevention trials. Biometrika 1986; 73:1-11.
38. Borgan O, Langholz B, Samuelsen SO, Goldstein L, Pagoda J. Exposure stratified case-cohort designs. Lifetime Data Analysis 2000; 6:39-58.
39. Gilbert PB, Peterson ML, Follmann D, Hudgens MG, Francis DP, Gurwith M, Heyward WL, Jobes DV, Popovic V, Self SG, Sinangil F, Burke D, Berman PW. Correlation between immunologic responses to a recombinant glycoprotein 120 vaccine and incidence of HIV-1 infection in a Phase 3 HIV-1 preventive vaccine trial. Journal of Infectious Diseases 2005; 191:666-677.
40. Salk JE, Menke Jr WJ, Francis Jr T. A clinical, epidemiological and immunological evaluation of vaccination against epidemic influenza. American Journal of Hygiene 1943; 42:57-93.
41. Ennis FA, Yi-Hua Q, Schild GC. Antibody and cytotoxic T lymphocyte responses of humans to live and inactivated influenza vaccines. Journal of General Virology 1982; 58:273-281.
42. Clements ML, Tierney EL, Murphy BR. Response of seronegative and seropositive adult volunteers to live attenuated cold-adapted reassortant influenza A virus vaccine. Journal of Clinical Microbiology 1985; 21:997-999.
43. Gorse GJ, Belshe RB. Enhancement of anti-influenza A virus cytotoxicity following influenza A virus vaccination in older, chronically ill adults. Journal of Clinical Microbiology 1990; 28:2539-2550.
44. Treanor JJ, Roth FK, Betts RF. Use of live cold-adapted influenza A H1N1 and H3N2 virus vaccines in seropositive adults. Journal of Clinical Microbiology 1990; 28:596-599.
45. Gorse GJ, O'Connor TZ, Newman FK, Mandava MD, Mendelman PM, Wittes J, Peduzzi PN. Immunity to influenza in older adults with chronic obstructive pulmonary disease. Journal of Infectious Diseases 2004; 190:11-19.
46. Catanzaro AT, Koup RA, Roederer M et al. Safety and immunogenicity evaluation of a multiclade HIV-1 candidate vaccine delivered by a replication-defective recombinant adenovirus vector. Journal of Infectious Diseases 2006; 194:1638-1649.
47. Daniels MJ, Hughes MD. Meta-analysis for the evaluation of potential surrogate markers. Statistics in Medicine 1997; 16:1965-1982.
48. Gail MH, Pfeiffer R, Van Houwelingen HC, Carroll RJ. On meta-analytic assessment of surrogate outcomes. Biostatistics 2000; 1:231-246.