CD4 cell count as a surrogate endpoint in HIV clinical trials: A meta-analysis of studies of the AIDS clinical trials group

AIDS

Volumn 12, Issue 14, 1998, Pages 1823-1832

  Hughes, Michael D ^a,e   Daniels, Michael J ^b   Fischl, Margaret A ^c   Kim, Soyeon ^a   Schooley, Robert T ^d  

^a CENTER FOR BIOSTATISTICS IN AIDS RESEARCH   (United States)

^b Department of Statistics Center for Statistics and Applications in Forensic Evidence Iowa State University 2438 Osborn Dr Ames   (United States)

^c UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF COLORADO   (United States)

^e HARVARD SCHOOL OF PUBLIC HEALTH   (United States)

Author keywords

CD4 cell count; Clinical trials; HIV infection; Surrogate endpoint

Indexed keywords

CD4 ANTIGEN; DIDANOSINE; NEVIRAPINE; SAQUINAVIR; ZALCITABINE; ZIDOVUDINE;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; LYMPHOCYTE COUNT; META ANALYSIS; NONBIOLOGICAL MODEL; PHASE 2 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; REGRESSION ANALYSIS; TREATMENT OUTCOME;

EID: 0032189130     PISSN: 02699370     EISSN: None     Source Type: Journal    
DOI: 10.1097/00002030-199814000-00014     Document Type: Article

References (42)

1. Hammer SM, Katzenstein DA, Hughes MD, et al.: A trial comparing nucleoside monotherapy with combination therapy in HIV-infected adults with CD4 cell counts from 200-500 per cubic millimeter. N Engl J Med 1996, 335:1081-1090.
2. DELTA Coordinating Committee: DELTA: a randomised double-blind controlled trial comparing combinations of zidovudine plus didanosine or zalcitabine or zidovudine alone in HIV infected individuals. Lancet 1996, 343:283-291.
3. CAESAR Coordinating Committee: Randomised trial of addition of lamivudine or lamivudine plus loviride to zidovudine-containing regimens for patients with HIV-1 infection: the CAESAR trial. Lancet 1997, 349:1413-1421.
4. Hammer SM, Squires KE, Hughes MD, et al.: A controlled trial of two nucleoside analogues plus indinavir in persons with human immunodeficiency virus infection and CD4 cell counts of 200 per cubic millimeter or less. N Engl J Med 1997, 337:725-733.
5. Machado SG, Gail MH, Ellenberg SS: On the use of laboratory markers as surrogates for clinical endpoints in the evaluation of treatment for HIV infection. J Acquir Immune Defic Syndr 1990, 3:1065-1073.
6. Lagakos SW, Hoth DF: Surrogate markers in AIDS: Where are we? Where are we going? Ann Intern Med 1992, 116:599-601.
7. Hughes MD, DeGruttola V, Welles SL: Evaluating surrogate markers. J Acquir Immune Defic Syndr Hum Retrovirol 1995, 10 (suppl 2):S1-S8.
8. De Gruttola V, Fleming T, Lin DY, Coombs R: Validating surrogate markers: are we being naive? J Infect Dis 1997, 175:237-246.
9. Fleming T, DeMets D: Surrogate end points in clinical trials: are we being misled? Ann Intern Med 1996, 125:605-613.
10. Mellors JW, Munoz A, Giorgi JV, et al.: Plasma viral load and CD4+ lymphocytes as prognostic markers in HIV-1 infection. Ann Intern Med 1997, 126:946-954.
11. Hughes MD, Johnson VA, Hirsch MS, et al.: Monitoring plasma HIV-1 RNA levels in addition to CD4+ lymphocyte count improves assessment of antiretroviral therapeutic response. Ann Intern Med 1997, 126:929-938.
12. O'Brien WA, Hartigan PM, Daar ES, Simberkoff MS, Hamilton JD, for the VA Cooperative Study Group on AIDS: Changes in plasma HIV-1 RNA levels and CD4+ lymphocyte counts predict both response to antiretroviral therapy and therapeutic failure. Ann Intern Med 1997, 126:939-945.
13. Choi S, Lagakos SW, Schooley TT, Volberding PA: CD4 + lymphocytes are an incomplete surrogate marker for clinical progression in persons with asymptomatic HIV infection taking zidovudine. Ann Intern Med 1993, 118:674-680.
14. De Gruttola V, Wulfsohn M, Fischl MA, Tsiatis A: Modeling the relationship between survival and CD4 lymphocytes in patients with AIDS and AIDS-related complex. J Acquir Immune Defic Syndr 1993, 6:359-365.
15. Lin DY, Fischl MA, Schoenfeld DA: Evaluating the role of CD4-lymphocyte counts as surrogate endpoints in human immunodeficiency virus clinical trials. Stat Med 1993, 12:835-842.
16. Freedman LS, Graubard BL, Schatzkin A: Statistical validation of intermediate endpoints for chronic diseases. Stat Med 1993, 11:167-178.
17. Daniels MJ, Hughes MD: Meta-analysis for the evaluation of potential surrogate markers. Stat Med 1997, 16:1965-1982.
18. Fischl MA, Parker CB, Pettinelli C, et al.: A randomized controlled trial of a reduced daily dose of zidovudine in patients with acquired immunodeficiency syndrome. N Engl J Med 1990, 323:1009-1014.
19. Fischl MA, Richman DD, Hansen N, et al.: The safely and efficacy of zidovudine in the treatment of subjects with mildly symptomatic human immunodeficiency virus type I (HIV) infection. Ann Intern Med 1990, 112:727-737.
20. Volberding PA, Lagakos SW, Koch MA, 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 1990, 322:941-949.
21. Volberding PA, Lagakos SW, Grimes JM, et al.: The duration of zidovudine benefit in persons with asymptomatic HIV infection: prolonged evaluation of protocol 019 of the AIDS Clinical Trials Group. JAMA 1994, 272:437-442.
22. Volberding PA, Lagakos SW, Grimes JM, et al.: Immediate versus deferred zidovudine in asymptomatic HIV-infected subjects with CD4 cell counts of 500 per microliter or greater. N Engl J Med 1995, 333:401-413.
23. Merigan TC, Amato DA, Balsley J, et al.: Placebo-controlled trial to evaluate zidovudine in treatment of human immunodeficiency virus infection in asymptomatic patients with hemophilia. Blood 1991, 4:900-906.
24. Follansbee S, Drew L, Olson R, Pollard R, Welter W, Salgo M: The efficacy of zalcitabine (ddC, HIVID) versus zidovudine (ZDV) as monotherapy in ZDV naive patients with advanced HIV disease: a randomized, double-blind, comparative trial (ACTG 114; N3300). IX International Conference on AIDS. Berlin, June 1993 [abstract PO-B26-2113].
25. Bozzette SA, Kanouse DE, Berry S, Duan N, for the Roche 3300/ACTG 114 Study Group: Health status and function with zidovudine or zalcitabine as initial therapy for AIDS. JAMA 1995, 273:295-301.
26. Dolin R, Amato DA, Fischl MA, et al.: Zidovudine compared with didanosine in patients with advanced HIV type 1 infection and little or no previous experience with zidovudine. Arch Intern Med 1995, 155:961-974.
27. Kahn JO, Lagakos SW, Richman DD, et al.: A controlled trial comparing continued zidovudine with didanosine in human immunodeficiency virus infection. N Engl J Med 1992, 327:581-587.
28. Allan JD, De Gruttola V, Cross A, et al.: Executive Summary of the Final Analysis of ACIG 118. Bethesda: National Institute of Infectious and Allergic Diseases; 1993.
29. Fischl MA, Olson RM, Follansbee SL, et al.: Zalcitabine compared with zidovudine in patients with advanced HIV-1 infection who received previous zidovudine therapy. Ann Intern Med 1993, 118:762-769.
30. Fischl MA, Stanley K, Collier AC, et al.: Combination and monotherapy with zidovudine and zalcitabine in patients with advance HIV disease. Ann Intern Med 1995, 122:24-32.
31. Collier AC, Coombs RW, Schoenfeld DA, et al.: Treatment of immunodeficiency virus infection with saquinavir, zidovudine and zalcitabine. N Engl J Med 1996, 334:1011-1017.
32. D'Aquila RT, Hughes MD, Johnson VA, et al.: Nevirapine, zidovudine, and didanosine compared with zidovudine and didanosine in patients with HIV-1 infection. A randomized, double-blind, placebo-controlled trial. Ann Intern Med 1996, 124:1019-1031.
33. Centers for Disease Control: Revision of the CDC surveillance case definition for acquired immunodeficiency syndrome. MMWR 1987, 36 (suppl 1):1S-15S.
34. Centers tor Disease Control: 1993 Revised classification system for HIV infection and expanded surveillance case definition for AIDS among adolescents and adults. MMWR 1992, 41 (RR-17):1-19.
35. Calvelli I, Denny TN, Paxton H, Gelman R, Kagan J: Guideline for flow cytometric immunophenotyping: a report from the National Institute of Allergy and Infectious Diseases, Division of AIDS. Cytometry 1993, 14:702-715.
36. Gelman R, Cheng SC, Kidd P, Waxdal M, Kagan J: Assessment of the effects of instrumentation, monoclonal antibody, and fluorochrome on flow cytometric immunophenotyping: a report based on 2 years of the NIAID DAIDS flow cytometry quality assessment program. Clin Immunol Immunopathol 1993, 66:150-162.
37. Lin DY, Fleming TR, DeGruttola V: Estimating the proportion of treatment effect explained by a surrogate marker. Stat Med 1997, 16:1515-1527.
38. O'Brien WA, Hartigan PM, 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 1996, 334:426-431.
39. Hughes MD, Struthers L, Shear M: Evaluation of HIV-1 RNA as a surrogate endpoint in a clinical trial of saquinavir. Sixth European Conference on Clinical Aspects and Treatment of HIV Infection. Hamburg, October 1997 [abstract 327].
40. Montaner J, DeMasi R, Hill A, CAESAR Coordinating Committee: Validation of HIV-1 RNA and CD4 count as surrogate markers in the CAESAR trial. Sixth European Conference on Clinical Aspects and Treatment of HIV Infection. Hamburg, October 1997 [abstract 207].
41. Babiker A, for the DELTA Coordinating Committee and Virology Group: Can HIV-1 RNA viral load be used as a surrogate for clinical endpoints in HIV disease? Sixth European Conference on Clinical Aspects and Treatment of HIV Infection. Hamburg, October 1997 [abstract 103].
42. Kim S, for the HIV Surrogate Marker Collaborative Group: Meta-analysis to investigate the value of HIV RNA level and CD4 cell count as surrogate endpoints for evaluating treatments. Sixth European Conference on Clinical Aspects and Treatment of HIV Infection. Harmburg, October 1997 [abstract 328].