Heterogeneity of the baseline risk within patient populations of clinical trials: A proposed evaluation algorithm

American Journal of Epidemiology

Volumn 148, Issue 11, 1998, Pages 1117-1126

  Ioannidis, John P A ^a   Lau, Joseph ^b  

^a NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^b TUFTS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Algorithms; Clinical trials; Epidemiologic methods; Meta analysis; Models, statistical; Randomization; Risk; Study design

Indexed keywords

COTRIMOXAZOLE; PENTAMIDINE; ZALCITABINE; ZIDOVUDINE;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ADULT; ALGORITHM; ARTICLE; CAUSE OF DEATH; CLINICAL TRIAL; DATA BASE; GENETIC HETEROGENEITY; HEALTH HAZARD; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; INFECTION RISK; MAJOR CLINICAL STUDY; META ANALYSIS; PNEUMOCYSTIS PNEUMONIA; PREDICTION; RISK ASSESSMENT; RISK FACTOR; STATISTICAL ANALYSIS; TREATMENT OUTCOME;

EID: 0032401967     PISSN: 00029262     EISSN: None     Source Type: Journal    
DOI: 10.1093/oxfordjournals.aje.a009590     Document Type: Article

References (35)

1. Glasziou PP, Irwig LM. An evidence based approach to individualising treatment. BMJ 1995;311:1356-9.
2. Bailey KR. Generalizing the results of randomized clinical trials. Controlled Clin Trials 1994;15:15-23.
3. Rothwell PM. Can overall results of clinical trials be applied to all patients? Lancet 1995;345:1616-19.
4. Ioannidis JP, Cappelleri JC, Schmid CH, et al. Impact of epidemic and individual heterogeneity on the population distribution of disease progression rates: an example from patient populations in trials of human immunodeficiency virus infection. Am J Epidemiol 1996;144:1074-85.
5. Cappelleri JC, Ioannidis JP, Schmid CH, et al. Large trials vs. meta-analyses of small trials: how do their results compare? JAMA 1996;276:1332-8.
6. Smith GD, Song F, Sheldon TA. Cholesterol lowering and mortality: the importance of considering initial level of risk. BMJ 1993;306:1367-73.
7. Boissel JP, Collet JP, Lievre M, et al. An effect model for the assessment of drug benefit: example of antiarrhythmic drugs in postmyocardial infarction patients. J Cardiovasc Pharmacol 1993;22:356-63.
8. Ioannidis JP, Sacks HS, Cappelleri JC, et al. Clinical efficacy of antiretroviral changes in treatment-experienced HIV infected patients: a metaanalysis. Online J Curr Clin Trials 1997;May 15. (Document no. 204).
9. Ioannidis JP, Cappelleri JC, Skolnik PR, et al. A meta-analysis of the relative efficacy and toxicity of Pneumocystis carinii prophylactic regimens. Arch Intern Med 1996;156:177-88.
10. Yusuf S, Zucker D, Peduzzi P, et al. Effect of coronary artery bypass graft surgery on survival: overview of 10-year results from randomised trials by the Coronary Artery Bypass Graft Surgery Trialists Collaboration. Lancet 1994;344:563-70.
11. McIntosh MW. The population risk as an explanatory variable in research synthesis of clinical trials. Stat Med 1996;15: 1713-28.
12. Yusuf S, Collins R, Peto R. Why do we need some large, simple randomized trials? Stat Med 1984;3:409-22.
13. Ioannidis JP, Lau J. The impact of high-risk patients on the results of clinical trials. J Clin Epidemiol 1997;50:1089-98.
14. Bozzette SA, Finkelstein DM, Spector SA, et al. A randomized trial of three antipneumocystis agents in patients with advanced human immunodeficiency virus infection. The NIAID AIDS Clinical Trials Group. N Engl J Med 1995;332: 693-9.
15. Kahn JO, Lagakos SW, Richman DD, et al. A controlled trial comparing continued zidovudine with didanosine in human immunodeficiency virus infection. The NIAID AIDS Clinical Trials Group. N Engl J Med 1992;327:581-7.
16. Fischl MA, Stanley K, Collier AC, et al. Combination and monotherapy with zidovudine and zalcitabine in patients with advanced HIV disease. The NIAID AIDS Clinical Trials Group. Ann Intern Med 1995;122:24-32.
17. Cox DR. Regression models and life tables (with discussion). J R Stat Soc 1972;34:187-220.
18. Nagelkerke NJ. A note on a general definition of the coefficient of determination. Biometrika 1991;78:691-2.
19. Cox DR, Snell EJ. The analysis of binary data. 2nd ed. London, England: Chapman and Hall Ltd, 1989.
20. Swindells S, Currier JS, Williams P. Correlation of viral load and risk for opportunistic infection. (Abstract 359). In: Abstracts of the Fourth Conference on Retroviruses and Opportunistic Infections, Washington, DC, January 22-26, 1997. Arlington, VA: American Society for Microbiology, 1997.
21. Saah AJ, Hoover DR, Peng Y, et al. Predictors for failure of Pneumocystis carinii pneumonia prophylaxis. JAMA 1995; 273:1197-202.
22. Wynia MK, Ioannidis JP, Lau J. Analysis of life-long strategies to prevent Pneumocystis carinii pneumonia in patients with variable HIV progression rates. AIDS 1998;12:1317-25.
23. Coombs RW, Welles SL, Hooper C, et al. Association of plasma human immunodeficiency virus type 1 RNA level with risk of clinical progression in patients with advanced infection. The NIAID AIDS Clinical Trials Group (ACTG) 116B/ 117 Study Team. J Infect Dis 1996;174:704-12.
24. Mellors JW, Rinaldo CR Jr, Gupta P, et al. Prognosis in HIV-1 infection predicted by the quantity of virus in plasma. Science 1996;272:1167-70.
25. Ioannidis JP, Cappelleri JC, Lau J, et al. Predictive value of viral load measurements in asymptomatic untreated HIV-1 infection: a mathematical model. AIDS 1996;10:255-62.
26. Rubin D. Meta-analysis: literature synthesis or effect-size surface estimation? J Educ Stat 1992;17:363-74.
27. Oxman AD, Guyatt GH. A consumer's guide to subgroup analyses. Ann Intern Med 1992;116:78-84.
28. Yusuf S, Wittes J, Probstfield J, et al. Analysis and interpretation of treatment effects in subgroups of patients in randomized clinical trials. JAMA 1991;266:93-8.
29. Coronary Drug Project Research Group. Influence of adherence to treatment and response of cholesterol on mortality in the Coronary Drug Project. N Engl J Med 1980;303:1038-41.
30. Kelsey JL, Whittemore AS, Evans AS, et al, eds. Methods in observational epidemiology. 2nd ed. New York, NY: Oxford University Press, 1996:348-54.
31. Copeland KT, Checkoway H, McMichael AJ, et al. Bias due to misclassification in the estimation of relative risk. Am J Epidemiol 1977;105:488-95.
32. Harrell FE Jr, Lee KL, Mark DB. Multivariate prognostic models: issues in developing models, evaluating assumptions and adequacy, and measuring and reducing errors. Stat Med 1996;15:361-87.
33. Schmid CH, Lau J, McIntosh M, et al. An empirical study of the effect of the control rate as a predictor of treatment efficacy in meta-analysis of clinical trials. Stat Med 1998 (in press).
34. Lau J, Ioannidis JP, Schmid CH. Summing up evidence: one answer is not always enough. Lancet 1998;351:123-7.
35. Ioannidis JP, Cappelleri JC, Lau J. Issues in comparisons between meta-analyses and large trials. JAMA 1998;279: 1089-93.