Meta-analysis of clinical trials with rare events

Biometrical Journal

Volumn 57, Issue 4, 2015, Pages 633-648

  Böhning, Dankmar ^a   Mylona, Kalliopi ^a   Kimber, Alan ^a  

^a UNIVERSITY OF SOUTHAMPTON   (United Kingdom)

Author keywords

Mantel Haenszel estimation; Meta analysis; Poisson regression with random effects; Rare event analysis; Zero and double zero studies

Indexed keywords

MEDICAL APPLICATIONS; POISSON DISTRIBUTION; RANDOM PROCESSES; STATISTICAL MECHANICS;

CLINICAL TRIAL; DOUBLE ZEROS; MANTEL-HAENSZEL ESTIMATION; META-ANALYSIS; POISSON REGRESSION; POISSON REGRESSION WITH RANDOM EFFECT; RANDOM EFFECTS; RARE-EVENT ANALYSIS; STATISTICAL TOOLS; ZERO AND DOUBLE-ZERO STUDY;

REGRESSION ANALYSIS;

BIOMETRY; CLINICAL TRIAL (TOPIC); HUMAN; META ANALYSIS; POISSON DISTRIBUTION; PROCEDURES; REGRESSION ANALYSIS;

BIOMETRY; CLINICAL TRIALS AS TOPIC; HUMANS; POISSON DISTRIBUTION; REGRESSION ANALYSIS;

EID: 84933680039     PISSN: 03233847     EISSN: 15214036     Source Type: Journal    
DOI: 10.1002/bimj.201400184     Document Type: Article

References (34)

1. Aitkin, M. (1999). A general maximum likelihood analysis of variance components in generalized linear models. Biometrics 55, 117-128.
2. Arends, L. R., Vokó, Z. and Stijnen, T. (2003). Combining multiple outcome measures in a meta-analysis: an application. Statistics in Medicine 22, 1335-1353.
3. Bhaumik, D. K., Amatya, A., Normand, S.-T., Greenhouse, J., Kaizar, E., Neelon, B. and Gibbons, R. D. (2012). Meta-analysis of rare binary adverse event data. Journal of the American Statistical Association 107, 555-567.
4. Böhning, D., Kuhnert, R. and Rattanasiri, S. (2008). Meta-Analysis of Binary Data Using Profile Likelihood. Chapman & Hall, London, UK.
5. Böhning, D. and Sarol, J. Jr., (2000). Estimating risk difference in multicenter studies under baseline-risk heterogeneity. Biometrics 56, 304-308.
6. Bracken, M. B. (2007). Rosiglitazone and cardiovascular risk. The New England Journal of Medicine 357, 937-938.
7. Breslow, N. E. and Day, N. E. (1987). Statistical Methods in Cancer Research. Volume 11 - The Design and Analysis of Cohort Studies. International Agency for Research on Cancer, Lyon, FR.
8. Cai, T., Parast, L. and Ryan, L. (2010). Meta-analysis for rare events. Statistics in Medicine 29, 2078-2089.
9. Cameron, A. C. and Trivedi, P. K. (1998). Regression Analysis of Count Data. Cambridge University Press, Cambridge, UK.
10. Clayton, D. and Hills, M. (1993). Statistical Models in Epidemiology. Oxford University Press, Oxford, UK.
11. Dahabreh, I. J. (2008). Meta-analysis of rare events: an update and sensitivity analysis of cardiovascular events in randomized trials of Rosiglitazone. Clinical Trials 5, 116-120.
12. Diamond, G. A., Bax, L. and Kaul, S. (2007). Uncertain effects of Rosiglitazone on the risk for myocardial infraction and cardiovascular death. Annals of Internal Medicine 147, 578-581.
13. Friedrich, J. O., Beyene, J. and Adhikari, N. K. J. (2009). Rosiglitazone: can meta-analysis accurately estimate excess cardiovascular risk given the available data? Re-analysis of randomized trials using various methodological approaches. BMC Research Notes 2, 5.
14. Friedrich, J. O., Neill, K. J. A., and Beyene, J. (2007). Inclusion of zero total event trials in meta-analyses maintains analytic consistency and incorporates all available data. BMC Medical Research Methodology 7, 5.
15. Hilbe, J. M. (2011). Negative Binomial Regression, (2nd edn.), Cambridge University Press, Cambridge, UK.
16. Home, P. D., Pocock, S. J., Beck-Nielsen, H., Gomis, R., Hanefeld, M., Jones, N. P., Komajda, M., McMurray, J. J. and RECORD Study Group. (2007). Rosiglitazone evaluated for cardiovascular outcomes - an interim analysis. The New England Journal of Medicine 357, 28-38.
17. Jewell, N. P. (2005). Statistics for Epidemiology. Chapman and Hall/CRC, Boca Raton, FL.
18. Kaul, S. and Diamond, G. A. (2011). Rosiglitazone and cardiovascular risk: Nissen and Wolski 2010 updated meta-analysis revisited. Journal of the American College of Cardiology 57, E552-E552.
19. Keus, F., Wetterslev, J., Gluud, C., Gooszen, H. G. and van Laarhoven, C. J. H. M. (2009). Robustness assessments are needed to reduce bias in meta-analyses that include zero-event randomized trials. The American Journal of Gastroenterology 104, 546-551.
20. Kiefer, J. and Wolfowitz, J. (1956). Consistency of the maximum likelihood estimator in the presence of infinitely many incidental parameters. Annals of Mathematical Statistics 27, 887-906.
21. Lambert, D. (1992). Zero-inflated Poisson regression, with an application to defects in manufacturing. Technometrics 34, 1-14.
22. Mannucci, E., Monami, M., Di Bari, M., Lamanna, C., Gori, F., Gensini, G. F. and Marchionni, N. (2009). Cardiac safety profile of Rosiglitazone: a comprehensive meta-analysis of randomized clinical trials. International Journal of Cardiology 143, 135-140.
23. Nissen, S. E. and Wolski, K. (2007). Effect of Rosiglitazone on the risk of myocardial infarction and death from cardiovascular causes. The New England Journal of Medicine 356, 2457-2471.
24. Nissen, S. E. and Wolski, K. (2010). Rosiglitazone revisited: an updated meta-analysis of risk for myocardial infaction and cardiovascular mortality. Archives of Internal Medicine 170, 1191-1201.
25. Rücker, G., Schwarzer, G., Carpenter, J. and Olkin, I. (2009). Why add anything to nothing? The arcsine difference as a measure of treatment effect in meta-analysis with zero cells. Statistics in Medicine 28, 721-738.
26. SAS Institute, Inc. (2008). SAS/STAT(R) 9.2 User's Guide, (2nd edn.), SAS Insitute, Inc, Cary, NC.
27. Self, S. G. and Liang, K. Y., (1987). Large sample properties of the maximum likelihood estimator and the likelihood ratio test on the boundary of the parameter space. Journal of the American Statistical Association 82, 605-611.
28. Shuster, J. J., Jones, L. S., and Salmon, D. A. (2007). Fixed vs. random effects meta-analysis in rare event studies: the Rosigliazone link with myocardial infarction and cardiac death. Statistics in Medicine 26, 4375-4385.
29. Singh, S., Loke, Y. K., and Furberg, C. D. (2007). Long-term risk of cardiovascular events with Rosiglitazone: a meta-analysis. Journal of the American Medical Association 298, 1189-1195.
30. Stijnen, T., Hamza, T. H., and Ozdemir, P. (2010). Random effects meta-analysis of event outcome in the framework of the generalized linear mixed model with applications in sparse data. Statistics in Medicine 29, 3046-3067.
31. Sweeting, M. J., Sutton, A. J., and Lambert, P. C., (2004). What to add to nothing? Use and avoidance of continuity corrections in meta-analysis of sparse data. Statistics in Medicine 23, 1351-1375.
32. Tian, L., Cai, T., Pfeffer, M. A., Piankov, N., Cremieux, P. Y., and Wei, L. J. (2009). Exact and efficient inference procedure for meta-analysis and its application to the analysis of independent 2 x 2 tables with all available data but without artificial continuity correction. Biostatistics 10, 275-281.
33. Winkelmann, R. (2003). Econometric Analysis of Count Data. Springer, New York, NY.
34. Zelterman, D. (2006). Models for Discrete Data (revised edn.). Oxford University Press, Oxford, UK.