Are multiple primary outcomes analysed appropriately in randomised controlled trials? A review

Contemporary Clinical Trials

Volumn 45, Issue , 2015, Pages 8-12

  Vickerstaff, Victoria ^a   Ambler, Gareth ^a   King, Michael ^a   Nazareth, Irwin ^a   Omar, Rumana Z ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Clinical trials; Multiple outcomes; Multiplicity; Neurology; Psychiatry

Indexed keywords

ARTICLE; BONFERRONI CORRECTION; DUNNET CORRECTION; GENERAL PRACTICE; HOCHBERG BENJAMIN ADJUSTMENT; HOLM ADJUSTMENT; HUMAN; MATHEMATICAL ANALYSIS; MEDICAL LITERATURE; MULTIPLE PRIMARY OUTCOME; NEUROLOGY; OUTCOME ASSESSMENT; PSYCHIATRY; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAMPLE SIZE; SECONDARY OUTCOME; SEQUENTIAL ADJUSTMENT; SIDAK CORRECTION; STATISTICAL ANALYSIS; BIOASSAY; METHODOLOGY; PROCEDURES; STANDARDS;

DATA INTERPRETATION, STATISTICAL; ENDPOINT DETERMINATION; HUMANS; NEUROLOGY; PSYCHIATRY; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SAMPLE SIZE;

EID: 84956740546     PISSN: 15517144     EISSN: 15592030     Source Type: Journal    
DOI: 10.1016/j.cct.2015.07.016     Document Type: Article

References (36)

1. De Los Reyes A., Kundey S.M.A., Wang M. The end of the primary outcome measure: a research agenda for constructing its replacement. Clin. Psychol. Rev. 2011, 31(5):829-838.
2. Blakesley R.E., Mazumdar S., Dew M.A., Houck P.R., Tang G., Reynolds C.F., et al. Comparisons of methods for multiple hypothesis testing in neuropsychological research. Neuropsychology 2009, 23(2):255-264.
3. Teixeira-Pinto A., Siddique J., Gibbons R., Normand S.-L. Statistical approaches to modeling multiple outcomes in psychiatric studies. Psychiatr. Ann. 2009, 39(7):729.
4. Tyler K.M., Normand S.L., Horton N.J. The use and abuse of multiple outcomes in randomized controlled depression trials. Contemp. Clin. Trials 2011, 32(2):299-304.
5. Mayo N.E., Scott S. Evaluating a complex intervention with a single outcome may not be a good idea: an example from a randomised trial of stroke case management. Age Ageing 2011, 40(6):718-724.
6. Pocock S., Geller N., Tsiatis A. The analysis of multiple endpoints in clinical trials. Biometrics 1987, 43(3):487-498.
7. Alosh M., Bretz F., Huque M. Advanced multiplicity adjustment methods in clinical trials. Stat. Med. 2014, 33(4):693-713.
8. Moyé L.A. Multiple Analyses in Clinical Trials: Fundamentals for Investigators 2003, Springer Science & Business Media.
9. Bagiella E. Clinical trials in rehabilitation: single or multiple outcomes?. Arch. Phys. Med. Rehabil. 2009, 90(11):S17-S21.
10. Offen W., Chuang-Stein C., Dmitrienko A., Littman G., Maca J., Meyerson L., et al. Multiple co-primary endpoints: medical and statistical solutions: a report from the multiple endpoints expert team of the Pharmaceutical Research and Manufacturers of America. Drug Inf. J. 2007, 41(1):31-46.
11. Wittchen H.-U., Jacobi F., Rehm J., Gustavsson A., Svensson M., Jönsson B., et al. The size and burden of mental disorders and other disorders of the brain in Europe 2010. Eur. Neuropsychopharmacol. 2011, 21(9):655-679.
12. ICH E9 Expert Working Group Statistical principles for clinical trials: ICH harmonised tripartite guideline. Stat. Med. 1999, 18(15):1903-1942.
13. Pogue J., Devereaux P., Thabane L., Yusuf S. Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes. PLoS One 2012, 7(4):e34785.
14. Dmitrienko A., D'Agostino R. Traditional multiplicity adjustment methods in clinical trials. Stat. Med. 2013, 32(29):5172-5218.
15. Bretz F., Hothorn T., Westfall P. Multiple Comparisons Using R 2010, CRC Press.
16. Abdi H. Bonferroni and Šidák corrections for multiple comparisons. Encyclopedia of Measurement and Statistics 2007, Sage, Thousand Oaks, CA. N.J. Salkind (Ed.).
17. Holm S. A simple sequentially rejective multiple test procedure. Scand. J. Stat. 1979, 65-70.
18. Hochberg Y. A sharper Bonferroni procedure for multiple tests of significance. Biometrika 1988, 75(4):800-802.
19. Pocock S. Clinical trials with multiple outcomes - a statistical perspective on their design, analysis and interpretation. Control. Clin. Trials 1997, 18:530-545.
20. Hommel G. A stagewise rejective multiple test procedure based on a modified Bonferroni test. Biometrika 1988, 75(2):383-386.
21. Goldstein H. Multilevel Statistical Models 2011, Wiley. com.
22. McCulloch C. Joint modelling of mixed outcome types using latent variables. Stat. Methods Med. Res. 2008, 17(1):53-73.
23. Haase R.F., Ellis M.V. Multivariate analysis of variance. J. Couns. Psychol. 1987, 34(4):404.
24. Stata Statistical Software: Release.12 2011, StataCorp StataCorp L, College Station, TX.
25. WHO International Standards for Clinical Trial Registries (November 2012. Report No). http://www.who.int/iris/bitstream/10665/76705/1/9789241504294_eng.pdf?ua=1:%202012.
26. Vitiello B., Elliott G.R., Swanson J.M., Arnold L.E., Hechtman L., Abikoff H., et al. Blood pressure and heart rate over 10 years in the multimodal treatment study of children with ADHD. Am. J. Psychiatr. 2012, 169(2):167-177.
27. Odekerken V.J., van Laar T., Staal M.J., Mosch A., Hoffmann C.F., Nijssen P.C., et al. Subthalamic nucleus versus globus pallidus bilateral deep brain stimulation for advanced Parkinson's disease (NSTAPS study): a randomised controlled trial. Lancet Neurol. 2013, 12(1):37-44.
28. Yoon F.B., Fitzmaurice G.M., Lipsitz S.R., Horton N.J., Laird N.M., Normand S.L. Alternative methods for testing treatment effects on the basis of multiple outcomes: simulation and case study. Stat. Med. 2011, 30(16):1917-1932.
29. Yang M., Goldstein H., Browne W., Woodhouse G. Multivariate multilevel analyses of examination results. J. R. Stat. Soc. A Stat. Soc. 2002, 165(1):137-153.
30. Ma X. Stability of school academic performance across subject areas. J. Educ. Meas. 2001, 38(1):1-18.
31. Mohan J., Twigg L., Taylor J. Mind the double gap using multivariate multilevel modelling to investigate public perceptions of crime trends. Br. J. Criminol. 2011, 51(6):1035-1053.
32. Phillips A., Haudiquet V. ICH E9 guideline 'Statistical principles for clinical trials': a case study. Stat. Med. 2003, 22(1):1-11.
33. Schulz K.F., Altman D.G., Moher D. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMC Med. 2010, 8(1):18.
34. Chan A.-W., Tetzlaff J.M., Altman D.G., Laupacis A., Gøtzsche P.C., Krleža-Jerić K., et al. SPIRIT 2013 statement: defining standard protocol items for clinical trials. Ann. Intern. Med. 2013, 158(3):200-207.
35. Hopewell S., Clarke M., Moher D., Wager E., Middleton P., Altman D.G., et al. CONSORT for reporting randomized controlled trials in journal and conference abstracts: explanation and elaboration. PLoS Med. 2008, 5(1):e20.
36. Products CfPM Points to Consider on Multiplicity Issues in Clinical Trials 2002, The European Agency for the Evaluation of Medicinal Products (EMEA), London.