Advertisement

Analysis of Randomized Controlled Clinical Trials

  • Robert N. Foley
Protocol
Part of the Methods in Molecular Biology™ book series (MIMB, volume 473)

Abstract

When analyzing the results of a trial, the primary outcome variable must be kept in clear focus. In the analysis plan, consideration must be given to comparing the characteristics of the subjects, taking account of differences in these characteristics; intention-to-treat analysis; interim analyses and stopping rules; mortality comparisons; composite outcomes; research design including run-in periods, factorial, stratified, and crossover designs; number needed to treat; power issues; multivariate modeling; and hypothesis-generating analyses.

Key words

Randomized controlled trials analysis intention to treat research design stopping rules multivariate modeling 

References

  1. 1.
    1. The Diabetes Control and Complications Trial Research Group. (1993) The effect of intensive treatment of diabetes on the development and progression of long-term complications in insulin-dependent diabetes mellitus. N Engl J Med 329, 977–986.CrossRefGoogle Scholar
  2. 2.
    2. Pocock, S. J. (1997) Group sequential methods in the design and analysis of clinical trials. Biometrika 64. 191–200.CrossRefGoogle Scholar
  3. 3.
    3. Haybittle, J. L. (1971) Repeated assessment of results in clinical trials of cancer treatment. Br J Radiol 44, 793–797.PubMedCrossRefGoogle Scholar
  4. 4.
    4. O'Brien, P.C., Fleming, T. R. (1979) A multiple testing procedure for clinical trials. Biometrics 35, 549–556.PubMedCrossRefGoogle Scholar
  5. 5.
    5. Singh, A. K, Szczech, L., Tang, K. L., Barnhart, H., Sapp, S., Wolfson, M., Reddan, D., CHOIR Investigators. (2006) Correction of anemia with epoetin alfa in chronic kidney disease. N Engl J Med 355, 2085–2098.PubMedCrossRefGoogle Scholar
  6. 6.
    6. Frick, M. H., Elo, O., Haapa, K., Heinonen, O. P., Heinsalmi, P., Helo, P., Huttunen, J. K., Kaitaniemi, P., Koskinen, P., Manninen, V., et al. (1987). Helsinki Heart Study: Primary-prevention trial with gemfibrozil in middle-aged men with dyslipidemia. Safety of treatment, changes in risk factors, and incidence of coronary heart disease. N Engl J Med 317, 1237–1245.PubMedCrossRefGoogle Scholar
  7. 7.
    7. Freemantle, N., Calvert, M., Wood, J., Eastaugh, J., Griffin, C. (2003) Composite outcomes in randomized trials: Greater precision but with greater uncertainty? JAMA 289, 2554–2559.PubMedCrossRefGoogle Scholar
  8. 8.
    8. Packer, M., O'Connor, C. M., Ghali, J. K., Pressler, M. L., Carson, P. E., Belkin, R. N., Miller, A. B., Neuberg, G. W., Frid, D., Wertheimer, J. H., Cropp, A. B., DeMets, D. L. (1996). Effect of amlodipine on morbidity and mortality in severe chronic heart failure. Prospective Randomized Amlodipine Survival Evaluation Study Group. N Engl J Med 335:1107–1114.PubMedCrossRefGoogle Scholar
  9. 9.
    9. Grizzle, J. E. (1965) The two-period change-over design and its use in clinical trials. Biometrics 21, 461–480.CrossRefGoogle Scholar
  10. 10.
    10. Sibbald, B., Roberts, C. (1998) Understanding controlled trials. Crossover trials. BMJ 316, 1719.PubMedCrossRefGoogle Scholar
  11. 11.
    11. Freeman, P. R. (1989) The performance of the two-stage analysis of two-treatment, two-period crossover trials. Stat Med 8, 1421–1432.PubMedCrossRefGoogle Scholar
  12. 12.
    12. Laupacis, A., Sackett, D. L., Roberts, R. S. (1988) An assessment of clinically useful measures of the consequences of treatment. N Engl J Med 318, 1728–1733.PubMedCrossRefGoogle Scholar
  13. 13.
  14. 14.
    14. Hansson, L., Zanchetti, A., Carruthers, S. G., Dahlof, B., Elmfeldt, D., Julius, S., Menard, J., Rahn, K. H., Wedel, H., Westerling, S. HOT Study Group. (1998). Effects of intensive blood-pressure lowering and low-dose aspirin in patients with hypertension: principal results of the Hypertension Optimal Treatment (HOT) randomised trial. Lance 351, 1755–1762.CrossRefGoogle Scholar

Copyright information

© Humana Press, a part of Springer Science+Business Media, LLC 2008

Authors and Affiliations

  1. 1.USRDSMinneapolisUSA

Personalised recommendations