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Evaluating treatment effects in group sequential multivariate longitudinal studies with covariate adjustment

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  • Neal O. Jeffries
  • James F. Troendle
  • Nancy L. Geller

Abstract

Jeffries et al. (2018) investigated testing for a treatment difference in the setting of a randomized clinical trial with a single outcome measured longitudinally over a series of common follow‐up times while adjusting for covariates. That paper examined the null hypothesis of no difference at any follow‐up time versus the alternative of a difference for at least one follow‐up time. We extend those results here by considering multivariate outcome measurements, where each individual outcome is examined at common follow‐up times. We consider the case where there is interest in first testing for a treatment difference in a global function of the outcomes (e.g., weighted average or sum) with subsequent interest in examining the individual outcomes, should the global function show a treatment difference. Testing is conducted for each follow‐up time and may be performed in the setting of a group sequential trial. Testing procedures are developed to determine follow‐up times for which a global treatment difference exists and which individual combinations of outcome and follow‐up time show evidence of a difference while controlling for multiplicity in outcomes, follow‐up, and interim analyses. These approaches are examined in a study evaluating the effects of tissue plasminogen activator on longitudinally obtained stroke severity measurements.

Suggested Citation

  • Neal O. Jeffries & James F. Troendle & Nancy L. Geller, 2023. "Evaluating treatment effects in group sequential multivariate longitudinal studies with covariate adjustment," Biometrics, The International Biometric Society, vol. 79(2), pages 1496-1506, June.
    • Handle: RePEc:bla:biomet:v:79:y:2023:i:2:p:1496-1506
    DOI: 10.1111/biom.13659
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    References listed on IDEAS

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    1. Neal O. Jeffries & James F. Troendle & Nancy L. Geller, 2018. "Detecting treatment differences in group sequential longitudinal studies with covariate adjustment," Biometrics, The International Biometric Society, vol. 74(3), pages 1072-1081, September.
    2. John M Lachin, 2014. "Applications of the Wei-Lachin Multivariate One-Sided Test for Multiple Outcomes on Possibly Different Scales," PLOS ONE, Public Library of Science, vol. 9(10), pages 1-14, October.
    3. Dei-In Tang & Nancy L. Geller, 1999. "Closed Testing Procedures for Group Sequential Clinical Trials with Multiple Endpoints," Biometrics, The International Biometric Society, vol. 55(4), pages 1188-1192, December.
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