Validation of surrogate endpoints in cancer clinical trials via principal stratification with an application to a prostate cancer trial.

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  • Additional Information
    • Source:
      Publisher: Wiley Country of Publication: England NLM ID: 8215016 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-0258 (Electronic) Linking ISSN: 02776715 NLM ISO Abbreviation: Stat Med Subsets: MEDLINE
    • Publication Information:
      Original Publication: Chichester ; New York : Wiley, c1982-
    • Subject Terms:
      Biomarkers* ; Neoplasms*; Endpoint Determination/*methods ; Randomized Controlled Trials as Topic/*methods; Algorithms ; Causality ; Clinical Trials, Phase III as Topic ; Humans ; Logistic Models ; Male ; Meta-Analysis as Topic ; Proportional Hazards Models ; Prostate-Specific Antigen ; Prostatic Neoplasms
    • Abstract:
      Increasing attention has been focused on the use and validation of surrogate endpoints in cancer clinical trials. Previous literature on validation of surrogate endpoints are classified into four approaches: the proportion explained approach; the indirect effects approach; the meta-analytic approach; and the principal stratification approach. The mainstream in cancer research has seen the application of a meta-analytic approach. However, VanderWeele (2013) showed that all four of these approaches potentially suffer from the surrogate paradox. It was also shown that, if a principal surrogate satisfies additional criteria called one-sided average causal sufficiency, the surrogate cannot exhibit a surrogate paradox. Here, we propose a method for estimating principal effects under a monotonicity assumption. Specifically, we consider cancer clinical trials which compare a binary surrogate endpoint and a time-to-event clinical endpoint under two naturally ordered treatments (e.g. combined therapy vs. monotherapy). Estimation based on a mean score estimating equation will be implemented by the expectation-maximization algorithm. We will also apply the proposed method as well as other surrogacy criteria to evaluate the surrogacy of prostate-specific antigen using data from a phase III advanced prostate cancer trial, clarifying the complementary roles of both the principal stratification and meta-analytic approaches in the evaluation of surrogate endpoints in cancer. Copyright © 2017 John Wiley & Sons, Ltd.
      (Copyright © 2017 John Wiley & Sons, Ltd.)
    • Contributed Indexing:
      Keywords: causal inference; monotonicity; prostate-specific antigen; surrogate paradox
    • Accession Number:
      0 (Biomarkers)
      EC 3.4.21.77 (Prostate-Specific Antigen)
    • Publication Date:
      Date Created: 20170510 Date Completed: 20180427 Latest Revision: 20191210
    • Publication Date:
      20240628
    • Accession Number:
      10.1002/sim.7318
    • Accession Number:
      28485043