Methods for conducting sensitivity analysis of trials with potentially nonignorable competing causes of censoring

Biometrics. 2001 Mar;57(1):103-13. doi: 10.1111/j.0006-341x.2001.00103.x.

Abstract

We consider inference for the treatment-arm mean difference of an outcome that would have been measured at the end of a randomized follow-up study if, during the course of the study, patients had not initiated a nonrandomized therapy or dropped out. We argue that the treatment-arm mean difference is not identified unless unverifiable assumptions are made. We describe identifying assumptions that are tantamount to postulating relationships between the components of a pattern-mixture model but that can also be interpreted as imposing restrictions on the cause-specific censoring probabilities of a selection model. We then argue that, although sufficient for identification, these assumptions are insufficient for inference due to the curse of dimensionality. We propose reducing dimensionality by specifying semiparametric cause-specific selection models. These models are useful for conducting a sensitivity analysis to examine how inference for the treatment-arm mean difference changes as one varies the magnitude of the cause-specific selection bias over a plausible range. We provide methodology for conducting such sensitivity analysis and illustrate our methods with an analysis of data from the AIDS Clinical Trial Group (ACTG) study 002.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Acquired Immunodeficiency Syndrome / drug therapy
  • Anti-HIV Agents / administration & dosage
  • Biometry*
  • Data Interpretation, Statistical
  • Humans
  • Models, Statistical
  • Randomized Controlled Trials as Topic / statistics & numerical data*
  • Sensitivity and Specificity
  • Zidovudine / administration & dosage

Substances

  • Anti-HIV Agents
  • Zidovudine