Multivariate survival analysis with doubly-censored data: Application to the assessment of accutane treatment for fibrodysplasia ossificans progressiva

Geoffrey Jones, David M Rocke

Research output: Contribution to journalArticle

7 Scopus citations

Abstract

Fibrodysplasia ossificans progressiva is a rare genetic disorder in which the joints of patients become disabled by the formation of heterotopic bone. Data are available on the status of 11 joints of each of 21 patients before, during and after treatment with Accutane. These are compared with data obtained by questionnaire from 40 untreated patients to determine the efficacy of the treatment. Both left- and right-censoring are present in each group, which, together with the multivariate nature of the data and the time-dependent treatment covariate, makes analysis difficult. We consider two alternative parametric models for incorporating within-subject dependence: a marginal model and a frailty model. Both analyses suggest that Accutane treatment is effective. We discuss and illustrate the differences between the two approaches. We also discuss the extent to which the conclusions are compromised by the observational nature of the study.

Original languageEnglish (US)
Pages (from-to)2547-2562
Number of pages16
JournalStatistics in Medicine
Volume21
Issue number17
DOIs
StatePublished - Sep 15 2002

ASJC Scopus subject areas

  • Epidemiology

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