Many clinical trials repeatedly measure several longitudinal outcomes on patients. Patient follow-up can discontinue due to an outcome-dependent event, such as clinical diagnosis, death, or dropout. Joint modeling is a popular choice for the analysis of this type of data.
View Article and Find Full Text PDFBackground: Missing data can complicate the interpretability of a clinical trial, especially if the proportion is substantial and if there are different, potentially outcome-dependent causes.
Methods: We aimed to obtain unbiased estimates, in the presence of a high level of missing data, for the intervention effects in a prodromal Alzheimer's disease trial: the LipiDiDiet study. We used a competing risk joint model that can simultaneously model each patient's longitudinal outcome trajectory in combination with the timing and type of missingness.
Joint models for longitudinal and survival data are increasingly used and enjoy a wide range of application areas. In this article, we focus on the application of joint models on clinical trial data with special interest in the treatment effect on the survival outcome. Within a joint model, the estimated treatment effect on the survival outcome is an aggregate comprising the indirect treatment effect through the longitudinal outcome and the direct treatment effect on the survival outcome.
View Article and Find Full Text PDFBackground: Many prodromal Alzheimer's disease trials collect two types of data: the time until clinical diagnosis of dementia and longitudinal patient information. These data are often analysed separately, although they are strongly associated. By combining the longitudinal and survival data into a single statistical model, joint models can account for the dependencies between the two types of data.
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