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Research output: Contribution to Journal/Magazine › Journal article › peer-review
Research output: Contribution to Journal/Magazine › Journal article › peer-review
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TY - JOUR
T1 - Quantifying the association between progression-free survival and overall survival in oncology trials using Kendall's tau.
AU - Weber, Enya
AU - Titman, Andrew Charles
PY - 2019/2/28
Y1 - 2019/2/28
N2 - This paper considers methods for estimating the association between progression-free and overall survival in oncology trials. Copula-based, non-parametric and illness-death model-based methods are reviewed. In addition, the approach based on an underlying illness-death model is generalized to allow general parametric models. The performance of these methods, in terms of bias and efficiency, is investigated through simulation and also illustrated using data from a clinical trial of treatments for colon cancer. The simulations suggest that the illness-death model-based method provides good estimates of Kendall's tau across several scenarios. In some situations copula-based methods perform well but their performance is sensitive to the choice of copula. The Clayton copula is most appropriate in scenarios which might realistically reflect an oncology trial, but the use of copula models in practice is questionable.
AB - This paper considers methods for estimating the association between progression-free and overall survival in oncology trials. Copula-based, non-parametric and illness-death model-based methods are reviewed. In addition, the approach based on an underlying illness-death model is generalized to allow general parametric models. The performance of these methods, in terms of bias and efficiency, is investigated through simulation and also illustrated using data from a clinical trial of treatments for colon cancer. The simulations suggest that the illness-death model-based method provides good estimates of Kendall's tau across several scenarios. In some situations copula-based methods perform well but their performance is sensitive to the choice of copula. The Clayton copula is most appropriate in scenarios which might realistically reflect an oncology trial, but the use of copula models in practice is questionable.
KW - Kendall's τ
KW - copula
KW - inverse probability of censoring weights
KW - multistate model
KW - overall survival
KW - progression-free survival
U2 - 10.1002/sim.8001
DO - 10.1002/sim.8001
M3 - Journal article
VL - 38
SP - 703
EP - 719
JO - Statistics in Medicine
JF - Statistics in Medicine
SN - 0277-6715
IS - 5
ER -