Discrimination performance in illness-death models with interval-censored disease data
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arXiv
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| Autores principales: | , , , |
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| Formato: | Preprint |
| Publicado: |
2025
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| _version_ | 1866912862298963968 |
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| author | Spreafico, Marta Rueten-Budde, Anja J. Putter, Hein Fiocco, Marta |
| author_facet | Spreafico, Marta Rueten-Budde, Anja J. Putter, Hein Fiocco, Marta |
| contents | In clinical studies, the illness-death model is often used to describe disease progression. A subject starts disease-free, may develop the disease and then die, or die directly. In clinical practice, disease can only be diagnosed at pre-specified follow-up visits, so the exact time of disease onset is often unknown, resulting in interval-censored data. This study examines the impact of ignoring this interval-censored nature of disease data on the discrimination performance of illness-death models, focusing on the time-specific Area Under the receiver operating characteristic Curve (AUC) in both incident/dynamic and cumulative/dynamic definitions. A simulation study with data simulated from Weibull transition hazards and disease state censored at regular intervals is conducted. Estimates are derived using different methods: the Cox model with a time-dependent binary disease marker, which ignores interval-censoring, and the illness-death model for interval-censored data estimated with three implementations - the piecewise-constant model from the msm package, the Weibull and M-spline models from the SmoothHazard package. These methods are also applied to a dataset of 2232 patients with high-grade soft tissue sarcoma, where the interval-censored disease state is the post-operative development of distant metastases. The results suggest that, in the presence of interval-censored disease times, it is important to account for interval-censoring not only when estimating the parameters of the model but also when evaluating the discrimination performance of the disease. |
| format | Preprint |
| id |
arxiv_https___arxiv_org_abs_2504_19726 |
| institution | arXiv |
| publishDate | 2025 |
| record_format | arxiv |
| spellingShingle | Discrimination performance in illness-death models with interval-censored disease data Spreafico, Marta Rueten-Budde, Anja J. Putter, Hein Fiocco, Marta Methodology In clinical studies, the illness-death model is often used to describe disease progression. A subject starts disease-free, may develop the disease and then die, or die directly. In clinical practice, disease can only be diagnosed at pre-specified follow-up visits, so the exact time of disease onset is often unknown, resulting in interval-censored data. This study examines the impact of ignoring this interval-censored nature of disease data on the discrimination performance of illness-death models, focusing on the time-specific Area Under the receiver operating characteristic Curve (AUC) in both incident/dynamic and cumulative/dynamic definitions. A simulation study with data simulated from Weibull transition hazards and disease state censored at regular intervals is conducted. Estimates are derived using different methods: the Cox model with a time-dependent binary disease marker, which ignores interval-censoring, and the illness-death model for interval-censored data estimated with three implementations - the piecewise-constant model from the msm package, the Weibull and M-spline models from the SmoothHazard package. These methods are also applied to a dataset of 2232 patients with high-grade soft tissue sarcoma, where the interval-censored disease state is the post-operative development of distant metastases. The results suggest that, in the presence of interval-censored disease times, it is important to account for interval-censoring not only when estimating the parameters of the model but also when evaluating the discrimination performance of the disease. |
| title | Discrimination performance in illness-death models with interval-censored disease data |
| topic | Methodology |
| url | https://arxiv.org/abs/2504.19726 |