How to measure temporal changes in care pathways for chronic diseases using health care registry data

BACKGROUND: Disease trajectories for chronic diseases can span over several decades, with several time-dependent factors affecting treatment decisions. Thus, there is a need for long-term predictions of disease trajectories to inform patients and healthcare professionals on the long-term outcomes and provide information on the need of future health care. Here, we propose a state transition model to describe and predict disease trajectories up to 25 years after diagnosis in men with prostate cancer (PCa), as a proof of principle. METHODS: States, state transitions, and transition probabilities were identified and estimated in Prostate Cancer data Base of Sweden (PCBaSe(Traject)), using nationwide population-based data from 118,743 men diagnosed with PCa. A state transition model in discrete time steps (i.e., 4 weeks) was developed and applied to capture all possible transitions (PCBaSe(Sim)). Transition probabilities were estimated for changes in both treatment and comorbidity. These models combined yielded parameter estimates to run an individual-level simulation based on the state-transition model to obtain prediction estimates. Predicted estimates were then compared to real world data in PCBaSe(Traject). RESULTS: PCBaSe(Sim) estimates for the cumulative incidence of first and second transitions, death from PCa and death from other causes were compared to observed transitions in PCBaSe(Traject). A good agreement was found between simulated and observed estimates. CONCLUSIONS: We developed a reliable and accurate simulation tool, PCBaSe(Sim) that provides information on disease trajectories for subjects with a chronic disease on an individual and population-based level. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1186/s12911-019-0823-y) contains supplementary material, which is available to authorized users.