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  • Simulating Progression-Free and Overall Survival for First-Line Doublet Chemotherapy With or Without Bevacizumab in Metastatic Colorectal Cancer Patients Based on Real-World Registry Data.

    Author(s)
    Degeling, Koen
    Wong, Hui-Li
    Koffijberg, Hendrik
    Jalali, Azim
    Shapiro, Jeremy
    Kosmider, Suzanne
    Wong, Rachel
    Lee, Belinda
    Burge, Matthew
    Tie, Jeanne
    Yip, Desmond
    Nott, Louise
    Khattak, Adnan
    Lim, Stephanie
    Caird, Susan
    Gibbs, Peter
    IJzerman, Maarten
    Griffith University Author(s)
    Caird, Susan
    Year published
    2020
    Metadata
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    Abstract
    BACKGROUND: Simulation models utilizing real-world data have potential to optimize treatment sequencing strategies for specific patient subpopulations, including when conducting clinical trials is not feasible. We aimed to develop a simulation model to estimate progression-free survival (PFS) and overall survival for first-line doublet chemotherapy with or without bevacizumab for specific subgroups of metastatic colorectal cancer (mCRC) patients based on registry data. METHODS: Data from 867 patients were used to develop two survival models and one logistic regression model that populated a discrete event simulation (DES). ...
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    BACKGROUND: Simulation models utilizing real-world data have potential to optimize treatment sequencing strategies for specific patient subpopulations, including when conducting clinical trials is not feasible. We aimed to develop a simulation model to estimate progression-free survival (PFS) and overall survival for first-line doublet chemotherapy with or without bevacizumab for specific subgroups of metastatic colorectal cancer (mCRC) patients based on registry data. METHODS: Data from 867 patients were used to develop two survival models and one logistic regression model that populated a discrete event simulation (DES). Discrimination and calibration were used for internal validation of these models separately and predicted and observed medians and Kaplan-Meier plots were compared for the integrated DES. Bootstrapping was performed to correct for optimism in the internal validation and to generate correlated sets of model parameters for use in a probabilistic analysis to reflect parameter uncertainty. RESULTS: The survival models showed good calibration based on the regression slopes and modified Hosmer-Lemeshow statistics at 1 and 2 years, but not for short-term predictions at 0.5 years. Modified C-statistics indicated acceptable discrimination. The simulation estimated that median first-line PFS (95% confidence interval) of 219 (25%) patients could be improved from 175 days (156-199) to 269 days (246-294) if treatment would be targeted based on the highest expected PFS. CONCLUSIONS: Extensive internal validation showed that DES accurately estimated the outcomes of treatment combination strategies for specific subpopulations, with outcomes suggesting treatment could be optimized. Although results based on real-world data are informative, they cannot replace randomized trials.
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    Journal Title
    Pharmacoeconomics
    DOI
    https://doi.org/10.1007/s40273-020-00951-1
    Note
    This publication has been entered in Griffith Research Online as an advanced online version.
    Subject
    Biomedical and clinical sciences
    Clinical sciences
    Economics
    Publication URI
    http://hdl.handle.net/10072/396645
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