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dc.contributor.authorDegeling, Koen
dc.contributor.authorWong, Hui-Li
dc.contributor.authorKoffijberg, Hendrik
dc.contributor.authorJalali, Azim
dc.contributor.authorShapiro, Jeremy
dc.contributor.authorKosmider, Suzanne
dc.contributor.authorWong, Rachel
dc.contributor.authorLee, Belinda
dc.contributor.authorBurge, Matthew
dc.contributor.authorTie, Jeanne
dc.contributor.authorYip, Desmond
dc.contributor.authorNott, Louise
dc.contributor.authorKhattak, Adnan
dc.contributor.authorLim, Stephanie
dc.contributor.authorCaird, Susan
dc.contributor.authorGibbs, Peter
dc.contributor.authorIJzerman, Maarten
dc.description.abstractBACKGROUND: 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.
dc.publisherSpringer Science and Business Media LLC
dc.subject.fieldofresearchClinical Sciences
dc.subject.fieldofresearchMedical and Health Sciences
dc.titleSimulating 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.
dc.typeJournal article
dc.type.descriptionC1 - Articles
dcterms.bibliographicCitationDegeling, K; Wong, H-L; Koffijberg, H; Jalali, A; Shapiro, J; Kosmider, S; Wong, R; Lee, B; Burge, M; Tie, J; Yip, D; Nott, L; Khattak, A; Lim, S; Caird, S; Gibbs, P; IJzerman, M, 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., Pharmacoeconomics, 2020
gro.description.notepublicThis publication has been entered in Griffith Research Online as an advanced online version.
gro.hasfulltextNo Full Text
gro.griffith.authorCaird, Susan

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