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

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, Brenda
dc.contributor.authorBurge, M
dc.contributor.authorTie, Jeanne
dc.contributor.authorYip, Desmond
dc.contributor.authorNott, L.
dc.date.accessioned2024-02-06T00:22:55Z
dc.date.issued2020
dc.date.updated2022-10-16T07:25:53Z
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.en_AU
dc.description.sponsorshipThis research was partly funded by the Netherlands Organisation for Health Research and Development (ZonMW) as part of the Translational Research Program (project number: 446001006). Roche Products Pty Limited (Australia) provided financial assistance for the development, installation and maintenance of the TRACC database. BioGrid Australia manages the TRACC database and provided data access and support.en_AU
dc.format.mimetypeapplication/pdfen_AU
dc.identifier.issn1170-7690en_AU
dc.identifier.urihttp://hdl.handle.net/1885/313240
dc.language.isoen_AUen_AU
dc.publisherAdis International Ltd.en_AU
dc.rights© 2020 The authorsen_AU
dc.sourcePharmacoEconomicsen_AU
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 Dataen_AU
dc.typeJournal articleen_AU
local.bibliographicCitation.issue11en_AU
local.bibliographicCitation.lastpage1275en_AU
local.bibliographicCitation.startpage1263en_AU
local.contributor.affiliationDegeling, Koen, University of Melbourne, Melbourne, VIC, Australiaen_AU
local.contributor.affiliationWong, Hui-li, The Walter and Eliza Hall Institute of Medical Researchen_AU
local.contributor.affiliationKoffijberg, Hendrik, Technical Medical Centre, University of Twente, Enschede, Netherlandsen_AU
local.contributor.affiliationJalali, Azim, Walter and Eliza Hall Institute of Medical Researchen_AU
local.contributor.affiliationShapiro, Jeremy, Cabrini Hospital Malvernen_AU
local.contributor.affiliationKosmider, Suzanne, Department of Medical Oncology, Western Health, Melbourne, VICen_AU
local.contributor.affiliationWong, Rachel, The Walter and Eliza Hall Institute of Medical Researchen_AU
local.contributor.affiliationLee, Brenda, Personalised Oncology Division, Walter and Eliza Hall Institute of Medical Research, Melbourne, VICen_AU
local.contributor.affiliationBurge, M, Royal Brisbane & Women's Hospitalen_AU
local.contributor.affiliationTie, Jeanne, Walter and Eliza Hall Institute of Medical Researchen_AU
local.contributor.affiliationYip, Desmond, College of Health and Medicine, ANUen_AU
local.contributor.affiliationNott, L., Royal Hobart Hospitalen_AU
local.contributor.authoremailu5086006@anu.edu.auen_AU
local.contributor.authoruidYip, Desmond, u5086006en_AU
local.description.embargo2099-12-31
local.description.notesImported from ARIESen_AU
local.identifier.absfor320402 - Applied immunology (incl. antibody engineering, xenotransplantation and t-cell therapies)en_AU
local.identifier.absfor321111 - Solid tumoursen_AU
local.identifier.absfor321104 - Cancer therapy (excl. chemotherapy and radiation therapy)en_AU
local.identifier.ariespublicationa383154xPUB17212en_AU
local.identifier.citationvolume38en_AU
local.identifier.doi10.1007/s40273-020-00951-1en_AU
local.identifier.scopusID2-s2.0-85089441743
local.identifier.thomsonIDWOS:000560931500001
local.identifier.uidSubmittedBya383154en_AU
local.publisher.urlhttps://link.springer.com/en_AU
local.type.statusPublished Versionen_AU

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