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.author | Degeling, Koen | |
dc.contributor.author | Wong, Hui-li | |
dc.contributor.author | Koffijberg, Hendrik | |
dc.contributor.author | Jalali, Azim | |
dc.contributor.author | Shapiro, Jeremy | |
dc.contributor.author | Kosmider, Suzanne | |
dc.contributor.author | Wong, Rachel | |
dc.contributor.author | Lee, Brenda | |
dc.contributor.author | Burge, M | |
dc.contributor.author | Tie, Jeanne | |
dc.contributor.author | Yip, Desmond | |
dc.contributor.author | Nott, L. | |
dc.date.accessioned | 2024-02-06T00:22:55Z | |
dc.date.issued | 2020 | |
dc.date.updated | 2022-10-16T07:25:53Z | |
dc.description.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). 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.sponsorship | This 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.mimetype | application/pdf | en_AU |
dc.identifier.issn | 1170-7690 | en_AU |
dc.identifier.uri | http://hdl.handle.net/1885/313240 | |
dc.language.iso | en_AU | en_AU |
dc.publisher | Adis International Ltd. | en_AU |
dc.rights | © 2020 The authors | en_AU |
dc.source | PharmacoEconomics | en_AU |
dc.title | 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 | en_AU |
dc.type | Journal article | en_AU |
local.bibliographicCitation.issue | 11 | en_AU |
local.bibliographicCitation.lastpage | 1275 | en_AU |
local.bibliographicCitation.startpage | 1263 | en_AU |
local.contributor.affiliation | Degeling, Koen, University of Melbourne, Melbourne, VIC, Australia | en_AU |
local.contributor.affiliation | Wong, Hui-li, The Walter and Eliza Hall Institute of Medical Research | en_AU |
local.contributor.affiliation | Koffijberg, Hendrik, Technical Medical Centre, University of Twente, Enschede, Netherlands | en_AU |
local.contributor.affiliation | Jalali, Azim, Walter and Eliza Hall Institute of Medical Research | en_AU |
local.contributor.affiliation | Shapiro, Jeremy, Cabrini Hospital Malvern | en_AU |
local.contributor.affiliation | Kosmider, Suzanne, Department of Medical Oncology, Western Health, Melbourne, VIC | en_AU |
local.contributor.affiliation | Wong, Rachel, The Walter and Eliza Hall Institute of Medical Research | en_AU |
local.contributor.affiliation | Lee, Brenda, Personalised Oncology Division, Walter and Eliza Hall Institute of Medical Research, Melbourne, VIC | en_AU |
local.contributor.affiliation | Burge, M, Royal Brisbane & Women's Hospital | en_AU |
local.contributor.affiliation | Tie, Jeanne, Walter and Eliza Hall Institute of Medical Research | en_AU |
local.contributor.affiliation | Yip, Desmond, College of Health and Medicine, ANU | en_AU |
local.contributor.affiliation | Nott, L., Royal Hobart Hospital | en_AU |
local.contributor.authoremail | u5086006@anu.edu.au | en_AU |
local.contributor.authoruid | Yip, Desmond, u5086006 | en_AU |
local.description.embargo | 2099-12-31 | |
local.description.notes | Imported from ARIES | en_AU |
local.identifier.absfor | 320402 - Applied immunology (incl. antibody engineering, xenotransplantation and t-cell therapies) | en_AU |
local.identifier.absfor | 321111 - Solid tumours | en_AU |
local.identifier.absfor | 321104 - Cancer therapy (excl. chemotherapy and radiation therapy) | en_AU |
local.identifier.ariespublication | a383154xPUB17212 | en_AU |
local.identifier.citationvolume | 38 | en_AU |
local.identifier.doi | 10.1007/s40273-020-00951-1 | en_AU |
local.identifier.scopusID | 2-s2.0-85089441743 | |
local.identifier.thomsonID | WOS:000560931500001 | |
local.identifier.uidSubmittedBy | a383154 | en_AU |
local.publisher.url | https://link.springer.com/ | en_AU |
local.type.status | Published Version | en_AU |
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