Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy

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dc.contributor.authorRozenbaum, Mark H.
dc.contributor.authorLarge, Samuel
dc.contributor.authorBhambri, Rahul
dc.contributor.authorStewart, Michelle
dc.contributor.authorYoung, Robert
dc.contributor.authorvan Doornewaard, Alexander
dc.contributor.authorDasgupta, Noel
dc.contributor.authorMasri, Ahmad
dc.contributor.authorNativi-Nicolau, Jose
dc.contributor.departmentMedicine, School of Medicine
dc.date.accessioned2024-04-01T14:19:20Z
dc.date.available2024-04-01T14:19:20Z
dc.date.issued2021
dc.description.abstractAim: Delayed diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) represents a missed opportunity for intervention. This study estimates the health benefits of timely diagnosis and treatment with tafamidis. Methods: A disease simulation model was developed to predict health outcomes under scenarios of timely and delayed diagnosis and treatment. Efficacy and quality of life (QoL) profiles were derived from the pivotal tafamidis trial and diagnostic delay durations from the literature. Results: Timely diagnosis and treatment were predicted to extend mean life expectancy by 5.46 and 7.76 years, relative to delayed diagnosis, for wild-type and hereditary ATTR-CM, respectively. Corresponding QALY gains were 4.50 and 6.22. Conclusion: Timely diagnosis and treatment with tafamidis are predicted to significantly improve survival and QoL for ATTR-CM patients.
dc.eprint.versionFinal published version
dc.identifier.citationRozenbaum MH, Large S, Bhambri R, et al. Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy. J Comp Eff Res. 2021;10(11):927-938. doi:10.2217/cer-2021-0071
dc.identifier.urihttps://hdl.handle.net/1805/39653
dc.language.isoen_US
dc.publisherBecaris
dc.relation.isversionof10.2217/cer-2021-0071
dc.relation.journalJournal of Comparative Effectiveness Research
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 Internationalen
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.sourcePublisher
dc.subjectATTR-CM
dc.subjectDiagnosis
dc.subjectDisease simulation model
dc.subjectHealth outcomes
dc.subjectTafamidis
dc.subjectTransthyretin amyloid cardiomyopathy
dc.titleEstimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy
dc.typeArticle
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