Estimating the Health Benefits of Timely Diagnosis and Treatment of Transthyretin Amyloid Cardiomyopathy
Open accessAbstract
Aim: 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.
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Overlapping symptoms associated with various diseases are a major contributor to diagnostic errors.
“Overlapping symptoms associated with various diseases are a major contributor to diagnostic errors [122,123].”
From Toward Safer Diagnoses: A SEIPS-Based Narrative Review of Diagnostic Errors (2026) · cites this paper as [123]
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Symptom burden in heart failure: assessment, impact on outcomes, and management
Alpert, C. M., Smith, M. A., Hummel, S. L., & Hummel, E. K. (2017) · Heart Failure Reviews · doi:10.1007/s10741-016-9581-4 · Cited by 304 · see citing works
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Toward Safer Diagnoses: A SEIPS-Based Narrative Review of Diagnostic Errors
Yen, C., Epling, J. W., Rockwell, M., & Vaughn-Cooke, M. (2026) · Diagnostics · doi:10.3390/diagnostics16020347 · Cited by 6 · see citing works
- Authors
- Rozenbaum, M. H., Large, S., Bhambri, R., Stewart, M., Young, R., Doornewaard, A. V., Dasgupta, N., Masri, A., & Nativi-Nicolau, J.
- Affiliations
- Pfizer (Netherlands) (NL)
- Pfizer (United Kingdom) (GB)
- Pfizer (United States) (US)
- Health Economics and Outcomes Research (United Kingdom) (GB)
- Indiana University Indianapolis (US)
- Oregon Health & Science University (US)
- University of Utah (US)
- ORCID
- Venue
- Journal of Comparative Effectiveness Research
- Publisher
- Becaris Publishing Limited
- Published
- 2021-08
- Volume
- 10
- Issue
- 11
- Pages
- 927-938
- ISSN
- 2042-6305 (ISSN-L)
- 2042-6313
- OpenAlex
- W3170918622
- Cited by
- 19
- References
- 1
- Citations by year
- 2026: 2 · 2025: 6 · 2024: 2 · 2023: 6 · 2022: 3
- FWCI
- 0.86
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- cc-by-nc-nd
- Locations
- Journal of Comparative Effectiveness Research (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- IUScholarWorks (Indiana University) (submittedVersion, cc-by-nc-nd)
- Funders
- Pfizer
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Amyloidosis: Diagnosis, Treatment, Outcomes
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Health Systems, Economic Evaluations, Quality of Life
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Heart Failure Treatment and Management
- Keywords
- transthyretin amyloid cardiomyopathy, tafamidis, timely diagnosis, delayed diagnosis, health benefits, quality-adjusted life years, life expectancy, wild-type ATTR-CM, disease simulation model, survival
- MeSH terms
- Humans, Cardiomyopathies, Prealbumin, Quality of Life, Amyloid Neuropathies, Familial, Delayed Diagnosis
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