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

Mark H. Rozenbaum; Samuel Large; Rahul Bhambri; Michelle Stewart; Robert Young; Alexander Van Doornewaard; Noel Dasgupta; Ahmad Masri; Jose Nativi-Nicolau

doi:10.2217/cer-2021-0071

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

Mark H. Rozenbaum, Samuel Large, Rahul Bhambri, Michelle Stewart, Robert Young, Alexander Van Doornewaard, Noel Dasgupta, Ahmad Masri, Jose Nativi-Nicolau

Knight Cardiovascular Institute

Research output: Contribution to journal › Article › peer-review

7 Scopus citations

Abstract

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.

Original language	English (US)
Pages (from-to)	927-938
Number of pages	12
Journal	Journal of Comparative Effectiveness Research
Volume	10
Issue number	11
DOIs	https://doi.org/10.2217/cer-2021-0071
State	Published - Aug 2021

Keywords

ATTR-CM
diagnosis
disease simulation model
health outcomes
tafamidis
transthyretin amyloid cardiomyopathy

ASJC Scopus subject areas

Health Policy

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@article{37ace0ee0dee4bcdafec0d325a3418f4,

title = "Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy",

abstract = "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.",

keywords = "ATTR-CM, diagnosis, disease simulation model, health outcomes, tafamidis, transthyretin amyloid cardiomyopathy",

author = "Rozenbaum, {Mark H.} and Samuel Large and Rahul Bhambri and Michelle Stewart and Robert Young and Doornewaard, {Alexander Van} and Noel Dasgupta and Ahmad Masri and Jose Nativi-Nicolau",

note = "Publisher Copyright: {\textcopyright} 2021 ",

year = "2021",

month = aug,

doi = "10.2217/cer-2021-0071",

language = "English (US)",

volume = "10",

pages = "927--938",

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T1 - Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy

AU - Rozenbaum, Mark H.

AU - Large, Samuel

AU - Bhambri, Rahul

AU - Stewart, Michelle

AU - Young, Robert

AU - Doornewaard, Alexander Van

AU - Dasgupta, Noel

AU - Masri, Ahmad

AU - Nativi-Nicolau, Jose

PY - 2021/8

Y1 - 2021/8

N2 - 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.

AB - 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.

KW - ATTR-CM

KW - diagnosis

KW - disease simulation model

KW - health outcomes

KW - tafamidis

KW - transthyretin amyloid cardiomyopathy

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

Abstract

Keywords

ASJC Scopus subject areas

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