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

Research output: Contribution to journalArticlepeer-review

2 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 languageEnglish (US)
Pages (from-to)927-938
Number of pages12
JournalJournal of Comparative Effectiveness Research
Volume10
Issue number11
DOIs
StatePublished - 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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