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

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posted on 11.08.2021, 13:36 by Mark H Rozenbaum, Samuel Large, Rahul Bhambri, Michelle Stewart, Robert Young, Alexander van Doornewaard, Noel Dasgupta, Ahmad Masri, Jose Nativi-Nicolau


Introduction: 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 (HRQoL) 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 HRQoL for ATTR-CM patients.

· Supplementary material file S1. Sensitivity and scenario analysis results.

· Supplementary material file S2. Technical appendix.

o S2.1 Model overview

o S2.2 NYHA class (disease) progression profiles

o S2.3 Disease-related mortality

· Supplementary Figure S1. Parametric models of overall survival – Pooled tafamidis arms (On-treatment).

· Supplementary Figure S2. Parametric models of overall survival – Placebo arm (Off-treatment).


This work was supported by Pfizer.