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

Abstract

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.

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Supplementary
material file S1. Sensitivity and scenario analysis results.

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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

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

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