Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial

Background Transthyretin cardiac amyloidosis (ATTR‐CM), found in 6% to 15% of cohorts with heart failure with preserved ejection fraction, has long been considered a rare disease with poor prognosis. New treatments have made it one of the few directly treatable causes of heart failure. This study so...

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Main Authors: Morris M. Kim, Mark Prasad, Yunwoo Burton, Clinton M. Kolseth, Yuanzi Zhao, Pranav Chandrashekar, Babak Nazer, Ahmad Masri
Format: Article
Language:English
Published: Wiley 2023-08-01
Series:Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
Subjects:
Online Access:https://www.ahajournals.org/doi/10.1161/JAHA.123.029705
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author Morris M. Kim
Mark Prasad
Yunwoo Burton
Clinton M. Kolseth
Yuanzi Zhao
Pranav Chandrashekar
Babak Nazer
Ahmad Masri
author_facet Morris M. Kim
Mark Prasad
Yunwoo Burton
Clinton M. Kolseth
Yuanzi Zhao
Pranav Chandrashekar
Babak Nazer
Ahmad Masri
author_sort Morris M. Kim
collection DOAJ
description Background Transthyretin cardiac amyloidosis (ATTR‐CM), found in 6% to 15% of cohorts with heart failure with preserved ejection fraction, has long been considered a rare disease with poor prognosis. New treatments have made it one of the few directly treatable causes of heart failure. This study sought to determine whether patients with ATTR‐CM, particularly those treated with tafamidis, have comparable survival to an unselected cohort with heart failure with preserved ejection fraction. Methods and Results We compared the clinical characteristics and outcomes between a single‐center cohort of patients with ATTR‐CM (n=114) and patients with heart failure with preserved ejection fraction enrolled in the TOPCAT (Treatment of Preserved Cardiac Function Heart Failure With an Aldosterone Antagonist) trial (n=1761, excluding Russia and Georgia). The primary outcome was a composite of all‐cause death, heart failure hospitalization, myocardial infarction, and stroke. Subgroup analysis of patients with ATTR‐CM treated with tafamidis was also performed. Patients with ATTR‐CM had higher rates of the primary composite outcome compared with patients enrolled in the TOPCAT trial (hazard ratio [HR], 1.44 [95% CI, 1.09–1.91]; P=0.01), with similar rates of all‐cause death (HR, 1.43 [95% CI, 0.99–2.06]; P=0.06) but higher rates of heart failure hospitalizations (HR, 1.62 [95% CI, 1.15–2.28]; P<0.01). Compared with patients enrolled in TOPCAT, patients with ATTR‐CM treated with tafamidis had similar rates of the primary composite outcome (HR, 1.30 [95% CI, 0.86–1.96]; P=0.21) and all‐cause death (HR, 1.10 [95% CI, 0.57–2.14]; P=0.78) but higher rates of heart failure hospitalizations (HR, 1.96 [95% CI, 1.27–3.02]; P<0.01). Conclusions Patients with ATTR‐CM treated with tafamidis have similar rates of all‐cause death compared with patients with heart failure with preserved ejection fraction, with higher rates of heart failure hospitalizations.
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spelling doaj.art-06f95632625c4ce8b8fab38b29d6eb5a2023-08-23T10:41:23ZengWileyJournal of the American Heart Association: Cardiovascular and Cerebrovascular Disease2047-99802023-08-01121510.1161/JAHA.123.029705Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT TrialMorris M. Kim0Mark Prasad1Yunwoo Burton2Clinton M. Kolseth3Yuanzi Zhao4Pranav Chandrashekar5Babak Nazer6Ahmad Masri7Center for Amyloidosis, Knight Cardiovascular Institute Oregon Health &amp; Science University Portland OR USACenter for Amyloidosis, Knight Cardiovascular Institute Oregon Health &amp; Science University Portland OR USACenter for Amyloidosis, Knight Cardiovascular Institute Oregon Health &amp; Science University Portland OR USACenter for Amyloidosis, Knight Cardiovascular Institute Oregon Health &amp; Science University Portland OR USACenter for Amyloidosis, Knight Cardiovascular Institute Oregon Health &amp; Science University Portland OR USACenter for Amyloidosis, Knight Cardiovascular Institute Oregon Health &amp; Science University Portland OR USADivision of Cardiology University of Washington Seattle WA USACenter for Amyloidosis, Knight Cardiovascular Institute Oregon Health &amp; Science University Portland OR USABackground Transthyretin cardiac amyloidosis (ATTR‐CM), found in 6% to 15% of cohorts with heart failure with preserved ejection fraction, has long been considered a rare disease with poor prognosis. New treatments have made it one of the few directly treatable causes of heart failure. This study sought to determine whether patients with ATTR‐CM, particularly those treated with tafamidis, have comparable survival to an unselected cohort with heart failure with preserved ejection fraction. Methods and Results We compared the clinical characteristics and outcomes between a single‐center cohort of patients with ATTR‐CM (n=114) and patients with heart failure with preserved ejection fraction enrolled in the TOPCAT (Treatment of Preserved Cardiac Function Heart Failure With an Aldosterone Antagonist) trial (n=1761, excluding Russia and Georgia). The primary outcome was a composite of all‐cause death, heart failure hospitalization, myocardial infarction, and stroke. Subgroup analysis of patients with ATTR‐CM treated with tafamidis was also performed. Patients with ATTR‐CM had higher rates of the primary composite outcome compared with patients enrolled in the TOPCAT trial (hazard ratio [HR], 1.44 [95% CI, 1.09–1.91]; P=0.01), with similar rates of all‐cause death (HR, 1.43 [95% CI, 0.99–2.06]; P=0.06) but higher rates of heart failure hospitalizations (HR, 1.62 [95% CI, 1.15–2.28]; P<0.01). Compared with patients enrolled in TOPCAT, patients with ATTR‐CM treated with tafamidis had similar rates of the primary composite outcome (HR, 1.30 [95% CI, 0.86–1.96]; P=0.21) and all‐cause death (HR, 1.10 [95% CI, 0.57–2.14]; P=0.78) but higher rates of heart failure hospitalizations (HR, 1.96 [95% CI, 1.27–3.02]; P<0.01). Conclusions Patients with ATTR‐CM treated with tafamidis have similar rates of all‐cause death compared with patients with heart failure with preserved ejection fraction, with higher rates of heart failure hospitalizations.https://www.ahajournals.org/doi/10.1161/JAHA.123.029705ATTR cardiomyopathycardiac amyloidosisheart failure with preserved ejection fraction
spellingShingle Morris M. Kim
Mark Prasad
Yunwoo Burton
Clinton M. Kolseth
Yuanzi Zhao
Pranav Chandrashekar
Babak Nazer
Ahmad Masri
Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial
Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
ATTR cardiomyopathy
cardiac amyloidosis
heart failure with preserved ejection fraction
title Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial
title_full Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial
title_fullStr Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial
title_full_unstemmed Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial
title_short Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial
title_sort comparative outcomes of a transthyretin amyloid cardiomyopathy cohort versus patients with heart failure with preserved ejection fraction enrolled in the topcat trial
topic ATTR cardiomyopathy
cardiac amyloidosis
heart failure with preserved ejection fraction
url https://www.ahajournals.org/doi/10.1161/JAHA.123.029705
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