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Introduction <p>Tafamidis is approved to treat transthyretin amyloid cardiomyopathy (ATTR-CM). Many patients with ATTR-CM present with a mixed phenotype of both cardiac and neurologic symptoms, but real-world effectiveness studies of tafamidis in this population are lacking. This study assessed survival and other outcomes in a real-world, contemporary cohort of tafamidis-treated and untreated patients with mixed-phenotype ATTR-CM.</p> Methods <p>The Transthyretin Amyloidosis Outcomes Survey (THAOS) was a longitudinal, observational, phase&#xa0;4 study of patients with transthyretin amyloidosis and asymptomatic carriers of pathogenic transthyretin gene variants and was completed in June 2023. This analysis included a contemporary cohort of patients enrolled in THAOS in 2019–2023 who were characterized as having mixed-phenotype ATTR-CM at enrollment. The tafamidis-treated cohort received the approved dose of tafamidis (meglumine 80&#xa0;mg/free acid 61&#xa0;mg) throughout the study, and the untreated cohort never received tafamidis.</p> Results <p>In tafamidis-treated (<i>n</i> = 116) and untreated patients (<i>n</i> = 223), respectively, median age at enrollment was 77.8 and 72.8&#xa0;years, and 42.2% and 77.6% had variant ATTR-CM. Survival rates at 30&#xa0;months were 81.5% (95%&#xa0;CI 66.7–90.2) in tafamidis-treated patients and 75.1% (95%&#xa0;CI 66.1–82.0) in untreated patients. Median yearly incidence of cardiovascular-related hospitalizations was 0.89 for tafamidis-treated and 1.70 for untreated patients, and median duration of cardiovascular-related hospitalizations was 7.0 and 11.5&#xa0;days, respectively. There were 13 (11.2%) and 40 (17.9%) deaths in the respective groups.</p> Conclusion <p>Patients with mixed-phenotype ATTR-CM treated with the approved dose of tafamidis had numerically higher survival rates, a numerically lower rate of cardiovascular-related hospitalizations, and fewer deaths than untreated patients. These data parallel recent results for patients with predominantly cardiac ATTR-CM from THAOS and extend results of ATTR-ACT to a contemporary, real-world, mixed-phenotype population.</p> Trial Registration <p>ClinicalTrials.gov identifier NCT00628745</p>

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Survival in a Contemporary, Real-World Cohort of Patients with Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: An Analysis from THAOS

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  • 发表日期：

    2025年7月9日

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  • Jonas Wixner,
  • Angela Dispenzieri,
  • Leslie Amass,
  • Martin Carlsson,
  • Steve Riley,
  • Evan Powers,
  • Jeffery W. Kelly,
  • Eve Cariou,
  • Jose Gonzalez Costello,
  • Martha Grogan,
  • Arnt V. Kristen,
  • David Slosky,
  • Edileide de BarrosCorreia,
  • Juan Gonzalez Moreno,
  • Marco Luigetti,
  • Marcia Waddington-Cruz,
  • Isabel Conceicao,
  • Michele Emden,
  • Darae Kim,
  • Miriam Freimer,
  • Alberta Warner,
  • Pablo Garcia Pavia,
  • Dianna Quan,
  • Igor Diemberger,
  • Hans Nienhuis,
  • Edward Miller,
  • Daniel Lenihan,
  • Michael Praktiknjo,
  • Cheng Yin Tan,
  • Dania Mohty,
  • Sasa Zivkovic,
  • Firas Al Badarin,
  • Ivailo Tournev,
  • Violaine Plante-Bordeneuve,
  • Mitsuharu Ueda,
  • Maria Alejandra Gonzalez Duarte Briseno,
  • Christopher Mueller,
  • Nowell Fine,
  • Robert Brunkhorst,
  • David Adams,
  • Olga Azevedo,
  • Chi-Chao Chao,
  • Jose Nativi Nicolau,
  • Jose Tallaj,
  • Roberto Fernandéz Torrón,
  • Jocelyn Inamo,
  • Sorina Badelita,
  • Michael Polydefkis,
  • Nitasha Sarswat,
  • James Tauras,
  • Teresa Coelho,
  • Valeria Lujan Salutto,
  • Diego Delgado,
  • Carsten Tschoepe,
  • Francisco Munoz Beamud,
  • Calogero Lino Cirami,
  • Stephen Gottlieb,
  • Brian Drachman,
  • Hector Ventura

摘要

Introduction

Tafamidis is approved to treat transthyretin amyloid cardiomyopathy (ATTR-CM). Many patients with ATTR-CM present with a mixed phenotype of both cardiac and neurologic symptoms, but real-world effectiveness studies of tafamidis in this population are lacking. This study assessed survival and other outcomes in a real-world, contemporary cohort of tafamidis-treated and untreated patients with mixed-phenotype ATTR-CM.

Methods

The Transthyretin Amyloidosis Outcomes Survey (THAOS) was a longitudinal, observational, phase 4 study of patients with transthyretin amyloidosis and asymptomatic carriers of pathogenic transthyretin gene variants and was completed in June 2023. This analysis included a contemporary cohort of patients enrolled in THAOS in 2019–2023 who were characterized as having mixed-phenotype ATTR-CM at enrollment. The tafamidis-treated cohort received the approved dose of tafamidis (meglumine 80 mg/free acid 61 mg) throughout the study, and the untreated cohort never received tafamidis.

Results

In tafamidis-treated (n = 116) and untreated patients (n = 223), respectively, median age at enrollment was 77.8 and 72.8 years, and 42.2% and 77.6% had variant ATTR-CM. Survival rates at 30 months were 81.5% (95% CI 66.7–90.2) in tafamidis-treated patients and 75.1% (95% CI 66.1–82.0) in untreated patients. Median yearly incidence of cardiovascular-related hospitalizations was 0.89 for tafamidis-treated and 1.70 for untreated patients, and median duration of cardiovascular-related hospitalizations was 7.0 and 11.5 days, respectively. There were 13 (11.2%) and 40 (17.9%) deaths in the respective groups.

Conclusion

Patients with mixed-phenotype ATTR-CM treated with the approved dose of tafamidis had numerically higher survival rates, a numerically lower rate of cardiovascular-related hospitalizations, and fewer deaths than untreated patients. These data parallel recent results for patients with predominantly cardiac ATTR-CM from THAOS and extend results of ATTR-ACT to a contemporary, real-world, mixed-phenotype population.

Trial Registration

ClinicalTrials.gov identifier NCT00628745