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

作者：Jonas Wixner, Angela Dispenzieri, Leslie Amass, Martin Carlsson, Steve Riley, Evan T. Powers, Jeffery W. Kelly, the THAOS investigators, Eve Cariou, José González‐Costello, Martha Grogan, Arnt V. Kristen, David Slosky, Edileide de BarrosCorreia, Juan González Moreno, Marco Luigetti, Márcia Waddington‐Cruz, Isabel Conceição, M. E. Roelofs-van Emden, Darae Kim, Miriam Freimer, Alberta L. Warner, Pablo García Pavía, Dianna Quan, Igor Diemberger, Hans Nienhuis, Edward J. Miller, Daniel J. Lenihan, Michael Praktiknjo, Cheng‐Yin Tan, Dania Mohty, Saša Živković, Firas Al Badarin, Ivailo Tournev, Violaine Planté‐Bordeneuve, Mitsuharu Ueda, Maria Alejandra Gonzalez Duarte Briseno, Christopher R. Mueller, Nowell M. Fine, Robert Brunkhorst, David Adams, Olga Azevedo, Chi‐Chao Chao, José Nativi Nicolau, José Tallaj, Roberto Fernández‐Torrón, Jocelyn Inamo, Sorina Bădeliță, Michael Polydefkis, Nitasha Sarswat, James M. Tauras, Teresa Coelho, Valeria Salutto, Diego Delgado, Carsten Tschöepe, Francisco Muñoz Beamud, Calogero Lino Cirami, Stephen S. Gottlieb, Brian Drachman, Hector Ventura · 发表于：Cardiology and Therapy · 年份：2025 · DOI：10.1007/s40119-025-00421-9 · 被引用次数：7 · 研究领域：Amyloidosis: Diagnosis, Treatment, Outcomes、Thyroid and Parathyroid Surgery、Coagulation, Bradykinin, Polyphosphates, and Angioedema

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 car...