ArticleCardiology and therapy2025
Survival in a Contemporary, Real-World Cohort of Patients with Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: An Analysis from THAOS.
Article in Cardiology and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00628745 (Transthyretin Amyloidosis Outcomes Survey), which is not on this map. Cited by 9 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Transthyretin Amyloidosis Outcomes Survey (THAOS): A Global, Multi-Center, Longitudinal, Observational Survey of Patients With Documented Transthyretin Gene Mutations or Wild-Type Transthyretin Amyloidosis.
Who cites it
9 citing papers in PubMed.
- Neurologic Stability During Tafamidis Treatment in Transthyretin Amyloidosis with a Mixed Phenotype: A Multicenter Real-World Study.Cardiology and therapy · 2026Article
- Vutrisiran Health Care Resource Utilization, Costs, Discontinuation, and Mortality: A Real-World Claims Analysis.Advances in therapy · 2026Article
- Sex Differences in a Contemporary, Real-World Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis.Cardiology and therapy · 2026Article
- Transthyretin Amyloid Cardiomyopathy in Women: Epidemiology, Diagnostic Delay, and Clinical Implications.Cardiology and therapy · 2026Review
- Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis.Cardiology and therapy · 2026Article
- Disease modifying treatment of hereditary transthyretin amyloidosis with polyneuropathy in Germany - expert consensus of the German society of amyloid diseases (DGAK) and the German neurological society (DGN).Neurological research and practice · 2026Review
- Contemporary Description of Clinical Characteristics and Outcomes in Patients with Hereditary ATTR Amyloidosis: Results from the Multicountry OverTTuRe Study.Cardiology and therapy · 2026Article
- Gut-Heart Axis: Microbiome Involvement in Wild-Type Transthyretin Amyloidosis.International journal of molecular sciences · 2026Article
- Transthyretin Kinetic Stabilizers for ATTR Amyloidosis: A Narrative Review of Mechanisms and Therapeutic Benefits.Cardiology and therapy · 2025Review
Corrections and comments
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Authors and funding
8 authors.
Funding
Abstract
introductionTafamidis 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.
methodsThe 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.
resultsIn 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.
conclusionPatients 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 registrationClinicalTrials.gov identifier NCT00628745.
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