Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis
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Discussion
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Implication
Randomized trial demonstrates reduced all-cause mortality in transthyretin amyloid cardiomyopathy treated with tafamidis, indicating robust real-world clinical benefits.
Key Points
- To compare survival outcomes between tafamidis-treated and untreated patients with transthyretin amyloid cardiomyopathy in a contemporary real-world matched cohort.
- Data were pooled from an early access cohort (NCT02791230) and the Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) between 2019 and 2023 for patients with a mixed or predominantly cardiac phenotype.
- A 1:1 propensity score matching approach matched 283 patients treated with approved-dose tafamidis to 283 untreated patients based on baseline age, New York Heart Association class, and phenotype.
- Estimated adjusted survival probabilities for all-cause mortality in treated vs untreated patients were 90.2% vs 76.0% at 30 months and 85.3% vs 67.5% at 42 months.
- Tafamidis treatment was associated with a 74% lower risk of all-cause mortality compared with no treatment (hazard ratio 0.26; 95% CI 0.13–0.51), with no new safety signals detected.
Cite This Study
Siepen et al. (2026) studied this question.