JACC. CardioOncology · 2025
A Multicenter Study of Contemporary Long-Term Tafamidis Outcomes in Transthyretin Amyloid Cardiomyopathy.
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Masri Ahmad, Bhattacharya Priyanka, Medoff Brent, Ejaz Ain U, Elman Miriam R, Chandrashekar Pranav, Ives Lauren, Santos Alfonsina Mirabal, Teruya Sergio L, Zhao Yuanzi, Huang Shuaiqi, Wang Xiaofeng, Sperry Brett W, Maurer Mathew S, Soman Prem, Hanna Mazen
Аннотация
BACKGROUND: Tafamidis improved survival and decreased cardiovascular hospitalizations in the ATTR-ACT trial. Due to improved recognition and earlier diagnosis, the epidemiology of transthyretin amyloid cardiomyopathy (ATTR-CM) is rapidly evolving.
OBJECTIVES: The authors sought to evaluate the contemporary long-term outcomes of patients with ATTR-CM treated with tafamidis.
METHODS: Patients with ATTR-CM who received at least 1 dose of tafamidis between 2018 and 2021 at 5 amyloidosis centers in the United States were enrolled. Primary outcome was all-cause mortality.
RESULTS: Among 624 patients, mean age was 76.9 ± 8.4 years, 12.5% were female, 17.5% were Black, and 17.5% had variant ATTR-CM. At the time of tafamidis start, 52% had NYHA functional class II, 34% had NYHA functional class III, 40% were in National Amyloidosis Center (NAC) Stage ≥II, 38% were in Columbia Stage ≥II, and the median NT-proBNP level was 1,914 (Q1-Q3: 957-3914) pg/mL. Over a median follow-up of 43.2 months (Q1-Q3: 25.2-52.8 months), 241 patients (38.6%) died. The probability of freedom from death at 65 months was 54.1% (95% CI: 47.4%-60.4%). Similarly, restricting the cohort to patients who received tafamidis within 6 months of their ATTR-CM diagnosis (n = 397, 63.6%) showed similar results, with a survival probability of 49.6% (95% CI: 37.6%-60.5%) at 65 months.
CONCLUSIONS: In a contemporary cohort of tafamidis-treated patients with ATTR-CM, 39% of patients died over a median of 43 months. Further work is needed to improve our understanding of ATTR-CM, its natural history, and how to further improve survival and prevent progression of heart failure.
OBJECTIVES: The authors sought to evaluate the contemporary long-term outcomes of patients with ATTR-CM treated with tafamidis.
METHODS: Patients with ATTR-CM who received at least 1 dose of tafamidis between 2018 and 2021 at 5 amyloidosis centers in the United States were enrolled. Primary outcome was all-cause mortality.
RESULTS: Among 624 patients, mean age was 76.9 ± 8.4 years, 12.5% were female, 17.5% were Black, and 17.5% had variant ATTR-CM. At the time of tafamidis start, 52% had NYHA functional class II, 34% had NYHA functional class III, 40% were in National Amyloidosis Center (NAC) Stage ≥II, 38% were in Columbia Stage ≥II, and the median NT-proBNP level was 1,914 (Q1-Q3: 957-3914) pg/mL. Over a median follow-up of 43.2 months (Q1-Q3: 25.2-52.8 months), 241 patients (38.6%) died. The probability of freedom from death at 65 months was 54.1% (95% CI: 47.4%-60.4%). Similarly, restricting the cohort to patients who received tafamidis within 6 months of their ATTR-CM diagnosis (n = 397, 63.6%) showed similar results, with a survival probability of 49.6% (95% CI: 37.6%-60.5%) at 65 months.
CONCLUSIONS: In a contemporary cohort of tafamidis-treated patients with ATTR-CM, 39% of patients died over a median of 43 months. Further work is needed to improve our understanding of ATTR-CM, its natural history, and how to further improve survival and prevent progression of heart failure.