A Multicenter Study of Contemporary Long-Term Tafamidis Outcomes in Transthyretin Amyloid Cardiomyopathy

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.

The authors sought to evaluate the contemporary long-term outcomes of patients with ATTR-CM treated with tafamidis.

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.

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.

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.