Contemporary real-world treatment of cardiac transthyretin amyloidosis with tafamidis: a long-term multicentre study

Tafamidis was the first drug approved for transthyretin amyloid cardiomyopathy (ATTR-CM), but contemporary real-world evidence is lacking.

This retrospective, multicentre study included ATTR-CM patients enrolled after commercial availability of tafamidis 61 mg in Germany (April 2020 to March 2021), with treatment initiation based on physician clinical judgement. Primary and secondary endpoints were all-cause mortality and the composite of all-cause mortality and/or heart failure (HF) hospitalization, respectively. The cohort comprised 329 patients (median age 80 [76-83] years, 85% male and 8% variant ATTR) and 80% received tafamidis. During a median follow-up of 39 [18-48] months, 21% of patients died and 42% reached the composite endpoint. Tafamidis-treated patients had a 3-year survival of 86% and an event-free survival of 63%. After propensity-score matching for age, New York Heart Association (NYHA) class, National Amyloidosis Centre (NAC) stage, and serum albumin, tafamidis was significantly associated with lower all-cause mortality (hazard ratio [HR] 0.28; 95%-confidence interval [CI] 0.13-0.61; P = .001) and fewer composite events (HR 0.56; 95%-CI 0.33-0.97; P = .039). In tafamidis-treated patients, primary and secondary endpoints were associated with advanced NAC stage (P < .031; P < .009), age ≥80 years (P = .022; P = .004), and serum albumin ≤42.5 g/L (P = .015; P < .001), whereas NYHA class ≥III predicted only the composite endpoint (P = .059; P < .001).

This study provides long-term real-world outcome data for ATTR-CM patients treated with tafamidis based on physician clinical judgement. Adverse outcomes were strongly predicted by low serum albumin, advanced NAC stage, and older age, emphasizing the importance of early diagnosis and timely therapy of ATTR-CM.