Functional Class and Biomarker Stability in Patients Treated with Tafamidis for Transthyretin Cardiac Amyloidosis

During a poster session at the 2020 American Heart Association virtual meeting, Pranav Chandrashekar, MD, Oregon Health & Science University, Portland, OR, and colleagues presented data that revealed decreased congestive heart failure (CHF) hospitalizations and improved survival outcomes in patients with transthyretin cardiac amyloidosis (ATTR-CM) treated with tafamidis.1

The benefits of tafamidis treatment have been reported previously in the ATTR-ACT trial in which tafamidis treatment improved survival and decreased hospitalizations due to CHF. Survival benefit emerged after approximately 18 months of treatment, with less clear benefit in those with New York Heart Association (NYHA) class III symptoms.2-4

In this real-world cohort of patients with ATTR-CM, the researchers evaluated the impact of treatment with tafamidis on short-term changes in NYHA class, biomarkers, and clinical outcomes.

Patients with ATTR-CM who were prescribed tafamidis were analyzed and prospectively assessed for NYHA class; N-terminal-pro B-type natriuretic peptide (NT-proBNP), troponin I, and outcomes of CHF hospitalizations and death data were also evaluated.4

In this study, tafamidis was prescribed to 51 patients with ATTR-CM (mean age, 73.3 ± 7 years, 100.0% male). Hereditary ATTR-CM was found in 6 of these patients, and 45 patients were found to have wild-type. At the time of treatment initiation with tafamidis, 6 patients were determined as NYHA class I (11.8%), 24 patients were determined as class II (47.1%), 20 patients were class III (39.2%), and 1 patient was class IV (2%). There were no significant differences before and after tafamidis treatment in time-averaged NT-proBNP, time-averaged median NYHA functional class, and time-averaged median troponin I.

After initiating treatment with tafamidis, patients were assessed over a median follow-up of 8 months (interquartile range [IQR], 6.0-11 months); within this time frame, approximately one-quarter of the patients (25.5%, 13 patients) were admitted for CHF hospitalization. In addition, a total of 15.7% of patients died at a median of 9.5 months (IQR, 2.5-12.5 months).

Tafamidis-treated ATTR-CM patients had stable functional status and biomarkers, including patients with NYHA class III symptoms. The rate of death at 10 months appeared comparable to that found in the ATTR-ACT trial in this real-world population, despite a higher proportion of patients with worse NYHA functional class.


  1. Chandrashekar P, Dale Z, Rashdan L, et al. Functional class, biomarker stability, and clinical outcomes of patients with transthyretin cardiac amyloidosis treated with tafamidis. Presented at: American Heart Association Scientific Sessions 2020; November 13-20, 2020. Abstract MP346.
  2. Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018;379:1007-1016.
  3. Li B, Alvir J, Stewart M. Extrapolation of survival benefits in patients with transthyretin amyloid cardiomyopathy receiving tafamidis: analysis of the tafamidis in transthyretin cardiomyopathy clinical trial. Cardiol Ther. 2020;9:535-540.
  4. Damy T, Garcia-Pavia P, Hanna M, et al. Efficacy and safety of tafamidis doses in the tafamidis in transthyretin cardiomyopathy clinical trial (ATTR-ACT) and long-term extension study. Eur J Heart Fail. 2020; DOI: 10.1002/ejhf.2027. Published online ahead of print.

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