Tafamidis in Transthyretin Cardiomyopathy Clinical Trial - ATTR-ACT

Contribution To Literature:

Highlighted text has been updated as of November 20, 2023.

The ATTR-ACT trial showed that tafamidis was superior at preventing major adverse events and functional decline in amyloidosis.


The goal of the trial was to evaluate tafamidis compared with placebo among patients with transthyretin amyloid cardiomyopathy. Tafamidis binds to transthyretin, which prevents tetramer dissociation and amyloidosis.

Study Design

  • Randomized
  • Parallel
  • Placebo
  • Blinded

Patients with amyloidosis were randomized to tafamidis (n = 264) versus placebo (n = 177). Tafamidis was given at a dose of 80 mg daily or 20 mg daily.

  • Total number of enrollees: 441
  • Duration of follow-up: 30 months
  • Mean patient age: 75 years
  • Percentage female: 9%

Inclusion criteria:

  • Patients 18-90 years of age with transthyretin amyloid cardiomyopathy

Exclusion criteria:

  • New York Heart Association class IV heart failure
  • Light chain amyloidosis
  • Liver or heart transplantation
  • Implanted cardiac device
  • Previous treatment with tafamidis
  • Renal insufficiency
  • Malnutrition

Principal Findings:

The primary outcome of all-cause death occurred in 29.5% of the tafamidis group compared with 42.9% of the placebo group (p < 0.05).

Secondary outcomes:

  • Cardiovascular-related hospitalization: 0.48 per year for tafamidis vs. 0.70 per year for placebo (p < 0.05)
  • Tafamidis vs. placebo was associated with a lower rate of decline for the 6-minute walk test and the Kansas City Cardiomyopathy Questionnaire–Overall Summary test (p < 0.001 for each).

Mean change in cardiac function from baseline to 30 months for tafamidis vs. placebo:

  • Left ventricular ejection fraction (LVEF): 2.09% (p = 0.13)
  • LV global longitudinal strain: -1.02% (p = 0.005)
  • Septal E/e’: -3.11 (p = 0.01)
  • Lateral E/e’: -2.35 (p = 0.006)


Among patients with transthyretin amyloid cardiomyopathy, tafamidis was superior to placebo at preventing death, cardiovascular-related hospitalizations, or functional decline. Tafamidis appears to be an effective treatment for transthyretin amyloid cardiomyopathy. Decline in LVEF was numerically less with tafamidis vs. placebo. Other measures of cardiac function were significantly improved with tafamidis vs. placebo, such as LV global longitudinal strain, septal E/e’, and lateral E/e’.


Shah SJ, Fine N, Garcia-Pavia P, et al. Effect of Tafamidis on Cardiac Function in Patients With Transthyretin Amyloid Cardiomyopathy: A Post Hoc Analysis of the ATTR-ACT Randomized Clinical Trial. JAMA Cardiol 2023;Nov 15:[Epub ahead of print].

Maurer MS, Schwartz JH, Gundapaneni B, et al., on behalf of the ATTR-ACT Study Investigators. Tafamidis Treatment for Patients With Transthyretin Amyloid Cardiomyopathy. N Engl J Med 2018;379:1007-16.

Editorial: Quarta CC, Solomon SD. Stabilizing Transthyretin to Treat ATTR Cardiomyopathy. N Engl J Med 2018;379:1083-4.

Presented by Dr. Claudio Rapezzi at the European Society of Cardiology Congress, Munich, Germany, August 27, 2018.

Clinical Topics: Heart Failure and Cardiomyopathies, Acute Heart Failure

Keywords: Amyloid, Cardiomyopathies, ESC18, ESC Congress, Heart Failure

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