Transthyretin Amyloidosis Cardiomyopathy Clinical Trial - ATTR-ACT

Contribution To Literature:

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

Description:

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).

Interpretation:

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.

References:

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: Geriatric Cardiology, Heart Failure and Cardiomyopathies, Prevention, Acute Heart Failure

Keywords: ESC Congress, ESC18, Amyloid, Amyloidosis, Cardiomyopathies, Geriatrics, Heart Failure, Prealbumin, Primary Prevention


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