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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