From the Guidelines
Tafamidis is the recommended first-line therapy for the treatment of TTR amyloid cardiomyopathy, administered as either tafamidis meglumine 80 mg daily or tafamidis 61 mg daily, due to its proven ability to improve cardiovascular outcomes and reduce mortality 1. The current treatment guidelines for TTR amyloid cardiomyopathy prioritize disease-modifying therapies, with tafamidis being the only therapy to demonstrate improved cardiovascular outcomes in ATTR-CM patients 1.
Key Considerations
- Tafamidis works by binding to the thyroxin-binding site of TTR, preventing its dissociation and subsequent amyloid formation.
- The ATTR-ACT clinical trial showed that tafamidis reduced all-cause mortality and cardiovascular-related hospitalization in patients with NYHA class I to III symptoms 1.
- Tafamidis is expected to have greater benefit when administered early in the disease course, as it prevents but does not reverse amyloid deposition.
- Patients with noncardiac disease that is not expected to limit survival should be selected for tafamidis treatment, as the survival curves separate after 18 months 1.
Treatment Administration
- Tafamidis is available in two formulations: tafamidis meglumine 20-mg capsules and tafamidis 61-mg capsules.
- The FDA-approved dose for tafamidis meglumine is 80 mg (4 capsules) once daily, while the FDA-approved dose for the new formulation is 61 mg once daily 1.
Additional Therapies
- Supportive care focuses on managing heart failure symptoms with careful diuretic use, while traditional heart failure medications like beta-blockers and ACE inhibitors are often poorly tolerated.
- Anticoagulation is recommended for patients with atrial fibrillation regardless of CHADS2-VASC score due to high thromboembolism risk.
- Cardiac device therapy should be considered on an individual basis, and heart transplantation may be appropriate for selected younger patients with isolated cardiac involvement.
From the FDA Drug Label
VYNDAQEL and VYNDAMAX are transthyretin stabilizers indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. The recommended dosage is either: • VYNDAQEL 80 mg orally once daily, or • VYNDAMAX 61 mg orally once daily
The current guidelines for treatment of TTR amyloid cardiomyopathy include the use of transthyretin stabilizers such as tafamidis, with a recommended dosage of either VYNDAQEL 80 mg orally once daily or VYNDAMAX 61 mg orally once daily 2, 2, 2. The goal of treatment is to reduce cardiovascular mortality and cardiovascular-related hospitalization.
From the Research
Current Guidelines for Treatment of TTR Amyloid Cardiomyopathy
The current guidelines for the treatment of TTR amyloid cardiomyopathy (ATTR-CM) include the use of tafamidis, a TTR stabilizer, which has been approved for use in several countries 3, 4. Tafamidis has been shown to reduce all-cause mortality and frequency of cardiovascular-related hospitalizations in patients with ATTR-CM 3, 5.
Treatment Options
- Tafamidis: a TTR stabilizer that inhibits the formation of TTR amyloid fibrils 3, 4
- Diflunisal: an off-label agent that has been studied in ATTR-CM patients 6, 7
- Patisiran and inotersen: RNA interference therapies that are currently approved for ATTR-polyneuropathy of hereditary origin and are under investigation for ATTR-CM 6, 7
- Novel therapies: including monoclonal antibodies and gene silencers, which are in the early development phases 6, 7
Clinical Considerations
- Early diagnosis and treatment are crucial in improving survival in patients with ATTR-CM 5
- Treatment selection is based on subtype and presence of cardiac or neurological manifestations 7
- Side effects, monitoring, and administration of therapies should be carefully considered 7