ABSTRACT

Introduction and Objective: The treatment of transthyretin (TTR) cardiac amyloidosis is based on two fundamental strategies: supportive therapy and disease-modifying therapy. Supportive therapy aims to prevent and control disease complications, providing support to affected organs and improving patient quality of life. In contrast, disease-modifying therapy focuses on interrupting or slowing the production and accumulation of amyloid fibrils responsible for tissue damage. This therapy employs drugs that act at different stages of amyloidogenesis: silencing the TTR gene to reduce protein production, stabilizing circulating TTR to prevent its dissociation and monomer formation, and destroying and reabsorbing already formed amyloid deposits. The benefits, efficacy, and safety of these therapies as treatments for cardiac amyloidosis will be rigorously evaluated.

Methods: An literature review of studies published between 2013 and 2024 was conducted in electronic databases such as PubMed, Google Scholar, ELSEVIER, UpToDate, and ClinicalKey, with the primary objective of evaluating the efficacy and clinical relevance of fafamidis in cardiac amyloidosis.

Results: Clinical trials have shown that tafamidis meglumine is a safe and effective medication for the treatment

of ATTRv30M and ATTR-CM. The drug has been associated with a reduction in the rate of cardiovascular-related hospitalizations, improved cardiac function, and better quality of life in patients.

Conclusions: Tafamidis has proven to be an effective and safe therapy for transthyretin cardiac amyloidosis, significantly improving patient survival and quality of life. Although it faces accessibility challenges due to its cost, it stands as a cornerstone in the treatment of this disease. Continuous research is crucial to optimize its use and develop complementary therapies.

Key words: Cardiac amyloidosis; Tafamidis; Tetramer; Monomers; Transthyretin stabilizer; V30M mutation.