Transthyretin cardiac amyloidosis (ATTR-CA) is typically a late-onset disease caused by the deposit of transthyretin amyloid fibrils throughout the heart. When this occurs, various cardiac sequelae can develop, including hypotension, conduction abnormalities, and valvular lesions. The cardiomyopathy caused by ATTR-CA (ATTR-CM) has proven difficult to treat. Until recently, symptomatic management was the only therapeutic option, and many therapies used to treat congestive heart failure were ineffective or even detrimental to patients with ATTR-CM. In addition, treatment was limited to heart and liver transplantation. As a result, prognosis was poor. Recently, a few drug therapies have come to light as potential treatment modalities for ATTR-CM, most notably tafamidis, sold under the brand names Vyndaqel and Vyndamax. After the phase III Transthyretin Amyloidosis Cardiomyopathy trial displayed the drug's efficacy, it was given breakthrough therapy designation and was approved by the Food and Drug Administration on May 6, 2019, for the treatment of ATTR-CA. This novel therapy, as well as various other therapies in the pipeline, such as inotersen and patisiran, provide hope where, until recently, there was little. Unfortunately, the exorbitant cost of these new therapies may present a barrier to long-term treatment for some patients. However, by further improving diagnostic algorithms and incorporating these new treatments into our existing therapeutic modalities, patients with ATTR-CA should be able to live far longer than previously expected. Finally, further research combining these novel treatment modalities must be done, as they may prove to be additive or even synergistic in their treatment of ATTR amyloidosis.
Trial registration: ClinicalTrials.gov NCT03336580.
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