Therapy of ATTR Cardiac Amyloidosis: Current Indications

Transthyretin cardiac amyloidosis is a restrictive cardiomyopathy caused by extracellular deposition in the heart of amyloid fibrils derived from plasma transthyretin (ATTR), either in its hereditary (ATTRh) or acquired (ATTRwt) forms. Cardiac amy-loidosis has a very poor prognosis if therapy is not started promptly. Therefore, it is very important to recognize cardiac amyloidosis early in order to imme-diately start a treatment capable of modifying the prognosis. Treatment of cardiac amyloidosis is not easy, often requiring a multidisciplinary team. New RNA-interfering drugs (such as patisiran) have been devised and are effective in the treatment of ATTRh amyloidosis. Tafamidis (a stabilizer of the native tetra -mer structure of TTR) is recommended to treat patients with genetic testing-proven hereditary hTTR-cardiomyopathy or wild-type TTR cardiomyopathy and NYHA Class I or II to reduce symptoms, CV hos-pitalization and mortality (Class I, level of evidence B). Patisiran should be considered in ATTRh cardio-myopathy with polyneuropathy. Thus, this review is intended to be a simple practical guide for the treat-ment of ATTR cardiac amyloidosis. (Curr Probl Car-diol 2023;48:101487.)

Automated summary

Transthyretin cardiac amyloidosis is a restrictive cardiomyopathy caused by the extracellular deposition of amyloid fibrils derived from plasma transthyretin. Early recognition and treatment are crucial for improving the prognosis. RNA-interfering drugs and stabilizers of the native tetramer structure of TTR have shown effectiveness in the treatment of different forms of cardiac amyloidosis.