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ATTR Amyloidosis: Current and Emerging Management Strategies : JACC: CardioOncology State-of-the-Art Review

ABSTRACT: Transthyretin cardiac amyloidosis (ATTR-CA) is increasingly diagnosed owing to the emergence of noninvasive imaging and improved awareness. Clinical penetrance of pathogenic alleles is not complete and therefore there is a large cohort of asymptomatic transthyretin variant carriers. Screening strategies, monitoring, and treatment of subclinical ATTR-CA requires further study. Perhaps the most important translational triumph has been the development of effective therapies that have emerged from a biological understanding of ATTR-CA pathophysiology. These include recently proven strategies of transthyretin protein stabilization and silencing of transthyretin production. Data on neurohormonal blockade in ATTR-CA are limited, with the primary focus of medical therapy on judicious fluid management. Atrial fibrillation is common and requires anticoagulation owing to the propensity for thrombus formation. Although conduction disease and ventricular arrhythmias frequently occur, little is known regarding optimal management. Finally, aortic stenosis and ATTR-CA frequently coexist, and transcatheter valve replacement is the preferred treatment approach. Central Illustration Highlights • ATTR-CA is increasingly recognized owing to improved awareness and emergence of noninvasive diagnostic pathways.• Clinical penetrance of pathogenic alleles is incomplete, with limited data for screening or management of asymptomatic carriers.• Biological understanding of ATTR-CA pathology has led to effective targeted therapies of TTR stabilizers and silencers.• Potential future treatments include CRISPR, amyloid extraction/degradation, and inhibition of amyloid seeding.

SUBMITTER: Griffin J

PROVIDER: S-EPMC8543085 | biostudies-literature |

REPOSITORIES: biostudies-literature

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