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

Jan M Griffin; Julie L Rosenthal; Justin L Grodin; Mathew S Maurer; Martha Grogan; Richard K Cheng

doi:10.1016/j.jaccao.2021.06.006

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

JACC CardioOncol. 2021 Oct 19;3(4):488-505. doi: 10.1016/j.jaccao.2021.06.006. eCollection 2021 Oct.

Authors

Jan M Griffin¹, Julie L Rosenthal², Justin L Grodin³, Mathew S Maurer¹, Martha Grogan⁴, Richard K Cheng⁵

Affiliations

¹ Columbia University Irving Medical Center, New York, New York, USA.
² Mayo Clinic, Phoenix, Arizona, USA.
³ Division of Cardiology, Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, Texas, USA.
⁴ Mayo Clinic, Rochester, Minnesota, USA.
⁵ University of Washington Medical Center, Seattle, Washington, USA.

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.

Keywords: 6MWT, 6-minute walk test; AF, atrial fibrillation; AL, light chain amyloid; AS, aortic stenosis; ASO, antisense oligonucleotide; ATTR-CA, transthyretin cardiac amyloidosis; ATTRv, variant transthyretin cardiac amyloidosis; ATTRwt, wild-type transthyretin cardiac amyloidosis; CMR, cardiac magnetic resonance; DCCV, direct current cardioversion; HF, heart failure; LVEF, left ventricular ejection fraction; NT-proBNP, N-terminal pro–B-type natriuretic peptide; SAP, serum amyloid P component; TAVR, transcatheter aortic valve replacement; amyloidosis; cardiomyopathy; heart failure; siRNA, small interfering RNA.

Publication types

Review