Aaron M Wolfson1, Kevin S Shah2, Jignesh K Patel3,4. 1. Sarver Heart Center, University of Arizona, 1501 North Campbell Avenue, Tucson, AZ, 85724, USA. 2. University of Utah Health Sciences Center, 30 North 1900 East, Rm 4A!00, Salt Lake City, UT, 84132, USA. 3. Heart Transplant Program, Cardiac Amyloidosis Program, 8536 Wilshire Blvd, 3rd floor, Beverly Hills, CA, 90211, USA. Jignesh.Patel@cshs.org. 4. Smidt Cedars-Sinai Heart Institute, 127 S San Vicente Blvd, Los Angeles, CA, 90048, USA. Jignesh.Patel@cshs.org.
Abstract
PURPOSE OF REVIEW: While morbidity and mortality remain high for amyloid cardiomyopathy (AC), increased awareness, earlier diagnosis, and advances in treatment have improved patient outcomes. This review will discuss the pathophysiology, contemporary diagnostic strategies, and novel and investigational therapeutic strategies for light-chain (AL) and transthyretin (ATTR) AC. RECENT FINDINGS: Diagnostic strategies for AC now include cardiac magnetic resonance imaging and bone scintigraphy. Proteosome inhibitor therapy is now front-line therapy for AL AC followed by autologous stem cell transplantation. Emerging disease-modifying strategies for ATTR AC include the recently FDA-approved TTR-stabilizer, tafamadis. ATTR gene-silencing therapy and amyloid fibril degradation therapy are two other strategies under investigation. Heart transplantation and durable mechanical circulatory support remain a final potential option; however, contemporary outcomes are improving with better patient selection. Patient outcomes for AC are expected to improve as increased awareness leads to earlier diagnosis and prompt treatment with emerging pharmacotherapy or advanced heart therapies.
PURPOSE OF REVIEW: While morbidity and mortality remain high for amyloid cardiomyopathy (AC), increased awareness, earlier diagnosis, and advances in treatment have improved patient outcomes. This review will discuss the pathophysiology, contemporary diagnostic strategies, and novel and investigational therapeutic strategies for light-chain (AL) and transthyretin (ATTR) AC. RECENT FINDINGS: Diagnostic strategies for AC now include cardiac magnetic resonance imaging and bone scintigraphy. Proteosome inhibitor therapy is now front-line therapy for AL AC followed by autologous stem cell transplantation. Emerging disease-modifying strategies for ATTRAC include the recently FDA-approved TTR-stabilizer, tafamadis. ATTR gene-silencing therapy and amyloid fibril degradation therapy are two other strategies under investigation. Heart transplantation and durable mechanical circulatory support remain a final potential option; however, contemporary outcomes are improving with better patient selection. Patient outcomes for AC are expected to improve as increased awareness leads to earlier diagnosis and prompt treatment with emerging pharmacotherapy or advanced heart therapies.
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