Where Are We with Cardiac Amyloidosis - Education Partner Program - OnDemand-Where Are We with Cardiac Amyloidosis

Video Transcription

Video Summary

In the webinar led by cardiologists Dr. Justin Roden and Dr. Kevin Alexander, and nurse practitioner Amy Brunel, key insights into cardiac amyloidosis, particularly ATTRCA, were shared. The discussion explored current diagnostic practices and emerging therapies. Dr. Roden emphasized the pathophysiology of ATTRCA, noting that amyloid formation results from the destabilization of TTR tetramers, leading to misfolding and aggregation into fibrils that deposit in organs. Diagnostic strategies include free light chain assays, bone scintigraphy, and tissue biopsies, with MRI being useful for early detection.<br /><br />Dr. Alexander covered emerging therapies, including transthyretin stabilizers like tefamidase and aciramis, which have shown mortality benefits in clinical trials. Silencer therapies like patisserin are also noteworthy for addressing protein production in the liver. Newer therapies under investigation include CRISPR-Cas9-based gene editing and antibody approaches aimed at amyloid clearance.<br /><br />Case vignettes examined nuanced challenges in managing amyloidosis, including overlapping pathologies like aortic stenosis, genotype-positive cases, and the need for tailored screening and treatment strategies. The importance of early intervention and multidisciplinary care was emphasized to improve outcomes.<br /><br />Audience engagement highlighted questions about optimal screening protocols for genotype-positive, phenotype-negative patients, the role of MRI in monitoring, and the use of older treatments like diflunisal. The panel concluded with gratitude extended to the organizing and supporting bodies for advancing the conversation on amyloidosis management.

Keywords

cardiac amyloidosis

ATTRCA

diagnostic practices

emerging therapies

transthyretin stabilizers

silencer therapies

CRISPR-Cas9

multidisciplinary care

early intervention

MRI

genotype-positive

aortic stenosis