Transthyretin Cardiac Amyloidosis

A new AI model accurately detected cardiac amyloidosis from echocardiograms with higher accuracy vs. other amyloidosis risk scores, according to research published in the European Heart Journal.

Among patients with transthyretin amyloidosis with cardiomyopathy, vutrisiran, an RNA interference agent, reduced risk for death and recurrent CV events compared with placebo, according to the results of the HELIOS-B trial.

Alnylam Pharmaceuticals announced the positive top-line results of the phase 3 HELIOS-B trial of its investigational RNA interfering therapy vutrisiran for the treatment of transthyretin amyloidosis with cardiomyopathy.

A common cardiac amyloidosis-causing gene believed to be present in around 3% of the Black U.S. population is associated with increased risk for HF hospitalization and death, which increases with age, a speaker reported.

While currently there is no cure for systemic amyloidosis, treatment options have recently expanded and have led to improved survival for both wild-type transthyretin and light-chain type amyloidosis that most commonly affect the heart.

Dhruv S. Kazi, MD, cardiologist and cardiovascular health economist at the Smith Center for Outcomes Research, Beth Israel Deaconess Medical Center, spoke with Healio about how tafamidis (Vyndamax, FoldRx/Pfizer) has changed the outlook of amyloidosis, but cost may remain a substantial barrier to access.

Interest in transthyretin cardiac amyloidosis, or ATTR-CM, has grown in part due to three areas of recent advancement: imaging, recognition of the lesser-known disease and drug approvals.

Taimur Sher, MBBS, MD a hematologist, internist and oncologist at the Mayo Clinic Cancer Center who leads the Multispecialty Amyloidosis Clinic at Mayo Clinic in Jacksonville, Florida, spoke with Healio about how to recognize and diagnose transthyretin amyloidosis, the newest FDA-approved treatments and the importance of genetic counseling in disease management.