Amyloidosis

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.

Amyloidosis, a disease in which misfolded proteins form amyloid fibrils that deposit in tissues and organs including the heart, is a vexing condition with devastating consequences. Encouragingly, new treatment options for cardiac amyloidosis have emerged recently and there is increasing awareness of this disease, once considered very rare. However, the cost of these therapies has raised concerns about the ability of patients to access such therapies.

PHILADELPHIA — Tafamidis was associated with improvements in a variety of metrics of quality of life in patients with transthyretin amyloid cardiomyopathy, according to new data from the ATTR-ACT trial.