Welcome back to another AJMC Payer Perspectives series. In this opening episode titled, ‘Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Clinical Burden and Diagnostic Challenges,’ moderator, Dr. Ryan Haumschild, and panelist, Dr. Kevin Alexander, introduce a discussion focused on the clinical and economic burden of transthyretin amyloid cardiomyopathy (ATTR-CM), with an emphasis on diagnosis, treatment access, and payer considerations.
Dr. Alexander describes ATTR-CM as a progressive systemic disease in which the transthyretin protein misfolds and deposits as amyloid fibrils, primarily in the heart and peripheral nervous system. The condition carries significant morbidity and mortality, with patients experiencing worsening heart failure symptoms, including shortness of breath, leg swelling, and exercise intolerance, along with frequent hospitalizations and functional decline.