Gender and Racial Distribution of Transthyretin Stabilizer Therapy in Patients With Transthyretin Cardiac Amyloidosis

ransthyretin amyloid cardiomyopathy (ATTR-CM) is characterized by the misfolding, deposition, and accumulation of transthyretin amyloid fibrils within the myocardium.Progressive infiltration leads to significantly thickened and rigid ventricular walls, diastolic dysfunction, conduction abnormalities, and eventually heart failure. 1 This increasingly under-recognized condition predominantly affects older adults, often diagnosed as restrictive cardiomyopathy in the eighth decade of life. 2 Historically, median survival for these patients has been poor due to the limited management options besides supportive care and, in some circumstances, organ transplant.1,2 Recently, the therapeutic landscape has evolved with the innovation of transthyretin stabilizing agents, which have significantly extended patient survival and enhanced quality of life.3,4 Following the landmark ATTR-ACT (Transthyretin Amyloidosis Cardiomyopathy Clinical Trial) in 2018, tafamidis became established as first line therapy for ATTR-CM.1,3 This benzoxazole derivative binds to thyroxine-binding sites of transthyretin tetramers and inhibits monomer formation.Patients who received tafamidis experienced a marked reduction in all-cause mortality, slowed progression of left ventricular systolic and diastolic dysfunction, decreased cardiovascular-related hospitalizations, and significantly lower rate in functional decline.1,3 Later, the ATTRibute-CM study in 2024 unveiled acoramidis, a novel therapy with more potent and robust (>90%) ATTR stabilization efficacy, which similarly demonstrated marked improvement hierarchical composite of morbidity, mortality, and functional capacity. 4 Here, our database-related study aims to provide updates in the current trends in gender and racial/ethnic distribution of ATTR-CM patients undergoing treatment with transthyretin stabilizers to promote improved representation and access to life-prolonging therapies.This study's data was collected from the TriNetX United States Collaborative Network, which provides access to electronic medical records (diagnoses, procedures, medications, laboratory values, and genomic information) from approximately 130 million patients from 73 health care organizations.This globally federated research database contains only aggregate, deidentified electronic medical record data per the deidentification standard defined in Section §164.514(a) of the HIPAA Privacy Rule.Therefore, this retrospective cohort study was exempt from approval by the University of Massachusetts Medical School institutional review board. What is the clinical question being addressed?How do current patterns of transthyretin stabilizer use across demographic groups relate to disparities in clinical outcomes among patients with ATTR-CM?What is the main finding?Racial minorities remain under-represented in ATTR-CM treatment groups and experience increased shortterm mortality, despite no difference in cardiovascular events.