Approximately one-third of patients with transthyretin cardiac amyloidosis without heart failure developed symptoms of this condition after a median follow-up period of 3.7 years, according to a study published in the Journal of the American College of Cardiology: Cardiooncology. 

Transthyretin amyloid cardiomyopathy is a progressive disease that often results in early mortality. Advancements in noninvasive imaging modalities have increased early detection rates of this disease, although data regarding the effectiveness of early treatment is scarce. 

Studies indicate that individuals with transthyretin amyloid cardiomyopathy are highly susceptible to developing heart failure, especially among the elderly. Patients who develop heart failure tend to suffer from an extremely poor prognosis, having an estimated survival period of only 2 to 4 years after diagnosis. 

Nevertheless, transthyretin stabilizers such as tafamidis have shown promise in reducing hospitalizations and mortality in patients with heart failure associated with transthyretin amyloid cardiomyopathy. Because most studies only investigate the efficacy of these stabilizers in patients who have already developed heart failure, its benefits in early initiation are unknown. 

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The authors of the study set out to track the progress of patients with transthyretin cardiac amyloidosis who do not have heart failure and to investigate if disease-modifying therapies affect clinical outcomes. 

The research team recruited patients with the above condition from 4 European hospitals. Participants were required to be in functional class I according to the New York Heart Association guidelines. They also had to be free from any heart failure symptoms or heart failure medications. Among the 118 patients recruited, 53 were on transthyretin stabilizers. 

The results of the study indicate that 1 in 3 patients without heart failure developed heart failure symptoms after a median follow-up period of 3.7 years, demonstrating that this condition is highly likely to occur at some point in life. Nevertheless, patients who were on transthyretin stabilizers were less likely to develop heart failure and demonstrated improved survival. 

“Our results provide support to consider initiating stabilizing agents at early stages of the disease,” the authors concluded. 


Gonzales-Lopez E, Escobar-Lopez L, Obici L, et al. Prognosis of transthyretin cardiac amyloidosis without heart failure symptoms. J Am Coll Cardiol CardioOnc. Published online November 4, 2022. doi:10.1016/j.jaccao.2022.07.007