Novel strategies for the diagnosis and treatment of cardiac amyloidosis

Expert Rev Cardiovasc Ther. 2015 Nov;13(11):1195-211. doi: 10.1586/14779072.2015.1093936. Epub 2015 Oct 23.


Systemic amyloidoses are rare, complex diseases caused by misfolding of autologous protein. The presence of heart involvement is the most important prognostic determinant. The diagnosis of amyloid cardiac involvement relies on echocardiography and magnetic resonance imaging, while scintigraphy with bone tracers is helpful in differentiating light chain amyloidosis from other types of amyloidosis involving the heart. Although these diseases are fatal, effective treatments exist that can alter their natural history, provided that they are started before irreversible cardiac damage has occurred. Refined diagnostic techniques, accurate patients' stratification based on biomarkers of cardiac dysfunction, the availability of novel, more powerful drugs, and ultimately, the unveiling of the cellular mechanisms of cardiac damage created a favorable environment for a dramatic improvement in the treatment of this disease that we expect in the next few years.

Keywords: RNA-targeted therapy; amyloidosis; biomarkers; cardiac toxicity; chemotherapy; diagnosis.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Amyloidosis / diagnosis
  • Amyloidosis / therapy*
  • Cardiomyopathies / diagnosis
  • Cardiomyopathies / therapy*
  • Echocardiography
  • Humans
  • Magnetic Resonance Imaging / methods
  • Treatment Outcome