Unfolding Cardiac Amyloidosis -From Pathophysiology to Cure

Curr Med Chem. 2019;26(16):2865-2878. doi: 10.2174/0929867325666180104153338.


Deposition of amyloidogenic proteins leading to the formation of amyloid fibrils in the myocardium causes cardiac amyloidosis. Although any form of systemic amyloidosis can affect the heart, light-chain (AL) or transthyretin amyloidosis (ATTR) account for the majority of diagnosed cardiac amyloid deposition. The extent of cardiac disease independently predicts mortality. Thus, the reversal of arrest of adverse cardiac remodeling is the target of current therapies. Here, we provide a condensed overview on the pathophysiology of AL and ATTR cardiac amyloidoses and describe treatments that are currently used or investigated in clinical or preclinical trials. We also briefly discuss acquired amyloid deposition in cardiovascular disease other than AL or ATTR.

Keywords: Amyloidosis; desmin; fibrils; heart failure; immunoglobulin light chains; posttranslational modification; pre-amyloid oligomers; transthyretin..

Publication types

  • Review

MeSH terms

  • Amyloid Neuropathies, Familial / drug therapy*
  • Amyloid Neuropathies, Familial / physiopathology
  • Amyloid Neuropathies, Familial / therapy
  • Animals
  • Heart Diseases / drug therapy*
  • Heart Diseases / physiopathology
  • Heart Diseases / therapy
  • Humans
  • Immunoglobulin Light Chains / metabolism
  • Immunoglobulin Light-chain Amyloidosis / drug therapy*
  • Immunoglobulin Light-chain Amyloidosis / physiopathology
  • Immunoglobulin Light-chain Amyloidosis / therapy
  • Immunotherapy
  • Liver Transplantation
  • Prealbumin / metabolism
  • Protein Multimerization / drug effects
  • Stem Cell Transplantation


  • Immunoglobulin Light Chains
  • Prealbumin

Supplementary concepts

  • Amyloidosis, Hereditary, Transthyretin-Related