Transthyretin cardiac amyloidosis: A treatable form of heart failure with a preserved ejection fraction

Trends Cardiovasc Med. 2021 Jan;31(1):59-66. doi: 10.1016/j.tcm.2019.12.003. Epub 2019 Dec 17.

Abstract

Cardiac amyloidosis (CA) is considered a rare disease with poor prognosis and limited therapeutic options. However, non-biopsy diagnostic modalities as well as emerging therapies are challenging this long-held belief. Radionuclide bone scintigraphy is increasingly being used in the diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CA). As such, it is expected that the number of patients diagnosed with ATTR-CA will continue to rise. Emerging therapies decrease the progressive morbidity and mortality associated with ATTR-CA. The importance of early recognition of ATTR-CA is imperative as prompt initiation of these novel agents is essential to maximize their therapeutic potential. Herein, we outline the current approach to diagnosis of ATTR-CA and review the therapeutic management of the disease.

Keywords: Amyloidosis; Cardiomyopathy; Heart failure; Inotersen; Patisiran; Tafamidis; Transthyretin.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Amyloid Neuropathies, Familial / diagnosis
  • Amyloid Neuropathies, Familial / mortality
  • Amyloid Neuropathies, Familial / physiopathology
  • Amyloid Neuropathies, Familial / therapy*
  • Cardiomyopathies / diagnosis
  • Cardiomyopathies / mortality
  • Cardiomyopathies / physiopathology
  • Cardiomyopathies / therapy*
  • Disease Progression
  • Early Diagnosis
  • Heart Failure / diagnosis
  • Heart Failure / mortality
  • Heart Failure / physiopathology
  • Heart Failure / therapy*
  • Humans
  • Prognosis

Supplementary concepts

  • Amyloidosis, Hereditary, Transthyretin-Related