Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy

Eur J Heart Fail. 2021 Jun;23(6):895-905. doi: 10.1002/ejhf.2198. Epub 2021 May 24.

Abstract

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a life-threatening condition with a heterogeneous clinical presentation. The recent availability of treatment for ATTR-CM has stimulated increased awareness of the disease and patient identification. Stratification of patients with ATTR-CM is critical for optimal management and treatment; however, monitoring disease progression is challenging and currently lacks best-practice guidance. In this report, experts with experience in treating amyloidosis and ATTR-CM developed consensus recommendations for monitoring the course of patients with ATTR-CM and proposed meaningful thresholds and frequency for specific parameters. A set of 11 measurable features across three separate domains were evaluated: (i) clinical and functional endpoints, (ii) biomarkers and laboratory markers, and (iii) imaging and electrocardiographic parameters. Experts recommended that one marker from each of the three domains provides the minimum requirements for assessing disease progression. Assessment of cardiac disease status should be part of a multiparametric evaluation in which progression, stability or improvement of other involved systems in transthyretin amyloidosis should also be considered. Additional data from placebo arms of clinical trials and future studies assessing ATTR-CM will help to elucidate, refine and define these and other measurements.

Keywords: Amyloidosis; Cardiac imaging; Heart failure; Laboratory markers; Monitoring tools; Transthyretin amyloid cardiomyopathy.

Publication types

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

MeSH terms

  • Amyloid Neuropathies, Familial* / diagnosis
  • Cardiomyopathies* / diagnosis
  • Consensus
  • Heart Failure*
  • Humans
  • Prealbumin / genetics

Substances

  • Prealbumin