Transthyretin-Related Amyloidosis Medication

Updated: Jan 26, 2017
  • Author: Jefferson R Roberts, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Medication Summary

Liver transplantation remains the gold standard treatment for transthyretin-related amyloidosis (ATTR), and multi-organ transplantation (heart, liver and kidney) has been successful in slowing the natural course of the disease. The US Food and Drug Administration (FDA) has not approved any medical treatments for ATTR–familial amyloid polyneuropathy (ATTR-FAP). In Europe, tafamidis is approved for stage I FAP. Research continues to identify and refine effective medical treatments for preventing the deposition of amyloid fibrils.

Cautious, closely monitored medical treatment for ATTR cardiomyopathy and consideration of implantable cardioverter-defibrillator (ICD) placement are required, as the restrictive pathophysiology of the heart failure differs from the more common hypertrophic or dilated varieties and standard treatments such as beta-blockers, angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blockers (ARBs), or diuretics could worsen symptoms. Digoxin can increase local amyloid fibril levels in the heart [19]