The heart and the kidneys are the most commonly involved organs in systemic
amyloidosis. Cardiac involvement is associated with an increased morbidity, treatment intolerance, and poorer overall survival. The most common types of
amyloidosis that are associated with cardiac involvement include light chain (
AL) amyloidosis and
transthyretin (TTR)
amyloidosis (both mutant and wild type). The traditional first-line treatment for
AL amyloidosis includes
alkylator-based
chemotherapy or high-dose
melphalan followed by autologous
stem cell transplantation (ASCT). Novel agents, including
proteasome inhibitors,
immunomodulators, and
monoclonal antibodies, have shown promising activity in both frontline and relapsed settings. Orthotopic
heart transplantation (OHT) followed by ASCT has led to superior outcomes compared to OHT alone. Orthotopic
liver transplantation (OLT) is the first-line treatment for TTR
amyloidosis. However, progression of cardiac
amyloidosis after OLT is often noted due to deposition of wild TTR. Combined OLT and OHT also has a role in treatment and leads to superior outcomes in carefully selected candidates. Pharmacologic agents, including
diflunisal,
tafamidis, small interfering
ribonucleic acid, and
doxycycline, have shown promising activity in stabilizing TTR from misfolding into fibrils and are being actively investigated. Best supportive care and management of
heart failure symptoms with
diuretics are a mainstay of treatment in all cardiac
amyloidosis subtypes. Robust data on the benefit of
angiotensin-converting enzyme inhibitors,
angiotensin receptor blockers, or beta blockers in
amyloid cardiomyopathy is lacking.