Treatment | Mechanism of action | Clinical outcome |
Plasma cell-directed therapies | Eliminate the production of amyloidogenic LC | Patients with advanced cardiac amyloidosis often die despite HR, whereas patients with less severe cardiac involvement may experience rapid improvement in cardiac biomarkers and cardiac function when FLC can be reduced by 50%–90%7 |
Cardiac support therapies and/or heart transplantation | Ameliorate symptoms of heart failure while patient is treated with other therapies or awaiting heart transplantation | Patients with isolated cardiac deposition of amyloid may respond best to transplantation, but these treatments are supportive and not disease modifying |
Amyloid-directed therapies | Inhibit amyloid fibril formation or neutralise and clear circulating and deposited LC aggregates | Published clinical trial data have demonstrated cardiac and other organ responses with anti-LC45 and anti-SAP47 immunotherapy Preliminary data suggest a potential survival benefit of doxycycline |
FLC, free light chain; HR, haematological response; LC, light chain; SAP, serum amyloid P component.