Cardiac Amyloidosis

No definitive therapy is available for cardiac amyloidosis. Treatment with alkylating agents such as melphalan has improved survival. Patients who do not respond to melphalan have benefited from high-dose chemotherapy with cyclophosphamide followed by peripheral stem cell transplantation. Most patients who have symptomatic cardiac involvement do not survive long enough to complete chemotherapy and therefore may not have the opportunity to show treatment benefit. Early diagnosis and treatment before the organ dysfunction has become irreversible are therefore essential to allow adequate duration of therapy. Lifesaving intervention with cardiac transplantation provides a window of opportunity in which other measures can be instituted.