What is the treatment for cardiac amyloidosis?

From the Guidelines

Treatment for cardiac amyloidosis should focus on managing the underlying cause and controlling symptoms, with daratumumab-CyBorD emerging as the standard of care for newly diagnosed AL amyloidosis, as shown in the ANDROMEDA study 1. The treatment approach for cardiac amyloidosis depends on the type of amyloidosis, with light chain (AL) amyloidosis requiring chemotherapy regimens like bortezomib, cyclophosphamide, and dexamethasone to target abnormal plasma cells, and autologous stem cell transplantation considered for eligible patients 1. Some key points to consider in the treatment of cardiac amyloidosis include:

• The use of daratumumab-CyBorD as the preferred induction therapy before HDM/SCT, offering great promise to the majority of patients who may not be candidates for SCT 1.
• The consideration of bortezomib-based regimens in combination with dexamethasone and an alkylating agent for patients who are not candidates for SCT 1.
• The importance of supportive care, including fluid and sodium restriction, regular monitoring of cardiac function, and management of arrhythmias 1.
• The need for individualized treatment plans based on amyloidosis type, disease stage, and patient characteristics 1.
• The potential use of tafamidis, patisiran, and inotersen for transthyretin (ATTR) amyloidosis, which can stabilize the transthyretin protein and slow disease progression [@Example@].

From the FDA Drug Label

VYNDAQEL and VYNDAMAX are indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. The treatment for cardiac amyloidosis is tafamidis (PO), which is indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization 2.

• The recommended dosage is either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally once daily or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally once daily 2.
• Key benefits of tafamidis include reducing cardiovascular mortality and cardiovascular-related hospitalization 2 2.

From the Research

Treatment Options for Cardiac Amyloidosis

The treatment for cardiac amyloidosis depends on the type of amyloidosis and the extent of cardiac involvement.

• For light-chain (AL) amyloidosis, treatment options include:
  • Bortezomib, cyclophosphamide, and dexamethasone (BCD) regimen, which has shown to be effective in achieving hematologic and cardiac responses 3, 4
  • Bortezomib, lenalidomide, and dexamethasone (VRD) regimen, which has also shown promising results in inducing deep hematologic responses 5
• For patients with severe cardiac impairment, bortezomib-containing regimens have been shown to be effective in reducing involved free light chain (iFLC) levels and improving cardiac function 6

Management Considerations

• Early diagnosis and treatment are crucial in managing cardiac amyloidosis, as effective therapies are more effective in early stages of disease before significant end-organ amyloid deposition has occurred 7
• Heart transplantation should be considered in carefully selected patients with end-stage cardiac amyloidosis, as transplant outcomes in these patients are now similar to those for other cardiomyopathies 7
• Aggressive screening of at-risk groups and identification of early signs of disease are essential in initiating timely treatment and improving patient outcomes 7