From the Guidelines
Amyloidosis is a complex condition characterized by abnormal protein deposits in various organs, and its management should prioritize early diagnosis and treatment to prevent further organ damage and improve quality of life, with treatment options varying based on the type of amyloidosis and affected organs. The symptoms of amyloidosis can vary depending on the organs affected, but common symptoms include fatigue, weight loss, swelling in the legs and ankles, shortness of breath, numbness or tingling in hands and feet, enlarged tongue, skin changes, and heart rhythm abnormalities 1. For AL amyloidosis, the most common type, chemotherapy medications like bortezomib (Velcade), cyclophosphamide, and dexamethasone are often used to suppress abnormal plasma cells, and autologous stem cell transplantation may be recommended for eligible patients 1. In contrast, for ATTR amyloidosis, medications such as tafamidis (Vyndaqel, 20-80mg daily), patisiran (Onpattro, administered intravenously every 3 weeks), or inotersen (Tegsedi, weekly subcutaneous injection) can stabilize or slow disease progression 1. Some key points to consider in the management of amyloidosis include:
- Early diagnosis and treatment are crucial to prevent further organ damage and improve quality of life 1
- Treatment options vary based on the type of amyloidosis and affected organs 1
- Supportive care is essential and includes medications like diuretics for fluid retention, pain management, and dietary modifications 1
- Organ transplantation may be considered in severe cases, but the prognosis is generally poor for cardiac transplantation in AL amyloidosis 1 The most recent and highest quality study recommends a comprehensive and multidisciplinary approach to the management of cardiac amyloidosis, including the use of bortezomib-based regimens and autologous stem cell transplantation for eligible patients 1.
From the FDA Drug Label
VYNDAQEL and VYNDAMAX are prescription medicines used to treat adults with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) to reduce death and hospitalization related to heart problems. The symptoms of amyloidosis are not explicitly listed in the provided drug labels. Treatment options for amyloidosis include VYNDAQEL and VYNDAMAX, which are used to treat adults with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) to reduce death and hospitalization related to heart problems 2, 2, 2.
From the Research
Symptoms of Amyloidosis
- Organ dysfunction, morbidity, and early mortality are characteristic of light chain amyloidosis (AL) 3
- Cardiac and/or renal dysfunction are common, with some patients experiencing Mayo stage IIIB cardiac disease 3
- Hepatic AL amyloidosis can present with spontaneous hepatic hematoma and lack of significant serum light chain elevation 4
- Amyloidosis can cause progressive organ damage, with the most common type being immunoglobulin light chain amyloidosis (AL) caused by clonal plasma cells that produce misfolded light chains 5
Treatment Options for Amyloidosis
- Daratumumab in combination with cyclophosphamide, bortezomib, and dexamethasone is a standard frontline AL therapy 3
- Daratumumab, bortezomib, and limited-duration dexamethasone (Dara-Vd) is an alternative frontline regimen that produces rapid and deep hematologic and organ responses 3
- High-dose melphalan and stem cell transplantation is recommended for carefully selected patients 6
- Bortezomib-based regimens, including bortezomib, oral melphalan, and dexamethasone (BMDex), and bortezomib, cyclophosphamide, and dexamethasone (CyBorD or VCd), are recommended for transplant-ineligible patients 6
- The combination of bortezomib and dexamethasone (BD) is a valid option for patients with severe heart or kidney impairment 7
- Novel therapeutics, including proteosome inhibitors, immunomodulatory agents, and monoclonal antibodies, have shown promising results for relapsed/refractory disease 6