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In AL amyloidosis, chemotherapy and immunotherapy
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In AA amyloidosis, treatment of the underlying disease
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In amyloidosis caused by transthyretin protein deposits, medications that stabilize transthyretin or reduce production of the protein
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Sometimes, organ transplantation
Treatment to decrease or control symptoms and complications of amyloidosis can improve quality of life for people with all forms of amyloidosis. Specific treatments to slow or stop amyloid formation can help in certain forms of amyloidosis.
For AL amyloidosis, treatment focuses on the underlying plasma cell disorder. High doses of melphalan and stem cell transplantation can lead to prolonged remissions and improved survival. However, only 15 to 20% of people with newly diagnosed AL amyloidosis are eligible to receive this treatment because of increased risk of side effects. Alternatively, combining medications of different classes (daratumumab, cyclophosphamide, bortezomib, and dexamethasone [called dara-VCd]) is highly effective in treating AL amyloidosis. Other combinations of immunotherapy drugs are used in treating people with AL amyloidosis who relapse.
For AA amyloidosis, treating the underlying disease can reduce amyloid deposits. For AA amyloidosis caused specifically by familial Mediterranean fever, colchicine is very effective.
For amyloidosis caused by transthyretin protein deposits, medications such as diflunisal, tafamidis, and acoramidis can stabilize transthyretin protein (preventing it from forming amyloid fibrils) and thus slow progression of hereditary and wild type transthyretin amyloid disease. Tafamidis and acoramidis are particularly useful if the heart is affected. Gene therapies that reduce transthyretin production (such as patisiran and vutrisiran) can improve nervous system effects of hereditary disease. In transthyretin amyloid heart disease, gene therapies extend survival and reduce the frequency of heart-related hospitalizations.
Organ transplants (for example, a kidney or the heart) have extended the lives of some people with organ failure due to amyloidosis.
In hereditary transthyretin amyloidosis, liver transplantation may be used. Liver transplantation can slow progression of the disease because the liver is where the mutant protein is produced. The liver removed from a person with hereditary transthyretin amyloidosis is sometimes transplanted into people with a fatal liver disease such as cirrhosis or liver cancer. Such a “domino transplant” is possible because a liver from a person with familial transthyretin amyloidosis is otherwise a normally functioning liver. Although people who receive a liver from a person with familial transthyretin amyloidosis may eventually develop amyloidosis themselves, the transplant can save their lives in the short term.
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