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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 the production of the protein
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Sometimes organ transplantation
Treatment to reduce or control symptoms and complications of amyloidosis can improve the quality of life for people with all forms of amyloidosis. Specific treatments to slow or stop amyloid formation may help in certain forms of amyloidosis.
Before 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, you can combine medicines from different classes (daratumumab, cyclophosphamide, bortezomiband dexamethasone [called dara-VCd]) is highly effective in treating AL amyloidosis. Other combinations of immunotherapy drugs are used to treat people with AL amyloidosis who relapse.
Before AA amyloidosis, treating the underlying disease can reduce amyloid deposits. For AA amyloidosis caused specifically by familial Mediterranean fever, colchicine is very effective.
Before amyloidosis caused by transthyretin protein deposits, medicine such as diflunisal, tafamidisand acoramidis may stabilize transthyretin protein (preventing it from forming amyloid fibrils) and thus slow the progression of hereditary and wild-type transthyretin amyloid disease. Tafamidis and acoramidis are particularly useful if the heart is affected. Gene therapies that reduce the production of transthyretin (eg patisiran and vutrisiran) can improve the nervous system effects of inherited disease. In transthyretin amyloid heart disease, gene therapies prolong survival and reduce the frequency of cardiac-related hospitalizations.
Organ transplants (for example, a kidney or the heart) have prolonged the lives of some people with organ failure due to amyloidosis.
IN hereditary transthyretin amyloidosis, liver transplantation can be used. Liver transplantation can slow the 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 someone with familial transthyretin amyloidosis may eventually develop amyloidosis themselves, the transplant may save their lives in the short term.
