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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 deposits of the transthyretin protein, medications that stabilize transthyretin or reduce production of the protein.
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Sometimes organ transplant.
Treatment to reduce or control the 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.
For AL amyloidosis, Treatment focuses on the underlying plasma cell disorder. High doses of melphalan and stem cell transplant 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 due to the increased risk of side effects. Alternatively, combine medications from different classes (daratumumab, cyclophosphamide, bortezomiband dexamethasone [called dara-VCd]) is very effective in the treatment of AL amyloidosis. Other immunotherapy drug combinations are used to treat people with AL amyloidosis who relapse.
For AA amyloidosis, Treatment of the underlying disease can reduce amyloid deposits. For AA amyloidosis caused specifically by familial Mediterranean fever, colchicine It is very effective.
For amyloidosis caused by deposits of the protein transthyretin, medications like diflunisal, tafamidisand acoramidis can stabilize the transthyretin protein (preventing it from forming amyloid fibrils) and therefore slow the progression of hereditary and wild-type transthyretin amyloid disease. tafamidis and acoramidis They are particularly useful if the heart is affected. Gene therapies that reduce transthyretin production (such as patisiran and vutrisirán) can improve the nervous system effects of hereditary diseases. In transthyretin amyloid heart disease, gene therapies extend survival and reduce the frequency of heart-related hospitalizations.
Organ transplants (for example, a kidney or heart) have extended the lives of some people with organ failure due to amyloidosis.
In hereditary transthyretin amyloidosis, liver transplant can be used. Liver transplant can slow the progression of the disease because it is in the liver where the mutant protein is produced. The liver taken from a person with hereditary transthyretin amyloidosis is sometimes transplanted into people with a fatal liver disease such as cirrhosis or liver cancer. This “domino transplant” is possible because the liver of a person with familial transthyretin amyloidosis is an otherwise normally functioning liver. Although people who receive a liver from a person with familial transthyretin amyloidosis may develop amyloidosis, the transplant can save their life in the short term.
