The treatment approach to amyloidosis depends on the type of amyloidosis a patient has as well as their current state of health.
Treatment of primary or AL amyloidosis
The treatments used in primary amyloidosis are the same as those used to treat multiple myeloma and include chemotherapy, corticosteroids and proteosome inhibitors. The aim of chemotherapy is to suppress the bone marrow cells and prevent production of the amyloid protein. The most commonly prescribed drug regimen includes melphalan and dexamethasone, a well tolerated and effective combination that is associated with a rapid response time.
An agent called bortezomib, one of a relatively new class of drugs called the proteosome inhibitors, has been shown to be effective in the treatment of primary amyloidosis and examples of the corticosteroid medications used include lenalidomide and thalidomide.
These treatments are all aimed at slowing disease progression and prolonging survival, but none can provide a cure. However, research has now started to focus on the effects of autologous stem cell transplantation in combination with high-dose chemotherapy and so far, the results seem to be promising.
Treatment of secondary or AA amyloidosis
Secondary amyloidosis is usually associated with conditions of chronic inflammation such as rheumatoid arthritis or inflammatory bowel syndrome. Typically, the deposited protein is the acute phase protein serum amyloid A. Medical or surgical therapy of the underlying condition can be helpful in preventing the progression of this amyloid. For example, an antibiotic may be administered in the case of infection or an anti-inflammatory agent in the case of arthritis.
Treatment of familial amyloidosis
This rare form of amyloidosis is caused by inherited mutations in genes that make the proteins that form fibrils. The main protein involved is the thyroid hormone-binding protein transthyretin. In most cases, the site of transthyretin production is the liver and liver transplant is an effective therapy. Two new agents called diflunisal and tafamidis are being investigated for their effectiveness in stabilizing the transthyretin and preventing it from depositing in organs.
Reviewed by Sally Robertson, BSc