Treatments and drugs

By Mayo Clinic Staff

Though there's no cure for amyloidosis, treatment may help manage signs and symptoms and limit further production of amyloid protein.

Treating primary systemic amyloidosis

Researchers are studying several therapies to determine their place in the treatment of amyloidosis. Ask your doctor whether these treatments are appropriate for you. For primary systemic amyloidosis, some of these include:
  • Medicines. Therapies include melphalan (Alkeran) or cyclophosphamide (Cytoxan) — chemotherapy agents that are also used to treat certain types of cancer — and dexamethasone, a corticosteroid used for its anti-inflammatory effects. These drugs interrupt the growth of the abnormal cells that produce amyloid protein.

    Researchers are investigating other types of chemotherapy regimens for amyloidosis. Several medications that are used in the treatment of multiple myeloma are being tested to see if they're effective in treating amyloidosis. These include bortezomib (Velcade), thalidomide (Thalomid) and a thalidomide derivative called lenalidomide (Revlimid). These drugs may be used alone or in combination with dexamethasone.

    Ask your doctor what clinical trials may be available to you. Undergoing chemotherapy for amyloidosis may cause side effects similar to those of chemotherapy for cancer treatment, such as nausea, vomiting, hair loss, infection and extreme fatigue.

  • Peripheral blood stem cell transplantation. Peripheral blood stem cell transplantation involves using high-dose chemotherapy and transfusion of previously collected immature blood cells (stem cells) to replace diseased or damaged marrow. These cells are your own (autologous transplant). Autologous transplant is the preferred approach. Not everyone is a candidate for this aggressive treatment, and it has serious risks, including death. In some research, results from stem cell transplants have been promising, although it's unclear yet whether overall survival rates are better for those given chemotherapy alone compared with those given chemotherapy and stem cell transplantation. Discuss the potential risks and benefits of this treatment with your doctor.

Treating secondary amyloidosis

If you have secondary amyloidosis, the goal of therapy is to treat the underlying condition — for example, taking an anti-inflammatory medication for inflammatory arthritis.

Treating hereditary amyloidosis

For hereditary amyloidosis, one possible therapy may be liver transplantation because the protein that causes this form of amyloidosis is made in the liver. In addition, researchers are investigating new drugs to stop the progression of inherited amyloidosis.

Treating complications

Because amyloidosis can cause a number of complications, you may also need treatment for those specific conditions, based on your signs and symptoms and affected organs.

For example, if amyloidosis affects your heart or kidneys, you may be asked to follow a low-salt diet to control fluid retention. Or your doctor may prescribe diuretics and other medications. In some cases, your doctor might prescribe medication for pain control.

If any of your organs are severely damaged from amyloid deposits, you may need an organ transplant. Although such a transplant won't cure you because amyloid deposits could potentially collect in the new organ as well, a transplant could prolong your life.

Aug. 06, 2011

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