Treatment

The treatment of amyloidosis depends on many factors. In many cases, a team of doctors will work with the patient to determine the best treatment plan.

Often, the most effective treatment for amyloidosis is the treatment and control of the underlying disease, if present.

This section outlines treatments that are the standard of care (the best treatments available) for amyloidosis. Patients are also encouraged to consider clinical trials as a treatment option when making treatment plan decisions. A clinical trial is a research study to test a new treatment to prove it is safe, effective, and possibly better than standard treatment. Your doctor can help you review all treatment options. For more information, visit the Clinical Trials section.

Medication and chemotherapy

Medication may be used to control the problems associated with amyloidosis. Steroids, alone or in combination with other drugs, have been shown to be effective.

Chemotherapy is the use of drugs to kill abnormal cells and is most commonly used for the treatment of cancer. However, chemotherapy drugs are also useful in other conditions, including amyloidosis, when they are used to kill the abnormal plasma cells. Systemic chemotherapy is delivered through the bloodstream, targeting cells throughout the body. Chemotherapy may be given by a medical oncologist, a doctor who specializes in giving chemotherapy to treat cancer. Some people may receive chemotherapy in their doctor's office; others may go to the hospital. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a specific time.

The side effects of chemotherapy depend on the individual and the dose used, but can include fatigue, risk of infection, nausea and vomiting, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished.

Some of the most common chemotherapy drugs used to treat amyloidosis are chlorambucil (Leukeran), melphalan (Alkeran), and cyclophosphamide (Cytoxan, Neosar). Side effects can include nausea, vomiting, and fatigue, but are typically treatable when they do occur. Low levels of red blood cells (anemia) can occur with melphalan and similar drugs. Late effects are uncommon with these types of treatments.

Newer drugs being evaluated for amyloidosis include lenolidamide (Revlimid) and bortezomib (Velcade). These drugs are currently being studied in clinical trials to determine their effectiveness in amyloidosis.

Learn more about chemotherapy and preparing for treatment. The medications used to treat diseases are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications. Learn more about your prescriptions by using searchable drug databases.

Immunotherapy

Immunotherapy (also called biologic therapy) is designed to boost the body's natural defenses. It uses materials either made by the body or in a laboratory to bolster, target, or restore immune system function. Alpha interferon is a form of biologic therapy given as injections under the skin. This is sometimes used to help relieve symptoms caused by the condition, but it can have significant side effects including fatigue, depression, and flu-like symptoms. Learn more about immunotherapy.

Surgery

Surgery, including organ transplantation, may be used to treat amyloidosis. Liver transplantation has been effective in certain types of hereditary amyloidosis. Learn more about what to expect during surgery.

Dietary therapy

Dietary therapy may be used to decrease the production of amyloid proteins or to lessen the burden on affected organs. For example, if amyloidosis affects the heart or kidneys, a low-sodium diet may be recommended. Even though amyloid is a protein, there is no link between dietary intake of proteins and amyloidosis.

Stem cell transplantation/bone marrow transplantation

A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because blood stem cells are typically what is being transplanted, not the actual bone marrow tissue.

In some cases, stem cell transplantation may be used to introduce healthy bone marrow cells that will not make amyloid proteins. The real impact of stem cell transplantation on amyloidosis is still being studied in clinical trials.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO).

In an ALLO transplant, stem cells are obtained from a donor whose tissue matches the patient’s on a genetic level; this testing is called HLA-typing. Most often, a patient’s brother or sister serves as the donor, although unrelated donors can serve as the donor too. Millions of people worldwide have volunteered to donate stem cells for patients who do not have matched family members; your health care team will search a computer registry to look for a match. In addition, a donation of stem cells derived from umbilical cord blood is sometimes considered if family donors are not available.

In an AUTO transplant, the patient’s own stem cells are used. The stem cells are obtained from the patient when he or she is in remission from previous treatment. The stem cells are then frozen until they are needed, usually after the high-dose treatment (explained below) is completed.

In both types, the goal of transplantation is to destroy abnormal plasma cells and have replacement blood stem cells create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy the abnormal cells which are making the amyloid protein. This also destroys the patient’s bone marrow tissue and suppresses the patient’s immune system so that, in an ALLO transplant, the donor cells are not rejected by the body. After the high-dose treatment is given, blood stem cells are infused into the patient’s vein to replace the bone marrow and restore normal blood counts from donor cells. Sometimes, ALLO transplants can also be performed after giving lower doses of chemotherapy and/or radiation therapy that are still sufficient to suppress the immune system and allow growth of the donor cells. (These transplants, sometimes termed “mini-transplants” or “reduced intensity transplants” have less immediate side effects, allowing the procedure to be used for older patients.)

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of disease, results of any previous treatment, and patient’s age and general health.

For both ALLO and AUTO transplant types, the replacement cells engraft (begin to make new blood cells) and turn into healthy, blood-producing tissue in 10 days to three weeks. Destroying the patient’s own marrow reduces the body’s natural defenses, temporarily leaving the patient at an increased risk of infection. Until the patient’s immune system is back to normal, patients may need antibiotics and blood transfusions.

In an ALLO transplant, another major risk is that the donor’s cells will recognize the patient’s body as foreign, causing graft-versus-host disease (GVHD). GVHD may be a serious complication of allogeneic transplants and can be fatal. Other side effects may include liver problems, diarrhea, infections, and rashes. However, GVHD can also be a benefit, in that the donor cells can recognize the abnormal cells as foreign and destroy these cells, a mechanism that is one of the major reasons why ALLO transplantation generally works so well over the long term. The risk of GVHD can be reduced with exact HLA-type matching and the use of preventive drugs.

In an AUTO transplant, there is little risk of GVHD because the replacement stem cells are the patient’s own cells.

Learn more about bone marrow and stem cell transplantation.

Find out more about common terms used during treatment.