Amyloidosis: Treatment Options

Approved by the Cancer.Net Editorial Board, 02/2015

ON THIS PAGE: You will learn about the different ways doctors use to treat people with this condition. To see other pages, use the menu on the side of your screen.

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

This section outlines treatments that are the standard of care (the best known treatments available) for amyloidosis. When making treatment plan decisions, patients are also encouraged to consider clinical trials as an option. A clinical trial is a research study to test a new approach to treatment to evaluate whether it is safe, effective, and possibly better than the standard treatment. Clinical trials may test such approaches as a new drug, a new combination of standard treatments, or new doses of current therapies. Your doctor can help you review all treatment options. For more information, see the About Clinical Trials and Latest Research sections.

Treatment overview

Different types of doctors often work together to create a patient’s overall treatment plan that combines different types of treatments, which is especially important because amyloidosis affects so many different organs. This is called a multidisciplinary team.

The treatment team is often made up of a:

  • Cardiologist, a doctor who treats heart conditions
  • Gastroenterologist, a doctor who specializes in conditions of the gastrointestinal tract
  • Pulmonologist, a doctor who specializes in conditions of the lungs
  • Nephrologist, a doctor who treats kidney problems
  • Hematologist, a doctor who specializes in blood disorders
  • Neurologist, a doctor who focuses on issues involving the brain and nervous system

Many times, the hematologist will take the lead in coordinating this multidisciplinary team. Health care teams may also include a variety of other health care professionals, including physician assistants, nurses, social workers, pharmacists, counselors, dietitians, and others.

Descriptions of the most common treatment options for amyloidosis are listed below. Treatment options and recommendations depend on several factors, including the type of amyloidosis, the type of underlying disease, possible side effects, and the patient’s preferences and overall health. Your care plan will also include treatment for symptoms and side effects. Take time to learn about all of your treatment options and be sure to ask questions about things that are unclear. Also, talk about the goals of each treatment with your doctor and what you can expect while receiving the treatment. Learn more about making treatment decisions.

Medication and chemotherapy

Medication may be used to control the problems associated with amyloidosis. Steroids have been shown to work well, and they are typically combined with other drugs.

Chemotherapy is the use of drugs to destroy abnormal cells, usually by stopping the cells’ ability to grow and divide. It is most commonly used to treat cancer; however, chemotherapy is also useful for other conditions, including amyloidosis. Chemotherapy may be given by a medical oncologist, a doctor who specializes in giving chemotherapy to treat cancer, or a hematologist.

During treatment for amyloidosis, chemotherapy is used to destroy abnormal cells in the blood. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein using a needle, a shot under the skin, or a pill or capsule that is swallowed (orally).

A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. A patient may receive one drug at a time or combinations of different drugs at the same time.

Some of the most common types of chemotherapy used to treat amyloidosis are cyclophosphamide (Cytoxan, Neosar) and melphalan (Alkeran) combined with dexamethasone (multiple brand names) and prednisone (multiple brand names).

Newer drugs being evaluated for amyloidosis include bortezomib (Velcade), lenalidomide (Revlimid), and pomalidomide (Pomalyst). Researchers continue to study these drugs to find out how well each one treats amyloidosis. Although no known therapy can reverse the buildup of amyloid proteins in tissues or organs, studies researching the combination of bortezomib, dexamethasone, and melphalan have shown that they may help the organs affected by amyloidosis work better.

The side effects of chemotherapy depend on the individual and the dose used, but they can include nausea, vomiting, and fatigue. These are generally manageable when they do occur, and they usually go away once treatment is finished. In addition, melphalan and similar drugs may lower the level of red blood cells, causing anemia. Although uncommon, some types of chemotherapy may cause long-term side effects, including damage to the bone marrow and other organs.

Learn more about the basics of 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.

Surgery

Surgery used to treat amyloidosis may include organ transplantation. Liver transplantation has been effective in treating certain types of hereditary amyloidosis. Kidney and heart transplantation may also work well. However, organ transplantation may not be appropriate for all patients, especially those who have large amounts of amyloid proteins that have been deposited in their organs. Talk with your doctor or a center with expertise in organ transplantation about your options.

Dietary therapy

Dietary therapy may be used to reduce the effect of the amyloidosis on a specific part of the body. This means doctors will recommend what to eat and drink and what to avoid. For example, if amyloidosis affects the heart or kidneys, a low-sodium diet may be recommended. It is important to note that, even though amyloid is a protein, there is no link between amyloidosis and eating protein-rich foods.

Stem cell transplantation/bone marrow transplantation

A bone marrow/stem cell transplant is a medical procedure in which the plasma cells in the bone marrow that produce amyloid protein are first destroyed by high doses of chemotherapy and then replaced by highly specialized cells, called hematopoietic stem cells, that develop into healthy bone marrow. Hematopoietic stem cells are blood-forming cells found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a peripheral blood stem cell transplant, rather than bone marrow transplant, because it is the stem cells collected from the bloodstream that are typically being transplanted, not the actual bone marrow tissue.

For some people with amyloidosis who have preserved organ function, peripheral blood stem cell transplantation may be useful. During this procedure, the patient is given high doses of chemotherapy to destroy blood cells that are making the amyloid proteins. The patient then receives healthy peripheral blood stem cells, which allows the body to start making healthy blood cells again. Survival can be significantly improved with high-dose chemotherapy and peripheral blood stem cell transplantation.  However, many patients are not candidates because of the severity of the amyloid protein buildup causing other organ dysfunction(s).

Before recommending stem cell 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.

Learn more about the basics of stem cell and bone marrow transplantation.

Getting care for symptoms and side effects

Amyloidosis and its treatment often cause side effects. Relieving a person’s symptoms and side effects is an important part of care. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.

Palliative care is any treatment that focuses on reducing symptoms, improving quality of life, and supporting patients and their families. Any person, regardless of age or type and stage of cancer, may receive palliative care. It works best when palliative care is started as early as needed.

People often receive treatment for amyloidosis and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, dietary therapy (see above), relaxation techniques, emotional support, and other therapies. You may also receive palliative treatments similar to those meant to eliminate the disease, such as chemotherapy. Talk with your doctor about the goals of each treatment in your treatment plan.

Before treatment begins, talk with your health care team about the possible side effects of your specific treatment plan and palliative care options. And during and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem so it can be addressed as quickly as possible. Learn more about palliative care

If treatment fails

Recovery from amyloidosis is not always possible. If the disease cannot be cured or controlled, it may be called advanced or terminal.

This diagnosis is stressful, and advanced disease is difficult to discuss for many people. However, it is important to have open and honest conversations with your doctor and health care team to express your feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families. Making sure a person is physically comfortable and free from pain is extremely important.

Patients who have advanced disease and who are expected to live less than six months may want to consider a type of palliative care called hospice care. Hospice care is designed to provide the best possible quality of life for people who are near the end of life. You and your family are encouraged to think about where you would be most comfortable: at home, in the hospital, or in a hospice environment. Nursing care and special equipment can make staying at home a workable alternative for many families. Learn more about advanced care planning.

After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and loss.

The next section in this guide is About Clinical Trials, and it offers more information about research studies that are focused on finding better ways to care for people with amyloidosis. Or, use the menu on the side of your screen to choose another section to continue reading this guide.