Amyloidosis: Treatment Options

Approved by the Cancer.Net Editorial Board, 08/2016

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.

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

This section tells you the treatments that are the standard of care for amyloidosis. “Standard of care” means the best treatments known. When making treatment plan decisions, patients are also encouraged to consider clinical trials as an option. A clinical trial is a research study that tests a new approach to treatment. Doctors want to learn if it is safe, effective, and possibly better than the standard treatment. Clinical trials can test a new drug, a new combination of standard treatments, or new doses of standard drugs or other treatments. Your doctor can help you consider all your treatment options. To learn more about clinical trials, 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. This is especially important because amyloidosis affects so many different organs. This is called a multidisciplinary team.

The treatment team often includes these experts:

  • Hematologist. A doctor who specializes in blood disorders

  • 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

  • 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.

Medications used to treat amyloidosis

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

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 hematologist or a medical oncologist, a doctor who specializes in giving chemotherapy to treat cancer.

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 1 drug at a time or combinations of different drugs at the same time.

Some of the 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).

Side effects of chemotherapy

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. Some types of chemotherapy may cause uncommon long-term side effects, including damage to the bone marrow and other organs.

Anemia is common in people with amyloidosis, especially those who are receiving chemotherapy, such as melphalan. Anemia is an abnormally low level of red blood cells (RBCs). RBCs have an iron-containing protein called hemoglobin that carries oxygen to all parts of the body. If the level of RBCs is too low, parts of the body do not get enough oxygen and cannot work properly. Most people with anemia feel tired or weak.

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.

Targeted therapy

Targeted therapy is a treatment that targets specific genes, proteins, or the tissue environment that contributes to the development of amyloidosis. This type of treatment blocks the growth and spread of abnormal cells while limiting damage to healthy cells.

Recent studies show that not all abnormalities have the same targets. To find the most effective treatment, your doctor may run tests to identify genes, proteins, and other factors. This helps doctors better match each patient with the most effective treatment whenever possible. In addition, many research studies are taking place now to find out more about specific molecular targets and new treatments directed at them. Learn more about the basics of targeted treatments.

For amyloidosis, targeted therapies include anti-angiogenesis therapy, monoclonal antibodies, and proteasome inhibitors.

  • Immunomodulatory drugs. Drugs in this class include lenalidomide (Revlimid), pomalidomide (Pomalyst), and thalidomide (Synovir, Thalomid).

  • Monoclonal antibodies. A monoclonal antibody is a type of targeted therapy. It recognizes and attaches to a specific protein in the abnormal cells, and it does not affect cells that don’t have that protein. Drugs in this class include daratumumab (Darzalex) and elotuzumab (Empliciti). A second type of monoclonal antibody directly targets the amyloid itself. There are currently several of these antibodies in clinical trials.

  • Proteasome inhibitors. Proteasome inhibition is a type of targeted therapy. The drugs in this class target specific enzymes called proteasomes that digest proteins in the cells. This class includes bortezomib (Velcade), ixazomib (Ninlaro), and carfilzomib (Kyprolis).

Researchers continue to study these drugs to find out how well each treats amyloidosis. These drugs do not reverse the buildup of amyloid proteins in tissues or organs. However, studies researching the combination of bortezomib, dexamethasone, and melphalan have shown that they may help the organs affected by amyloidosis work better.

Recent studies have started to examine the role of treatments that can improve organ function by directly targeting the amyloid deposits. This type of treatment is still being studied in clinical trials.

Side effects of targeted therapy

Targeted therapy may cause different side effects from those usually linked with chemotherapy. Patients receiving targeted therapy may develop skin, hair, nail, and/or eye problems. Learn more about the skin reactions to targeted therapy.

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.

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 some normal 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 allow 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 cannot receive this treatment because the amyloid protein buildup has affected the function of other organs.

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 the 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 disease, 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. 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 6 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. It offers more information about research studies that are focused on finding better ways to care for people with amyloidosis. Or, use the menu to choose another section to continue reading this guide.