ON THIS PAGE: You will learn about the different ways doctors use to treat people with this condition. Use the menu to see other pages.

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. Learn more about clinical trials in the About Clinical Trials and Latest Research sections of this guide.

How amyloidosis is treated

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, nurse practitioners, 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. Talk with your doctor about the goals of each treatment and what you can expect while receiving the treatment. These types of talks are called “shared decision making.”

Shared decision making is when you and your doctors work together to choose treatments that fit the goals of your/your child’s care. Shared decision making is particularly important for amyloidosis because there are different treatment options. Learn more about making treatment decisions.

Therapies using medication

Treatments using medication are used to destroy abnormal cells. Medication may be given through the bloodstream to reach abnormal cells throughout the body. When a drug is given this way, it is called systemic therapy. Medication may also be given locally, which is when the medication is applied directly to the cancer or kept in a single part of the body.

This type of medication is generally prescribed by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist, a doctor who specializes in treating blood disorders.

Systemic therapy is the use of medication to destroy abnormal cells. This type of medication is given through the bloodstream to reach abnormal cells throughout the body. Systemic therapies are generally prescribed by a hematologist or a medical oncologist, a doctor who specializes in treating cancer with medication.

Medications are often given through an intravenous (IV) tube placed into a vein using a needle or as a pill or capsule that is swallowed (orally). If you are given oral medications, be sure to ask your health care team about how to safely store and handle it.

The types of medications used for amyloidosis include:

• Chemotherapy

• Targeted therapy

Each of these types of therapies is discussed in more detail below. A person may receive 1 type of medication at a time or a combination of medications given at the same time. They can also be given as part of a treatment plan that includes surgery and/or bone marrow/stem cell transplantation.

It is also important to let your doctor know if you are taking any other prescription or over-the-counter medications or supplements. Herbs, supplements, and other drugs can interact with cancer medications, causing unwanted side effects or reduced effectiveness. Learn more about your prescriptions by using searchable drug databases.

Chemotherapy

Chemotherapy is the use of drugs to destroy abnormal cells, usually by keeping the abnormal cells from growing, dividing, and making more cells. It is most commonly used to treat cancer. However, chemotherapy is also useful for other, non-cancerous conditions, including amyloidosis.

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 the steroids 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 fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite, and diarrhea. These side effects usually go away after treatment is finished.

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.

Talk with your health care team about potential side effects of the chemotherapy you are prescribed and how they can be managed. Learn more about the basics of chemotherapy.

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 and limits 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 shown that people with newly diagnosed AL amyloidosis, the four-drug combination of subcutaneous daratumumab, bortezomib, cyclophosphamide, and dexamethasone is safe and effective. This treatment is now considered standard of care for most patients.

Other research 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. Talk with your health care team about how these can be relieved. 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 potential treatment options.

Bone marrow transplantation/stem cell transplantation

A bone marrow transplant is a medical procedure in which the bone marrow that contains the plasma cells that produce amyloid protein are destroyed by high doses of chemotherapy and then replaced by highly specialized cells. These cells, called hematopoietic stem cells, develop into healthy bone marrow. Hematopoietic stem cells are blood-forming cells found both in the bloodstream and in the bone marrow. This procedure is also called a peripheral blood stem cell transplant. This is 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 general health.

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

Physical, emotional, and social effects of amyloidosis

Amyloidosis and its treatment cause physical symptoms and side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative care or supportive care. It is an important part of your care that is included along with treatments intended to slow, stop, or eliminate amyloidosis.

Palliative care focuses on improving how you feel during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or type of illness, may receive this type of care. And it often works best when it is started right after a diagnosis. People who receive palliative care along with treatment for amyloidosis often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies.

Before treatment begins, talk with your doctor about the goals of each treatment in the treatment plan being recommended. You should also talk about the possible side effects of the specific treatment plan and palliative care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.

During treatment, your health care team may ask you to answer questions about your symptoms and side effects and to describe each problem. Be sure to tell the health care team if you are experiencing a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.

Learn more about the importance of tracking side effects in another part of this guide. Learn more about palliative care in a separate section of this website.

If treatment does not work

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 some 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 has special skills, experience, and knowledge to support patients and their families. Making sure a person is physically comfortable, free from pain, and emotionally supported is extremely important.

Patients who have advanced disease and who are expected to live less than 6 months may want to consider 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. Use the menu to choose a different section to read in this guide.