Amyloidosis - Introduction
ON THIS PAGE: You will find some basic information about an illness called amyloidosis, which is not cancer, and the parts of the body it may affect. This is the first page of Cancer.Net’s Guide to Amyloidosis. Use the menu to see other pages. Think of that menu as a roadmap to this entire guide.
About amyloidosis
Amyloidosis is a rare and serious illness. Amyloidosis occurs when normal proteins in the body become misshapen and clump together. These misshapen proteins are called amyloid deposits or fibrils. Over time, the amyloid deposits build up in organs and tissues in the body. Eventually, this buildup cause symptoms and organ failure because the organs and tissues are not able to work as well as they should.
Amyloid protein deposits can be found in specific organs, such as the lung, skin, bladder, or bowel, or they can be systemic. "Systemic" means that the deposits may be found throughout the body. Systemic amyloidosis is the most common.
Amyloidosis is a rare disorder. Although it is not a type of cancer, it may be associated with certain blood cancers like multiple myeloma . Because amyloidosis is rare, it has been difficult to study. However, doctors and researchers have begun to understand more about amyloidosis over the past few decades. Research continues to learn more about this illness, which can be life-threatening.
Types of amyloidosis
There are different types of amyloidosis. They are named for the type of protein that is affected by the illness. The types of amyloidosis include:
-
AL amyloidosis. This is the most common type of amyloidosis in the United States. AL amyloidosis is a disorder of the plasma cells. Plasma cells are a type of white blood cell responsible for the production of immunoglobulins or antibodies, which are a type of protein that fights infection. The amyloid proteins that build up in the tissues in this condition are known as light chains. They can either be kappa or lambda light chains. "AL" stands for amyloid and light chain.
In AL amyloidosis, the light chain proteins are misshapen and there are too many of them. These are deposited in tissues and can damage one or more organs. The heart, kidneys, nerves, and gastrointestinal system are the most common organs affected. Because AL amyloidosis is associated with the overproduction of plasma cell proteins, it is linked to multiple myeloma .
-
AA amyloidosis . AA amyloidosis is also called "autoimmune amyloidosis," "secondary amyloidosis," or “inflammatory amyloidosis.” In this condition, the amyloid protein that builds up in the tissues is called the A protein. AA amyloidosis is associated with some chronic diseases, such as diabetes, tuberculosis, rheumatoid arthritis, and inflammatory bowel disease. It may also be linked to aging. AA amyloidosis can affect the spleen, liver, kidneys, adrenal glands, and lymph nodes. Lymph nodes are tiny, bean-shaped organs that fight infection.
-
Hereditary or familial amyloidosis . Hereditary amyloidosis is rare. It can be passed from generation to generation within a family. The proteins produced in hereditary amyloidosis may cause problems with the heart and may cause carpal tunnel syndrome and eye abnormalities. The most common subtypes involve a protein called transthyretin (TTR). This is sometimes called ATTR amyloidosis.
This section covers AL, AA, and hereditary amyloidosis. Other types of amyloidosis include beta-2 microglobulin amyloidosis, which occurs in some patients with chronic kidney problems, and types of amyloidosis located in specific areas of the body.
The next section in this guide is Statistics . It helps explain how many people are diagnosed with amyloidosis and general survival rates. Use the menu to choose a different section to read in this guide.
Amyloidosis - Statistics
ON THIS PAGE: You will find information about the estimated number of people who will be diagnosed with amyloidosis each year. You will also read general information on surviving the disease. Remember survival rates depend on several factors, and no 2 people are the same. Use the menu to see other pages.
Every person is different, with different factors influencing their risk of being diagnosed with this disorder and the chance of recovery after a diagnosis. It is important to talk with your doctor about any questions you have around the general statistics provided below and what they may mean for you individually. The original sources for these statistics are provided at the bottom of this page.
How many people are diagnosed with amyloidosis?
Because amyloidosis is rare, the diagnosis is often delayed or the condition is not diagnosed. Therefore, it is difficult to know exactly how many people are affected by this disease. It is estimated that about 4,000 people in the United States develop amyloid and light chain (AL) amyloidosis each year. The disease is typically diagnosed between the ages of 50 and 65. However, people as young as 20 have also been diagnosed with AL amyloidosis.
Hereditary and autoimmune amyloidosis (AA) are less common than AL amyloidosis. Some research notes that the number of cases of AA amyloidosis are declining due to better treatments of the underlying inflammatory conditions.
What is the survival rate for amyloidosis?
The survival rate for AL amyloidosis varies based on several factors. These include a person’s age and general health and how well the treatment plan works. Other factors than can affect outcomes include how early the condition is diagnosed and if and how the heart is affected.
Talk with your doctor if you have any questions about this information. Learn more about understanding statistics .
Statistics and information adapted from the National Organization for Rare Disorders and Medscape websites. (All sources accessed February 2023.)
The next section in this guide is Risk Factors. It describes the factors that may increase the chance of developing amyloidosis. Use the menu to choose a different section to read in this guide.
Amyloidosis - Risk Factors
ON THIS PAGE: You will find out more about the factors that increase the chance of developing this condition. Use the menu to see other pages.
A risk factor is anything that increases a person’s chance of developing a disease. Although risk factors often influence the development of a disease, most do not directly cause the disease itself. Some people with several risk factors never develop amyloidosis, while others with no known risk factors do.
Most people who develop amyloidosis have no known risk factors. The cause is often not known. The following factors may raise a person’s risk of developing amyloidosis:
-
Age. The risk of amyloidosis increases as a person gets older. For AL amyloidosis, the majority of people diagnosed are older than 40.
-
Gender. Amyloidosis is more common in men than in women.
-
Other diseases. As explained in the Introduction , amyloidosis is sometimes linked with another disease. For instance, research shows that 12% to 15% of people with multiple myeloma also develop AL amyloidosis.
-
Family history. Hereditary amyloidosis can run in families. This may be due to a genetic change, called a mutation, that is passed down from generation to generation.
The next section in this guide is Symptoms and Signs . It explains what body changes or medical problems amyloidosis can cause. Use the menu to choose a different section to read in this guide.
Amyloidosis - Symptoms and Signs
ON THIS PAGE: You will find out more about body changes and other things that can signal a problem that may need medical care. Use the menu to see other pages.
The symptoms of amyloidosis can vary widely, depending on the specific organ or number of organs affected by the buildup of amyloid protein. People with amyloidosis may experience the following symptoms or signs. A symptom is something that only the person experiencing it can identify and describe, such as fatigue, nausea, or pain. A sign is something that other people can identify and measure, such as a fever, rash, or an elevated pulse. Together, signs and symptoms can help describe a medical problem.
Sometimes, people with amyloidosis do not have any of the signs and symptoms detailed below. Or, the cause of a symptom may be another medical condition. All of these factors can make diagnosing amyloidosis challenging because the symptoms may be similar to those of numerous other diseases and conditions that are more common than amyloidosis.
Symptoms of amyloidosis are usually determined by the organ or function that is affected by the protein buildup. For example:
-
Kidneys. Amyloidosis in the kidneys will reduce the kidneys’ ability to filter waste and break down proteins. As a result, large amounts of protein may be found in the urine, causing “foamy” urine. The kidneys may even stop working. Decreased urine output and changes in creatinine clearance tests, a blood test to measure kidney function, may be present.
-
Liver. Amyloidosis may cause the liver to grow larger and affect its ability to function normally. This may cause pain in the upper abdomen, swelling in the abdomen, and changes in liver enzymes that can be found using blood tests.
-
Heart. Amyloidosis of the heart may cause an irregular heartbeat, called an arrhythmia; enlarge the heart; and cause poor heart function, resulting in fluid buildup, an irregular heartbeat, shortness of breath, or chest pain.
-
Gastrointestinal tract. Amyloidosis of the gastrointestinal tract may cause problems with the digestion and absorption of food nutrients, diarrhea or constipation, bleeding, blockages, and a thickened tongue, called macroglossia. It may also cause problems with the esophagus, including gastroesophageal reflux disease (GERD).
-
Thyroid gland. Amyloidosis of the thyroid gland may cause a goiter, which is a noncancerous swelling of the thyroid gland.
-
Lungs. Amyloidosis of the lungs may cause problems with breathing, including shortness of breath.
-
Nervous system. Disorders of peripheral nerves are the most common neurological complication of amyloidosis. Patients may experience painful paresthesias (unusual sensations), numbness and balance difficulties, vomiting, diarrhea, constipation, sweating, or sexual problems. Numbness, tingling, or weakness in the arms or legs may develop. This condition is known as peripheral neuropathy . Carpal tunnel syndrome may also occur.
Other general symptoms of amyloidosis include:
-
Fatigue , which is extreme exhaustion or tiredness. It is a common problem for people with amyloidosis. Patients who feel fatigue often say that even a small effort, such as walking across a room, can seem like too much.
-
Unexplained weight loss
-
Anemia , which is a low level of red blood cells
-
Weak hand grip, which may arise from carpal tunnel syndrome
-
Skin changes, such as a rash around the eyes
-
Clay-colored stools
-
Joint pain
-
Risk of infection . People with AL amyloidosis are at increased risk of getting an infection. This is because this type of amyloidosis causes abnormalities in plasma cells. Plasma cells produce immunoglobulins, which help fight infection. A condition called leukopenia occurs when the body does not have enough white blood cells and is less able to fight off infections. Some white blood cells, called neutrophils, can destroy harmful bacteria and fungi. People with leukopenia or neutropenia , a low level of neutrophils, are more likely to develop a serious infection.
If you are concerned about any changes you experience, please talk with your doctor. Your doctor will ask how long and how often you’ve been experiencing the symptom(s), in addition to other questions. This is to help find out the cause of the problem, called a diagnosis.
If amyloidosis is diagnosed, relieving symptoms remains an important part of your care and treatment. This may also be called "palliative care" or "supportive care." It is often started soon after diagnosis and continued throughout treatment. Be sure to talk with your health care team about symptoms you experience, including any new symptoms or a change in symptoms.
The next section in this guide is Diagnosis . It explains what tests may be needed to learn more about the cause of the symptoms. Use the menu to choose a different section to read in this guide.
Amyloidosis - Diagnosis
ON THIS PAGE: You will find a list of common tests, procedures, and scans that doctors use to find the cause of a medical problem. Use the menu to see other pages.
Doctors use many tests to find, or diagnose, amyloidosis. Doctors may also do tests to learn which treatments could work best. A biopsy is the only sure way for the doctor to diagnose amyloidosis. Imaging tests may be used to find out whether organs, such as the heart or kidneys, are affected.
How amyloidosis is diagnosed
This section describes options for diagnosing amyloidosis. Not all tests listed below will be used for every person. Your doctor may consider these factors when choosing a diagnostic test:
-
Your signs and symptoms
-
Your age and medical condition
-
The results of earlier medical tests
In addition to a physical examination, the tests listed below may be used to diagnose amyloidosis. After these diagnostic tests are done, your doctor will review all of the results with you.
-
Laboratory tests. Doctors may take samples of the patient’s blood and urine to run tests to learn more about the patient’s disease and general health.
-
Bone marrow aspiration and biopsy. These 2 procedures are similar and often done at the same time to examine the bone marrow. Bone marrow has both a solid and a liquid part. A bone marrow aspiration removes a sample of the fluid with a needle. A bone marrow biopsy is the removal of a small amount of solid tissue using a needle.
A pathologist then analyzes the sample(s). A pathologist is a doctor who specializes in interpreting laboratory tests and evaluating cells, tissues, and organs to diagnose disease. A common site for a bone marrow aspiration and biopsy is the pelvic bone, which is located in the lower back by the hip. Doctors generally give a type of medication called “anesthesia” beforehand to numb the area. Anesthesia is medication that blocks the awareness of pain. Stronger types of anesthesia can also be used to lessen the pain.
-
Biopsy. A biopsy is the removal of a small amount of tissue for examination under a microscope. Other tests can suggest that amyloid proteins are present, but only a biopsy can make a definite diagnosis. A pathologist then analyzes the sample(s). When amyloidosis is suspected, tissue samples are most often taken from abdominal fat or bone marrow (see below). A sample may also be taken from the liver, nerves, heart, kidneys, or rectum. However, these are more invasive procedures, and a patient may need to stay in the hospital for these tests.
-
Ultrasound. An ultrasound uses sound waves to create a picture of the internal organs. Areas affected by amyloid protein make different echoes of the sound waves than healthy tissue. When the sound waves bounce back to the computer and are changed into images, the doctor can find these areas inside the body. An ultrasound of the abdominal area may be used to look for enlarged organs.
-
Heart evaluation. A heart evaluation, including an electrocardiogram (EKG or ECG) and an echocardiogram (echo) , will look for structural abnormalities in the heart and examine the motion of the walls of the heart.
-
Molecular testing. Your doctor may recommend running laboratory tests to identify specific genes, proteins, and other unique factors. Results of these tests can help determine your treatment options .
The next section in this guide is Types of Treatment . Use the menu to choose a different section to read in this guide.
Amyloidosis - Types of Treatment
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.
Amyloidosis - About Clinical Trials
ON THIS PAGE: You will learn more about clinical trials, which are the main way that new medical approaches are tested to see how well they work. Use the menu to see other pages.
What are clinical trials?
Doctors and scientists are always looking for better ways to care for patients with amyloidosis. To make scientific advances, doctors create research studies involving volunteers, called clinical trials. Every drug that is now approved by the U.S. Food and Drug Administration (FDA) was tested in clinical trials.
Clinical trials are used for all types and stages of amyloidosis. Many focus on new treatments to learn if a new treatment is safe, effective, and possibly better than the existing treatments. These types of studies evaluate new drugs, different combinations of treatments, new approaches to surgery, and new methods of treatment.
People who participate in clinical trials can be some of the first to get a treatment before it is available to the public. However, there are some risks with a clinical trial, including possible side effects and the chance that the new treatment may not work. People are encouraged to talk with their health care team about the pros and cons of joining a specific study.
Some clinical trials study new ways to relieve symptoms and side effects during treatment. Others study ways to manage the late effects that may happen a long time after treatment. Talk with your doctor about clinical trials for symptoms and side effects.
Deciding to join a clinical trial
People decide to participate in clinical trials for many reasons. For some patients, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other patients volunteer for clinical trials because they know that these studies are the only way to make progress in treating amyloidosis. Even if they do not benefit directly from the clinical trial, their participation may benefit future patients with the disease.
Insurance coverage and the costs of clinical trials differ by location and by study. In some programs, some of the expenses from participating in the clinical trial are reimbursed. In others, they are not. It is important to talk with the research team and your insurance company first to learn if and how your child’s treatment in a clinical trial will be covered. Learn more about health insurance coverage of clinical trials .
Sometimes people have concerns that, in a clinical trial, they may receive no treatment by being given a placebo or a “sugar pill.” When used, placebos are usually combined with standard therapy when they are used. Study participants will always be told when a placebo is used in a study. Find out more about placebos in clinical trials.
Patient safety and informed consent
To join a clinical trial, patients must participate in a process known as informed consent. During informed consent, the doctor should:
-
Describe all of the treatment options so that the person understands how the new treatment differs from the standard treatment.
-
List all of the risks of the new treatment, which may or may not be different from the risks of standard treatment.
-
Explain what will be required of each person in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.
-
Describe the purposes of the clinical trial and what researchers are trying to learn.
Clinical trials also have certain rules called "eligibility criteria" that help structure the research and keep patients safe. You and the research team will carefully review these criteria together.
Patients who participate in a clinical trial may stop participating at any time for personal or medical reasons. This may include that the new treatment is not working or there are serious side effects. Clinical trials are also closely monitored by experts who watch for any problems with each study. It is important that patients participating in a clinical trial talk with their doctor and researchers about who will be providing their treatment and care during the clinical trial, after the clinical trial ends, and/or if the patient chooses to leave the clinical trial before it ends.
Finding a clinical trial
Research through clinical trials is ongoing for many types of serious conditions. For specific topics being studied for amyloidosis, learn more in the Latest Research section.
Cancer.Net offers a lot of information about clinical trials in other areas of the website, including a complete section on clinical trials and places to search for clinical trials for a specific diagnosis .
In addition, you can find a free video-based educational program about clinical trials, in another section of this website.
The next section in this guide is Latest Research . It explains areas of scientific research for amyloidosis. Use the menu to choose a different section to read in this guide.
Amyloidosis - Latest Research
ON THIS PAGE: You will read about the scientific research being done to learn more about this condition and how to treat it. Use the menu to see other pages.
Doctors are working to learn more about amyloidosis, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with amyloidosis. The following areas of research may include new options for patients through clinical trials. Always talk with your doctor about the diagnostic and treatment options best for you.
-
Combination chemotherapy/other medication . New combinations of steroids and chemotherapy are being studied to treat amyloidosis.
-
Peripheral blood stem cell transplantation . Based on research in some medical centers, peripheral blood stem cell transplantation may be offered to patients more often for amyloidosis. See the Types of T reatment section for more information.
-
Palliative care/supportive care . Clinical trials are underway to find better ways of reducing symptoms and side effects of current amyloidosis treatments to improve comfort and quality of life for patients.
Looking for More About the Latest Research?
If you would like additional information about the latest areas of research regarding amyloidosis, explore these related items that take you outside of this guide:
- To find clinical trials specific to your diagnosis, talk with your doctor or search online clinical trial databases now .
- Visit the website of Conquer Cancer, the ASCO Foundation to find out how to help support research for cancer and other cancer-related conditions. Please note that this link takes you to a separate ASCO website.
The next section in this guide is Coping with Treatment . It offers some guidance in how to cope with the physical, emotional, social, and financial changes that amyloidosis and its treatment can bring. Use the menu to choose a different section to read in this guide.
Amyloidosis - Coping with Treatment
ON THIS PAGE: You will find out more about steps to take to help cope with physical, social, and emotional side effects. Use the menu to see other pages.
Every treatment can cause side effects or changes to your body and how you feel. For many reasons, people don’t experience the same side effects even when given the same treatment. This can make it hard to predict how you will feel during treatment.
As you prepare to start treatment for amyloidosis, it is normal to fear treatment-related side effects . It may help to know that your health care team will work to prevent and relieve side effects. This part of treatment is called “palliative care" or "supportive care.” It is an important part of your treatment plan, regardless of your age or disease.
Coping with physical side effects
Common physical side effects from each treatment option for amyloidosis are described in the Types of Treatment section. Learn more about side effects of amyloidosis and its treatment, along with ways to prevent or control them . Changes to your physical health depend on several factors, including the disease state, the length and dose of treatment, and your general health.
Talk with your health care team regularly about how you are feeling. It is important to let them know about any new side effects or changes in existing side effects. If they know how you are feeling, they can find ways to relieve or manage your side effects to help you feel more comfortable and potentially keep any side effects from worsening.
You may find it helpful to keep track of your side effects so it is easier to explain any changes with your health care team. Learn more about why tracking side effects is helpful .
Sometimes, side effects can last after treatment ends. Doctors call these long-term side effects. They call side effects that occur months or years after treatment late effects . Treating long-term side effects and late effects is an important part of survivorship care. Learn more by reading the Follow-up Care section of this guide or talking with your doctor.
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. Although amyloid is a protein, there is no link between amyloidosis and eating protein-rich foods.
Coping with emotional and social effects
You can have emotional and social effects after a diagnosis of amyloidosis. This may include dealing with a variety of emotions, such as anxiety or anger, or managing your stress level. Sometimes, people find it difficult to express how they feel to their loved ones. Some have found that talking to a social worker, counselor, or member of the clergy can help them develop more effective ways of coping and talking about amyloidosis.
You can also find coping strategies for emotional and social effects in a separate section of this website. This section includes many resources for finding support and information to meet your needs.
Coping with the costs of care
Treatment can be expensive. It is may be a source of stress and anxiety for people with amyloidosis and their families. In addition to treatment costs, many people find they have extra, unplanned expenses related to their care. For some people, the high cost stops them from following or completing their treatment plan. This can put their health at risk and may lead to higher costs in the future. Patients and their families are encouraged to talk about financial concerns with a member of their health care team. Learn more about managing financial considerations , in a separate part of this website.
Coping with barriers to care
Different groups of people experience different rates of new cancer cases and experience different outcomes from their diagnosis. These differences are called “health disparities.” Disparities are caused in part by real-world barriers to quality medical care and more often negatively affect racial and ethnic minorities , poor people , sexual and gender minorities (LGBTQ+) , adolescent and young adult populations , older adults , and people who live in rural areas or other underserved communities .
If you are having difficulty getting the care you need, talk with a member of your health care team or explore other resources that help support medically underserved people .
Caring for a loved one with amyloidosis
Family members and friends often play an important role in taking care of a person with amyloidosis. This is called being a caregiver. Caregivers can provide physical, practical, and emotional support to the patient, even if they live far away. Being a caregiver can also be stressful and emotionally challenging. One of the most important tasks for caregivers is caring for themselves .
Caregivers may have a range of responsibilities on a daily or as-needed basis, including:
-
Providing support and encouragement
-
Giving medications
-
Helping manage symptoms and side effects
-
Coordinating medical appointments
-
Providing a ride to appointments
-
Assisting with meals
-
Helping with household chores
-
Handling insurance and billing issues
A caregiving plan can help caregivers stay organized and help identify opportunities to delegate tasks to others. It may be helpful to ask the health care team how much care will be needed at home and with daily tasks during and after treatment. Use this 1-page fact sheet to help make a caregiving action plan. This free fact sheet is available as a PDF, so it is easy to print.
Learn more about caregiving or read the ASCO Answers Guide to Caring for a Loved One in English or Spanish .
Talking with your health care team about side effects
Before starting treatment, talk with your doctor about possible side effects. Ask:
-
Which side effects are most likely?
-
When are they likely to happen?
-
What can we do to prevent or relieve them?
Be sure to tell your health care team about any side effects that happen during treatment and afterward, too. Tell them even if you don’t think the side effects are serious. This discussion should include physical, emotional, social, and financial effects of amyloidosis.
Looking for More on How to Track Side Effects?
Cancer.Net offers several resources to help you keep track of your symptoms and side effects. Please note that these links will take you to other sections of Cancer.Net:
-
Cancer.Net Mobile: The free Cancer.Net mobile app allows you to securely record the time and severity of symptoms and side effects.
-
ASCO Answers Managing Pain: Get this 32-page booklet about the importance of pain relief that includes a pain tracking sheet to help patients record how pain affects them. The free booklet is available as a PDF, so it is easy to print.
-
ASCO Answers Fact Sheets: Read 1-page fact sheets on anxiety and depression , constipation , diarrhea , and rash that provide a tracking sheet to record the timing and severity of the side effect. These free fact sheets are available as a PDF, so they are easy to print, fill out, and give to your health care team.
The next section in this guide is Follow-up Care . It explains the importance of checkups after you finish amyloidosis treatment. Use the menu to choose a different section to read in this guide.
Amyloidosis - Follow-Up Care
ON THIS PAGE: You will read about your medical care after amyloidosis treatment is completed and why this follow-up care is important. Use the menu to see other pages.
Care for people diagnosed with amyloidosis does not end when active treatment has finished. Your health care team will continue to check to see if amyloidosis has come back, manage side effects, and monitor your overall health. This is called follow-up care.
Your follow-up care may include regular physical examinations, medical tests, or both. Doctors want to keep track of your recovery in the months and years ahead.
Rehabilitation may be recommended, and this could mean any of a wide range of services, such as physical therapy, career counseling, pain management, nutritional planning, and/or emotional counseling. The goal of rehabilitation is to help people regain control over many aspects of their lives and remain as independent as possible. Learn more about rehabilitation.
Watching for recurrence
One goal of follow-up care is to check for a recurrence, which means that the amyloidosis has come back. For most people with amyloidosis, the condition will recur and their body will start producing abnormal amyloid proteins again. During follow-up care, a doctor familiar with your medical history can give you personalized information about your risk of recurrence. Your doctor will ask specific questions about your health. Some people may have blood tests or imaging tests done as part of their regular follow-up care, but testing recommendations depend on several factors, including the type of amyloidosis first diagnosed and the types of treatment given.
The anticipation before having a follow-up test or waiting for test results may add stress to you or a family member. This is sometimes called "scanxiety." Learn more about how to cope with this type of stress.
Managing long-term and late side effects
Most people expect to experience side effects when receiving treatment. However, it is often surprising that some side effects may linger beyond the treatment period. These are called long-term side effects. In addition, other side effects called late effects may develop months or even years afterwards. Long-term and late effects can include both physical and emotional changes.
Talk with your doctor about your risk of developing such side effects based on your individual treatment plan and your overall health. If you had a treatment known to cause specific late effects, you may also have certain physical examinations, scans, or blood tests to help find and manage them. For example, long-term follow-up care for amyloidosis usually includes regular and careful monitoring of liver and kidney function.
Keeping personal health records
You and your doctor should work together to develop a personalized follow-up care talk with your doctor about sure to ask about any concerns you have about your future physical or emotional health. ASCO offers forms to help create a treatment summary to keep track of the treatment you received and develop a follow-up care plan once treatment is completed.
This is also a good time to decide who will lead your follow-up care. Some people continue to see their hematologist, while others transition back to the general care of their primary care doctor or another health care professional. This decision depends on several factors, including side effects, health insurance rules, and your personal preferences.
If a doctor who was not directly involved in your amyloidosis care will lead your follow-up care, be sure to share your treatment summary and follow-up care plan forms with them, as well as all future health care providers. Details about your amyloidosis treatment are very valuable to the health care professionals who will care for you throughout your lifetime.
Making healthy lifestyle choices
People treated for amyloidosis are encouraged to follow established guidelines for good health, such as maintaining a healthy weight, not smoking, eating a balanced diet, and having recommended screening tests. Talk with your doctor to develop a plan that is best for your needs. Moderate physical activity can help rebuild your strength and energy level. Your doctor can help you create an appropriate exercise plan based upon your needs, physical abilities, and fitness level. Learn more about making healthy lifestyle choices .
The next section offers Questions to Ask the Health Care Team to help start conversations with the health care providers taking care of you. Use the menu to choose a different section to read in this guide.
Amyloidosis - Questions to Ask the Health Care Team
ON THIS PAGE: You will find some questions to ask your doctor or other members of your health care team to help you better understand your diagnosis, treatment plan, and overall care. Use the menu to see other pages.
Talking often with the doctor is important to make informed decisions about your health care. These suggested questions are a starting point to help you learn more about your care and treatment. You are also encouraged to ask additional questions that are important to you. You may want to print this list and bring it to your next appointment, or download Cancer.Net’s free mobile app for a digital list and other interactive tools to manage your care. It may also be helpful to ask a family member or friend to come with you to appointments to help take notes.
Questions to ask after getting a diagnosis
-
Can you explain my pathology report (laboratory test results) to me?
-
What organs, tissues, and/or parts of my body are affected?
-
How does this type of amyloidosis usually progress? Is it possible to slow down amyloidosis? Is it curable?
Questions to ask about choosing a treatment
-
What are my treatment options?
-
Can my symptoms be controlled with medication alone?
-
What clinical trials are available for me? Where are they located, and how do I find out more about them?
-
What treatment plan do you recommend? Why?
-
What is the goal of each treatment? Is it to eliminate the underlying disease, help me feel better, or both?
-
Who will be part of my health care team, and what does each member do?
-
Who will be leading my overall treatment?
Questions to ask about managing side effects
-
What are the possible side effects of each treatment, both in the short term and the long term?
-
How will this treatment affect my daily life? Will I be able to work, exercise, and perform my usual activities?
-
Could this treatment affect my sex life? If so, how and for how long?
-
Could this treatment affect my ability to become pregnant or have children? If so, should I talk with a fertility specialist before treatment begins?
-
If I’m worried about managing the costs of care, who can help me?
-
What support services are available to me? To my family?
-
If I have questions or problems, who should I call?
Questions to ask about planning follow-up care
-
Are there any signs and symptoms I should watch for?
-
What long-term side effects or late effects are possible based on the treatment I received?
-
What follow-up tests will I need, and how often will those tests be needed?
-
How do I get a treatment summary and follow-up care plan to keep in my personal records?
-
Who will be leading my follow-up care?
-
What support services are available to me after treatment has finished? To my family?
The next section in this guide is Additional Resources . It offers some more resources on this website that may be helpful to you. Use the menu to choose a different section to read in this guide
Amyloidosis - Additional Resources
ON THIS PAGE: You will find some helpful links to other areas of Cancer.Net that provide information about care and treatment for people with amyloidosis and other serious medical conditions. This is the final page of Cancer.Net’s Guide to Amyloidosis. To go back and review other pages, use the menu.
Cancer.Net includes many other sections about the medical and emotional aspects of being diagnosed with amyloidosis and other potentially life-threatening conditions, both for the patient and their family members and friends. This website is meant to be a resource for you and your loved ones from the time of diagnosis, through treatment, and beyond.
Beyond this guide, here are a few links to help you explore other parts of Cancer.Net:
-
Search for a cancer specialist in your local area using this free database of doctors from the American Society of Clinical Oncology.
-
Learn what medical phrases and terms used in cancer care and treatment mean.
-
Read more about the first steps to take when you are diagnosed with a potentially serious medical condition .
-
Find out more about clinical trials as a treatment option.
-
Get information about managing the financial costs of medical care.
-
Learn more about coping with the emotions that being diagnosed with a serious illness can bring, including those within a family or a relationship.
-
Find a national, not-for-profit advocacy organization that may offer additional information, services, and support for people with this condition.
-
Explore what to do when you finish treatment .
-
Download Cancer.Net Mobile , a free app that includes a symptom and side effect tracker, medication reminders, and other interactive resources.
-
To find a range of information and insights from different voices on timely topics, visit the Cancer.Net Blog .
This is the end of Cancer.Net’s Guide to Amyloidosis. Use the menu to choose a different section to read in this guide.
