How Is Amyloidosis Treated?

Amyloidosis is a rare disease in which an abnormal protein called amyloid builds up in the organs. The heart, kidneys, liver, spleen, nervous system, or digestive tract are all organs that may be impacted. Amyloidosis may affect only one organ or several. It may eventually lead to organ failure.^1,2

There are several types of amyloidosis. Some are caused by a genetic mutation and some are caused by an underlying health condition. Sometimes the cause is unknown. The type of amyloidosis you have determines which treatment may be right for you.¹

Types of amyloidosis

Some of the most common types of amyloidosis are:^1-3

• AL or light-chain amyloidosis, the most common type that affects many organs
• AA amyloidosis, which mostly affects the kidneys and is typically caused by other conditions such as rheumatoid arthritis or inflammatory bowel disease
• ATTR or transthyretin amyloidosis, which includes the most common inherited form (hereditary or familial ATTR) and age-related ATTR (wild-type ATTR)

Other types include dialysis-related, APOA1, gelsolin, fibrinogen, and lysozyme amyloidosis.

Treatment goals

Amyloidosis cannot be cured, so treatment goals focus on 3 things:^1,4

• To try and slow how much and how fast the disease advances
• To reduce symptoms
• To prolong life

Types of amyloidosis treatments

Inherited forms of amyloidosis and those caused by genetic mutations are treated differently than forms caused by underlying conditions. If your amyloidosis is caused by an underlying condition, your doctor will probably try to treat that condition as a way to control the amyloidosis.

Amyloidosis is a complex disease and treatment options often depend on a person's overall health as well as their type of amyloidosis. Being treated at a center specializing in the condition, or at least having your treatment plan created by a specialist, is recommended. Some people have a treatment plan built by a specialist and managed by their local doctor. Treatment options may include:^1,2

• Chemotherapy
• Bone marrow or stem cell transplant
• Drugs
• Clinical studies

Chemotherapy

Some of the same drugs that treat cancer may also be used for complications of AL amyloidosis. Two older drugs used include melphalan (Evomela®) and dexamethasone (Dextenza®). Sometimes high-dose melphalan is given along with stem cell treatment. Some newer drugs include bortezomib (Velcade®) and lenalidomide (Revlimid®). In some cases, bortezomib, cyclophosphamide (Cytoxan®), and dexamethasone may be used together.²

Bone marrow transplant

Bone marrow transplant is also called stem cell transplant. This is another treatment option for AL amyloidosis, and is generally done in combination with chemotherapy. During a bone marrow transplant, healthy stem cells are removed from your blood or bone marrow.¹

You then get chemotherapy to kill the abnormal cells in your bone marrow (where blood cells are made). Then, your doctor will put the healthy stem cells back into your body. These healthy cells replace the unhealthy ones.¹

Heart medicines

Many times, amyloidosis attacks the heart. If your heart is affected, your doctor may prescribe blood thinners to reduce the risk of clots. You may also need drugs to control your heart rate or increase urination. These 2 types of drugs help ease stress on the heart. A well-balanced, low-salt diet may be necessary too.^2,4

ATTR treatments

Several drugs are used to treat transthyretin amyloidosis. These drugs work either by turning off the TTR gene or stabilizing the TTR protein. This stops more amyloid plaque from building up in the organs. Patisiran (Onpattro®), inoteresen (Tegsedi®), and eplontersen (Wainua™️) are TTR gene silencers that treat peripheral nerve disease caused by hereditary ATTR amyloidosis. Tafamidis meglumine (Vyndaqel®) treats heart disease caused by ATTR. In some cases of hereditary ATTR, liver transplant may be an option when the disease is not too far advanced.^2,4,5

Other treatments

AA amyloidosis caused by an underlying condition may be treated in a number of ways. Kidney transplant may help some people with kidney disease due to AA amyloidosis. A medical device called Lixelle® is being studied as a treatment for a type of dialysis-related amyloidosis.

There are a few clinical studies looking at more drugs that may help people with different forms of amyloidosis.