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Amyloidosis is a group of diseases in which abnormal proteins, called amyloid proteins, accumulate in organs or organ systems such as the heart, kidneys, nervous system or gastrointestinal tract. There are different types of amyloidosis, dependent on the type of protein being deposited in tissues.

The most common form of amyloidosis in the United States is called primary amyloid. In primary amyloid, the protein being deposited is a part of the antibody protein called the light chain.

Primary amyloid is now a treatable disease. The mainstays of therapy are the same agents used to treat a related bone marrow cancer, called multiple myeloma. Treatment options include chemotherapy, corticosteroid medicines, biologic agents, and in some instances, autologous stem cell transplantation. The goal of therapy is to halt the buildup of amyloid protein in organs and to prolong life.

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Fortunately, amyloidosis is rare, affecting about eight out of every 1 million people annually. The majority of those affected are more than 40 years of age and 60 percent are male, although the condition can affect anyone. The cause is unknown.

Types of Amyloidosis

There are three main types of amyloidosis:

  • Primary Amyloid This is a plasma cell disorder originating in the bone marrow. Abnormal cells produce antibody proteins which deposit as amyloid. It is the most common type of amyloidosis in the United States, affecting 2,000 people each year.

    This condition, which is usually treated with chemotherapy, is not considered to be a type of cancer. However, it may be associated with multiple myeloma, which is a type of bone cancer. The severity of the illness often depends on the number and extent of organ involvement.
  • Secondary Amyloid This condition is caused by a chronic infection or inflammatory disease such as rheumatoid arthritis, familial Mediterranean fever (an intestinal disorder), osteomyelitis (infection of bone) or inflammatory disorders of the bowel. The amyloid deposits in this type of the disease are made up of a protein called the AA protein. Medical or surgical treatment of the underlying chronic infection or inflammatory disease can slow or stop the progression of this type of amyloid.
  • Familial (or Hereditary) Amyloid This rare form of amyloid is the only type that is inherited. It affects families of nearly every ethnic background. The deposits in this type are most commonly made up of the transthyretin protein produced in the liver. The condition results from a mutation in this protein. Liver transplant is used to cure this form of amyloid.

Our Approach to Amyloidosis

UCSF is dedicated to delivering the most advanced treatment options for amyloidosis with care and compassion. There are several types of amyloidosis, and the best therapy depends on the type and severity of the patient's condition. Treatments range from medications such as antibiotics to stem cell or liver transplants.

We are also dedicated to discovering even better treatments for amyloidosis through research. Interested patients may have the option to participate in clinical trials, which evaluate potential new therapies.

UCSF Health medical specialists have reviewed this information. It is for educational purposes only and is not intended to replace the advice of your doctor or other health care provider. We encourage you to discuss any questions or concerns you may have with your provider.