What is Amyloidosis?

Amyloidosis is a group of diseases in which many different types of proteins accumulate in the body organs and tissue. These deposits damage the structure and function of the tissues and cause serious disease, which is often fatal if it affects major organs and is not treated.

There are three major types of amyloidosis that are all very different from one another.

AL Amyloidosis is a plasma cell disorder and is the most common type of amyloidosis in developed countries. The bone marrow controls cells that produce antibodies (proteins), which normally serve their purpose of fighting infection and disease, then are broken down and recycled in the body. In the case of AL amyloidosis, the light chain proteins are not broken down, build up in the bloodstream, and ultimately deposit in organs and tissues. The heart, kidneys, gastrointestinal track and nerves are most often affected.

Possible treatment options for AL amyloidosis include IV Melphalan with or without autologeous stem cell transplant or melphalan combined with dexamethasone. There currently are a number of open clinical trials investigating new drugs and chemotherapy agents.

AA Amyloidosis results from increased levels of a protein due to chronic infection or inflammatory disease such as rheumatoid arthritis, familial Mediterranean fever or osteomyelitis. The kidneys are the main site of amyloid deposition.

Treatment of the underlying infection or disease can slow or stop the progressive build up of amyloid proteins. There are also potential new drug treatments to allow dissolution of the amyloid proteins

Hereditary Amyloidosis is the only type of amyloidosis that can be inherited. There are several different types of protein, such as Transthyretin (TTR), Fibrinogen Aa, Apolipoprotein AI and AII and Lysozyme involved that are produced by mutated genes. Most often the proteins are manufactured in the liver, thus liver transplants have been used for treatment. There are drugs being investigated for the TTR form of this disease that may keep the TTR protein from breaking down into amyloid proteins.

For Patient Support and Awareness Information: www.amyloidosis.org

www.hematology.org