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Amyloidosis
Treatment

Treatment of amyloidosis is given to improve symptoms and extend life. Treatment can limit further production of amyloid proteins and, in some instances, promote the breakdown of amyloid proteins in affected organs. The type of treatment required varies depending on the type of amyloidosis and the patient's symptoms.

With secondary amyloid, the main goal of therapy is to treat the underlying condition — for example, taking an anti-inflammatory medication for rheumatoid arthritis or antibiotics for an infection.

In hereditary amyloid, liver transplantation has been the most effective therapy. The new liver does not produce the abnormal amyloid proteins and consequently the disease improves. Investigational drugs are also being evaluated to try and prevent this type of amyloid protein from depositing in organs.

For primary amyloid, treatments include the same agents used to treat multiple myeloma, such as chemotherapy, corticosteroid medicines (lenalidomide or thalidomide) and/or bortezomib (Velcade). These treatments slow organ deterioration and some have been shown to prolong life, but none provide a cure.

Because primary amyloid is such a difficult disease to treat and survival is limited, researchers have begun to investigate the use of high-dose chemotherapy with autologous stem cell transplantation as a means of prolonging survival. The initial results with autologous stem cell transplantation are encouraging.

Autologous Stem Cell Transplantation

Patients with primary amyloid undergo an extensive work-up to evaluate organ function and the effects that amyloidosis has had on the body. Those with adequate heart, liver and lung function are encouraged to proceed to autologous stem cell transplantation.

High-dose melphalan chemotherapy is administered over one day. Then the patient's own stem cells (bone marrow) are re-administered two to three days later. An additional three to four weeks are spent in the hospital awaiting recovery and growth of the bone marrow.

The hope is that this therapy will delay progression of the disease, and in some cases, improve symptoms through removal of the abnormal proteins from the organs. However, this therapy is not a cure, and amyloidosis will return in everyone. That said, we have had patients who have been successfully treated with stem cell transplantation and when their disease progressed, have been able to receive another stem cell transplant.

Several new investigational agents are being evaluated in the treatment of multiple myeloma, another plasma cell disorder. The hope is that some of these agents also may be effective in treating amyloidosis. For patients who are not candidates for stem cell transplantation, these agents may prove to be the best available treatment.

Patients with hereditary amyloid should be referred to the Liver Transplant Clinic for evaluation.

Reviewed by health care specialists at UCSF Medical Center.

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