Amyloidosis

Amyloidosis

Signs and Symptoms
Diagnosis
Treatment

Treatment

Although there currently is no cure for amyloidosis, treatment 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 symptoms that are present.

With secondary amyloidosis, 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 amyloidosis, liver transplantation has been the most effective therapy. The new liver does not produce the abnormal amyloid proteins and consequently the disease improves.

For primary amyloidosis, treatment includes chemotherapy. Until recently, low-dose chemotherapy had been the treatment of choice. Low-dose oral chemotherapy, usually melphalan (also called Alkeran), has improved the median lifespan for patients with amyloidosis to approximately one and a half years.

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

Stem Cell Transplantation

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

High-dose melphalan chemotherapy is administered over one day. Then the patient's own stem cells are re-administered two to three days later and 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 amyloidosis, and in some cases, allow for improved symptoms through removal of the abnormal proteins from the organs. However, this therapy is not curative 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), and the hope is that some of these agents also may be affective in treating amyloidosis. For patients who are not candidates for stem cell transplantation these agents may prove to be the best available treatment.

Reviewed by health care specialists at UCSF Medical Center.
Last updated May 8, 2007