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Amyloidosis treatment

How is amyloidosis treated?

There is a range of treatments available for amyloidosis with much research being carried out around the world to find new treatments.

Once a firm diagnosis has been made and the subtype of amyloidosis is identified, your doctors will discuss their recommended treatment regime with you. Regardless of the type of amyloidosis you have the goals of treatment are:

  • to stop or slow the production of the amyloid protein
  • to preserve and support affected organs and tissues
  • to improve your quality of life.
AA amyloidosis

AA amyloidosis is managed by controlling the underlying inflammatory disease and therefore reducing the production of the amyloid protein SAA. If the SAA level can be reduced to almost normal and remains there for a long time there is a chance that the existing amyloid will eventually regress, improving the organ function. New treatments that interfere with the process of amyloid deposition are also being studied in AA.

AL amyloidosis

Over the past 10 years an increasing range of therapies has been developed. Even though treatment for AL amyloidosis is still not thought to be curative, many patients are living long and active lives. In deciding on the best treatment for you, your medical team will take into account a number of factors including your age, general health and the extent to which your organs have been affected by the disease. They will also consider potential complications of therapy.

The aim of treatment is to rapidly reduce the free light chains that are causing the production of amyloid. Targeting the plasma cells within the bone marrow, which are producing the free light chains, achieves this. Once the production of the amyloid protein is slowed or stopped, the amyloid fibrils already deposited in the organs may slowly move out of the affected organs. The function of the affected organs may then slowly improve. Sometimes, however, the organs may be damaged to the point where the organ function does not improve greatly. At this time there are no specific treatments that can directly clear amyloid deposits from the organs and tissues of the body.

Hereditary amyloidosis

Liver transplantation may be considered to remove the source of the amyloid-forming TTR variant. However, transplantation can be limited by the presence of amyloid in the heart, especially in an older patient. Liver transplant is only suitable for a minority of patients with ATTR. Drugs are being trialled at this time to stop the formation of the amyloid protein.

Senile systemic amyloidosis

Treatment is generally aimed at treating the symptoms of the disease. Because the heart is the most commonly affected organ, this disease will be monitored and treated by a cardiologist. As chemotherapy may actually disadvantage these patients, careful review is required to clarify the diagnosis particularly if the amyloid is found only in the heart.

Tests will be performed throughout your treatment to see whether there is evidence of your amyloid responding to treatment. Treatment regimes may be changed or started at any time if the required results are not achieved or if you are experiencing severe side effects or toxicities with the treatment. Your medical team will continually check any affected organs to see how they are standing up to the treatment and whether there has been an improvement in performance status.