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 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.
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.
Treatment for AL amyloidosis includes:
- steroids such as dexamethasone and prednisolone
- novel treatments, such as thalidomide, bortezomib (also known as Velcade), and lenalidomide (also known as Revlimid)
- an autologous stem cell transplant
- treatments to preserve and support the function of affected organs that may be used in conjunction with the treatments above.
Some of these treatments are not funded in Australia by the Pharmaceutical Benefits Scheme (PBS). There may be some other funding sources for this medication, or you may have to fund the full price yourself. Speak to your doctor about all of your options.
Although amyloidosis is not a cancer, chemotherapy drugs are used in the treatment of AL amyloidosis to destroy the amyloid-producing plasma cells in the bone marrow. Chemotherapy literally means therapy with chemicals. It may involve the use of a single drug or combinations of drugs (combination chemotherapy). These drugs are usually given in several cycles (or courses) with a rest period of a few weeks in between each cycle. This is to allow the body to recover from the side effects of the drugs. Chemotherapy may be given in tablet form or by intravenous injection. Patients receiving oral treatment can usually take these drugs at home.
Whatever the type of chemotherapy, it is important to appreciate that improvement in amyloid- related symptoms is often slow and may not be apparent for 12-18 months. In addition to chemotherapy, supportive measures can help to reduce symptoms, maintain general wellbeing, and assist the function of affected organs.
Cortico-steroids are hormones that are produced naturally by the body in the adrenal glands. They can also be made in the laboratory and they play an important role in treatment. Manufactured cortico-steroids such as prednisolone, dexamethasone, and methylprednisolone are commonly used in combination with chemotherapy in the treatment of AL amyloidosis. Cortico-steroids can cause a rise in the blood sugar, mood alterations, increased appetite, fluid retention and a decreased immune system. Many of the side effects of cortico-steroids are temporary and should pass once you finish taking them.
New and novel treatments
Thalidomide is a drug that works in a number of ways to interfere with the growth and survival of the light chain-producing plasma cells in the bone marrow. Thalidomide is taken daily in tablet form. Bortezomib (Velcade) is a type of drug called a proteasome inhibitor. The proteasome has an important role in all cells and especially in the abnormal plasma cells in AL amyloidosis. The drugs inhibit the proteasome causing the abnormal cell to die while leaving the normal healthy cell less affected. Bortizomib is given by intravenous injection. While you do not need to be admitted for this treatment, it does require frequent visits to the hospital. Lenalidomide is a new drug derived from Thalidomide. It is given in tablet form for three weeks out of four, often in combination with dexamethasone and sometimes with chemotherapy.
Autologous stem cell transplant
Stem cell transplantation is an intensive procedure with a number of significant risks in patients with AL amyloidosis, so it is usually considered only for younger patients with good heart and kidney function. If your medical team feels the risk of you undertaking a transplant may be too high at diagnosis they may still suggest that your stem cells are collected and stored in case a stem cell transplant becomes an appropriate treatment choice in the future.
Slowing or stopping the production of amyloid may not be sufficient in itself to repair the damaged organ. When the damage is considered permanent an organ transplant may be considered. Kidney and heart transplants are sometimes offered in some treatment centres. To stop the amyloid protein depositing in the new organ, chemotherapy or stem cell transplantation is essential, either before or after successful organ transplantation.
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.
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.
Side effects of treatment
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