Patients will usually only see the relevant specialists for the care of their unique disease and affected organs. These specialists may not always be in the same clinic or building but will work together to deliver the best treatment for the individual patient.
Treatments have progressed considerably over the last 20 years. Although AL amyloidosis remains an extremely serious disease and is considered incurable, many patients are enjoying long periods of remission.
All patients diagnosed with AL amyloidosis have a slightly different presentation. Organ damage and age can mean patients are fragile and highly sensitive to treatment. Therefore, an individualized approach is used when the treatment team is selecting the most effective and best tolerated treatment regimen for each patient.
Historically the treatment of AL amyloidosis has been directed at the underlying bone marrow disorder with the aim of reducing as quickly as possible the free light chains which are causing the production of the amyloid protein.
How does chemotherapy work in AL amyloidosis
- The chemotherapy drugs have been traditionally borrowed from those proven to work in treating multiple myeloma. This is because drugs that kill cancerous plasma cells (i.e. multiple myeloma) are also good at killing the benign plasma cells underlying AL amyloidosis. Killing the plasma cells results in less amyloid forming free light chain being produced
- Once the production of light chains is greatly reduced or stopped, referred to as a haematological response, it has been shown that the amyloid already deposited in the organs may slowly leech out of those organs (organ response), improving function. This is often an extremely slow process and it may take 12 months or more after the end of treatment.
Types of chemotherapy
- Traditional chemotherapy drugs (Melphalan, Cyclophosphamide) with or without an autologous stem cell transplant, were for many years the only treatments available. These treatments are still very effective today
- Corticosteroids (dexamethasone), while not traditional chemotherapy, are also very effective at killing plasma cells
- Newer chemotherapy drugs such as the proteasome inhibitors (bortezomib, ixazomib), immunomodulatory drugs (thalidomide, lenalidomide, pomalidomide)
- Most patients take one or two chemotherapy drugs plus steroid medication. The drugs work together to destroy the plasma cells that make the light chain proteins.
N.B. Unfortunately not all drugs are registered for use in Australia or available through the Australian Pharmaceutical Benefit PBS scheme. There may be other ways of funding these treatments which the doctors will discuss with their patient
Side effects of treatment
Treatments can cause a number of side effects which may include:
- Retention of fluid in feet and legs
- Worsening of heart failure
- Hair loss
Patents will be closely monitored throughout treatment and constantly offered support and information about their treatment and side effects.
If a patient is worried about any new symptoms they should contact their doctor or specialized nurse at once.
Other treatments under investigation
More recently different approaches targeting the actual amyloid deposits have been developed and tested in clinical trials with the hope that they will complement chemotherapy agents. For example, doxycycline, an antibiotic, has been shown to disrupt amyloid fibrils and is being investigated in clinical trials.
Anti-amyloid fibril antibodies which may enhance removal of amyloid deposits already in the organs are being tested in clinical trials.
How is the progress of treatment monitored?
The aim of treatment is to reduce the production of light chains and support and improve organ function.
- Blood tests (free light chain assay, serum protein electrophoresis) and urine tests (urine protein electrophoresis) will be performed from time to time to measure the haematological response to treatment (CR, VGPR or PR). This is assessing whether the amyloid forming light chain protein is being reduced by the treatment.
- Blood tests will be run routinely to check on any negative effects from the treatment.
- Other tests mentioned above will be run routinely to check on the state of the patient’s organs and general health (Organ response).
What happens if the drug treatment is not working?
If the elected treatment is shown not to slowing the production of the amyloid protein fairly rapidly or the patient is having difficulty tolerating the treatment, other drugs may be suggested or the dosages of the drugs may be altered.