Amyloidosis Treatment
AL Amyloidosis Treatments
The primary goal of treating AL amyloidosis is to minimize amyloid deposition in the body’s tissues/organs. While better treatment options are still vitally needed, there are many more options today than just a few years ago.
The treatment physicians usually consist of a Hematologist (who prescribes/manages the specific treatment aimed at reducing light chain production) and specialists who help manage the associated organ dysfunction (such as a Cardiologist). Specific therapies often include treatment with a steroid (e.g. dexamethasone, prednisone) with chemotherapy drugs (such as melphalan).
Newer chemotherapy drugs which your Hematologist may prescribe include lenalidomide and bortezomib. The exact regimen depends on the specifics of your case, and balancing the beneficial effects of treatment with the potential side-effects. Virtually all of these treatments are oral (pills) and do not require hospitalization.
Another mechanism to treat amyloidosis involves giving stronger chemotherapy to try to further eliminate the abnormal cells which produce the light chains. In this treatment approach, the chemotherapy is so strong that it depletes the body’s own normal bone marrow cells. Because of this, stem cells (usually collected from the patient before the chemotherapy is given) are given back to a patient after the chemotherapy, a procedure called a “bone marrow transplant” or a “stem cell transplant.” While this had been the preferred treatment approach for many years, a landmark study published in 2007 found that survival was generally better with conventional medical therapy (as described above) than with bone marrow transplants. This is likely due to the side-effects and toxicities associated with treatment with the very strong chemotherapy which is administered during a bone marrow transplant. For this reason, we recommend that most patients with AL amyloidosis undergo conventional therapy – though there are some cases (such as when disease progresses despite treatment with conventional therapy) in which bone marrow transplant represents the best option.
In addition, with a goal of helping both current and future patients with amyloidosis, the Stanford University Amyloid Center also actively participates in and leads clinical trials. Your treatment team will tell you about any clinical trials which you are eligible for, and explain the potential benefits and risks of any trial to you.
ATTR Amyloidosis Treatments (‘Familial’ or ‘Senile’ Amyloidosis)
Treatment for ATTR amyloidosis depends on whether it is part of the ‘Familial’ (genetic mutation) form or the ‘Senile’ form.
For Familial ATTR amyloidosis, the key to treatment is early diagnosis. This is most effective if individuals who carry the mutation are identified by genetic screening when a family member is known to carry the disease. The genetic testing is available through the Stanford University Amyloid Center. If an individual is identified as carrying the genetic mutation, he/she will be carefully screened for manifestations of the disease, and screening will continue on an annual or semi-annual basis. If early disease is identified, progression can be halted with liver transplantation, as the liver produces the abnormal transthyretin protein.
In Senile ATTR amyloidosis, treatment is generally aimed at treating the symptoms of the disease. Because the heart is the most commonly affected organ, your disease will be monitored and treated by a Cardiologist.
New treatment modalities are constantly being developed and tested. We are currently enrolling patients affected by both forms of ATTR amyloidosis in a registry aimed at better understanding the natural course of the disease. Upcoming clinical trials may soon aim at inhibiting the transthyretin protein from forming into amyloid fibrils with a new medication.