For Patients – Major Treatments

Treatment of amyloidosis 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 patient's symptoms.

For AL Amyloidosis, also called primary amyloidosis, treatment includes agents to stop the growth of the abnormal cells that produce the amyloid protein. Treatment is not a cure but with newer modalities of treatment, patient outcomes have improved.

• The drugs used are similar to those used to treat multiple myeloma, such as chemotherapy [cyclophosphamide, melphalan], proteasome inhibitors [bortezomib, carfilzomib], immunomodulatory drugs [lenalidomide, pomalidomide] and corticosteroids.
• High-dose chemotherapy with autologous stem cell transplantation is also a treatment modality. Patients undergo an extensive work-up to evaluate organ function and the effects that amyloidosis has had on the body. In those with adequate heart, liver and lung function, high dose chemotherapy followed by autologous stem cell rescue is considered.

  Your own stem cells are collected from the blood after giving a growth factor. About two weeks later you are admitted to the hospital to receive high-dose melphalan chemotherapy over one day. Then your own stem cells, previously collected, are re-administered via a vein two to three days later. An additional two 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 disease and improve survival. However, this therapy is not a cure.

Several new investigational agents are being evaluated in the treatment of multiple myeloma, another plasma cell disorder. The hope is that some of these agents also may be effective in treating AL amyloidosis.

Investigational agents are also being evaluated to remove amyloid protein that has deposited in the organs.

Treatment for other types of amyloidosis

In AA amyloidosis [secondary amyloidosis], the main goal of therapy is to treat the underlying condition — for example, taking an anti-inflammatory or disease modifying medication for rheumatoid arthritis or antibiotics for an infection.

In hereditary amyloid, since the protein [mutant transthyretin] that causes this form of amyloidosis is made in the liver, liver transplantation is an option. The new liver does not produce the abnormal amyloid protein and consequently the disease improves.

Investigational drugs are also being evaluated to try and prevent this type of amyloid protein from depositing in organs.

Heart Transplant and Kidney Transplant

In selected patients with amyloidosis, heart transplant and kidney transplant have been performed.

Management of symptoms

• Symptoms such as fluid retention are treated with medications to remove fluid [diuretics] and low-salt diet.
• In cases of low blood pressure/dizziness on standing – medications to help stabilize the blood pressure may be considered.

At the UCSF Comprehensive Amyloid Center, a multidisciplinary team evaluates the patient and then discusses further with the patient and the patient’s caregivers the appropriate treatment options.