Treatments for amyloidosis — the buildup of a protein called amyloid in your body's tissues or organs — can help limit further production of amyloid protein and manage signs and symptoms (supportive care).

Your treatment options depend, in part, on the type of amyloidosis you have. As you adjust to your diagnosis and make decisions about your treatment, it may help to understand more about some common treatment options. Clinical trials that are testing newer treatment possibilities are another option to consider.

For AL amyloidosis, treatment options include:

  • Chemotherapy. With AL amyloidosis, your bone marrow produces abnormal plasma cells — a type of white blood cell that makes proteins called antibodies to fight infections. Many of the same chemotherapy drugs and combinations that treat multiple myeloma — a cancer that forms in plasma cells — are also used to treat AL amyloidosis. The goal is to stop the growth of the abnormal cells that produce amyloid.
  • Autologous blood stem cell transplantation (ASCT). This is another option for some people. This procedure involves collecting your own stem cells from your blood and storing them for a short time while you have high-dose chemotherapy. The stem cells are then returned to your body through a vein.

    Because stem cell transplant has life-threatening risks, ASCT is most appropriate for those whose disease isn't advanced and whose heart isn't greatly affected.

AA amyloidosis treatments target the underlying infection or inflammatory condition that's causing the amyloidosis. Examples include:

  • Anti-inflammatory medications to treat rheumatoid arthritis
  • Surgery to treat a tumor
  • Antibiotics to treat an ongoing infection
  • A drug called colchicine if you have familial Mediterranean fever

Treatment can't reverse severe kidney problems. But if you have more-moderate kidney issues, treatment may also help reduce those issues.

Treatments for hereditary amyloidosis depend on the subtype of amyloidosis.

  • Liver transplant. This may be an option for those whose hereditary amyloidosis is caused by a protein made in the liver, including the most common hereditary amyloidosis, ATTR.

    A liver transplant can help reduce or stop the amyloid deposits and may ease nerve and digestive issues. However, heart problems may continue, and sometimes a heart transplant also is recommended. For some types, a kidney transplant may be recommended.

  • Patisiran (Onpattro). This drug is delivered through a vein every few weeks and may improve nerve symptoms. It works by targeting and interfering with the expression of mutated TTR genes responsible for some subtypes of hereditary amyloidosis.
  • Inotersen (Tegsedi). This drug — injected once a week — also may improve nerve symptoms. It works by interfering with the liver's ability to produce TTR. Side effects include reducing the amount of platelets in the blood (thrombocytopenia). Platelets are what help your blood to clot, to stop bleeding.

Treatments for amyloidosis caused by dialysis may include changing your mode of dialysis or having a kidney transplant.

To manage ongoing signs and symptoms of amyloidosis, your doctor may also recommend:

  • Pain medication
  • Fluid retention medication (diuretic) and a low-salt diet, to help the kidneys
  • Blood-thinning medication
  • Drugs to ease diarrhea, constipation, nausea or vomiting
  • Medication to control your heart rate

To help ensure you're receiving the best treatments for you, ask questions about your options and share any symptoms, side effects or concerns that you have.

May 01, 2019