Treatment of Amyloidosis

1. Medications for Amyloidosis

Medications play a crucial role in slowing down or stopping the production of amyloid proteins, particularly in AL and ATTR amyloidosis. Different drugs are used depending on the specific type of amyloidosis.
Chemotherapy (For AL Amyloidosis – Light Chain Amyloidosis)
In AL amyloidosis, abnormal plasma cells in the bone marrow produce misfolded light chains, which form amyloid deposits. To stop this, chemotherapy drugs are used to destroy these plasma cells, reducing the production of abnormal proteins.
Common chemotherapy drugs used include:

  • Bortezomib – A proteasome inhibitor that targets and destroys abnormal plasma cells.
  • Cyclophosphamide – A chemotherapy agent that reduces amyloid production.
  • Dexamethasone – A steroid that helps control inflammation and improves the effectiveness of chemotherapy.

In some cases, combination therapy (e.g., Bortezomib + Cyclophosphamide + Dexamethasone) is used to maximize the effectiveness of treatment. Chemotherapy helps slow down amyloid buildup and can prolong organ function in patients with AL amyloidosis.
Targeted Drugs for ATTR Amyloidosis (Transthyretin Amyloidosis)
ATTR amyloidosis is caused by mutations in the TTR (transthyretin) protein, leading to amyloid deposits in the heart and nerves. Unlike AL amyloidosis, chemotherapy is not effective for this type. Instead, specialized TTR-stabilizing and gene-silencing drugs are used:

Tafamidis (Vyndaqel)

  • Function: Binds to transthyretin (TTR) and prevents it from misfolding into amyloid.
  • Used For: Both hereditary (mutant TTR) and wild-type (age-related) ATTR amyloidosis, especially for heart involvement.

Patisiran & Inotersen (Gene-Silencing Drugs)

  • Function: These drugs block the production of TTR protein at the genetic level, preventing amyloid buildup.
  • Used For: Hereditary ATTR amyloidosis (especially with nerve damage).

These newer drugs have improved survival rates and quality of life for ATTR amyloidosis patients by reducing the progression of nerve and heart damage.

2. Organ Transplant for Severe Cases

In some severe cases of amyloidosis, organ function deteriorates to the point where medications alone are not enough. In such cases, an organ transplant may be necessary to replace a damaged organ and prevent further amyloid buildup.
Kidney Transplant

  • Used For: Patients with renal (kidney) failure due to AL or AA amyloidosis.
  • Why It Helps: Since amyloidosis damages the kidney’s ability to filter waste, a kidney transplant can restore normal kidney function and improve survival.

Liver Transplant

  • Used For: Hereditary ATTR amyloidosis (mutations in the TTR gene).
  • Why It Helps: The liver is the main producer of TTR protein, which causes ATTR amyloidosis. By replacing the liver, doctors stop the production of defective TTR protein, preventing further amyloid buildup.

Heart Transplant

  • Used For: Advanced cardiac amyloidosis where the heart is severely damaged.
  • Why It Helps: When amyloid deposits stiffen the heart muscle, it cannot pump blood effectively, leading to heart failure. A heart transplant can restore proper heart function.

Although transplants can prolong life, they are only performed in carefully selected patients, as amyloidosis can sometimes affect multiple organs and increase surgical risks.

3. Stem Cell Transplant (For AL Amyloidosis)

Autologous Stem Cell Transplant (ASCT) is an advanced treatment option for certain patients with AL amyloidosis. This procedure replaces the bone marrow’s abnormal plasma cells with healthy stem cells, allowing the body to produce normal proteins instead of amyloid-forming ones.
How It Works:

  • The patient’s own stem cells are collected from the bone marrow.
  • High-dose chemotherapy is given to destroy the abnormal plasma cells.
  • The healthy stem cells are reinfused into the body to rebuild normal blood and immune function.

Who Can Receive a Stem Cell Transplant?

  • Patients under 70 years old with good organ function.
  • Patients with AL amyloidosis but without severe heart disease.

A successful stem cell transplant can stop disease progression and, in some cases, even lead to long-term remission.

4. Supportive Care to Manage Symptoms

Since amyloidosis affects multiple organs, supportive treatments are essential for improving quality of life and managing symptoms.
Managing Fluid Retention and Swelling

  • Diuretics (water pills) like furosemide help reduce swelling in the legs caused by heart or kidney failure.
  • Salt restriction in the diet helps control fluid buildup.

Blood Pressure and Heart Rate Management

  • Patients with cardiac amyloidosis may experience low blood pressure or irregular heartbeats.
  • Beta-blockers and calcium channel blockers are sometimes used, but in AL amyloidosis, these medications must be given cautiously because the heart is highly sensitive to drug side effects.

Physical Therapy and Lifestyle Changes

  • Physical therapy helps patients with nerve damage (neuropathy) maintain mobility and balance.
  • Exercise programs help prevent muscle weakness and improve circulation.
  • Nutritional support is critical for patients with digestive involvement, ensuring they receive proper calories and nutrients.
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