Treatment for Amyloidosis
Treatment of AL amyloidosis can help reduce symptoms, reduce heart failure-related hospitalization, and improve the length and quality of life.
Treatment plans are tailored to each patient and are based upon disease progression and seriousness of the patient’s organ, tissue, and nerve involvement. Currently, treatment options are focused on reducing the production of light chain protein from the bone marrow; however, newer treatments are in clinical development, such as CAEL-101, a novel antibody that may be able to clear the deposited protein in various organs and promote quicker recovery.
Treatment plans include both supportive treatment and source treatment.
Supportive treatment is helpful for various symptoms, including cardiac and kidney problems, and can make a significant improvement in a patient’s quality of life. Examples include:
- Cardiac medication
- Preserving kidney function
- Symptomatic treatment of neuropathy
- Geriatric assessments
- Psychological support
Reversing any damage to the organs and other parts of the body is difficult to achieve. However, if treatment begins during the early onset of clinical symptoms, the overall success rate is higher, so early detection is essential.
A bone marrow transplant is often considered the preferred treatment for AL because it can provide long-term control of the underlying disease. However, only a minority of AL patients are eligible.
For the majority of AL amyloidosis patients, other chemotherapy-based treatments are considered. These treatment regimens are tailored for each patient based on organ function, symptoms, and preferences.
Patients with AL amyloidosis have benefited from the development of new drugs for myeloma, many of which work effectively on the plasma cells that cause AL amyloidosis. Often, drug combinations are more effective than single drugs in attacking the abnormal plasma cells. Dosage is tailored to each patient. Categories of drugs that may be used include:
- Traditional chemotherapy drugs: Melphalan, Cyclophosphamide (Cytoxan™), Bendamustine (Treanda™)
- Proteasome inhibitors: Bortezomib (Velcade™), MLN9708 (Ixazomib™), Carfilzomib (Kyprolis™)
- Immunomodulators: Thalidomide (Thalidomid™), Lenalidomide (Revlimid™), Pomalidomide (Pomalyst™).
- Monoclonal antibodies: daratumumab (Darzalex)
Some of these drugs are still considered investigational for AL amyloidosis and should not be used outside of a sponsored clinical trial. Most of them are given with a steroid, such as dexamethasone, which seems to increase their effectiveness.
Treatment for ATTR can be divided into three categories:
- TTR silencers, such as patisiran or inotersen, which reduce TTR production in the liver
- TTR stabilizers, such as tafamidis, which stabilize the TTR protein structure and prevent it from breaking up and forming amyloid fibrils
- ATTR fibril disruptors, which remove existing amyloid deposits from the heart and other tissues
At UT Southwestern, our patients have access to leading-edge therapies through our experienced physicians. In 2018, the FDA approved two medications for hATTR amyloid polyneuropathy: patisiran (an siRNA) and inotersen (an antisense oligonucleotide). In May 2019, the FDA approved tafamidis for hATTR or ATTRwt cardiomyopathy.
To treat heart failure in people with ATTR amyloidosis, our physicians also use loop diuretics such as furosemide. Depending on each patient’s symptoms, we might also prescribe other heart failure medications, which can help the heart pump more blood to the body by relaxing blood vessels and reducing blood pressure. We monitor these patients very closely for any side effects.
Several new medications are available through clinical trials to treat ATTR amyloidosis. At UT Southwestern, eligible patients have access to these promising and advanced treatments.
For patients with cardiac amyloidosis, UT Southwestern physicians focus on managing heart failure and treating the underlying condition that is causing the amyloid deposits.
Because these patients may not tolerate certain medications commonly used in heart failure or other heart conditions, it’s important to have a precise diagnosis for safe, effective treatment.
We may recommend:
- Lifestyle changes: Limiting salt intake or making other dietary changes
- Water pills (diuretics): Medicine that increases the amount of water and salt removed from the body as urine
- Medications to treat atrial fibrillation (irregular heartbeat): Beta-blockers or other medicines
- Heart transplant
- Implantable cardioverter-defibrillator (AICD) or pacemaker: Electronic devices that monitor and correct an arrhythmia