How to Treat ATTR
Transthyretin amyloidosis (ATTR) is a rare and serious disease that happens when abnormal proteins called amyloid build up in the body’s organs, especially the heart and nerves. There are two main types of ATTR: hereditary ATTR (hATTR), which is passed down through families, and wild-type ATTR (wtATTR), which happens with age and has no known genetic cause.
Medical research has come a long way in recent years. There are several types of ATTR treatment options that can help manage the disease, slow its progression and improve quality of life. A popular option is Vyndamax, which we'll look at first.
1. Vyndamax (tafamidis)
Vyndamax is one of the first FDA-approved treatments specifically for ATTR with cardiomyopathy (when ATTR affects the heart). It works by stabilizing the TTR protein, preventing it from breaking apart and forming harmful amyloid clumps. By keeping the protein stable, Vyndamax helps slow the progression of the disease.
This medication is taken once daily as a pill and is typically well-tolerated. It’s an important option for people with both hereditary and wild-type ATTR that affects the heart. Studies show it can improve survival and reduce hospitalizations related to heart problems.
2. Patisiran (Onpattro)
Patisiran is a type of treatment called an RNA interference (RNAi) therapy. It’s used mainly for hATTR with polyneuropathy—when the disease causes nerve damage and symptoms like numbness, tingling or muscle weakness.
This medicine works by stopping the liver from making faulty TTR protein, which lowers the amount of amyloid in the body. It’s given as an infusion (through a vein) once every three weeks.
People who take patisiran often see improvements in nerve function, mobility and overall quality of life.
3. Inotersen (Tegsedi)
Inotersen is another RNA-targeting treatment for hATTR with polyneuropathy. Like patisiran, it helps reduce the production of the abnormal TTR protein in the liver.
What makes inotersen different is that it’s given as a weekly injection under the skin, which some patients may find more convenient than infusions. It has been shown to help slow nerve damage and improve daily function.
However, because it can affect the kidneys and platelets (which help blood clot), patients on inotersen need regular blood tests to stay safe.
4. Diflunisal (off-label use)
Diflunisal is actually a nonsteroidal anti-inflammatory drug (NSAID), similar to ibuprofen. But in the case of ATTR, it’s sometimes used off-label (not officially approved for this purpose) to stabilize the TTR protein and reduce amyloid formation.
Though not as powerful or targeted as newer drugs, it may be an option for people who don’t have access to other treatments. It’s taken by mouth, usually twice a day. However, long-term use of NSAIDs can affect the kidneys and stomach, so it should be used carefully under a doctor’s supervision.
5. Heart and Liver Transplant
In some cases, especially for younger patients with hATTR, a liver transplant might be considered. Since the liver makes the abnormal TTR protein, replacing it with a healthy liver can stop the production of amyloid at its source.
Similarly, heart transplant may be an option for people with severe cardiomyopathy (heart damage) from ATTR.
These procedures are not common and come with risks, but they may be considered for eligible patients in certain situations.
6. Supportive and Symptom-Based Care
Besides medications, another important part of ATTR treatment is managing symptoms. This may include:
- Pain relief medications for nerve pain.
- Physical therapy to improve strength and mobility.
- Diuretics or other heart medications for people with ATTR cardiomyopathy.
- Pacemakers or defibrillators for heart rhythm problems.
- Nutritional support if digestion or appetite is affected.
Your healthcare team can tailor a plan that combines different types of ATTR treatment options depending on your symptoms and type of ATTR.
Final Thoughts
Living with transthyretin amyloidosis can be challenging, but treatment options are improving every year. Whether it’s Vyndamax to stabilize the TTR protein, RNA therapies like patisiran and inotersen or supportive care to manage symptoms, there are now many types of ATTR treatment options available.
If you’ve been diagnosed with ATTR, talk to your doctor about genetic testing, treatment eligibility and which therapies may work best for you. Early diagnosis and treatment can make a big difference in managing this condition and improving quality of life.
Keep reading to learn about the signs of a heart attack due to ATTR-CM.