Talk to Your Doctor About Treatment for ATTR
Transthyretin amyloidosis (ATTR) is a rare condition where a protein called transthyretin becomes unstable and forms clumps, known as amyloid, that build up in the body’s organs, especially the heart and nerves. This can lead to serious health problems over time. There are two main types: hereditary ATTR (hATTR), which is passed down through families, and wild-type ATTR (wtATTR), which usually develops with age.
While ATTR is serious, there are now several effective treatment options that can help slow its progression and improve quality of life. Here's a closer look at the most common treatment for ATTR available today.
1. Vyndamax: A First-Line Option for Heart-Related ATTR
Vyndamax (tafamidis) is currently one of the most widely used medications for treating ATTR, especially in patients who have cardiomyopathy—a form of ATTR that affects the heart. It works by stabilizing the TTR protein and preventing it from breaking apart and forming amyloid clumps. This action helps reduce the buildup of amyloid in the heart, slowing the progression of the disease.
Vyndamax is taken as a once-daily pill and is generally well-tolerated. Clinical studies have shown that it can improve survival rates and reduce hospitalizations in people with ATTR cardiomyopathy. Because of these benefits, Vyndamax is often the first recommended treatment for ATTR when the heart is primarily affected.
2. RNA Interference Therapy: Patisiran (Onpattro)
Patisiran is a type of RNA interference therapy designed for people with hereditary ATTR, particularly those who have nerve damage caused by the disease. This medication works by stopping the liver from producing the faulty TTR protein in the first place. By reducing the amount of abnormal TTR protein in the body, patisiran can help lower the level of amyloid deposits.
This medication is given through an intravenous infusion every three weeks. Patients treated with patisiran often see improvement in nerve function, walking ability and quality of life. It may also help reduce heart involvement in patients who have both nerve and heart symptoms. Although it requires regular clinic visits for infusions, the benefits for many patients are significant.
3. Antisense Therapy: Inotersen (Tegsedi)
Inotersen is another option for treating hereditary ATTR, particularly when the disease affects the nerves. It works in a similar way to patisiran by targeting the messenger RNA that carries the genetic instructions for making the TTR protein. This reduces the amount of abnormal protein produced by the liver.
Unlike patisiran, inotersen is given as a weekly injection under the skin, which some patients may find more convenient than infusions. However, this medication does require regular blood monitoring to check for possible side effects, such as low platelet counts or kidney problems. When used properly and with close supervision, inotersen can help slow the progression of nerve damage and improve daily function.
4. Non-Hormonal Stabilizer: Diflunisal
Diflunisal is a nonsteroidal anti-inflammatory drug (NSAID) that has shown some ability to stabilize the TTR protein and reduce amyloid formation. Although not officially approved by the FDA for ATTR, it is sometimes used "off-label" as an alternative treatment for patients who do not have access to or cannot tolerate other medications.
Because it is a traditional NSAID, diflunisal may not be suitable for people with kidney problems, ulcers or certain heart conditions. Still, for some patients, it can offer modest benefits in slowing the disease’s progression.
5. Organ Transplantation: Liver and Heart
In advanced cases of hereditary ATTR, especially when the liver is producing most of the abnormal TTR protein, a liver transplant may be considered. Replacing the liver can stop the source of faulty protein production. This approach is more commonly used in younger patients or those with early-stage disease, as the risks of surgery can be high.
In some cases where the heart has been severely affected, a heart transplant may also be an option. This is a more complex decision and is typically reserved for patients who meet specific medical criteria and are in otherwise good health.
6. Supportive Treatments for Managing Symptoms
In addition to medications that target the underlying disease, many patients also benefit from supportive treatments that manage the symptoms of ATTR. These may include medications to relieve nerve pain, drugs to help the heart pump more effectively and devices like pacemakers for heart rhythm problems. Physical therapy, dietary support and regular monitoring are also important parts of managing the condition day to day.
Choosing the Right Treatment for ATTR
Finding the best treatment for ATTR depends on the type of ATTR you have, which organs are affected and how far the disease has progressed. Some patients benefit from a single medication, while others may need a combination of treatments to manage symptoms and slow the disease. Early diagnosis and treatment can make a big difference in outcomes, so it’s important to work closely with a healthcare provider who has experience treating amyloidosis.
ATTR is a serious condition, but with new treatment options and ongoing research, people living with this disease have more hope than ever before.
Keep reading to learn about the warning signs of a heart attack due to ATTR-CM.