If you’ve been diagnosed with hereditary transthyretin amyloidosis (hATTR), you may have questions about your treatment options and wonder whether there are advantages to switching hATTR treatments. All of the treatment options for hATTR are designed to slow disease progression, and the best treatment is often the one that fits your specific symptoms and lifestyle most seamlessly.

One MyAmyloidosisTeam member discussed the possibility of switching their hATTR treatment, writing, “I’m going to approach my cardiologist when I meet with her again in March. She’s also a scientist, so hopefully I’ll get some good feedback about it and possibly switch 🤞🙏.”

To learn more about switching hATTR treatments, MyAmyloidosisTeam spoke with two amyloidosis specialists, Dr. Ronald Witteles and Dr. Rodney H. Falk. Dr. Witteles is a professor of medicine at Stanford University with specialties in heart failure and amyloidosis. Dr. Falk is an associate professor at Harvard Medical School who specializes in cardiovascular medicine, including cardiac amyloidosis.

What Is hATTR?

Amyloidosis causes an abnormal protein — known as amyloid — to build up in the body’s organs and tissues. Amyloidosis is a serious and rare disease that can affect various organs and cause a broad range of symptoms. One type of amyloidosis is hATTR. In people with hATTR, the condition is hereditary (passed down in families). It is caused by a mutation (change) in a gene (known as a TTR gene) that regulates a transthyretin protein (TTR protein).

The buildup of hATTR amyloidosis deposits interferes with organ function and typically affects the heart, the digestive tract (gastrointestinal system), and the nervous system. The condition can seriously impact quality of life, but in many cases, it can also be effectively treated.

There is another type of ATTR amyloidosis that isn’t hereditary. This is called wild-type ATTR or wild-type amyloidosis.

Is One Medication Better Than Another?

Several medications have been approved by the U.S. Food and Drug Administration (FDA) for treating hATTR, including:

• Acoramidis (Attruby)
• Eplontersen (Wainua)
• Patisiran (Onpattro)
• Tafamidis (Vyndamax)
• Vutrisiran (Amvuttra)

“The only wrong option is to be on [no medication].”

— Dr. Ronald Witteles

Some medications are approved for hereditary and wild type ATTR, which can cause cardiomyopathy (heart damage). Others are approved for hATTR, which can cause polyneuropathy (nerve damage). Some medications are approved for both wild-type ATTR and hATTR.

There are also medications that are prescribed off-label for hATTR. This means the drug itself has been approved by the FDA as a safe treatment, but it hasn’t been approved specifically for the condition it’s being used for. Doctors often prescribe treatments off-label when there’s evidence that they may be effective.

In severe cases, your doctor may recommend a liver transplant, because TTR buildup mostly occurs in the liver.

Both Dr. Witteles and Dr. Falk stressed that when it comes to the medications used in the treatment of hATTR, one isn’t better than another.

“The only wrong option is to be on none of them,” Dr. Witteles said.

According to Dr. Falk, studies have shown that these medications are more or less equally beneficial in treating hATTR. “If you look at the clinical trials, the results are pretty much the same,” he said.

Some health conditions have a first-line therapy — a specific treatment plan that is typically used initially when someone is first diagnosed with a health condition. However, in people with hATTR, any treatment option may be used first. No single treatment is considered a first-line therapy. “It’s not that we know, well, this one’s better than this one, and you start with this one,” said Dr. Witteles.

Why Switch Treatments?

Research shows that people with hATTR switch treatments for various reasons, including:

• Concern about disease progression
• Medication side effects
• Medication preferences
• Access to new medications

You can discuss treatment options with your doctor to determine if switching medications is appropriate for you.

Switching Treatments Because of Disease Progression

One of the main reasons you or your doctor may want to switch hATTR treatments is if your condition is worsening. “Sometimes people will switch if they are on one and they feel like they’re getting worse or their doctor feels like they’re getting worse,” Dr. Witteles said.

Dr. Falk emphasized that switching medications may or may not improve someone’s hATTR. “It’s not entirely clear whether changing medications does anything because they all work pretty much,” he said. However, the medications work in different ways. “They have different mechanisms,” Dr. Falk said.

“We have to listen to the patients. They may have side effects of certain medications, not very often for the TTR medications.”

— Dr. Rodney H. Falk

Both Dr. Witteles and Dr. Falk said that it wasn’t clear if switching medications will improve someone’s condition. “We actually don’t know that that does anything,” said Dr. Witteles.

Dr. Falk explained that sometimes hATTR may be progressing due to individual factors that aren’t related to the specific medication someone is taking. In those cases, he said, switching medications may not improve someone’s condition: “There are some other subtleties there, which people don’t really address very much, that may be causing progressive disease that are unrelated to the therapies they’re getting and won’t get any better by changing therapies.”

Switching Treatments Due to Side Effects

Some people may experience unpleasant side effects due to a medication for hATTR and want to switch treatments. Fortunately, many people tolerate hATTR treatments well, Dr. Falk said. However, it’s possible that someone may not tolerate a treatment as well as they would like. “We also have to listen to the patients. They may have side effects of certain medications, not very often for the TTR medications,” he said.

Treatments for hATTR may be taken as a pill, an injection, or an infusion.

While these drugs are generally well tolerated, some can lower vitamin A levels, so your doctor may recommend vitamin A supplements, which could influence your preference for one drug over another. Always discuss possible side effects with your doctor before trying a new treatment, and be sure to report any unwanted side effects you experience.

You May Prefer How a Certain Medication Is Taken

Medications for hATTR are taken in different ways. Depending on the medication, an hATTR treatment may be taken in one of these ways:

• Orally — As a tablet or capsule
• Subcutaneously — As an under-the-skin injection
• Intravenously — As an infusion into your vein at a medical facility

Treatment to control hATTR is lifelong. The way you take a specific treatment could be a concern for some people. As Dr. Witteles explained, “There’s no reason to think that this is something that people could come off of in the future, because this protein is constantly being made by the body. So as soon as you come off the treatment, you just have that same protein that could deposit there again.”

Medications for hATTR that are administered by injection or infusion are taken less frequently than oral medications, which may appeal to people who don’t want to remember to take a pill every day. Some people may prefer one method over another, and that could be a reason to switch treatments.

Treatment Plans May Be Affected by Other Factors

The choice of medications and potentially switching medications for hATTR may be determined based on other factors. “I saw a patient this morning with TTR for the first time. He happened to have an arrhythmia that he was unaware of, and the drug I would usually use affects the liver. But he’d been on some other medication that had just recently had some abnormalities in his liver function tests, so now that becomes a focus,” Dr. Falk said.

Always keep up with any follow-up appointments. Your doctor will monitor your condition and determine if any of your individual factors — such as organ function — may indicate that you should switch treatments. Your treatment plan is a living document: As new medications emerge and your health evolves, your doctor should work with you to ensure your therapy matches your current needs.

Talk to Your Doctor if You’re Concerned About Your Treatment

If you’re not satisfied with your treatment plan, it’s good to talk to your doctor about your options. Your doctor can discuss available treatment options that may be appropriate for you. It’s important to communicate openly with your healthcare team about any changing symptoms you may be experiencing.

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