Transthyretin Cardiac Amyloidosis (ATTR-CM) Treatment: Medication, Lifestyle Changes, and More

Treatment options for transthyretin cardiac amyloidosis (ATTR-CM), also known as transthyretin amyloid cardiomyopathy, have grown considerably in recent years. This condition is caused by an irregular protein, called transthyretin (TTR), that’s produced in the liver and builds up in the heart. This buildup causes the heart walls to stiffen, making it harder for the heart to pump blood, eventually leading to heart failure and frequently to early death.

Disease-modifying drugs have become a cornerstone of ATTR-CM treatment in recent years. These new advanced medications can slow or stop the buildup of new TTR proteins in the heart.

A decade ago, “management was primarily focused on treating heart failure symptoms,” says Ajay Vallakati, MBBS, an advanced heart failure and transplant cardiologist at the Ohio State University Wexner Medical Center in Dublin, Ohio. “Now we have TTR stabilizers and TTR gene silencers,” which can help stop ATTR-CM from getting worse.

There isn't much existing research that directly compares the efficacy of stabilizers versus silencers, so it’s important to have a conversation with your doctor to decide which option may be right for you, or if it makes sense for you to take both of them.

Regardless of what you decide, you’ll reap the most protection by starting treatment as soon as possible. “Early diagnosis and initiation of treatment are crucial, as they offer the greatest potential benefit,” Dr. Vallakati says.

TTR Stabilizers

Stabilizers are oral medications that stop TTR proteins from clumping inside the heart.

With ATTR-CM, “the TTR protein that is secreted from the liver breaks loose and gets stuck inside the heart muscle, and stabilizers prevent it from breaking loose,” explains Ashesh Parikh, DO, a cardiologist with Texas Health Presbyterian Hospital Plano and with Texas Health Physicians Group.

Medications in this class include:

• Acoramidis (Attruby)
• Tafamidis (Vyndagel, Vyndamax)

TTR Silencers

Silencers work differently than stabilizers, but they still protect the body from the effects of TTR to slow or stop the progression of ATTR-CM. These injectable medications “create a barrier or a halo around the proteins [that are released by the liver], preventing the proteins from settling in the heart,” Dr. Parikh explains.

• Vutrisiran (Amvuttra)

• Inotersen (Tegsedi)
• Patisiran (Onpattro)

Off-Label Treatments

“These things need to be treated separately from a person’s ATTR-CM,” Parikh says.

Diuretics

Medication in this class include:

• Torsemide (Demadex, Soaanz)
• Bumetanide (Bumex)

Aldosterone Agonists

Also called aldosterone receptor agonists, these oral medications keep the body from producing aldosterone, a hormone that raises blood pressure. That’s helpful for some people with ATTR-CM, since heart failure and fluid retention can cause a person’s blood pressure to go up.

• Spironolactone (Aldactone and CaroSpir)
• Eplerenone (Inspra)
• Finerenone (Kerendia)

Common side effects can include cough, dizziness, headache, diarrhea, or high potassium levels. Spironolactone may also cause enlarged breasts (regardless of sex), erection problems, or vaginal bleeding after menopause.

Antiarrhythmics

Anticoagulants

Medications in this class include:

• Warfarin (Jantoven, Coumadin)
• Dabigatran (Pradaxa)
• Apixaban (Eliquis)
• Edoxaban (Lixiana, Savaysa)
• Heparin

Liver Transplant

“With the development of gene silencers, medications are now available that decrease the production of TTR in the liver,” says Vallakati. “As a result, liver transplantation is no longer necessary for most patients.”

Heart Transplant

Patients who develop advanced heart failure from ATTR-CM may require a heart transplant.

“These patients often experience shortness of breath at rest,” Vallakati says. “Heart transplantation improves their symptoms and increases survival.”

But heart transplantation won’t cure ATTR-CM. “After heart transplantation, we continue treatment with stabilizers, silencers, or both to prevent new deposition of TTR protein in the transplanted heart,” says Vallakati.

Kidney Transplant

Limit Salt Intake

Eat a Heart-Healthy Diet

Stay Active

It’s not always easy to get regular exercise, especially if you have symptoms like shortness of breath or fatigue. But any activity that you can do will improve your endurance, even if it’s just short walks around the block. Your doctor can help you decide on an exercise plan that’s safe for you. They may also recommend physical therapy or cardiac rehabilitation, which can help you work up to being more active.

EGCG is unlikely to pose safety concerns for most people, but always ask your doctor before trying a new complementary or integrative approach.

• Treatment options for ATTR-CM, a cardiac condition caused by abnormal proteins accumulating in the heart, have expanded considerably in recent years.
• Therapeutic approaches now include disease-modifying medications — TTR stabilizers and silencers — that can slow or stop disease progression by treating its root cause.
• Many people with ATTR-CM will require other drugs and therapies to help manage the symptoms and complications of the disease.
• Early diagnosis and treatment provide the greatest benefits, and ongoing care may involve mental health support and conversations with your doctor about long-term management.