A liver transplant was traditionally offered to patients with hereditary transthyretin amyloid cardiomyopathy (ATTR-CM) as a way to slow or stop disease progression. However, as more effective, non-invasive treatments for ATTR-CM have emerged, liver transplants have become far less common than they once were.
Who is eligible for a liver transplant in ATTR-CM?
There are two types of ATTR-CM: hereditary ATTR-CM (hATTR-CM), where the disease is the result of a fault or mutation in the transthyretin protein, and wild-type ATTR-CM (wATTR-CM), which is associated with aging. A liver transplant is only an option for people living with hATTR-CM, as it removes the source of the faulty protein.
However, not all hATTR-CM patients qualify for a liver transplant. Research has found that patients with some specific mutations have had less favorable results after a transplant. A liver transport is also a major operation with potential risks such as bleeding, infection and complications. The criteria for receiving an organ transplant can exclude many patients with ATTR-CM.
Read more about ATTR-CM symptoms
When is a liver transplant recommended in ATTR-CM?
These days doctors rarely recommend liver transplants for people living with hATTR-CM, as effective drug therapies have emerged. Tafamidis, for example, has been shown to give patients with ATTR-CM longer survival rates than those of liver transplants. In rare occasions, a liver transplant may be recommended in conjunction with a heart transplant as a solution to heart failure and liver failure. This double organ transplant is only considered in patients strong enough to manage the toll of such a significant surgical intervention.
What are the benefits of a liver transplant?
Transthyretin protein is produced in the liver. In hATTR-CM it is either mutated or faulty, causing it to misfold, form clumps and become deposited throughout the body. The main benefit of a liver transplant is that it removes the source of these proteins. When no more misfolded protein is produced, disease progression stops. However, a transplant doesn’t address the problem of the existing amyloid deposits that continue to cause damage in the heart and other organs.
In recent years, patients with hATTR-CM who receive a liver transplant have still been shown to experience a progression of cardiomyopathy, or heart failure.
A liver transplant has its limitations and is no longer considered as an effective treatment for ATTR-CM. They were considered standard of care at a time when other treatments were less effective in slowing disease progression.
Newer alternatives to liver transplants in ATTR-CM
In 2019, the U.S. Food and Drug Administration (FDA) approved tamafidis to treat ATTR-CM. It slows or stops disease progression by stabilizing the transthyretin protein, stopping it from misfolding and forming amyloid fibrils.
In 2024, the FDA approved acoramidis to treat the disease. Like tamafidis, it is a TTR protein stabilizer, and has been found to significantly reduce cardiovascular death and hospitalizations in patients with ATTR-CM.
In 2025, vutrisiran received FDA approval as a treatment for ATTR-CM. Vutrisiran binds to transthyretin messenger RNA, which prevents its translation into protein.
Other promising therapies are currently in development. In addition, symptom management is a key aspect of caring for people with ATTR-CM and can have a significant impact on quality of life.
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