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Ask The Experts: Treating rare heart disease ATTR-CM

There is currently no cure for ATTR-CM, but with proper supportive treatment to manage its symptoms and improve survival, most patients do enjoy a reasonable quality of life. PHOTO: GETTY IMAGES

Q: I am a 60-year-old male recently diagnosed with Transthyretin Amyloid Cardiomyopathy (ATTR-CM). What are my treatment options? Is this disease curable?

A: In general, the treatment for ATTR-CM can be divided into two aspects.

First, there is the supportive treatment of heart failure, as well as electrical conduction block and rhythm disorders, which arise from the deposition or infiltration of abnormal transthyretin (TTR) protein into different parts of the heart - such as the heart valves or muscle.

Second, there are specific treatment agents that try to prevent or reduce this abnormal protein from depositing into the heart, significantly the heart muscle, along with agents that help to remove them from the heart.

How does treatment help?

Depending on the location and extent of this abnormal protein infiltration into the heart, symptoms may start and progress differently for different patients.

In the early disease stage, you would likely not have many symptoms. With more deposition, the normal contraction and relaxation of your heart muscles would be affected, and symptoms of heart failure start to appear - namely, shortness of breath with minimal effort, fatigability and fluid retention in the body. You may also experience leg swelling and breathlessness when lying flat.

The main aim of treatment at this juncture is to help you to get rid of excess fluid and maintain fluid balance by giving you diuretics.

Some patients may also experience a slow heart rate and recurrent fainting spells if the abnormal protein is deposited on the conduction system of the heart. In this situation, a pacemaker would be necessary.

Patients are generally also prone to developing atrial fibrillation, a fast and irregular heart rhythm that would precipitate heart failure and increase the risk of getting a stroke. Medications would be required to control this rhythm disorder, as well as blood thinners to reduce the risk of a stroke.

What are my options for treatment?

Specific treatments for ATTR-CM are limited and costly.

A therapeutic agent has recently been approved in Singapore. This medication has been shown to improve symptoms and survival, and is the first to be approved for ATTR-CM. It helps to stabilise the TTR protein and, in turn, helps to reduce the formation and subsequent deposition of the abnormal TTR protein into the heart.

There are other medications currently going through different phases of clinical trials, and more therapeutic agents are expected to be available in the future.

Unfortunately, there is no cure for ATTR-CM, a chronic progressive disorder, as yet. Nevertheless, most patients enjoy reasonable quality of life with supportive treatment for many years.

Hopefully, with the availability of specific treatment agents, the progression of the disease can be slowed, and patients can maintain a stable state of health for many more years.

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