You may not have heard of diseases that occur when a type of protein in our body accumulates incorrectly and gets deposited in important organs like the heart and nerves. These are often rare diseases. In medicine, this type of protein deposition is called amyloidosis . Today, we are going to talk about a rare disease called ATTR-CM, which specifically affects the heart, that is, in which these proteins are deposited in the heart muscle, and the modern drug called Amvuttra used for it.
What exactly is ATTR-CM?
Simply put, ATTR-CM is a condition in which, due to a genetic defect or naturally occurring with aging, the protein TTR, which is produced in our body, changes shape and clumps together and deposits itself in the walls of the heart muscle. The full name for this is Transthyretin Amyloid Cardiomyopathy.
Think about it, our heart walls need to be flexible to pump properly. But when these proteins build up, the walls of the heart become thick and stiff . We call this cardiomyopathy. This makes the heart unable to pump blood properly. Over time, this can even progress to heart failure.
Some people with ATTR-CM do not experience any symptoms, but others experience symptoms similar to those of a normal heart attack.
- Slight shortness of breath (dyspnea)
- Swelling, especially in the legs and ankles
- Confusion or desperation
- Increased heart rate or irregular heartbeat
- Chest pain
This can make you feel tired quickly when doing things like climbing stairs, doing household chores, or walking a short distance.
In addition, other symptoms unrelated to the heart may also occur. For example, weakness, numbness or pain in the arms and legs, excessive sweating , dizziness or fainting, and digestive problems.
How does Amvuttra medicine work?
To understand this, we first need to know a little about a protein called TTR. TTR (Transthyretin) is a protein that is mainly produced in our liver. Its main function is to transport thyroid hormones and vitamin A throughout the body.
In people with ATTR-CM, a genetic defect causes the TTR protein to be produced in an incorrect shape. This incorrect shape of the protein accumulates and deposits in the heart.
The drug Amvuttra works as a gene silencer . That is, this drug goes directly to the liver and reduces the activity of the gene that makes the faulty TTR protein . It works like turning down the volume on a radio.
This reduces the amount of faulty TTR protein produced by the liver, which in turn reduces the deposition of new proteins in organs like the heart.
Amvuttra is a subcutaneous injection. This vaccine is usually given by a healthcare professional every three months .
How was the success of this medicine tested?
Like any new drug, the effectiveness of AMVUTTRA has been confirmed through a large study. In this study, 655 ATTR-CM patients were given the Amvuttra vaccine and the other group was given a placebo vaccine containing no drug every 12 months for three years.
Researchers looked at several factors to measure whether this drug was effective.
- Six-minute walk test: This measures how far you can walk at a normal pace in 6 minutes. This distance decreases as your heart condition worsens.
- New York Heart Association (NYHA) classification: This measures the severity of symptoms of heart failure. There are four classes (I, II, III, IV). The higher the class number, the more severe the disease.
| NYHA Class | Description |
|---|---|
| Class I | There are no restrictions on normal physical activities. There is no fatigue, shortness of breath, or chest pain. |
| Class II | Physical activity is slightly limited. There is no discomfort when resting, but fatigue and tiredness may occur during normal activities. |
| Class III | Physical activity is significantly limited, and symptoms occur even during lighter than normal work. |
| Class IV | Symptoms are present even at rest. Any physical activity makes the condition worse. |
- Quality of Life Questionnaire (KCCQ-OS): This measures the impact of the disease on the patient's quality of life.
What did the research find?
At the end of the three-year treatment period, the researchers analyzed the results.
The main finding was that patients who received Amvuttra had a 28% lower risk of death or sudden hospitalization or emergency room (ETU) visit due to heart failure , compared to those who received a placebo (a dummy vaccine).
Another important thing is that at the end of 42 months, the risk of death from any cause was reduced by 35% in the group that received Amvuttra.
Additionally, patients who received Amvuttra had a controlled decline in walking ability and quality of life.
But remember, these research results may not be the same for everyone. You and your doctor will need to decide together whether Amvuttra is right for you and whether the benefits outweigh the side effects.
Let's learn about the results and side effects.
How long does it take to see results?
Amvuttra is a medicine that works over time. Therefore, the results are not seen immediately. It may take several months or more to see any improvement in symptoms.
How to manage side effects?
One of the main side effects of Amvuttra is a decrease in vitamin A levels in the blood. This is because the TTR protein is what transports vitamin A throughout the body. When the drug reduces TTR production, the transport of vitamin A also decreases.
- Your doctor will regularly order blood tests to check your vitamin A levels. It is very important to get these tests done on time.
- Add foods rich in vitamin A (e.g. carrots, sweet potatoes, spinach, eggs, milk, red bell peppers ) to your diet.
- If you notice any changes in your vision (especially decreased night vision), inform your doctor immediately.
- Depending on your vitamin A level, your doctor may recommend taking a vitamin A supplement.
These are not the only side effects of Amvuttra. If you have any other symptoms that are bothering you, be sure to talk to your doctor .
Take-Home Message
- ATTR-CM is a rare but serious heart disease caused by the deposition of misfolded proteins in the heart.
- Amvuttra is a modern medicine used for this purpose and is injected every three months.
- This drug works by reducing the production of the defective protein in the liver.
- Research has shown that this can reduce deaths, hospitalizations, and improve the quality of life of patients.
- Since this can lead to low vitamin A levels, it is important to maintain regular contact with your doctor and undergo regular tests.
- Before starting any treatment, carefully discuss the pros and cons with your doctor.
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