Do you just feel numbness or tingling in your hands and feet? Or do you have muscle weakness and difficulty walking? Sometimes, these symptoms can be caused by a rare condition that we haven't heard of yet. Today, we're going to talk about one such condition and a new treatment for it.
What is hATTR? How does Amvuttra work?
Simply put, hATTR (Hereditary Transthyretin-Mediated Amyloidosis) is a hereditary disease. It belongs to a group of diseases called amyloidosis. In these diseases, certain proteins in our body change their shape, accumulate in abnormal ways, and deposit in various organs.
In hATTR disease, a protein called Transthyretin (TTR) is deposited in this way. TTR is actually a good protein that is produced in our liver and helps transport thyroid hormones and vitamin A throughout the body. However, a person with hATTR disease has a small change in the TTR gene, that is, a mutation. As a result, the TTR protein that is produced is in the wrong shape. This misshapen protein accumulates and is deposited in places like the nerves, heart, and digestive system as deposits called amyloid .
When these deposits accumulate around the nerves, they become damaged. We call this polyneuropathy . That's when the symptoms mentioned earlier, such as inflammation, numbness, pain, and difficulty walking, occur.
The important thing is that even if you inherit the gene variant associated with this disease from a family member, it does not mean that you will definitely develop hATTR disease.
The drug Amvuttra works by reducing the amount of this abnormal TTR protein produced by the liver. This reduces the accumulation of amyloid deposits in the body, helping to control symptoms and prevent or delay the disease from getting worse.
| Common symptoms seen in hATTR disease | |
|---|---|
| Nerve-related Symptoms | |
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| Digestive symptoms | |
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| Other Common Symptoms | |
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Your doctor will also recommend changes to your diet and exercise routine, along with treating your symptoms. Until recently, the only treatment options for this disease were symptom control and, if necessary, liver or heart transplants. But modern drugs like Amvuttra now allow you to control the disease by reducing the TTR protein, which is the underlying cause of the disease.
How do you use this medicine?
Amvuttra is an injection that is given under the skin . It is given by a health professional, such as a doctor or a trained nurse. It is usually given four times a year (every three months) . You can arrange to receive the vaccine at your doctor's office, at a clinic, or even at home.
How has this medicine been researched?
Like any new medicine, Amvuttra has been tested in clinical trials to determine its effectiveness and safety. In this trial, a group of patients who received Amvuttra were compared with a group of patients who received a placebo , a dummy treatment that contained no active ingredient. This is how we can determine whether the medicine really works.
The study included patients with hATTR disease and the resulting polyneuropathy. Their nerve weakness, muscle strength, and ability to perform daily activities were measured using special scoring systems.
According to the results of the study, patients who received Amvuttra improved their neurological function and quality of life. While their neurological scores decreased and symptoms improved, those who received the placebo experienced an increase in symptoms.
But remember, the results of these studies may not be the same for everyone. You should discuss with your doctor whether this medication is right for you and whether the benefits outweigh the risks.
When will I start feeling the effects of the medicine?
This is not a quick fix. As the medication gradually starts to work in your body, you may notice a reduction or some improvement in your symptoms within a few months . The long-term goal of this treatment is to control the progression of the disease. Therefore, it is very important to keep an eye on your symptoms.
Tell your doctor to watch out for these things:
- The ability to walk without assistance
- Numbness and tingling in the limbs
- Body reflexes
- Controlling blood pressure while standing
Even if you don't notice an immediate change, don't miss getting your vaccinations on time. Your doctor may also perform various blood tests, scans, or physical examinations to monitor your progress.
Side effects and how to manage them?
The most common side effects seen with Amvuttra are:
- Pain in the arms or legs
- Joint pain
- Difficulty breathing
In addition, Amvuttra can reduce your vitamin A levels . Therefore, your doctor will perform a blood test to check your vitamin A levels before and during treatment. If you notice any changes in your vision, especially night vision problems , inform your doctor immediately.
These are not the only possible side effects. If you have any new symptoms that make you feel uncomfortable, tell your doctor immediately.
Can there be problems with other medications?
Amvuttra is not known to cause any significant interactions with other foods, beverages, or medications. However, it is best to tell your doctor about all medications, vitamins, herbal products, and supplements you are taking.
Take-Home Message
- hATTR is a rare, inherited condition that damages nerves.
- Amvuttra is a modern treatment that helps control the progression of this disease.
- This is a vaccine given by a health professional every three months.
- Before starting treatment, discuss the pros and cons with your doctor.
- It is very important to monitor vitamin A levels during treatment and to notify the doctor immediately if there are any changes in vision.
- Tell your doctor immediately about any side effects or discomfort.
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