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First, let's see, why doesn't bleeding stop in hemophilia?
To put it simply, there are special proteins that help our blood clot. We call these " clotting factors ." Just like mortar is needed to hold bricks together when building a wall, these clotting factors are essential to stop bleeding. A person with hemophilia is deficient in one of these clotting factors.- People with hemophilia A lack a substance called Factor VIII.
- People with hemophilia B lack a substance called Factor IX.
So what is this new medicine called Qfitlia (fitusiran)?
This is a drug that works in a very strange way. We talked earlier about the fact that blood clotting requires something called thrombin. In our own body, there is another protein that controls the production of this thrombin, that is, it stops the production of more thrombin. It is called antithrombin . Think about it this way. You have a person in your body who helps blood clot (thrombin). But there is another person who controls and prevents that person from working, who prevents it from working. In hemophilia, the person who helps (thrombin) is initially produced less. So when the person who prevents (antithrombin) works normally, blood clotting decreases even more. What Qfitlia does is reduce the amount of antithrombin that prevents it. Then, the amount of thrombin that your body naturally produces is allowed to work properly without being hindered. What this does is, it encourages blood clotting by using the body's own system, not by giving you the clotting factor that you have reduced. This is the first antithrombin-reducing drug to be used in the treatment of hemophilia.Let's further explain the difference between these two treatment methods.
You can easily understand this difference from the table below.| Characteristic | Traditional treatment (Factor Replacement) | Qfitlia (fitusiran) treatment |
|---|---|---|
| Functionality | The clotting factor that is lacking in the body is given externally. | It reduces antithrombin, which prevents blood clotting, and allows the body to utilize more of its own thrombin. |
| How to give | Often intravenously. | Under the skin (subcutaneous). |
| Frequency | It may be necessary several times a week. | Once a month or every two months (6-12 times a year). |
| For those with inhibitors | Normal factor therapy does not work, and other special treatments (bypassing agents) are needed. | This treatment can also be used successfully in people with inhibitors (antibodies against the factors). |
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Check Availability →Is this medicine suitable for me and how do I use it?
If you have hemophilia A or B and are over 12 years of age, your doctor may consider whether this medicine is right for you. The most important thing is that it can also be used in people who have developed inhibitors (resistance) to previous factor therapy. However, before starting this treatment, your doctor will do several blood tests . 1. Antithrombin level: This measures how much antithrombin is in your blood. The dose of Qfitlia is determined based on this value. 2. Liver function: Your liver is monitored before and during treatment. This medicine is given as an injection under the skin (subcutaneously). This means that it is not given into a vein. You or a caregiver can give it to you at home after training. It is given in the skin of the abdomen, thigh, or back of the upper arm. You should avoid areas with bruises or cuts. If you are already taking other hemophilia treatments, you may need to stop or change those treatments after starting Qfitlia. Your doctor will explain this to you clearly. Never stop taking your current medications or stop taking Qfitlia without your doctor's advice. Doing so may increase your risk of bleeding.Should we also be aware of the side effects? How can we prevent them?
Like any medication, Qfitlia can have some side effects. It's important to be aware of them and take steps to prevent them.- Blood Clots: Because this medication promotes blood clotting, it is very rare for unwanted blood clots to form inside the veins. Your doctor will determine the right dose for your antithrombin level to reduce this risk.
- Elevated liver enzymes: Some people may have elevated liver enzymes . This is why doctors regularly monitor the condition of the liver with blood tests .
- Gallstones and Gallbladder Inflammation: If you suddenly experience symptoms such as severe stomach pain, nausea, and vomiting , inform your doctor immediately.
- Infections: There is a small risk of infection at the injection site. Therefore, it is important to always inject cleanly and as directed by your doctor. Good hygiene practices, such as frequent hand washing, are also important.
💡 Take-Home Message
- Qfitlia (fitusiran) is a new treatment for hemophilia A and B that works in a different way.
- This works by reducing the amount of antithrombin, which prevents blood clotting, instead of giving the body's low clotting factor externally.
- This medication is an injection under the skin, not into a vein, and is usually given infrequently, such as once a month or every two months.
- Your doctor will determine whether this treatment is right for you after several blood tests.
- To avoid side effects, it is essential to have blood tests (especially antithrombin and liver function) at regular intervals as prescribed by your doctor.
- Avoid stopping Qfitlia treatment without medical advice for any reason.
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