A new drug for hemophilia? Let's learn about Qfitlia (fitusiran)?

If you have hemophilia, you probably know how frustrating and frightening it can be to bleed constantly, sometimes for no reason, and have trouble stopping bleeding from even the smallest cuts. You may already be taking medication to treat your bleeding, or you may be taking medication to prevent it. However, with the advancement of medical science, there are now many effective, new treatments for this condition. So today, let's talk about a new, different drug. It's called Qfitlia (fitusiran).

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.

When these factors decrease, the production of another important step in the blood clotting process, called thrombin , decreases. Thrombin is the main factor that ultimately forms a blood clot and stops bleeding. So when thrombin decreases, it becomes difficult to stop bleeding when it occurs. Do you understand? Usually, what we have been doing as treatment for so long is to artificially make the clotting factor (factor 8 or 9) that is lacking in your body and give it as an injection into a vein. But the Qfitlia drug we are talking about today works in a completely different way.

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 needs something called thrombin to clot. In our own bodies, 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 of it this way. You have a blood clotting factor (thrombin) in your body that helps your blood clot. But there is another factor (antithrombin) that controls and prevents it from working. In hemophilia, the production of the factor that helps (thrombin) is initially low. So when the factor that prevents (antithrombin) works normally, the 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. This means that it uses the body's own system to encourage blood clotting, rather than giving you the clotting factor that you have lost. 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.

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.

It is very important to have regular blood tests as your doctor recommends. Antithrombin levels are usually checked at the first, third, fifth, and sixth months of treatment. This is how the right dose of medication can be determined for you and how 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.

Studies have shown that people with hemophilia (both those with and without inhibitors) who took Qfitlia had significantly fewer bleeding episodes than those who did not take any preventive treatment. Also, people with inhibitors who switched from other treatments to Qfitlia had fewer bleeding episodes. But keep in mind that these results may vary from person to person.

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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