Hemophilia and Alhemo: A modern treatment to control bleeding

Have you ever heard of a condition called hemophilia ? This is a rare but very serious condition. Simply put, it is a condition in which the blood does not clot properly in our bodies. This means that even a small injury, fall, or cut can sometimes cause significant bleeding. Sometimes, spontaneous bleeding can occur for no apparent reason.

What exactly is hemophilia?

Imagine that when we bleed, our bodies have a small team of workers working to stop it. We call these workers 'factors'. These are actually special types of proteins. These factors come together to form a blood clot and stop the bleeding.

There are two main types of hemophilia:

• Hemophilia A: These people do not have enough of the protein called factor VIII , which is needed for blood clotting.
• Hemophilia B : These people lack a protein called factor IX .

This is usually treated by your doctor giving you the deficient factor intravenously. Treatment to prevent bleeding is called “prophylaxis,” and treatment when bleeding occurs is called “on-demand.”

But, there's a small problem here...

In some patients, the body starts to work against these factors that we give through vaccination. As if it thinks they are an enemy and attacks them. We call this the formation of “inhibitors” . When this happens, the power of the factor therapy that we give is reduced. So, treating people with these types of ‘inhibitors’ is a little more complicated.

What happens with this new drug called Alhemo?

Alhemo(concizumab-mtci) is a new drug approved to reduce or prevent bleeding, especially in patients with hemophilia A or hemophilia B who have the previously mentioned 'inhibitors'.

Simply put, our bodies naturally have a protein that prevents blood from clotting. It's called TFPI (Tissue Factor Pathway Inhibitor) . Think of it like a 'traffic cop' in our bodies; it controls the blood clotting process. What Alhemo does is slightly interfere with the work of this 'cop' called TFPI. Then, the body increases the production of another factor that helps blood clot (factor Xa), and the ability to form blood clots improves. This greatly reduces the risk of bleeding.

How to use Alhemo?

Alhemo is a daily subcutaneous injection . It comes in a prefilled pen, so it's very easy to use. If your doctor decides that you can give yourself this injection at home, they will show you exactly how to do it.

• Dose: A slightly higher dose (loading dose) is given on the first day of treatment. After that, you should take the prescribed maintenance dose daily. The dose is determined based on your weight, so you should tell your doctor if your weight changes.
• Tests: Four weeks after starting treatment, you will have a blood test to check if the amount of medicine in your body is correct. Based on the results, your doctor may adjust your dosage.
• Vaccination sites: The vaccine is given in the abdomen (2 inches around the navel) or thigh. Do not inject in the same place every day, but change the place. Do not inject in areas that are sore, bruised, red, have eczema, or have scars .

What are the side effects? How to deal with them?

Like any medicine, Alhemo can have side effects.

Common side effects

Because this is a vaccine, minor injection site reactions may occur. For example:

• Bruising, redness, itching, or slight bleeding at the injection site.
• To help with this, you can place a cool compress on the area.
• This can also be reduced by changing the injection site every day.
• Do not take any painkillers or other medications without consulting your doctor.

Serious side effects – be especially careful about this!

These blood-clotted hands, legs, lungs, heartOr it can form in the veins in places like the brain. If you are preparing for any surgery, you should also tell your doctor about it.

If you experience any of the following symptoms, seek medical advice immediately without delay:

• Heart palpitations
• Chest pain or tightness
• Difficulty breathing
• Difficulty speaking, changes in vision
• Pain or swelling in the legs

How do you get a special medicine like this?

Alhemo is not a medicine that you can buy at a regular pharmacy. These are a type of 'specialty medicine'. There is a special process to obtain these.

After your doctor prescribes the medication, you will be contacted by a specialty pharmacy. They will be the ones who will give you the details on how to have the medication shipped to your home. You may also need to get prior authorization from your insurance company. A representative from the company that makes the medication may also be able to help with this process. Your doctor will guide you through all of this.