Have you ever noticed that your urine is a little dark, maybe even bloody, in the morning? Do you struggle with feeling tired all the time, feeling pale? These could be symptoms of a rare blood disorder that many people have not heard of called PNH, or Paroxysmal Nocturnal Hemoglobinuria. Don't be scared when you hear this name. Let's talk about everything simply and clearly.
What exactly is PNH?
Simply put, PNH is when our own immune system mistakenly attacks and destroys our own healthy red blood cells. Imagine, what is happening here is like an army that is supposed to protect a country, attacking the people of that country.
This is because there is a genetic mutation in some of the cells in our bone marrow. As a result, the red blood cells produced by that bone marrow are slightly weaker. These weak cells can be easily recognized and destroyed by our immune system.
- Destruction of red blood cells: In medical terms, this breakdown of red blood cells is called hemolysis .
- Urine color changes: Hemoglobin is a protein found in red blood cells that carries oxygen. When the cells break down, this hemoglobin is released into the blood, filtered by the kidneys, and excreted in the urine. This is called hemoglobinuria . This is why urine appears dark, brown, or blood-colored. This is often most obvious in morning urine.
But keep in mind that the symptoms of PNH are not the same for everyone. Some people may not notice any change in urine color at all.
Another serious complication of this disease is an increased risk of blood clots . Some of these clots can be life-threatening. Also, this disease can affect not only red blood cells, but also white blood cells that fight infection and platelets that stop bleeding.
What are the treatments for PNH disease?
Treatment for PNH depends on your symptoms and the severity of your condition. Your doctor will discuss with you which treatment is best for you. The table below lists some of the main treatments currently available.
| Treatment method | Simply put, what happens? |
|---|---|
| Androgen therapy | These are a type of male hormone. They stimulate the bone marrow and increase the production of red blood cells. They help reduce anemia. |
| Anticoagulants | They prevent blood clots from forming in the blood vessels. These are available as injections or pills. |
| Blood transfusions | When red blood cell or platelet levels become very low, the deficiency is compensated for by external blood transfusions. |
| Bone marrow transplant | This is the only way to completely cure PNH. But it is very risky. Therefore, it is only used for the most severe cases. |
| Complement Inhibitors | They target the part of the immune system that destroys red blood cells and stop that process. Soliris and Ultomiris are these types of drugs. |
| Growth factors | It helps to increase the production of red or white blood cells by stimulating the bone marrow. |
| Other medications | The doctor also prescribes folic acid, iron supplements, steroids, and immunosuppressants as needed. |
So what is this Empaveli?
Empaveli is a new and highly effective complement inhibitor drug used to treat PNH.
Think of our immune system as a chain of interconnected proteins. One part of this chain is called the `Complement system`. In PNH, this system becomes overactive and destroys red blood cells.
Older drugs (e.g. eculizumab) targeted a site called C5 in this chain. They only stopped the destruction inside the blood vessels (intravascular hemolysis).
But this new drug, called Empaveli, targets an earlier site, called C3. The advantage of this is that it controls the damage inside the blood vessels, as well as the damage inside organs like the liver and spleen (extravascular hemolysis).
Therefore, Empaveli may be particularly beneficial for patients who have not fully recovered from previous medications and who continue to have anemia.
How to use Empaveli?
Empaveli is a medicine that you can use at home.
- Method: This is a subcutaneous injection .
- Frequency: Usually given twice a week .
- Dosage: Your doctor may adjust your dosage by checking the level of an enzyme called `Lactate Dehydrogenase (LDH)` in your blood. LDH levels measure how much red blood cells are being broken down.
- How to use: There are two ways to use this. One is to use a small pump that delivers the medicine into the body over 30-60 minutes. The other is a one-time injector system that is applied to the abdomen.
Don't worry, your doctor or a trained nurse will first teach you how to use this.
Things to know about the effectiveness and safety of this drug
The success of Empaveli has been confirmed in two major clinical studies, PEGASUS and PRINCE.
Simply put, in both studies, patients who took Empaveli had significantly higher hemoglobin levels than those who took other treatments. They also had significantly fewer blood transfusions.
Side effects and safety
The main side effects seen with Empaveli are:
- Pain, redness, or swelling at the injection site: This is normal. You may find relief by applying an ice pack or applying warm water.
- Increased risk of infections: Because this medicine suppresses part of the immune system, there is a risk of becoming susceptible to certain bacterial infections (especially encapsulated bacteria).
To reduce this risk, your doctor will give you all the vaccinations you need before you start taking Empaveli. You should also keep a Patient Safety Card with you while you are taking the medicine and for 2 months after you stop taking it. This will help people who are treating you know that you are taking this medicine in case of an emergency.
What is the Empaveli REMS program?
This is a bit of a complicated word, but simply put, it means a special protection program . Due to the risk of infection with drugs like Empaveli, such programs are implemented by government agencies.
Under this `REMS (Risk Evaluation and Mitigation Strategy)` program:
1. The doctor who prescribes your medication must be registered in this program.
2. The pharmacy that gives you the medicine must also be certified for this.
3. Doctor, you need to be well informed about the risks of the medication and the symptoms of an infection (fever, body aches, severe headache).
This ensures that you are fully aware of the benefits and risks of this medication and are using it safely.
Take-Home Message
- PNH is a rare but manageable disease in which your own immune system destroys red blood cells.
- Empaveli is a highly effective, new treatment for PNH that stops the destruction of blood cells.
- This medicine can cause infection, so be sure to get the shots your doctor gives you. Be aware of any signs of infection.
- Empaveli is provided under a special mechanism of action (REMS). Always keep the Patient Safety Card you receive with you.
- If you have any questions about your condition or treatment, please speak to your doctor without hesitation.
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