Do you have symptoms like constant fatigue, shortness of breath when walking a little bit, or dark brown urine in the morning? Sometimes these can be symptoms of a rare blood disorder called Paroxysmal Nocturnal Hemoglobinuria, or PNH as we doctors call it. Don't be scared when you hear this name. Although it is a bit complicated, let's talk about this disease and a new treatment for it called Fabhalta in simple and detailed terms.
What exactly is PNH?
Simply put, PNH is a very rare blood disorder caused by a genetic defect. Our red blood cells (RBCs) have a protective protein coating around them. However, in PNH, this protective coating is not formed.
Think of our red blood cells as soldiers without armor. Without this protection, our body's own immune system becomes confused and starts attacking and destroying these healthy red blood cells, thinking they are enemies. This premature breakdown of red blood cells is what we medically call hemolysis . This destruction can occur both inside the blood vessels (intravascular) and outside the blood vessels (extravascular).
When red blood cells are destroyed in this way, the main symptoms can be anemia and blood clots. These blood clots can sometimes be life-threatening. A person with PNH may experience many of these symptoms, or they may have just a few.
| Common symptoms of PNH disease | |
|---|---|
| Constant fatigue or weakness | Difficulty breathing |
| Headache | Irregular or rapid heartbeat |
| Stomach pain | Easily bruised skin |
| Dark-colored (brown) urine, especially at night or in the morning | Redness, pain, and swelling in a specific area of the skin (may be a sign of a blood clot) |
Treatment for PNH includes blood transfusions, folic acid or iron tablets for anemia, anticoagulants, and drugs that protect red blood cells from attack by the immune system. The treatment you receive will depend on how many symptoms you have and how severe they are.
How does the medicine Fabhalta work?
Drugs such as eculizumab (Soliris) or ravulizumab (Ultomiris) that are usually given as injections for PNH work by blocking a part of our immune system called the complement system, called C5. While these stop intravascular hemolysis, some people may continue to have anemia due to extravascular hemolysis.
This is where the first oral medication called Fabhalta becomes important.
Think of our immune system as an army defending our country. In PNH, parts of this army get confused and attack our own healthy red blood cells. Fabhalta blocks a protein called factor B that causes this confusion. This stops the immune system from attacking the red blood cells. This increases the number of healthy red blood cells, reduces symptoms, and reduces the need for blood transfusions.
How should I use this medicine?
Fabhalta is a capsule that is swallowed whole twice a day, with or without food. Do not break, crush, or chew the capsule. If you miss a dose, take it as soon as you remember and then take the remaining doses as usual. If you are already taking another PNH medicine (such as eculizumab or ravulizumab), your doctor will explain the correct way to switch to Fabhalta.
Very important: It is essential that you have received all your required vaccinations at least two weeks before starting this treatment. This includes the pneumococcal vaccine and meningitis vaccine. If you are unable to receive your vaccinations and are unable to start Fabhalta, your doctor will give you the vaccinations and start you on a course of antibiotics at the same time.
Even if your symptoms improve, do not stop taking Fabhalta without medical advice . If you do, your red blood cells may start breaking down faster again.
How have the effects of this medicine been studied?
Two main studies were conducted to confirm the efficacy and safety of Fabhalta. Both included patients with PNH and the anemia that resulted from it.
1. APPLY-PNH study: This involved people who had anemia despite taking other PNH medications (eculizumab or ravulizumab) for at least 6 months. They were divided into two groups, one group was given Fabhalta, and the other group continued to take the old medication.
2. APPOINT-PNH study: This involved patients who had not previously received any other complementary medicine for PNH. They were all given Fabhalta.
The results of these studies, after 24 weeks, are very encouraging.
| Benefit received | Result |
|---|---|
| Increased hemoglobin level | More than 82% of patients who took Fabhalta had a hemoglobin increase of 2 g/dL, compared to none of those who took the other drugs. |
| Avoiding blood transfusions | 95% of patients who took Fabhalta did not need external blood transfusions during the study period, compared to only 50% of those who took the other drugs. |
Remember, despite the results of these studies, your results may vary from person to person. It's best to discuss the benefits and risks of the medication with your doctor before making a decision.
How do you know if the medicine is working?
If Fabhalta treatment is successful, your red blood cell and hemoglobin levels will start to increase. You may experience things like:
- Increased energy and decreased fatigue
- Decreased dark color of urine
- Increased appetite and decreased stomach pain
- Reducing the need for external blood transfusions
- Being able to do daily tasks easily
Since the results of treatment vary from person to person, keep a record of your symptoms and discuss them with your doctor every time you see him.
Side effects and how to manage them?
Like any medicine, Fabhalta can cause side effects. The most common are headache, diarrhea, nausea, stomach pain, and skin rash. Symptoms of the common cold and other bacterial or viral infections may also occur.
Serious infections that need attention!
Because this medicine suppresses part of the immune system, there is a risk of serious infections from certain types of bacteria. Especially *Streptococcus pneumoniae*, *Neisseria meningitidis*, and *Haemophilus influenzae type B*. These infections can be life-threatening if not recognized and treated promptly.
The best way to prevent infection is to get all recommended vaccinations , wash your hands well, and stay away from people who are sick. If you experience any of the following symptoms, tell your doctor immediately or go to the nearest hospital emergency room (ETU).
| Warning signs of a serious infection | |
|---|---|
| Fever with or without phlegm, chest pain and cough, rapid heartbeat | Symptoms such as body aches and fever |
| Clammy skin | Severe headache with fever, nausea, and vomiting, or neck/back stiffness |
| Confusion | Vision changes |
If you are concerned about any side effects, talk to your doctor immediately. If you suspect poisoning from a medication, you can call the National Poisons Information Centre at the Colombo National Hospital .
What blood tests are required during treatment?
Your doctor will perform several blood tests to monitor your health and your response to the medication.
- Red blood cell (RBC) and hemoglobin levels: These are checked regularly in PNH. This helps to see how you are responding to treatment and to determine if a blood transfusion is needed.
- Blood fat levels: Fabhalta may increase your cholesterol and triglyceride levels. Therefore, your doctor will recommend a fasting lipid profile test before and during treatment.
Can there be problems with other medications?
Yes, some medications can interact with Fabhalta. Some medications, such as Rifampin, can reduce the effectiveness of Fabhalta. Taking Fabhalta with some medications, such as Gemfibrozil, is not recommended.
Therefore, it is extremely important to inform your doctor about all the medications you are taking – including prescription medications, over-the-counter medications, vitamins, traditional medicines, and even supplements.
Take-Home Message
- PNH is a rare condition in which the body's own immune system destroys red blood cells because they lack the protein layer that protects them.
- Fabhalta is a new oral drug for PNH. It works by stopping a malfunction in the immune system and protecting red blood cells.
- Before starting this medication, it is mandatory to have been vaccinated against diseases such as meningitis and pneumonia.
- If you develop signs of a serious infection during treatment, such as fever, severe headache, or confusion, seek medical advice immediately.
- Even if your symptoms improve, never stop taking the medication without your doctor's advice.
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