Has your doctor told you that you have a rare condition called Systemic Mastocytosis ? You may feel a little scared and worried when you hear that. It's very normal. But don't worry. There are now very good and effective treatments for such conditions. Today we are going to talk about the medicine Ayvakit (avapritinib) that you were prescribed. Let's understand everything about this disease and the medicine simply and clearly.
What exactly is Systemic Mastocytosis (SM)?
Simply put, Systemic Mastocytosis is a rare condition in which a type of cell called 'mast cells' in our body grows excessively. These mast cells are part of our immune system. Like our country's defense forces. When something foreign enters the body, these cells become activated and release chemicals like 'histamine'.
Imagine that when a mosquito bites you, the area becomes itchy and red, right? That's what happens because of this histamine. But in a person with Systemic Mastocytosis, these mast cells accumulate in an uncontrolled manner in the body. They accumulate in the skin, bone marrow, or internal organs like the liver and spleen. Because of this, the body can experience allergic symptoms, such as itching, redness of the skin, stomach aches, and dizziness, for no apparent reason.
The word "systemic" means that it can affect the entire body, not just one area. There are many different types of this disease, from mild, slow- growing (indolent) forms to advanced, cancerous forms.
How do I know if Ayvakit is right for me?
Your doctor will perform a series of tests to determine exactly what type of Systemic Mastocytosis you have. The results of those tests and your symptoms will determine whether Ayvakit is right for you. Ayvakit is currently approved to treat several types of Systemic Mastocytosis in adults.
| Type of disease | A simple explanation |
|---|---|
| Advanced Systemic Mastocytosis (AdvSM) - a severe, rapidly spreading type | |
| Aggressive Systemic Mastocytosis | Excessive accumulation of mast cells leads to dysfunction of organs such as the liver, digestive tract, and bones. |
| SM with an associated blood disorder (hematological neoplasm) | As mast cells increase, other blood-related diseases (e.g. myelodysplastic syndromes) may develop. |
| Mast cell leukemia | This is a very rare, fast-growing cancer in which mast cells accumulate in large numbers (more than 20%) in the tissues. |
| Indolent Systemic Mastocytosis (ISM) - a mild, slow-growing type | |
| Indolent Systemic Mastocytosis (ISM) | This is not cancer. Mast cells grow slowly. Although they can cause unpleasant symptoms, organ failure usually does not occur. |
Important: It can be dangerous to use Ayvakit if your blood platelet count is too low. Platelets are a type of cell that helps stop bleeding. Your doctor will check your platelet count regularly before you start taking this medicine and while you are taking it. This is to reduce the risk of serious side effects, such as bleeding in the brain.
How does this Ayvakit medicine work on the body?
Ayvakit is a medicine that belongs to a group of drugs called 'kinase inhibitors'. Kinases are a type of enzyme protein that helps send signals between cells in our body. These signals are very important for things like cell growth and division.
Many people with Systemic Mastocytosis have a mutation in a protein called KIT found in mast cells . This mutation causes the KIT protein to become overactive, causing the mast cells to grow uncontrollably.
The Ayvakit drug targets the mutated KIT protein and stops it from working . It's like silencing a tower that's sending out the wrong signal. This stops the overproduction of mast cells, reduces symptoms, and controls organ damage.
How should I use Ayvakit pills?
Ayvakit is a pill. The most important thing when taking it is to take it on an empty stomach . That means, you should take it at least an hour before a meal, or two hours after a meal. That's when the medicine is best absorbed by the body.
The commonly prescribed doses are as follows:
- For Advanced Systemic Mastitis TOS (AdvSM): 200mg once daily.
- For Indolent Systemic Mastocytosis (ISM): 25mg once daily.
This is usually a long-term treatment. Your doctor will tell you to keep taking the medicine until you are getting good results from it and are not having any serious side effects. If you have any side effects, your doctor may decide to lower your dose, temporarily stop it, or stop it completely.
Always follow your doctor's instructions exactly. Never stop taking your medication or change the dosage without talking to your doctor. Seek medical advice immediately if you experience any side effects that seem severe.
What does research say about the success of Ayvakit?
There is information about three clinical trials conducted to investigate the efficacy and safety of the Ayvakit drug.
For AdvSM (severe): In the two studies, EXPLORER and PATHFINDER, 57% of people who took Ayvakit, or more than half, had a reduction in symptoms and some recovery of function in the affected organs. Half of those who showed results maintained that improvement for 38 months, or more than 3 years.
For ISM (mild type): In the PIONEER study, those who took Ayvakit had significantly less symptoms (such as itching, stomach pain, and fatigue) than those who took placebo. This means that this drug has provided significant relief in improving quality of life.
These studies show that Ayvakit is a very effective treatment option for Systemic Mastocytosis. However, keep in mind that it does not completely cure the disease, and the results will not be the same for everyone.
Can there be problems with other medications? (Drug Interactions)
Yes, Ayvakit can interact with some medications. Especially those that affect the enzyme CYP3A in our liver. Some medications can increase the level of Ayvakit in the body and increase side effects, while other medications can decrease the level of Ayvakit and reduce its effectiveness.
Therefore, be sure to inform your doctor about all medications you are taking (medications for other diseases, vitamins, traditional medicines, supplements). He or she can then check for any problems and, if necessary, change your other medications or change the dosage of Ayvakit.
Also, if you are taking blood thinners, such as warfarin (Coumadin, Jantoven) , your risk of bleeding into the brain from Ayvakit may be slightly increased. Therefore, your doctor will monitor you more closely if you are taking such medications.
How to get Ayvakit medicine?
Ayvakit is a prescription-only medicine, usually prescribed by a hematologist or oncologist.
This is not a medication that you can get at a regular pharmacy . It is available at a 'specialty pharmacy'. Once your doctor has forwarded the prescription to that specialty pharmacy, they can ship the medication to your home or to your doctor's office.
Take-Home Message
- Systemic Mastocytosis (SM) is a rare condition caused by an excessive accumulation of mast cells in the body.
- Ayvakit is a highly effective, modern medicine that targets the root cause of this disease and treats it.
- This medicine should definitely be taken on an empty stomach (one hour before or two hours after a meal).
- It is very important to inform your doctor about all other medications, vitamins, and supplements you are taking.
- You should never stop taking medication or change the dosage without medical advice.
- Talk to your doctor immediately about any discomfort or side effects. Don't be afraid, know the right information, and with proper treatment, you can live a healthy life.
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