Imagine that only one part of your body, for example an arm, a leg, or a certain area of skin, grows abnormally faster than the rest. This is a very rare condition that some people are born with. In medical terms, we call this group of conditions PIK3CA-Related Overgrowth Spectrum, or PROS for short. This is something that really causes a lot of problems for the person who sees it, as well as the person who experiences it. Today, in this article, we will talk about what this PROS is and about Vijoice, the first drug approved specifically for this condition.
What exactly is PROS?
Simply put, PROS is a condition caused by genetic factors. We all have genes in our bodies that control the growth, division, and death of cells. `PIK3CA` is one such important gene. Think of this gene as a `switch` that controls the growth of our cells.
However, in a person with PROS, there is a defect, or mutation, in the `PIK3CA` gene. This causes the `switch` to be stuck in the `on` position. Then the protein in question starts working too fast and too much. As a result, some parts of the body, for example, the skin, blood vessels , bones , and brain, start to grow uncontrollably.
The important thing is that PROS does not affect every part of the body. It can vary from one part of the body to another, and from person to person. Therefore, the symptoms are also very diverse.
Until now, there was no complete cure for PROS. Treatments focused on managing the symptoms. For example, medications for pain, itching, and seizures, and surgery to remove the abnormal growths. However, there was a risk of those growths growing again after surgery.
How does the drug Vijoice (alpelisib) work?
This is where the drug Vijoice comes in. Vijoice is the first drug to target the underlying cause of PROS, the hyperactivity caused by the genetic mutation.
The active ingredient in this is alpelisib . This drug is actually used for some types of breast cancer . The reason for this is that those cancer cells grow due to overactivity of a pathway called PIK3, just like in PROS.
So what Vijoice does is block the PIK3 pathway, which acts like a 'switch' stuck in the 'on' position. This slows down the abnormal growth of cells, reduces the size of already growing tissues, and controls symptoms.
What are the benefits of this medicine?
Studies conducted on this drug (EPIK-P1 and EPIK-P3) have shown significant benefits for patients.
| Benefit | Simple explanation |
|---|---|
| Smaller growths (lesions) | It has been observed that many people who took the drug have had their abnormally developed fatty tumors (lipomas) and vascular malformations reduced in volume by 20% or more. |
| Reduced need for surgery | About 70% of children and 90% of adults taking Vijoice have not needed surgery. |
| Symptom relief | Patients have reported a significant reduction in symptoms such as pain, itching, and fatigue. |
| Making everyday tasks easier | About 81% of patients who participated in the studies reported that their ability to perform daily activities and quality of life had improved compared to before the drug. |
Important: These results may not be the same for everyone. It is best to discuss with your doctor whether this medication is right for you and what the benefits and risks are.
What are the most common side effects and how to manage them?
Like any medication, Vijoice can cause side effects . However, most of these are mild or moderate and can be managed.
| Side effect | How to manage and what to pay attention to |
|---|---|
| Diarrhea | Drink plenty of fluids such as water and juice. If you experience symptoms such as severe diarrhea, stomach cramps, or blood or mucus in your stool, tell your doctor immediately . |
| Dehydration | Diarrhea can cause dehydration. Watch out for symptoms like extreme dizziness, fatigue, and decreased urination. These can affect your kidneys, so tell your doctor right away. |
| Mouth sores (Stomatitis) | Mouth sores and pain may occur. Avoid mouthwashes containing alcohol. Rinse your mouth with lukewarm salt water. Stay away from spicy, acidic foods. |
| Increased blood sugar (Hyperglycemia) | Your doctor will check your blood sugar levels before you start taking this medicine and during your treatment. Tell your doctor if you have symptoms such as excessive thirst, frequent urination, or fatigue. People with diabetes mellitus should be especially careful about this. |
| Hair thinning (Alopecia) | Hair loss or thinning may occur during treatment. Use mild shampoos. Talk to your doctor about this. |
Can there be problems with other medications?
Yes. Vijoice can interact with some other medications. Simply put, some medications can reduce the effectiveness of Vijoice. Vijoice can also change the effectiveness of some other medications.
For example, medications such as carbamazepine for epilepsy, rifampin for tuberculosis, and herbal products such as St. John's wort can affect this.
Therefore, before you start this medication, it is essential to tell your doctor about all the medications you are currently taking (including vitamins, herbal products, and painkillers) so that he or she can make any necessary adjustments.
Take-Home Message
- PROS is a rare condition in which parts of the body grow abnormally due to a mutation in the gene called `PIK3CA`.
- Vijoice (alpelisib) is the first drug approved to target the underlying process that causes this disease.
- This can help shrink abnormal growths, reduce the need for surgery, and improve quality of life.
- Side effects such as diarrhea, increased blood sugar, and mouth sores may occur, and it is very important to manage them and inform the doctor.
- It is essential to inform your doctor about all other medications you are taking. Never start or stop taking any medication without medical advice.
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