Have you or someone you know ever been prescribed a medicine called 'Givosiran' by a doctor? When you hear that name, you may feel a little unfamiliar, that is, unfamiliarity, and maybe even a little scared. It's normal to feel that way. Because these are not the types of medicines we hear about every day. So today, let's talk about this medicine called Givosiran, what it is for, how to use it, and what precautions you need to take, in a very simple way that you can understand.
What is this Givosiran?
Simply put, Givosiran is a vaccine. It is commonly known by the brand name ``(GIVLAARI)``. This medicine is mainly used to treat a very rare disease called ``Acute Hepatic Porphyria`` .
Now you may be wondering what 'Acute Hepatic Porphyria' is. This is a genetic disease that occurs in our liver. Our body needs a chemical called 'porphyrin' to make hemoglobin in the blood. However, in people with this disease, due to a defect in that process, this chemical called porphyrin accumulates unnecessarily in the body. This can cause severe symptoms such as severe stomach pain, vomiting, muscle weakness, and mental problems.
This vaccine, called Givosiran, is very special. It's made using a very advanced technology called 'small interfering RNA' (siRNA) . This means that this drug goes directly into our cells and stops the genetic message that causes the harmful accumulation of porphyrin. It's like stopping someone who's giving you the wrong advice.
This is not like a regular painkiller. It is a very targeted medicine that goes to the root cause of the disease and treats it.
Things you need to tell your doctor before taking medicine
Before you start Givosiran, it is very important to talk openly with your doctor about your complete health history. Be sure to tell them about the following:
- Kidney disease: If you have any kidney-related condition.
- Liver disease: If you have other liver diseases in addition to your existing porphyria condition.
- Allergies: Tell your doctor if you are allergic to any other medicine, food, dyes, or preservatives, not just to Givosiran.
- Pregnancy: If you are pregnant or expecting a baby soon.
- If you are a breastfeeding mother: If you are breastfeeding your baby.
Based on this information, the doctor will decide whether this treatment is suitable for you or not and how to proceed.
How to get the medicine and what to be careful about
This is not a medicine you can take home and use.
- Administration: This is a subcutaneous injection .
- Location: This vaccine is only given in a medical facility, such as a hospital or clinic, by a trained healthcare professional.
- For children: If this medicine is used in young children, special care is required under the advice of a pediatrician.
- If you miss a dose: Your doctor will give you a date and time to get your vaccine. It is very important to get your treatment on time. If for some reason you cannot go on that day, call your doctor or clinic immediately and let them know.
- If you overdose: Since this is administered by a medical professional, the chances of an overdose are very low. However, if you suspect that something has happened, you should immediately call the National Poisons Information Centre at the Colombo National Hospital , or go to the Emergency Treatment Unit (ETU) of the nearest hospital.
Possible side effects of the medicine
Like any medicine, Givosiran can cause side effects in some people. It is important to be aware of these. Let's break these down into two parts.
| Side effects and symptoms | Description |
|---|---|
| Serious side effects that require immediate medical attention | |
| Allergic Reactions | Skin rash, eczema-like conditions, itching, swelling of the face, lips or tongue, difficulty breathing. If any of these occur, it is an emergency. |
| Symptoms of Liver Injury | Dark yellow or brown urine, yellowing of the eyes or skin (jaundice), pain in the upper right side of the stomach, nausea, loss of appetite, feeling lethargic, and fever. |
| Symptoms of Kidney Injury | Difficulty urinating or a significant change in the amount of urine you pass (passing very little or no urine at all). |
| Things that are usually not serious, but should be reported to the doctor if they persist | |
| Nausea | Feeling like your stomach is churning or you feel like vomiting. |
| Pain at the injection site | Redness, slight swelling, or pain at the injection site. |
| Tiredness | Feeling more tired than usual. |
Can there be problems with other medications?
Yes, it definitely can. We call that 'Drug Interactions'. The Givosiran vaccine can interact with some other medications.
So, tell your doctor about everything you use . That means:
- Medicines prescribed by doctors for other diseases.
- Medicines that can be purchased from the pharmacy without a prescription (e.g. painkillers, cold medicine).
- Vitamins, minerals or other supplements.
- Ayurvedic, local or traditional herbs.
- If you smoke or drink alcohol, tell me.
In particular, the psychiatric drug thioridazine should not be used with this vaccine at all. Also, caffeine-containing products and cough medicines such as dextromethorphan can cause problems with this.
This list is not complete, so it's best to be clear with your doctor about everything you're taking.
Take-Home Message
- Givosiran is a special injection given under the skin for a rare liver disease called 'Acute Hepatic Porphyria'.
- This medicine is only given in a hospital or clinic by a trained medical professional. It is not for home use.
- Before starting treatment, it is imperative to inform your doctor about all your medical conditions, allergies, and all other medications and supplements you are taking.
- Always be on the lookout for serious side effects, such as allergic reactions or signs of liver or kidney damage. If you notice any of these, inform your doctor immediately.
- Never miss blood tests and treatment appointments prescribed by your doctor.
- If you have any questions or concerns about this medication, the best person to ask about it is the doctor who is treating you.


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