Nitisinone
Tyrosinemias
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Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor
Controlled Drug Substance
No
Nitisinone
Summary
Nitisinone is used to treat hereditary tyrosinemia type 1, which is a genetic disorder affecting the breakdown of the amino acid tyrosine. This condition can lead to liver damage and other serious health issues.
Nitisinone works by blocking the breakdown of tyrosine, which is an amino acid, in the body. This prevents the formation of toxic byproducts that can damage the liver and other organs.
The usual starting dose of Nitisinone for adults is 1 mg to 2 mg per kilogram of body weight per day, divided into two doses. It is taken as a capsule or liquid, with or without food.
Common side effects of Nitisinone include elevated tyrosine levels, which can cause eye and skin issues. Stomach upset, including nausea and vomiting, may also occur but are not common.
Nitisinone can cause elevated tyrosine levels, leading to eye and skin problems. It should be used with caution in patients with liver problems. Regular monitoring of blood levels and liver function is important.
Indications and Purpose
How does Nitisinone work?
Nitisinone works by blocking the breakdown of tyrosine, an amino acid, in the body. This prevents the formation of toxic byproducts that can damage the liver and other organs. Think of it like stopping a leak in a pipe to prevent water damage. By reducing these harmful substances, Nitisinone helps improve liver function and reduces the risk of liver cancer.
Is Nitisinone effective?
Nitisinone is effective for treating hereditary tyrosinemia type 1, which is a genetic disorder affecting the breakdown of the amino acid tyrosine. It works by blocking the breakdown of tyrosine, reducing toxic byproducts. Clinical studies show Nitisinone improves liver function and reduces the risk of liver cancer in affected individuals.
What is Nitisinone?
Nitisinone is a medication used to treat hereditary tyrosinemia type 1, a genetic disorder affecting the breakdown of the amino acid tyrosine. It works by blocking the breakdown of tyrosine, reducing toxic byproducts. This helps improve liver function and reduces the risk of liver cancer in affected individuals. Nitisinone is taken as a capsule or liquid.
Directions for Use
For how long do I take Nitisinone?
Nitisinone is usually a long-term medication for managing hereditary tyrosinemia type 1, a genetic disorder affecting the breakdown of the amino acid tyrosine. You'll typically take Nitisinone every day as a lifelong treatment unless your doctor suggests otherwise. How long you'll need this medication depends on your body's response and any side effects you experience.
How do I dispose of Nitisinone?
Dispose of Nitisinone by taking it to a drug take-back program or collection site at a pharmacy or hospital. If you can't find a take-back program, you can throw it in the trash at home. Mix it with something undesirable like used coffee grounds, seal it in a plastic bag, and throw it away.
How do I take Nitisinone?
Take Nitisinone as prescribed by your doctor. It's usually taken once or twice daily, with or without food. You can swallow the capsule whole or open it and mix the contents with water or applesauce. If you miss a dose, take it as soon as you remember unless it's almost time for your next dose. Don't take two doses at once. Follow your doctor's specific advice about diet and fluid intake while taking this medication.
How long does it take for Nitisinone to start working?
Nitisinone begins working in your body shortly after you take it. However, you may not notice all the benefits right away. For hereditary tyrosinemia type 1, improvements in symptoms and liver function may take several weeks to become noticeable. How quickly the medication works can depend on your overall health and response to treatment.
How should I store Nitisinone?
Store Nitisinone at room temperature, away from moisture and light. Keep it in a tightly closed container. Don't store it in humid places like bathrooms. Always store Nitisinone out of children's reach to prevent accidental swallowing. Check the expiration date regularly and properly dispose of any unused or expired medication.
What is the usual dose of Nitisinone?
The usual starting dose of Nitisinone for adults is 1 mg to 2 mg per kilogram of body weight per day, divided into two doses. Your doctor may adjust your dose based on your response and needs. The maximum recommended dose is 2 mg per kilogram per day. Always follow your doctor's specific dosing instructions for your personal health needs.
Warnings and Precautions
Can I take Nitisinone with other prescription drugs?
Nitisinone can interact with other medications, increasing the risk of adverse effects. It's important to tell your doctor about all the medications you take. Your doctor can help you avoid any potential interactions and ensure your treatment is safe and effective. Regular monitoring of your health is important while taking Nitisinone.
Can Nitisinone be taken safely while breastfeeding?
Nitisinone is not recommended while breastfeeding due to limited safety data. We don't know if it passes into human breast milk. If you're taking Nitisinone and want to breastfeed, talk with your doctor about safer medication options that would allow you to nurse your baby safely.
Can Nitisinone be taken safely while pregnant?
Nitisinone isn't recommended during pregnancy due to limited safety data. Animal studies show potential risks, but human data is lacking. If you're pregnant or planning to become pregnant, talk with your doctor about the safest way to manage your condition. Your doctor can help create a pregnancy-specific treatment plan.
Does Nitisinone have adverse effects?
Adverse effects are unwanted reactions to a medication. Common adverse effects of Nitisinone include elevated tyrosine levels, which can cause eye and skin issues. Serious effects may include liver problems. If you notice any new or worsening symptoms, contact your doctor. Regular monitoring helps manage these effects and keep your treatment safe.
Does Nitisinone have any safety warnings?
Yes, Nitisinone has important safety warnings. It can cause elevated levels of tyrosine, which may lead to eye and skin problems. Regular monitoring of blood levels and eye exams are recommended. Not adhering to these warnings can result in serious complications. Always follow your doctor's advice and report any new symptoms immediately.
Is it safe to drink alcohol while taking Nitisinone?
It's best to avoid alcohol while taking Nitisinone. Alcohol can interfere with the medication's effectiveness and may worsen side effects like liver problems. If you do choose to drink occasionally, limit how much alcohol you consume and talk with your doctor about alcohol use while taking Nitisinone.
Is it safe to exercise while taking Nitisinone?
You can exercise while taking Nitisinone, but keep a few things in mind. This medication can cause elevated tyrosine levels, which may affect your energy levels. To exercise safely, drink plenty of water before, during, and after physical activity. Watch for signs of dizziness or unusual fatigue. If you notice these symptoms, slow down or stop exercising and rest.
Is it safe to stop Nitisinone?
Stopping Nitisinone suddenly can cause serious problems for your health condition. If you're taking it for a chronic condition, stopping could worsen your symptoms. Always talk with your doctor before stopping Nitisinone. They might suggest gradually reducing your dose or switching to a different medication to keep your condition under control.
Is Nitisinone addictive?
Nitisinone is not addictive or habit-forming. This medication doesn't cause dependency or withdrawal symptoms when you stop taking it. Nitisinone works by affecting the metabolism of certain amino acids, not brain chemistry, so it doesn't lead to addiction. You won't experience cravings for this medication or feel compelled to take more than prescribed.
Is Nitisinone safe for the elderly?
Elderly patients may be more vulnerable to the side effects of Nitisinone, such as liver problems. Regular monitoring of liver function and tyrosine levels is important. Specific risks include increased sensitivity to the medication's effects. Always consult your doctor about the safety of Nitisinone for elderly patients.
What are the most common side effects of Nitisinone?
Side effects are unwanted reactions to a medication. Common side effects of Nitisinone include elevated tyrosine levels, which can cause eye and skin issues. These effects vary from person to person. If you notice new symptoms after starting Nitisinone, they might be temporary or unrelated to the medication. Talk with your doctor before stopping any medication.
Who should avoid taking Nitisinone?
Don't take Nitisinone if you're allergic to it or its ingredients. Serious allergic reactions require immediate medical help. Nitisinone should be used with caution in patients with liver problems. Always consult your doctor about these concerns and follow their advice to ensure safe use of the medication.
