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Nitisinone
Nitisinone (brand name: Nitisinone or Nitisinone-2) is a medication used primarily in the treatment of tyrosinemia type 1, a rare genetic disorder that affects the body's ability to break down the amino acid tyrosine. By inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase, nitisinone reduces the accumulation of toxic metabolites, thus helping to manage the condition and prevent associated complications.
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Frequently Asked Questions
How quickly does nitisinone work?
Nitisinone may help reduce levels of toxic metabolites within a few days to weeks of starting treatment, but monitoring is necessary to assess its effectiveness.
Can I take nitisinone with food?
Yes, nitisinone can be taken with or without food; consistency in how it is taken is important.
What should I do if I miss a dose?
If you miss a dose, take it as soon as you remember. If it’s close to the time for your next dose, skip the missed dose and continue with your regular schedule. Do not double up doses.
Are there any dietary restrictions while using nitisinone?
Patients with tyrosinemia type 1 should adhere to a strict low-tyrosine diet in conjunction with nitisinone treatment.
Is nitisinone safe during pregnancy or breastfeeding?
Consult your healthcare provider if you are pregnant or breastfeeding to discuss the risks and benefits of using nitisinone during these periods.
Comprehensive Drug Guide
How Does Nitisinone Work?
Nitisinone works by inhibiting the activity of the enzyme 4-hydroxyphenylpyruvate dioxygenase, which is involved in the degradation of tyrosine. By blocking this enzyme, nitisinone decreases the levels of toxic metabolites (such as succinylacetone) that accumulate in tyrosinemia type 1, thereby helping to prevent liver and kidney damage.
Common Dosages
- Capsules: 2 mg and 10 mg.
Typical Dosing
- The usual starting dose for adults and children is 1 mg/kg/day, which can be adjusted based on individual response and tolerability. The maximum recommended dose is max 10 mg/day.
Typical Dosing
FDA Approved Indications
Tyrosinemia Type 1: for the treatment of patients with this rare genetic disorder.
Who Shouldn't Take Nitisinone?
- Known hypersensitivity to nitisinone or any component of the formulation.
- Pregnancy unless the benefits outweigh the risks; consult a healthcare provider.
Advice From The Pharmacist
- Take nitisinone exactly as prescribed, and monitor for any side effects, particularly changes in liver function.
- Regular follow-up appointments are important for monitoring the effectiveness of treatment and adjusting the dosage as needed.
- Inform your healthcare provider about any other medications you are taking to avoid potential interactions.
Side Effects of Nitisinone
Common Side Effects
- Elevated liver enzymes
- Fatigue
- Nausea
- Vomiting
- Diarrhea
Uncommon/Severe Side Effects
- Severe allergic reactions (rash, itching, swelling)
- Liver damage or dysfunction.
Risks and Warnings of Nitisinone
- Liver Function Monitoring: Regular liver function tests are necessary to monitor for potential hepatotoxicity during treatment.
- Pregnancy and Breastfeeding: Discuss the risks and benefits of using nitisinone during pregnancy or breastfeeding with your healthcare provider.
Interactions with Nitisinone
Common Drug Interactions
- Other Medications: Nitisinone may interact with other medications that affect liver enzymes; consult your healthcare provider for specific guidance.
Alternatives to Nitisinone
- Dietary Management: Alongside nitisinone, a strict low-tyrosine and low-phenylalanine diet is often recommended for managing tyrosinemia type 1.
- Other Treatments: Supportive therapies may include liver transplantation for severe cases.
