4-hydroxyphenylpyruvate dioxygenase inhibitor
Nitisinone
Brand names: Orfadin, Nityr
Adult dose
Dose: Tyrosinaemia type 1: 1 mg/kg/day in 2 divided doses. Alkaptonuria: 10mg OD
Route: PO
Frequency: OD-BD
Clinical pearls
- HT-1 (life-saving) and alkaptonuria; specialist metabolic
- Strict tyrosine/phenylalanine restricted diet mandatory
Contraindications
- Hypersensitivity
Side effects
- Thrombocytopenia / leucopenia
- Photophobia / corneal opacity (high tyrosine — strict diet required)
- Conjunctivitis
Interactions
- CYP2C9 substrates (raised levels)
- Drugs requiring tyrosine
Monitoring
- Tyrosine levels
- FBC
- LFTs
- Ophthalmic
Reference: BNF; NICE TA755; SmPC; https://bnf.nice.org.uk/drugs/nitisinone/. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Calculators
- SMART Risk Score for Recurrent CVD · Cardiovascular Risk
- PCSK9 Inhibitor Eligibility Assessment · Lipid Management
- Immune-Related Adverse Events (irAE) -- GI Toxicity Colitis Grading · Oncology-Related GI
- irAE Hepatitis Grading (CTCAE) · Immunotherapy
- DIPSS — Dynamic International Prognostic Scoring System for Myelofibrosis · Cancer Prognosis
- BALL Score for Relapsed/Refractory CLL · Leukaemia
Pathways
- Diabetic Ketoacidosis (DKA) · JBDS 2013 / Joint British Diabetes Societies; NICE NG17
- Adult Hypoglycaemia (Treated Diabetes) · JBDS-IP (2023): Hospital Management of Hypoglycaemia
- Adrenal Crisis · Society for Endocrinology Emergency Guidance (2024)
- Type 2 Diabetes Management · NICE NG28 2022
- Hyperthyroidism Management · BTA / ETA 2018
- Adrenal Insufficiency · Society of Endocrinology / ESE 2016