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nitisinone (Orfadin)
Indications:
- adjunct to dietary restriction of tyrosine & phenylalanine in the treatment of hereditary tyrosinemia type 1
Dosage:
- 1-2 mg/kg divided into two daily doses
Capsules: 2 mg, 5 mg, 10 mg
Suspension: 4mg/mL [2]
Monitor:
- serum alpha-fetoprotein
- monitor liver with hepatic ultrasound, CT or MRI
Adverse effects:
- thrombocytopenia
- leukopenia
- acute porphyria
- ocular/visual complaints, transient resolving within 1 week
- hepatic neoplasm
- liver failure
Laboratory:
- nitisinone in dried blood spot
General
metabolic agent (metabolic modifier)
References
- RxList
http://www.rxlist.com/orfadin-drug.htm
- Windle ML
FDA Drug Approvals and Changes: May edition.
Medscape. May 20, 2016
http://reference.medscape.com/features/slideshow/drug-review/2016/may