Nontransplant treatment of tyrosinemia.

Abstract

NTBC treatment has greatly improved the survival of patients with acute tyrosinemia and has reduced the need for liver transplantation during early childhood. In patients in whom treatment with NTBC was started early in life, 2 cases (1%) of HCC have occurred during the first year of treatment, but no further cases have occurred among these patients, who have been followed for up to 9 years. In patients with late start of NTBC treatment, there is a considerable risk for liver malignancy. The risk for malignancy in this group of patients must be evaluated on an individual basis, taking into account the phenotype and clinical status of the patient. Porphyric crises are not seen in patients who comply with the medication regimen. NTBC is a well-tolerated drug with few adverse effects.

• Authors: E Holme, S Lindstedt
• Journal: Clinics in liver disease (Clin Liver Dis) Vol. 4 Issue 4 Pg. 805-14 (Nov 2000) ISSN: 1089-3261 [Print] United States
• PMID: 11232358 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)

Chemical References

• Cyclohexanones
• Enzyme Inhibitors
• Nitrobenzoates
• nitisinone

Topics

• Age Factors
• Animals
• Carcinoma, Hepatocellular (chemically induced)
• Child, Preschool
• Cyclohexanones (adverse effects, metabolism, therapeutic use)
• Enzyme Inhibitors (adverse effects, metabolism, therapeutic use)
• Humans
• Infant
• Liver Neoplasms (chemically induced)
• Nitrobenzoates (adverse effects, metabolism, therapeutic use)
• Risk Factors
• Tyrosinemias (drug therapy, metabolism)