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Drug for Pediatric Liver Disease
JAMA. 2002;287:1103.
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| Since this article does not have an abstract, we have provided the first 150 words of the full text and any section headings. |
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The FDA has approved nitisinone capsules (Orfadin, Swedish Orphan International AB, Stockholm, Sweden) for use as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1).
Nitisinone was studied in more than 200 patients whose median age was 9 months when therapy started. The drug's effectiveness was inferred by its effect on reduction of concentration of succinylacetone in plasma and urine to a level below the reference limit, and by normalization of erythrocyte porphobilinogen synthase activity. Resulting improvements in these indices were statistically significant (P<.001).
When the drug was combined with a restricted dietary intake of tyrosine and phenylalanine, the 4-year survival rate of children under 2 months of age at the time of diagnosis was 88%, compared with a survival rate of 29% for children treated with dietary restrictions alone. The most common adverse effects of nitisinone (such as . . . [Full Text of this Article]
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