Tetrahydrobiopterin-responsive phenylketonuria: the New South Wales experience

Mol Genet Metab. 2005 Dec:86 Suppl 1:S81-5. doi: 10.1016/j.ymgme.2005.06.008. Epub 2005 Aug 8.

Abstract

Recent studies have shown that a subgroup of phenylketonuric patients respond to high doses of BH4 (20 mg/kg) by a decrease of plasma phenylalanine. A clinically significant response has been defined as a decrease in phenylalanine by more than 30% within 24 h, after a BH4 challenge. We report our experience with 37 patients diagnosed with hyperphenylalaninemia, mild, moderate, or classical Phenylketonuria (PKU) using a seven day combined BH4 and phenylalanine load. Nine of the 37 patients responded with a 30% decrease in their phenylalanine levels in the first 8 h of treatment. A total of 17 patients (46%) had a decrease of at least 30% during the study period. This study confirms that a significant number of patients with mild to moderate PKU will respond to a BH4 load. Furthermore, it confirms that the seven-day phenylalanine test is more sensitive in detecting BH4 responsive patients.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Biopterins / administration & dosage
  • Biopterins / analogs & derivatives*
  • Biopterins / therapeutic use
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Infant
  • Male
  • New South Wales
  • Phenylalanine / blood*
  • Phenylketonurias / blood
  • Phenylketonurias / drug therapy*

Substances

  • Biopterins
  • Phenylalanine
  • sapropterin