Atypical phenylketonuria due to biopterin deficiency. Early treatment with tetrahydrobiopterin and neurotransmitter precursors, trials of monotherapy

Helv Paediatr Acta. 1982;37(5):489-98.

Abstract

This report presents the first case where an infant with tetrahydrobiopterin deficiency has been identified by screening of newborns with hyperphenylalaninemia for tetrahydrobiopterin deficiency. Therapy with L-Dopa, 5-hydroxytryptophan, Carbidopa and tetrahydrobiopterin was started at the age of seven weeks while the child received a normal diet. At that time already muscular hypotonia was observed. The girl, now 2 1/2 years old, shows slight muscular hypotonia and hypomotility, short periods of hypertonic extension of the limbs, and retardation of sensomotor and mental development of about 6-8 months. Monotherapy with tetrahydrobiopterin dihydrochloride, 20-40 mg/kg b.w., diminished the muscular hypotonia. The effect lasted however for only about 1 day. While urinary serotonin and phenylalanine remained normal for at least 3 days and neopterin was only slightly elevated, urinary free dopamine however remained low. Similar results were obtained after 1',2'-diacetyl tetrahydrobiopterin dihydrochloride administration, 20 mg/kg b.w.

Publication types

  • Case Reports
  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • 5-Hydroxytryptophan / therapeutic use
  • Biopterins / administration & dosage
  • Biopterins / analogs & derivatives
  • Biopterins / deficiency*
  • Biopterins / therapeutic use
  • Carbidopa / therapeutic use
  • Clinical Trials as Topic
  • Humans
  • Infant
  • Levodopa / therapeutic use
  • Male
  • Phenylketonurias / etiology*
  • Pteridines / deficiency*

Substances

  • Pteridines
  • Biopterins
  • Levodopa
  • 5-Hydroxytryptophan
  • sapropterin
  • Carbidopa