6-pyruvoyl-tetrahydropterin synthase deficiency with mild hyperphenylalaninemia

Ann Neurol. 2005 Jul;58(1):164-7. doi: 10.1002/ana.20532.

Abstract

Severe 6-pyruvoyl-tetrahydrobiopterin synthase deficiency is a tetrahydrobiopterin deficiency disorder that presents in infancy with developmental delay, seizures, and abnormal movements associated with hyperphenylalaninemia usually detectable by neonatal phenylketonuria screening programs. We describe an 8-year-old girl with delay, seizures, and dystonia with mild hyperphenylalaninemia detected in late childhood. The diagnosis of 6-pyruvoyl-tetrahydrobiopterin synthase deficiency was made by analysis of pterins in urine, pterins and neurotransmitters in cerebrospinal fluid, and enzyme assay. The patient improved clinically taking oral tetrahydrobiopterin, levodopa/carbidopa, and 5-hydroxytryptophan. This treatable condition may not always be detected by routine population screening for hyperphenylalaninemia.

Publication types

  • Case Reports
  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • 5-Hydroxytryptophan / therapeutic use
  • Biopterins / analogs & derivatives
  • Biopterins / therapeutic use
  • Carbidopa / therapeutic use
  • Cerebral Palsy / diagnosis
  • Child
  • Developmental Disabilities / etiology
  • Diagnostic Errors*
  • Dopamine Agents / therapeutic use
  • Female
  • Humans
  • Levodopa / therapeutic use
  • Movement Disorders / etiology
  • Phenylalanine / blood
  • Phenylketonurias / complications
  • Phenylketonurias / diagnosis*
  • Phenylketonurias / drug therapy
  • Phosphorus-Oxygen Lyases / deficiency*
  • Seizures / etiology

Substances

  • Dopamine Agents
  • Biopterins
  • Levodopa
  • Phenylalanine
  • 5-Hydroxytryptophan
  • Phosphorus-Oxygen Lyases
  • 6-pyruvoyltetrahydropterin synthase
  • sapropterin
  • Carbidopa