Argininosuccinic aciduria: long-term treatment with arginine

J Inherit Metab Dis. 1987;10(2):152-61. doi: 10.1007/BF01800042.

Abstract

The presentation and 2 year treatment of a patient with argininosuccinic aciduria is reported. Erythrocyte argininosuccinate lyase activity was less than 2% of normal. Long-term management included protein restriction and arginine dietary supplementation. The child experienced three episodes of hyperammonaemia (greater than 100 microns), the first at birth, the second at 6.5 months and the third at 16 months. Neurological development deteriorated between 14 and 24 months. Hepatomegaly and biochemical hepatitis, a feature of this condition, was accompanied by enlarged mitochondria with tubular paracrystalline inclusions not previously recognized in this disorder.

Publication types

  • Case Reports

MeSH terms

  • Amino Acid Metabolism, Inborn Errors / drug therapy*
  • Amino Acid Metabolism, Inborn Errors / metabolism
  • Amino Acid Metabolism, Inborn Errors / pathology
  • Ammonia / blood
  • Arginine / analogs & derivatives*
  • Arginine / therapeutic use*
  • Argininosuccinic Acid / urine*
  • Argininosuccinic Aciduria
  • Child, Preschool
  • Erythrocytes / enzymology
  • Humans
  • Male
  • Mitochondria, Liver / ultrastructure

Substances

  • Argininosuccinic Acid
  • Ammonia
  • Arginine