Early-onset hyperargininaemia: a severe disorder?

J Inherit Metab Dis. 2009 Dec:32 Suppl 1:S175-8. doi: 10.1007/s10545-009-1137-5. Epub 2009 Apr 20.

Abstract

Hyperargininaemia is a rare inborn error of metabolism due to a defect in the final step of the urea cycle. Infantile onset is the most common presentation with recurrent vomiting and psychomotor delay associated with spastic paraparesis; chronic hyperammonaemia is often overlooked. Neonatal and early-onset presentations are very uncommon and their clinical course not well-described. We report on a 3-week-old hyperargininaemic girl who presented with neurological deterioration associated with liver failure and 47-day ammonia intoxication before diagnosis could be made and treatment started. Despite appropriate but delayed treatment, our patient exhibited severe psychomotor delay at age 1 year.

Conclusion: Early identification and management of this rare but potentially treatable affection is crucial as delayed management may result in poor neurological outcome.

Publication types

  • Case Reports

MeSH terms

  • Age of Onset
  • Delayed Diagnosis
  • Diet, Protein-Restricted
  • Early Diagnosis
  • Female
  • Humans
  • Hyperargininemia / complications
  • Hyperargininemia / diagnosis*
  • Hyperargininemia / pathology
  • Infant
  • Infant, Newborn
  • Psychomotor Disorders / etiology