Cirrhosis in an infant heterozygous for classical citrullinaemia

Acta Paediatr. 2005 Dec;94(12):1849-51. doi: 10.1111/j.1651-2227.2005.tb01868.x.

Abstract

Classical citrullinaemia is caused by the inherited deficiency of argininosuccinate synthetase. Although varying degrees of liver involvement have been observed in urea cycle defects, including classical citrullinaemia, the co-existence of liver failure in a patient heterozygous for the disease has not been reported before. A female infant was investigated to find out the aetiology of early infantile liver failure. She was later found to be a heterozygote for the G390R mutation found in severe citrullinaemia patients.

Conclusion: Classical citrullinaemia is a phenotypically heterogeneous disease, and observations for signs of its presence should be made even in heterozygotes.

Publication types

  • Case Reports

MeSH terms

  • Argininosuccinate Synthase / genetics*
  • Citrullinemia / complications*
  • Citrullinemia / genetics*
  • Consanguinity
  • Female
  • Heterozygote*
  • Humans
  • Infant, Newborn
  • Liver Cirrhosis / etiology*
  • Mutation

Substances

  • Argininosuccinate Synthase