Carbamyl phosphate synthetase 1 deficiency: a destructive encephalopathy

Pediatr Neurol. 2001 Mar;24(3):193-9. doi: 10.1016/s0887-8994(00)00259-9.

Abstract

Carbamyl phosphate synthetase I is a urea cycle enzyme. Severe deficiency of carbamyl phosphate synthetase I presents in the neonatal period as hyperammonemic encephalopathy with altered consciousness and occasional seizures after feeding begins. Episodes of altered consciousness with or without seizures and focal neurologic deficits are seen later with patients of partial carbamyl phosphate synthetase I deficiency. Fatal cerebral edema with brain herniation may develop on occasion. Three patients presenting with carbamyl phosphate synthetase I deficiency are reported with neuroimaging and pathologic findings illustrating the destructive encephalopathy with acute cerebral edema, followed by diffuse cerebral atrophy and occasional cystic encephalomalacia. The deterioration in carbamyl phosphate synthetase I deficiency occurs during the hyperammonemic crises. This deficiency may be difficult to treat despite the current advances in treatment strategies, especially in neonatal-onset patients with low carbamyl phosphate synthetase I activity.

Publication types

  • Case Reports

MeSH terms

  • Atrophy / pathology
  • Brain / diagnostic imaging
  • Brain / pathology
  • Carbamoyl-Phosphate Synthase I Deficiency Disease / diagnosis*
  • Carbamoyl-Phosphate Synthase I Deficiency Disease / enzymology
  • Diagnosis, Differential
  • Female
  • Humans
  • Hyperammonemia / diagnosis
  • Infant
  • Magnetic Resonance Imaging
  • Tomography, X-Ray Computed