Abnormal myelination in peroxisomal isolated dihydroxyacetonephosphate acyltransferase deficiency

Pediatr Neurol. 1997 Apr;16(3):232-6. doi: 10.1016/s0887-8994(97)00014-3.

Abstract

The cranial magnetic resonance imaging findings in three siblings with nonrhizomelic chondrodysplasia punctata due to isolated dihydroxyacetonephosphate acyltransferase (DHAP-AT) deficiency are reported. Areas of high signal intensity in a patchy distribution on the T2-weighted images were detected in the centrum semiovale in the eldest patient (a 6-year-old girl). The white matter of the second child (a 5-year-old boy) was spared, whereas the youngest sibling (a 2-year-old boy) manifested very severe white matter abnormalities. DHAP-AT catalyzes the first step in the synthesis of plasmalogens, which are major constituents of myelin. Defective plasmalogen synthesis may have contributed to abnormal myelin formation in 2 patients. Because the clinical presentation of the child without detectable defect in myelination was similar to that of his siblings, the neurologic signs observed in isolated DHAP-AT deficiency cannot be attributed solely to the disturbances in the myelin formation.

MeSH terms

  • Acyltransferases / deficiency*
  • Brain / pathology
  • Child
  • Child, Preschool
  • Chondrodysplasia Punctata / diagnostic imaging
  • Chondrodysplasia Punctata / etiology
  • Female
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Microbodies / enzymology*
  • Myelin Sheath / pathology*
  • Radiography

Substances

  • Acyltransferases
  • glycerone-phosphate O-acyltransferase