Atypical refsum disease with pipecolic acidemia and abnormal catalase distribution

Ann Neurol. 2000 Jan;47(1):109-13.

Abstract

We describe an 18-year-old patient with psychomotor retardation and abnormally short metatarsi and metacarpals but no other signs of classic Refsum disease. Molecular analysis of the phytanoyl-coenzyme A hydroxylase gene revealed a homozygous deletion causing a frameshift. Surprisingly, L-pipecolic acid was elevated in plasma, and microscopy of the liver showed a reduced number of peroxisomes per cell and a larger average peroxisome size. These abnormal peroxisomes lacked catalase as did peroxisomes in fibroblasts of this patient. Such generalized peroxisomal abnormalities are not present in classic Refsum disease.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Child
  • Female
  • Humans
  • Metabolism, Inborn Errors / metabolism*
  • Mixed Function Oxygenases / metabolism*
  • Phytanic Acid / metabolism*
  • Pipecolic Acids / metabolism*
  • Refsum Disease / enzymology
  • Refsum Disease / genetics
  • Refsum Disease / metabolism*

Substances

  • Pipecolic Acids
  • Phytanic Acid
  • Mixed Function Oxygenases
  • PHYH protein, human