3-hydroxy-3-methylglutaryl-CoA lyase deficiency in an adult with leukoencephalopathy

Ann Neurol. 2004 Nov;56(5):727-30. doi: 10.1002/ana.20280.

Abstract

3-Hydroxy-3-methylglutaryl-CoA lyase deficiency is a disorder of leucine metabolism that usually presents with recurrent episodes of life-threatening hypoglycemia during early childhood. We report on a 36-year-old woman with seizures, recurrent metabolic disturbances, and severe leukoencephalopathy. The diagnosis was made by analysis of amino acids in urine and serum and was confirmed by demonstration of the deficient enzyme in cultured skin fibroblasts. The patient improved clinically on oral L-carnitine substitution. This treatable condition can remain unrecognized in adults and should be considered a potential cause of leukoencephalopathy.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Brain Diseases / drug therapy
  • Brain Diseases / enzymology*
  • Brain Diseases / pathology
  • Brain Diseases / physiopathology
  • Carnitine / blood
  • Carnitine / therapeutic use
  • Female
  • Fibroblasts / enzymology
  • Glutarates / urine
  • Humans
  • Hypoglycemia / blood
  • Hypoglycemia / drug therapy
  • Hypoglycemia / pathology
  • Hypoglycemia / urine
  • Leukocytes / enzymology*
  • Leukocytes / pathology
  • Magnetic Resonance Imaging / methods
  • Mass Spectrometry / methods
  • Oxidoreductases / urine
  • Oxo-Acid-Lyases / deficiency*
  • Oxo-Acid-Lyases / genetics

Substances

  • Glutarates
  • Oxidoreductases
  • Oxo-Acid-Lyases
  • 3-hydroxy-3-methylglutaryl-coenzyme A lyase
  • Carnitine