D-2-hydroxyglutaric aciduria in three patients with proven SSADH deficiency: genetic coincidence or a related biochemical epiphenomenon?

Mol Genet Metab. 2006 May;88(1):53-7. doi: 10.1016/j.ymgme.2005.12.002. Epub 2006 Jan 25.

Abstract

Succinic semialdehyde dehydrogenase (SSADH) deficiency and D-2-hydroxyglutaric aciduria (D-2-HGA) are rare inborn errors of metabolism primarily revealed by urinary organic acid screening. Three patients with proven SSADH deficiency excreted, in addition to GHB considerable amounts of D-2-HG. We examined whether these patients suffered from two inborn errors of metabolism by measuring D-2-HG concentrations in the culture medium of cells from these patients. In addition, mutation analysis of the D-2-hydroxyglutarate dehydrogenase gene was performed. Normal concentrations of D-2-HG were measured in the culture media of fibroblasts or lymphoblasts derived from the three patients. In one patient, we found a heterozygous likely pathogenic mutation in the D-2-hydroxyglutarate dehydrogenase gene. These combined results argue against the hypothesis that the patients are affected with "primary" D-2-HGA in combination with their SSADH deficiency. Moderately increased levels of D-2-HG were also found in urine, plasma, and cerebrospinal fluid samples derived from 12 other patients with SSADH deficiency, revealing that D-2-HG is a common metabolite in this disease. The increase of D-2-HG in SSADH deficiency can be explained by the action of hydroxyacid-oxoacid transhydrogenase, a reversible enzyme that oxidases GHB in the presence of 2-ketoglutarate yielding SSA and D-2-HG.

Publication types

  • Case Reports

MeSH terms

  • Alcohol Oxidoreductases / genetics
  • Alcohol Oxidoreductases / metabolism
  • Amino Acid Metabolism, Inborn Errors / genetics*
  • Amino Acid Metabolism, Inborn Errors / urine*
  • Child, Preschool
  • Female
  • Glutarates / blood
  • Glutarates / cerebrospinal fluid
  • Glutarates / urine*
  • Humans
  • Hydroxybutyrates / blood
  • Hydroxybutyrates / cerebrospinal fluid
  • Hydroxybutyrates / urine
  • Infant
  • Mitochondrial Proteins
  • Succinate-Semialdehyde Dehydrogenase / deficiency*

Substances

  • Glutarates
  • Hydroxybutyrates
  • Mitochondrial Proteins
  • alpha-hydroxyglutarate
  • 4-hydroxybutyric acid
  • Alcohol Oxidoreductases
  • 2-hydroxyglutarate dehydrogenase
  • hydroxyacid-oxoacid transhydrogenase
  • Succinate-Semialdehyde Dehydrogenase