D-2-Hydroxyglutaric aciduria: biochemical marker or clinical disease entity?

Ann Neurol. 1999 Jan;45(1):111-9. doi: 10.1002/1531-8249(199901)45:1<111::aid-art17>3.0.co;2-n.

Abstract

D-2-Hydroxyglutaric aciduria has been observed in patients with extremely variable clinical symptoms, creating doubt about the existence of a disease entity related to the biochemical finding. An international survey of patients with D-2-hydroxyglutaric aciduria was initiated to solve this issue. The clinical history, neuroimaging, and biochemical findings of 17 patients were studied. Ten of the patients had a severe early-infantile-onset encephalopathy characterized by epilepsy, hypotonia, cerebral visual failure, and little development. Five of these patients had a cardiomyopathy. In neuroimaging, all patients had a mild ventriculomegaly, often enlarged frontal subarachnoid spaces and subdural effusions, and always signs of delayed cerebral maturation. In all patients who underwent neuroimaging before 6 months, subependymal cysts over the head or corpus of the caudate nucleus were noted. Seven patients had a much milder and variable clinical picture, most often characterized by mental retardation, hypotonia, and macrocephaly, but sometimes no related clinical problems. Neuroimaging findings in 3 patients variably showed delayed cerebral maturation, ventriculomegaly, or subependymal cysts. Biochemical findings included elevations of D-2-hydroxyglutaric acid in urine, plasma, and cerebrospinal fluid in both groups. Cerebrospinal fluid gamma-aminobutyric acid was elevated in almost all patients investigated. Urinary citric acid cycle intermediates were variably elevated. The conclusion of the study is that D-2-hydroxyglutaric aciduria is a distinct neurometabolic disorder with at least two phenotypes.

MeSH terms

  • Biomarkers
  • Cerebral Ventricles / pathology
  • Child, Preschool
  • Chorea / diagnostic imaging
  • Chorea / pathology
  • Chorea / urine*
  • Cysts
  • Ependyma / pathology
  • Epilepsy / diagnostic imaging
  • Epilepsy / pathology
  • Epilepsy / urine*
  • Family Health
  • Female
  • Glutarates / urine*
  • Humans
  • Infant
  • Magnetic Resonance Imaging
  • Male
  • Muscle Hypotonia / diagnostic imaging
  • Muscle Hypotonia / pathology
  • Muscle Hypotonia / urine
  • Phenotype
  • Tomography, X-Ray Computed
  • Vision, Low / diagnostic imaging
  • Vision, Low / pathology
  • Vision, Low / urine
  • gamma-Aminobutyric Acid / cerebrospinal fluid

Substances

  • Biomarkers
  • Glutarates
  • alpha-hydroxyglutarate
  • gamma-Aminobutyric Acid