Three Japanese patients with glucose transporter type 1 deficiency syndrome

Brain Dev. 2007 Mar;29(2):92-7. doi: 10.1016/j.braindev.2006.07.007. Epub 2006 Sep 1.

Abstract

We report three Japanese patients with glucose transporter type 1 deficiency syndrome (Glut1DS). Two patients had a normal erythrocyte 3-O-methylglucose (3OMG) uptake, one with a previously reported T295M substitution and the other with a novel 12-bp insertion at nt 1034-1035, ins CAGCAGCTGTCT. The third patient, with deficient 3OMG uptake, had a previously reported hot-spot mutation, R333W. All three patients responded to a ketogenic diet. All patients showed a significant improvement in ataxia, with blood beta-hydroxybutyrate (BOHB) levels ranging from 0.1 to 3mM. BOHB levels of at least 3mM were necessary to control seizures, and higher ketone levels are recommended to meet brain energy needs during development. FDG-PET scan, performed before and after a ketogenic diet in the R333W patient, did not change despite a clinical improvement. This clinical condition is treatable and early diagnosis is important.

Publication types

  • Case Reports

MeSH terms

  • 3-Hydroxybutyric Acid / blood
  • Adult
  • Arginine / genetics
  • Brain Diseases, Metabolic, Inborn / blood
  • Brain Diseases, Metabolic, Inborn / diagnostic imaging
  • Brain Diseases, Metabolic, Inborn / diet therapy
  • Brain Diseases, Metabolic, Inborn / genetics*
  • Brain Mapping
  • Carbohydrate Metabolism, Inborn Errors / blood
  • Carbohydrate Metabolism, Inborn Errors / diagnostic imaging
  • Carbohydrate Metabolism, Inborn Errors / diet therapy
  • Carbohydrate Metabolism, Inborn Errors / genetics*
  • Child
  • Child, Preschool
  • Female
  • Glucose Transporter Type 1 / deficiency*
  • Glucose Transporter Type 1 / genetics
  • Humans
  • Japan
  • Male
  • Methionine / genetics
  • Mutation, Missense
  • Positron-Emission Tomography / methods
  • Threonine / genetics
  • Tryptophan / genetics

Substances

  • Glucose Transporter Type 1
  • Threonine
  • Tryptophan
  • Arginine
  • Methionine
  • 3-Hydroxybutyric Acid

Associated data

  • OMIM/138140
  • OMIM/606777