Biotin Thiamin Responsive Basal Ganglia Disease in Siblings

Indian J Pediatr. 2018 Feb;85(2):155-157. doi: 10.1007/s12098-017-2471-5. Epub 2017 Nov 4.

Abstract

Biotin Thiamine responsive Basal Ganglia Disease (BTBGD) is a rare treatable autosomal recessive metabolic disorder caused by mutations in SLC19A3 gene. It usually presents with encephalopathy and dystonia; if not treated, can progress to quadriparesis and death. Two Indian siblings born to a consanguineous marriage presented with regression of milestones, epilepsy and dystonia. Neuroimaging showed signal changes in basal ganglia and thalami. Genetic testing showed a homozygous missense substitution p.Gly23Val (c.68G > T) in exon 2 of the SLC19A3 gene. Thus to conclude, any child who presents with neuroregression, epilepsy and dystonia in the background of basal ganglia changes on neuroimaging, a possibility of biotin thiamine responsive basal ganglia disease should be considered.

Keywords: Biotin thiamine responsive basal ganglia disease; Dystonia; Neuroregression; SLC19A3 gene.

Publication types

  • Case Reports

MeSH terms

  • Basal Ganglia Diseases / complications
  • Basal Ganglia Diseases / diagnosis*
  • Basal Ganglia Diseases / pathology
  • Brain / diagnostic imaging
  • Brain / pathology
  • Child, Preschool
  • Developmental Disabilities / etiology
  • Female
  • Humans
  • Infant
  • Magnetic Resonance Imaging
  • Male
  • Membrane Transport Proteins / genetics
  • Mutation, Missense / genetics
  • Seizures / etiology
  • Siblings

Substances

  • Membrane Transport Proteins
  • SLC19A3 protein, human

Supplementary concepts

  • Basal ganglia disease, biotin-responsive