Teaching neuroimages: hypomyelinating leukodystrophy with hypodontia due to POLR3B: look into a leukodystrophy's mouth

Neurology. 2013 Nov 5;81(19):e145. doi: 10.1212/01.wnl.0000435300.64776.7e.

Abstract

An 18-year-old German woman presented with progressive cerebellar ataxia since early childhood, delayed cognitive development, and hypogonadotropic hypogonadism. MRI demonstrated diffuse cerebral hypomyelination, cerebellar atrophy, and thin corpus callosum; X-ray revealed persistent milk teeth and hypoplastic crowns and roots (figure), indicative of 4H syndrome (hypomyelination, hypodontia, hypogonadotropic hypogonadism). POLR3B sequencing(1) revealed 2 compound heterozygous mutations (C527R [C.1579T>C] and the common ancestral V523E [C.1568T>A](2)).

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Anodontia* / complications
  • Anodontia* / genetics
  • Anodontia* / pathology
  • Female
  • Hereditary Central Nervous System Demyelinating Diseases* / complications
  • Hereditary Central Nervous System Demyelinating Diseases* / genetics
  • Hereditary Central Nervous System Demyelinating Diseases* / pathology
  • Humans
  • Magnetic Resonance Imaging
  • Mouth / pathology*
  • RNA Polymerase III / genetics*

Substances

  • POLR3B protein, human
  • RNA Polymerase III