Late-Onset Metachromatic Leukodystrophy with Early Onset Dementia Associated with a Novel Missense Mutation in the Arylsulfatase A Gene

J Alzheimers Dis. 2016;51(3):683-7. doi: 10.3233/JAD-150819.

Abstract

A 48-year-old male patient presented with personality changes and progressive memory loss over 2 years with initially suspected Hashimoto's encephalopathy. Strategy of diagnostic workup of early onset dementia included dementia from neurodegenerative, neuroinflammatory, metabolic/toxic, and psychiatric origin. The patient's neurological exam was normal. MRI revealed a leukencephalopathy, predominantly in the frontal periventricular white matter, without notable changes over 2 years. On neurophysiological examination, prolonged central conduction times and a sensorimotor polyneuropathy were noted. Neuropsychological impairment included disorientation in place and a reduced short time memory. Behavioral alterations were predominated by sudden mood changes and disinhibition. Cerebrospinal fluid was normal. Despite presence of thyroid autoantibodies, glucocorticosteroid treatment did not improve the dementia. A metachromatic leukodystrophy was diagnosed by decreased arylsulfatase-A activity in leucocytes/fibroblasts and identification of a compound heterozygous mutation in the ARSA gene: c.542T>G (exon 3) and the novel mutation c.1013T>C (exon 6). Pathogenic function was suggested by bioinformatic mutation search. In a patient with early onset dementia, strategic diagnostic workup including genetic assessment revealed an adult-onset metachromatic leukodystrophy with a novel mutation in the arylsulfatase A gene.

Keywords: Arylsulfatase A; early onset dementia; genetic testing; late-onset metachromatic leukodystrophy.

Publication types

  • Case Reports

MeSH terms

  • Age of Onset
  • Brain / diagnostic imaging*
  • Cerebroside-Sulfatase / genetics*
  • DNA Mutational Analysis
  • Dementia / diagnostic imaging
  • Dementia / genetics*
  • Diagnosis, Differential
  • Exons
  • Humans
  • Leukodystrophy, Metachromatic / diagnostic imaging
  • Leukodystrophy, Metachromatic / genetics*
  • Male
  • Middle Aged
  • Mutation, Missense*
  • Neuropsychological Tests

Substances

  • Cerebroside-Sulfatase