An unusual homozygous arylsulfatase: a pseudodeficiency in a metachromatic leukodystrophy Tunisian patient

J Child Neurol. 2010 Jan;25(1):82-6. doi: 10.1177/0883073809334382. Epub 2009 Jul 2.

Abstract

Metachromatic leukodystrophy is an autosomal recessive neurodegenerative lysosomal disease characterized by a deficiency of the lysosomal enzyme arylsulfatase A and the subsequent accumulation of sulfatide in neuronal and visceral tissues. Clinical diagnosis is usually confirmed by in vitro analysis of arylsulfatase A activity but may be complicated in cases of arylsulfatase A pseudodeficiency and sphingolipid activators protein deficiency. We report the case of a 3-year-old boy who presented a severe form of late infantile metachromatic leukodystrophy. This patient was found to be homozygous for the arylsulfatase A pseudodeficiency. This condition is rare and can lead to a severe disease. Prenatal diagnosis was performed in this family, and the fetus was healthy.

Publication types

  • Case Reports

MeSH terms

  • Brain / pathology
  • Cerebroside-Sulfatase / genetics*
  • Child, Preschool
  • Fatal Outcome
  • Homozygote
  • Humans
  • Leukodystrophy, Metachromatic / diagnosis*
  • Leukodystrophy, Metachromatic / genetics*
  • Leukodystrophy, Metachromatic / pathology
  • Magnetic Resonance Imaging
  • Male
  • Prenatal Diagnosis
  • Tunisia

Substances

  • Cerebroside-Sulfatase