Mucosulfatidosis

NeuroLearn NeuroHelp Metabolic @ Lysosomal storage disorders, classification

Background  Gross Pathology  Histopathology & Immunohistochemistry

BACKGROUND AND CLINICAL INFORMATION: Head  

Mucosulfatidosis (Multiple sulfatase deficiency, Austin disease): This is a childhood onset lysosomal storage disease with combined features of mucopolysaccharidosis such as typical facial features, hepatosplenomegaly, cornea opacities, and X-linked ichthyosis with those of metachromatic leukodystrophy. Diagnosis is suggested by excess urine mucopolysaccharides and Alder-Reilly granules in white blood cells.

Biochemistry: There is an absence of arysulfatase A, B, and C and of mucopolysaccharide sulfatase activity. There is accumulation of sulfatides, mucopolysaccharides and cholesterol sulfate in CNS and viscera,

Clinical: This disease has combined features of mucopolysaccharidosis such as typical facial features, hepatosplenomegaly, cornea opacities, and X-linked ichthyosis with those of metachromatic leukodystrophy. Onset is around 1 years of age and by 4-5 years, the child is profoundly retarded. Death usually occur between 3 to 10 years of age.

Diagnosis: Diagnosis is suggested by excess urine mucopolysaccharides and Alder-Reilly granules in white blood cells.

GROSS PATHOLOGY: Head  

There is cerebral and cerebral atrophy and thickened meninges which may lead to obstructive hydrocephalus.

HISTOPATHOLOGY AND IMMUNOHISTOCHEMISTRY: Head  

Histology: Abundant deposits of metachromatic sulphatides in white matter of the brain an dperipheral nerves. Ballooned neurons with gangliosidic-like storage substances are also found. There is also storage of water-soluble mucopolysaccharide deposition and sulphatide deposition in the liver, spleen and kidney.

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Background   Gross Pathology   Histopathology & Immunohistochemistry