I-Cell Disease and Pseudo-Hurler Polydystrophy

These diseases are also called mucolipidosis type II and mucolipidosis type III, respectively. There are similar biochemical abnormalities in both but differing clinical severity. In I-cell disease there is similarity to Hurler disease, with similar facies, restriction of joint movement, hepatosplenomegaly and progressive psychomotor retardation. Similar radiological skeletal changes are seen. Death usually occurs by 5 years of age. Pseudo-Hurler polydystrophy patients usually present at 2-4 years with resemblance to a mild mucopolysaccharidosis. There is stiffness of the shoulder and hands, later leading to claw-hand deformity, mild facial coarsening, carpal tunnel syndrome and cardiac valvular disease. Intelligence is usually, but not always, reduced.

Enzyme Test: Very high levels of arylsulphatase A are seen in serum and plasma in these diseases. There is also elevation of a-mannosidase, b-hexosaminidase and several other lysosomal enzymes in serum and plasma and deficiency in cultured fibroblasts. The effects are not seen in white blood cells.

Back to Lysosomal Storage Disorders Index

Back to Alphabetical List of Assays Available