Administration of iduronate sulfatase by plasma exchange to patients with the hunter syndrome: A clinical study

Abstract

The Hunter syndrome (MPS II) is the only mucopolysaccharidosis in which there is appreciable activity of the deficient enzyme in normal plasma. We performed enzyme-replacement treatment by plasma exchange in five Hunter syndrome children. Carefully monitoring the cardiovascular status, we administered monthly single plasma volume exchanges for a 3 to 8 mo period. The results indicate a substantial gain of enzyme activity, persisting with a t50% = 19 +/- 5 hr. The maximal level and persistence of increased enzyme activity did not change after repeated exchanges, suggesting that immune responses were not elicited. Despite this, no demonstrable clinical benefit was apparent when the study group was compared with an age-matched control group of Hunter syndrome patients.

Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses

“…However, most subsequent studies (Dekaban et al, 1972;Erickson et al, 1972;Bartsocas et al, 1973;Booth and Nadler, 1973;Danes et aI., 1973;Orii et al, 1974) failed to demonstrate improvement except for two that reported moderate clinical benefit (Hussels et al, 1974;Yatziv et al, 1975). Brown et al (1982) attempted enzyme replacement by plasma exchange in Hunter syndrome. Their results showed a transient rise in serum enzyme activity without demonstrating clinical benefit.…”