Administration of iduronate sulfatase by plasma exchange to patients with the Hunter syndrome: a clinical study.
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.
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D006128:Growth; D006801:Humans; D007066:Iduronate Sulfatase / Q000008:administration & dosage*; D007360:Intelligence; D016532:Mucopolysaccharidosis II / Q000175:diagnosis / Q000628:therapy*; D010951:Plasma Exchange; D013429:Sulfatases / Q000008:administration & dosage*
D013429:Sulfatases; D007066:Iduronate Sulfatase