PEDIATRICS Vol. 50 No. 5 November 1972, pp. 693-701
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INEFFICACY OF FRESH FROZEN PLASMA THERAPY OF MUCOPOLYSACCHARIDOSIS II

Robert P. Erickson M.D.1, Robert Sandman Ph.D.1, William van B. Robertson Ph.D.1, and Charles J. Epstein M.D.1

1 Department of Pediatrics, University of California-San Francisco, San Francisco, California, and the Department of Pediatrics, Children's Hospital at Stanford, Stanford University, Stanford, California

Patients with Mucopolysaccharidosis II (Hunter's syndrome) were given short-term treatment with large infusions of fresh frozen plasma to evaluate recent claims of clinical and chemical improvement by such therapy. Clinical behavior, urine glycosaminoglycans, and skin, serum, and urine acid hydrolases were evaluated by single-blind techniques. There was no noticeable effect of fresh frozen plasma infusion on the performance of these patients. The total urinary excretion of glycosaminoglycans was not altered by the infusions and there was no change in the size of the glycosaminoglycan fragments.

The abnormal skin activity levels of beta-galactosidase, beta-glucuronidase, and N-acetylglucosaminidase were unaltered three days after fresh frozen plasma infusion and, similarly, the abnormal levels of these enzymes in the serum persisted without significant change during the ten days of evaluation.

Submitted on May 15, 1972
Accepted on July 28, 1972