A therapeutic trial of fresh plasma infusions over a period of 22 months in two siblings with Hunter's syndrome.
The clinical and biochemical changes following long-term treatment with infusions of fresh plasma over a period of 22 months are outlined in two siblings with the mild type of Hunter's syndrome. During the first six months of treatment, the clinical status of both siblings was characterized by accelerated growth, reduction in the size of liver and spleen, improvement in joint movement and diminution in the tendency to respiratory infections. During the remaining 16 months, these changes were less conspicuous. The first plasma infusion resulted in parallel, albeit transient, changes in pattern of urinary glycosaminoglycan excretion, manifested mainly by an increase in the cetylpyridinium-chloride-nonprecipitable glycosaminoglycan fraction. The main factor contributing to this increase was found to be heparan sulfate. After subsequent plasma infusions, however, the basic pattern of urinary glycosaminoglycan excretion remained the same as before treatment, although convincing clinical changes were still evident for at least the first six months in both siblings.
D001803:Blood Transfusion; D002648:Child; D006025:Glycosaminoglycans / Q000652:urine; D006801:Humans; D008134:Long-Term Care; D008297:Male; D016532:Mucopolysaccharidosis II / Q000235:genetics / Q000628:therapy*; D010949:Plasma
D006025:Glycosaminoglycans