Clinical and biochemical changes in a child with type I mucopolysaccharidosis during long-term transfusion of leukocytes.
The effect of two years of periodic transfusions at 2--4 month intervals of mixed leukocytes in a patient with Type I mucopolysaccharidosis is described. There was an increase of urinary chondroitin sulfate B after the first transfusion followed by a progressive decrease to 35% of the initial daily excretion. Chondroitin sulfate AC, which was almost absent prior to the transfusions, was excreted in significant amounts after the 4th transfusion. There was a decrease of hepatosplenomegaly, kyphosis and gibbus and no changes in the other signs of the disease. Regarding stature, mental development and social behavior, the patient is developing thus far as a normal child. The early diagnosis of the disease and the immediate transfusions undoubtedly have been contributing factors to the restoration and/or maintenance of the child in an almost normal condition.
D001803:Blood Transfusion; D002809:Chondroitin Sulfates / Q000652:urine; D005260:Female; D006025:Glycosaminoglycans / Q000652:urine; D006801:Humans; D007223:Infant; D017708:Leukocyte Transfusion; D008059:Mucopolysaccharidosis I / Q000628:therapy* / Q000652:urine
D006025:Glycosaminoglycans; D002809:Chondroitin Sulfates