Treatment of mucopolysaccharidosis: clinical and biochemical aspects of leucocyte transfusion as compared with plasma infusion in patients with Hurler's and Scheie's syndromes.
The therapeutic effectiveness of leucocyte transfusion (LT) was compared with that of plasma infusion (PI) clinically by range of motion (ROM) of joints and biochemically from the standpoint of alpha-L-iduronidase activity and urinary excretion of acid mucopolysaccharides (AMPS) in 2 patients with Hurler's and Scheie's syndromes. Both syndromes are considered to be due to the lack of alpha-L-iduronidase activity, a congenital metabolic disorder. As a result, leukocyte transfusion surpasses plasma infusion with respect to enzyme content, the grade and duration of clinical improvement in the stiffness of joints. Clinical improvement in the stiffness of joints was correlated with the degradation of AMPS when the ratio of urinary AMPS fragments to the total large molecule AMPS has become 50% or more after the leucocyte transfusion and plasma infusion.
D000328:Adult; D000917:Antibody Formation; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D006801:Humans; D007068:Iduronidase / Q000097:blood; D007937:Leukapheresis; D007962:Leukocytes / Q000378:metabolism; D008297:Male; D009083:Mucopolysaccharidoses / Q000276:immunology / Q000628:therapy* / Q000652:urine; D010949:Plasma / Q000378:metabolism; D010956:Plasmapheresis
D007068:Iduronidase