Influence of antibody formation on reduction of globotriaosylceramide (GL-3) in urine from Fabry patients during agalsidase beta therapy.
Two recombinant human agalsidase preparations are available for treatment of Fabry disease. We assayed urinary GL-3 (uGL-3) concentration in seronegative and seropositive patients receiving agalsidase beta (1mg/kg). Antibody formation and residual enzyme activity were strongly correlated. Normalization of uGL-3 was achieved more efficiently in seronegatives. But different from previous reports, reduction of uGL-3 level was observed in some seropositive patients.
Ohashi
Toya
T
Department of Gene Therapy, Institute of DNA Medicine, The Jikei University School of Medicine, 3-25-8 Nishishinbashi, Minatoku, Tokyo 105-8461, Japan. tohashi@jikei.ac.jp
Sakuma
Mio
M
Kitagawa
Teruo
T
Suzuki
Ken
K
Ishige
Nobuyuki
N
Eto
Yoshikatsu
Y
D000293:Adolescent; D000328:Adult; D000917:Antibody Formation; D001323:Autoantibodies / Q000097:blood*; D002648:Child; D002675:Child, Preschool; D000795:Fabry Disease / Q000188:drug therapy / Q000276:immunology / Q000652:urine*; D006801:Humans; D007527:Isoenzymes / Q000276:immunology / Q000627:therapeutic use*; D008875:Middle Aged; D014281:Trihexosylceramides / Q000652:urine*; D000519:alpha-Galactosidase / Q000276:immunology / Q000627:therapeutic use*
D001323:Autoantibodies; D007527:Isoenzymes; D014281:Trihexosylceramides; C018549:globotriaosylceramide; D000519:alpha-Galactosidase; C459420:agalsidase beta