Enzyme replacement therapy (ERT) is the worldwide standard of care for a number of mucopolysaccharidosis (MPS) diseases. We report a kinetic study of plasmatic dermatan sulfate (DS) in a 3-year-old subject affected by a severe form of MPS II during the first 10 months of ERT with Idursulfase. A strong increase in the DS plasmatic concentration was measured immediately after the first enzyme infusion, with a maximum after 3 h, followed by a continuous decrease in the 8-15 days following the beginning of treatment. After this, a constant plasmatic content of DS concentration was observed. Overall, during the 10-month treatment period, ERT reduced the plasmatic concentration of DS up to ~80-85 %, but it was unable to totally remove it from the blood. We can suppose that immediately after the first enzyme administrations, a large amount of abnormal DS is removed from tissues reaching the blood compartment and eliminated via the urine, and thereafter only minimal changes are observed. The persistency of the residual amounts of DS with the actually recommended dosage in our Patient may suggest the opportunity to promote further studies with increased enzyme dosages to completely remove the accumulation of lysosomal DS.

Plasmatic kinetics of dermatan sulfate during enzyme replacement therapy with iduronate-2-sulfatase in a mucopolysaccharidosis II Patient / Volpi, Nicola; Zampini, L; Maccari, Francesca; Santoro, L; Galeotti, Fabio; Galeazzi, T; Gabrielli, O; Coppa, G. V.. - In: GLYCOCONJUGATE JOURNAL. - ISSN 0282-0080. - STAMPA. - 30(7):(2013), pp. 727-732. [10.1007/s10719-013-9471-0]

Plasmatic kinetics of dermatan sulfate during enzyme replacement therapy with iduronate-2-sulfatase in a mucopolysaccharidosis II Patient

VOLPI, Nicola;MACCARI, Francesca;GALEOTTI, FABIO;
2013

Abstract

Enzyme replacement therapy (ERT) is the worldwide standard of care for a number of mucopolysaccharidosis (MPS) diseases. We report a kinetic study of plasmatic dermatan sulfate (DS) in a 3-year-old subject affected by a severe form of MPS II during the first 10 months of ERT with Idursulfase. A strong increase in the DS plasmatic concentration was measured immediately after the first enzyme infusion, with a maximum after 3 h, followed by a continuous decrease in the 8-15 days following the beginning of treatment. After this, a constant plasmatic content of DS concentration was observed. Overall, during the 10-month treatment period, ERT reduced the plasmatic concentration of DS up to ~80-85 %, but it was unable to totally remove it from the blood. We can suppose that immediately after the first enzyme administrations, a large amount of abnormal DS is removed from tissues reaching the blood compartment and eliminated via the urine, and thereafter only minimal changes are observed. The persistency of the residual amounts of DS with the actually recommended dosage in our Patient may suggest the opportunity to promote further studies with increased enzyme dosages to completely remove the accumulation of lysosomal DS.
30(7)
727
732
Plasmatic kinetics of dermatan sulfate during enzyme replacement therapy with iduronate-2-sulfatase in a mucopolysaccharidosis II Patient / Volpi, Nicola; Zampini, L; Maccari, Francesca; Santoro, L; Galeotti, Fabio; Galeazzi, T; Gabrielli, O; Coppa, G. V.. - In: GLYCOCONJUGATE JOURNAL. - ISSN 0282-0080. - STAMPA. - 30(7):(2013), pp. 727-732. [10.1007/s10719-013-9471-0]
Volpi, Nicola; Zampini, L; Maccari, Francesca; Santoro, L; Galeotti, Fabio; Galeazzi, T; Gabrielli, O; Coppa, G. V.
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/11380/970500
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