Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3

Two different enzyme preparations are used for the treatment of Fabry disease patients, agalsidase alfa (Replagal, Shire) and agalsidase beta (Fabrazyme, Genzyme). Therapeutic efficacy of both products has been variable probably due to differences in gender, severity, age and other patient character...

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Veröffentlicht in:Molecular genetics and metabolism 2008-07, Vol.94 (3), p.319-325
Hauptverfasser: Vedder, Anouk C., Breunig, Frank, Donker-Koopman, Wilma E., Mills, Kevin, Young, Elisabeth, Winchester, Bryan, Ten Berge, Ineke J.M., Groener, Johanna E.M., Aerts, Johannes M.F.G., Wanner, Christoph, Hollak, Carla E.M.
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container_end_page 325
container_issue 3
container_start_page 319
container_title Molecular genetics and metabolism
container_volume 94
creator Vedder, Anouk C.
Breunig, Frank
Donker-Koopman, Wilma E.
Mills, Kevin
Young, Elisabeth
Winchester, Bryan
Ten Berge, Ineke J.M.
Groener, Johanna E.M.
Aerts, Johannes M.F.G.
Wanner, Christoph
Hollak, Carla E.M.
description Two different enzyme preparations are used for the treatment of Fabry disease patients, agalsidase alfa (Replagal, Shire) and agalsidase beta (Fabrazyme, Genzyme). Therapeutic efficacy of both products has been variable probably due to differences in gender, severity, age and other patient characteristics. We studied the occurrence of α-Gal A antibodies and their effect on urinary and plasma globotriaosylceramide (GL-3), plasma chitotriosidase and clinical outcome in 52 patients after 12 months of treatment with either 0.2mg/kg agalsidase alfa (10 males, 8 females) or beta (8 males, 5 females) or 1.0mg/kg agalsidase beta (10 males, 11 females). Antibodies were detected in 18/28 male patients after 6 months. None of the females developed antibodies. Following 12 months of 0.2mg/kg treatment, urinary GL-3 decreased in antibody negative (AB−) but increased in antibody positive (AB+) patients. Treatment with 1.0mg/kg gave a reduction in urinary GL-3 in both AB− and AB+ patients. Levels of plasma GL-3 and chitotriosidase decreased in all patient groups. Twelve months of 0.2mg/kg treatment did not change renal function or left ventricular mass. Further, no change in renal function was seen following 1.0mg/kg treatment and left ventricular mass decreased in both AB− and AB+ patients. In summary, α-Gal A antibodies frequently develop in male Fabry disease patients and interfere with urinary GL-3 excretion. Infusion of a dose of 1.0mg/kg results in a more robust decline in GL-3, less impact, if any of antibodies, stable renal function and reduction of LVMass.
doi_str_mv 10.1016/j.ymgme.2008.03.003
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Therapeutic efficacy of both products has been variable probably due to differences in gender, severity, age and other patient characteristics. We studied the occurrence of α-Gal A antibodies and their effect on urinary and plasma globotriaosylceramide (GL-3), plasma chitotriosidase and clinical outcome in 52 patients after 12 months of treatment with either 0.2mg/kg agalsidase alfa (10 males, 8 females) or beta (8 males, 5 females) or 1.0mg/kg agalsidase beta (10 males, 11 females). Antibodies were detected in 18/28 male patients after 6 months. None of the females developed antibodies. Following 12 months of 0.2mg/kg treatment, urinary GL-3 decreased in antibody negative (AB−) but increased in antibody positive (AB+) patients. Treatment with 1.0mg/kg gave a reduction in urinary GL-3 in both AB− and AB+ patients. Levels of plasma GL-3 and chitotriosidase decreased in all patient groups. Twelve months of 0.2mg/kg treatment did not change renal function or left ventricular mass. Further, no change in renal function was seen following 1.0mg/kg treatment and left ventricular mass decreased in both AB− and AB+ patients. In summary, α-Gal A antibodies frequently develop in male Fabry disease patients and interfere with urinary GL-3 excretion. Infusion of a dose of 1.0mg/kg results in a more robust decline in GL-3, less impact, if any of antibodies, stable renal function and reduction of LVMass.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>18424138</pmid><doi>10.1016/j.ymgme.2008.03.003</doi><tpages>7</tpages></addata></record>
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subjects Adult
Aged
alpha-Galactosidase - administration & dosage
alpha-Galactosidase - adverse effects
alpha-Galactosidase - antagonists & inhibitors
alpha-Galactosidase - immunology
Antibodies
Antibodies - blood
Antibodies - pharmacology
Antibody Formation - drug effects
Dose-Response Relationship, Drug
Enzyme replacement
Fabry disease
Fabry Disease - blood
Fabry Disease - drug therapy
Fabry Disease - immunology
Fabry Disease - urine
Female
Globotriaosylceramide
Heart Ventricles - pathology
Hexosaminidases - metabolism
Humans
Hypertrophy - chemically induced
Kidney - physiology
Male
Middle Aged
Treatment Failure
Trihexosylceramides - blood
Trihexosylceramides - metabolism
Trihexosylceramides - urine
title Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3
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