Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3
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- 作者：Anouk C. Vedder ; Frank Breunig ; Wilma E. Donker-Koopman ; Kevin Mills ; Elisabeth Young ; Bryan Winchester ; Ineke J.M. Ten Berge ; Johanna E.M. Groener ; Johannes M.F.G. Aerts ; Christoph Wanner ; Carla E.M.
- 关键词：Fabry disease ; Enzyme replacement ; Antibodies ; Globotriaosylceramide
- 刊名：Molecular Genetics and Metabolism
- 出版年：2008
- 出版时间：July 2008
- 年：2008
- 卷：94
- 期：3
- 页码：319-325
- 全文大小：231 K
文摘
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 ![]()
-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 0.2 mg/kg agalsidase alfa (10 males, 8 females) or beta (8 males, 5 females) or 1.0 mg/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 0.2 mg/kg treatment, urinary GL-3 decreased in antibody negative (AB−) but increased in antibody positive (AB+) patients.