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Treatment of Fabry disease with different dosing regimens of agalsidase: effects on antibody formation and GL-3.
Mol Genet Metab. 2008 Jul; 94(3):319-25.MG

Abstract

Two different enzyme preparations are used for the treatment of Fabry disease patients, agalsidase alpha (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 alpha-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 alppha (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, alpha-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.

Authors+Show Affiliations

Department of Internal Medicine/Endocrinology and Metabolism, Academic Medical Center, F4-224, Meibergdreef 9, 1105 AZ Amsterdam, The Netherlands.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial
Journal Article
Multicenter Study

Language

eng

PubMed ID

18424138

Citation

Vedder, Anouk C., et al. "Treatment of Fabry Disease With Different Dosing Regimens of Agalsidase: Effects On Antibody Formation and GL-3." Molecular Genetics and Metabolism, vol. 94, no. 3, 2008, pp. 319-25.
Vedder AC, Breunig F, Donker-Koopman WE, et al. Treatment of Fabry disease with different dosing regimens of agalsidase: effects on antibody formation and GL-3. Mol Genet Metab. 2008;94(3):319-25.
Vedder, A. C., Breunig, F., Donker-Koopman, W. E., Mills, K., Young, E., Winchester, B., Ten Berge, I. J., Groener, J. E., Aerts, J. M., Wanner, C., & Hollak, C. E. (2008). Treatment of Fabry disease with different dosing regimens of agalsidase: effects on antibody formation and GL-3. Molecular Genetics and Metabolism, 94(3), 319-25. https://doi.org/10.1016/j.ymgme.2008.03.003
Vedder AC, et al. Treatment of Fabry Disease With Different Dosing Regimens of Agalsidase: Effects On Antibody Formation and GL-3. Mol Genet Metab. 2008;94(3):319-25. PubMed PMID: 18424138.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Treatment of Fabry disease with different dosing regimens of agalsidase: effects on antibody formation and GL-3. AU - Vedder,Anouk C, AU - Breunig,Frank, AU - Donker-Koopman,Wilma E, AU - Mills,Kevin, AU - Young,Elisabeth, AU - Winchester,Bryan, AU - Ten Berge,Ineke J M, AU - Groener,Johanna E M, AU - Aerts,Johannes M F G, AU - Wanner,Christoph, AU - Hollak,Carla E M, Y1 - 2008/04/18/ PY - 2008/03/11/received PY - 2008/03/11/accepted PY - 2008/4/22/pubmed PY - 2008/9/16/medline PY - 2008/4/22/entrez SP - 319 EP - 25 JF - Molecular genetics and metabolism JO - Mol Genet Metab VL - 94 IS - 3 N2 - Two different enzyme preparations are used for the treatment of Fabry disease patients, agalsidase alpha (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 alpha-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 alppha (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, alpha-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. SN - 1096-7206 UR - https://www.unboundmedicine.com/medline/citation/18424138/Treatment_of_Fabry_disease_with_different_dosing_regimens_of_agalsidase:_effects_on_antibody_formation_and_GL_3_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1096-7192(08)00077-2 DB - PRIME DP - Unbound Medicine ER -