The DOI matches its Crossref record; 12 of the 12 references it cites were found in a registry or an index.
Sustained, Long-Term Renal Stabilization After 54 Months of Agalsidase β Therapy in Patients with Fabry Disease
Abstract
Fabry disease, an inherited deficiency of the lysosomal enzyme alpha-galactosidase A, causes progressive intralysosomal accumulation of globotriaosylceramide (GL-3) and premature death from renal, cardiac, and cerebrovascular manifestations. To determine the long-term safety and efficacy of recombinant human alpha-galactosidase A, an open-label, phase III extension study was conducted, involving 58 patients who had classic Fabry disease and completed a 20-wk, double-blind, randomized, placebo-controlled, phase III study of agalsidase beta and were transitioned to an extension trial to receive biweekly 1 mg/kg agalsidase beta for up to an additional 54 mo. GL-3 accumulation was evaluated in the capillary endothelia of the skin, kidney, and heart. Renal function was assessed. By month 54, all patients with optional kidney biopsies (n = 8) maintained complete GL-3 clearance in renal capillary endothelial cells and multiple cell types. Continued, complete clearance of skin (31 of 36) and heart (six of eight) capillary endothelium was demonstrated. Mean plasma GL-3 levels remained decreased in the normal range. Median serum creatinine and estimated GFR remained stable (normal) in patients with renal data at month 54 (n = 41). Six patients had renal disease progression; most (four of six) were older than 40 yr and had significant proteinuria at baseline and evidence of sclerotic glomeruli pretreatment. Adverse events were generally mild and unrelated to treatment. The most common treatment-related adverse events were infusion-associated reactions, which decreased over time. Long-term agalsidase beta therapy stabilizes renal function in patients without renal involvement at baseline, maintains reduction of plasma GL-3, and sustains GL-3 clearance in capillary endothelial cells and multiple renal cell types.
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Cites
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Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease
Eng, C. M., Guffon, N., Wilcox, W. R., Germain, D. P., Lee, P., Waldek, S., Caplan, L., Linthorst, G. E., & Desnick, R. J. (2001) · New England Journal of Medicine · doi:10.1056/nejm200107053450102 · Cited by 1,559
Self-citationBRANTON, M. H., SCHIFFMANN, R., SABNIS, S. G., MURRAY, G. J., QUIRK, J. M., ALTARESCU, G., GOLDFARB, L., BRADY, R. O., BALOW, J. E., AUSTIN, H. A., et al. (2002) · Medicine · doi:10.1097/00005792-200203000-00003 · Cited by 461
Long-Term Safety and Efficacy of Enzyme Replacement Therapyfor Fabry Disease
Wilcox, W. R., for the International Fabry Disease Study Group, Banikazemi, M., Guffon, N., Waldek, S., Lee, P., Linthorst, G. E., Desnick, R. J., & Germain, D. P. (2004) · The American Journal of Human Genetics · doi:10.1086/422366 · Cited by 404
Self-citationEng, C. M., Banikazemi, M., Gordon, R. E., Goldman, M., Phelps, R., Kim, L., Gass, A., Winston, J., Dikman, S., Fallon, J. T., et al. (2001) · The American Journal of Human Genetics · doi:10.1086/318809 · Cited by 385
Self-citationInborn Metabolic Diseases: Diagnosis and Treatment
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Self-citationNatural history of Fabry disease in females in the Fabry Outcome Survey
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Patients with Fabry disease on dialysis in the United States
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Cited by
Kernan, W. N., Ovbiagele, B., Black, H. R., Bravata, D. M., Chimowitz, M. I., Ezekowitz, M. D., Fang, M. C., Fisher, M., Furie, K. L., Heck, D. V., et al. (2014) · Stroke · doi:10.1161/str.0000000000000024 · Cited by 5,170
Fabry disease revisited: Management and treatment recommendations for adult patients
Ortiz, A., Germain, D. P., Desnick, R. J., Politei, J., Mauer, M., Burlina, A., Eng, C., Hopkin, R. J., Laney, D., Linhart, A., et al. (2018) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2018.02.014 · Cited by 650
Self-citationWaldek, S., Patel, M. R., Banikazemi, M., Lemay, R., & Lee, P. (2009) · Genetics in Medicine · doi:10.1097/gim.0b013e3181bb05bb · Cited by 353
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Metadata
- Authors
- Germain, D. P., Waldek, S., Banikazemi, M., Bushinsky, D. A., Charrow, J., Desnick, R. J., Lee, P., Loew, T., Vedder, A. C., Abichandani, R., Wilcox, W. R., & Guffon, N.
- Venue
- Journal of the American Society of Nephrology
- Published
- 2007-05
- Volume
- 18
- Issue
- 5
- Pages
- 1547-1557
- ISSN
- 1046-6673 (ISSN-L)
- OpenAlex
- W2041953538
- Cited by
- 438
- References
- 12
- FWCI
- 26.70
- Type
- journal article
- Language
- en
- Open access
- closed
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- Germain, D. P., Waldek, S., Banikazemi, M., Bushinsky, D. A., Charrow, J., Desnick, R. J., Lee, P., Loew, T., Vedder, A. C., Abichandani, R., Wilcox, W. R., & Guffon, N. (2007). Sustained, Long-Term Renal Stabilization After 54 Months of Agalsidase β Therapy in Patients with Fabry Disease. Journal of the American Society of Nephrology. https://doi.org/10.1681/asn.2006080816
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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