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| Content Provider | World Health Organization (WHO)-Global Index Medicus |
|---|---|
| Author | Desnick, Robert J. Linthorst, Gabor E. Banikazemi, Maryam Guffon, Nathalie Lee, Philip Wilcox, William R. Germain, Dominique P. Waldek, Stephen |
| Description | Country affiliation: United States Author Affiliation: Wilcox WR ( Cedars-Sinai Burns and Allen Research Institute and UCLA School of Medicine, Los Angeles, CA, USA.) |
| Abstract | Elsewhere, we reported the safety and efficacy results of a multicenter phase 3 trial of recombinant human alpha -galactosidase A (rh-alpha GalA) replacement in patients with Fabry disease. All 58 patients who were enrolled in the 20-wk phase 3 double-blind, randomized, and placebo-controlled study received subsequently 1 mg/kg of rh-alpha GalA (agalsidase beta, Fabrazyme, Genzyme Corporation) biweekly in an ongoing open-label extension study. Evidence of long-term efficacy, even in patients who developed IgG antibodies against rh- alpha GalA, included the continuously normal mean plasma globotriaosylceramide (GL-3) levels during 30 mo of the extension study and the sustained capillary endothelial GL-3 clearance in 98% (39/40) of patients who had a skin biopsy taken after treatment for 30 mo (original placebo group) or 36 mo (original enzyme-treated group). The mean serum creatinine level and estimated glomerular filtration rate also remained stable after 30-36 mo of treatment. Infusion-associated reactions decreased over time, as did anti-rh- alpha GalA IgG antibody titers. Among seroconverted patients, after 30-36 mo of treatment, seven patients tolerized (no detectable IgG antibody), and 59% had > or =4-fold reductions in antibody titers. As of 30 mo into the extension trial, three patients were withdrawn from the study because of positive serum IgE or skin tests; however, all have been rechallenged successfully at the time of this report. Thus, enzyme replacement therapy for 30-36 mo with agalsidase beta resulted in continuously decreased plasma GL-3 levels, sustained endothelial GL-3 clearance, stable kidney function, and a favorable safety profile. |
| ISSN | 00029297 |
| e-ISSN | 15376605 |
| Journal | The American Journal of Human Genetics |
| Issue Number | 1 |
| Volume Number | 75 |
| Language | English |
| Publisher | Cell Press (on behalf of American Society of Human Genetics) |
| Publisher Date | 2004-07-01 |
| Publisher Place | United States |
| Access Restriction | Open |
| Subject Keyword | Fabry Disease Drug Therapy Enzymology Isoenzymes Therapeutic Use Skin Metabolism Trihexosylceramides Alpha-galactosidase Biopsy Capillaries Drug Effects Double-blind Method Endothelium, Vascular Pathology Immunoglobulin E Blood Kidney Blood Supply Kidney Function Tests Metabolic Clearance Rate Placebos Recombinant Proteins Clinical Trial Clinical Trial, Phase Iii Comparative Study Randomized Controlled Trial Research Support, Non-u.s. Gov't Research Support, U.s. Gov't, P.h.s. Discipline Human Genetics |
| Content Type | Text |
| Resource Type | Article |
| Subject | Genetics Genetics (clinical) |
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