| Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease. | |
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MedLine Citation:
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PMID: 15466083 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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We report a patient with Pompe disease who developed reversible nephrotic syndrome during prolonged, high-dose, experimental, enzyme replacement therapy with recombinant human acid alpha-glucosidase (rhGAA). Because of the development of antibodies to rhGAA and concomitant clinical decline, escalating doses of rhGAA were administered as part of an experimental immune tolerance regimen. Histologic evaluation of kidney tissue revealed glomerular deposition of immune complexes containing rhGAA itself, in a pattern of membranous nephropathy. To our knowledge, this is the first reported case of nephrotic syndrome occurring during enzyme replacement therapy. The nephrotic syndrome gradually resolved after the rhGAA dose was decreased, indicating that decreasing the antigenic load can ameliorate glomerular immune complex deposition associated with enzyme replacement in a highly sensitized patient. |
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Authors:
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Tracy E Hunley; Deyanira Corzo; Martha Dudek; Priya Kishnani; Andrea Amalfitano; Yuan-Tsong Chen; Susan M Richards; John A Phillips; Agnes B Fogo; George E Tiller |
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Publication Detail:
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Type: Case Reports; Journal Article; Research Support, Non-U.S. Gov't |
Journal Detail:
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Title: Pediatrics Volume: 114 ISSN: 1098-4275 ISO Abbreviation: Pediatrics Publication Date: 2004 Oct |
Date Detail:
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Created Date: 2004-10-06 Completed Date: 2005-02-14 Revised Date: 2006-11-15 |
Medline Journal Info:
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Nlm Unique ID: 0376422 Medline TA: Pediatrics Country: United States |
Other Details:
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Languages: eng Pagination: e532-5 Citation Subset: AIM; IM |
Affiliation:
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Division of Pediatric Nephrology, Vanderbilt University Medical Center, C-4204 Medical Center North, Nashville, TN 37232-2584, USA. tray.hunley@mcmail.vanderbilt.edu |
Export Citation:
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| MeSH Terms | |
Descriptor/Qualifier:
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Antibodies
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blood,
immunology Child, Preschool Glycogen Storage Disease Type II / complications, drug therapy*, immunology Humans Immune Tolerance Kidney / pathology Male Nephrotic Syndrome / chemically induced*, immunology, pathology alpha-Glucosidases / adverse effects*, immunology, therapeutic use |
| Chemical | |
Reg. No./Substance:
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0/Antibodies; EC 3.2.1.20/alpha-Glucosidases |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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