Document Detail

Agalsidase benefits renal histology in young patients with Fabry disease.
MedLine Citation:
PMID:  23274955     Owner:  NLM     Status:  MEDLINE    
The effect of early-onset enzyme replacement therapy on renal morphologic features in Fabry disease is largely unknown. Here, we evaluated the effect of 5 years of treatment with agalsidase alfa or agalsidase beta in 12 consecutive patients age 7-33 years (median age, 16.5 years). We performed renal biopsies at baseline and after 5 years of enzyme replacement therapy; 7 patients had additional biopsies after 1 and 3 years. After a median of 65 months, biopsy findings from all patients showed total clearance of glomerular endothelial and mesangial cell inclusions, and findings from 2 patients showed complete clearance of inclusions from epithelial cells of the distal tubule. The 4 patients who received the highest dose of agalsidase exhibited substantial clearance of podocyte inclusions, and the youngest patient had nearly complete clearance of these inclusions. Linear regression analysis showed a highly significant correlation between podocyte globotriaocylceramide clearance and cumulative agalsidase dose (r=0.804; P=0.002). Microalbuminuria normalized in five patients. In summary, long-term enzyme replacement therapy in young patients can result in complete globotriaocylceramide clearance of mesangial and glomerular endothelial cells across all dosage regimens, and clearance of podocyte inclusions is dose-dependent.
Camilla Tøndel; Leif Bostad; Kristin Kampevold Larsen; Asle Hirth; Bjørn Egil Vikse; Gunnar Houge; Einar Svarstad
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Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  Journal of the American Society of Nephrology : JASN     Volume:  24     ISSN:  1533-3450     ISO Abbreviation:  J. Am. Soc. Nephrol.     Publication Date:  2013 Jan 
Date Detail:
Created Date:  2012-12-31     Completed Date:  2013-03-04     Revised Date:  2014-01-09    
Medline Journal Info:
Nlm Unique ID:  9013836     Medline TA:  J Am Soc Nephrol     Country:  United States    
Other Details:
Languages:  eng     Pagination:  137-48     Citation Subset:  IM    
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MeSH Terms
Enzyme Replacement Therapy*
Fabry Disease / drug therapy*,  pathology,  urine
Isoenzymes / administration & dosage,  therapeutic use*
Kidney / drug effects*,  pathology
Kidney Function Tests
Proteinuria / urine
Trihexosylceramides / urine
Young Adult
alpha-Galactosidase / administration & dosage,  therapeutic use*
Reg. No./Substance:
0/Isoenzymes; 0/Trihexosylceramides; 71965-57-6/globotriaosylceramide; EC 3.2.1.-/agalsidase alfa; EC 3.2.1.-/agalsidase beta; EC
Comment In:
Nat Rev Nephrol. 2013 Mar;9(3):125   [PMID:  23321565 ]

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