Primary Hyperoxaluria

Primary hyperoxalurias (PH) are inborn errors in the metabolism of glyoxylate and oxalate. PH type 1, the most common form, is an autosomal recessive disorder caused by a deficiency of the liver-specific enzyme alanine, glyoxylate aminotransferase (AGT) resulting in overproduction and excessive urin...

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Main Authors: Jérôme Harambat, Sonia Fargue, Justine Bacchetta, Cécile Acquaviva, Pierre Cochat
Format: Article
Language:English
Published: Wiley 2011-01-01
Series:International Journal of Nephrology
Online Access:http://dx.doi.org/10.4061/2011/864580
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author Jérôme Harambat
Sonia Fargue
Justine Bacchetta
Cécile Acquaviva
Pierre Cochat
author_facet Jérôme Harambat
Sonia Fargue
Justine Bacchetta
Cécile Acquaviva
Pierre Cochat
author_sort Jérôme Harambat
collection DOAJ
description Primary hyperoxalurias (PH) are inborn errors in the metabolism of glyoxylate and oxalate. PH type 1, the most common form, is an autosomal recessive disorder caused by a deficiency of the liver-specific enzyme alanine, glyoxylate aminotransferase (AGT) resulting in overproduction and excessive urinary excretion of oxalate. Recurrent urolithiasis and nephrocalcinosis are the hallmarks of the disease. As glomerular filtration rate decreases due to progressive renal damage, oxalate accumulates leading to systemic oxalosis. Diagnosis is often delayed and is based on clinical and sonographic findings, urinary oxalate assessment, DNA analysis, and, if necessary, direct AGT activity measurement in liver biopsy tissue. Early initiation of conservative treatment, including high fluid intake, inhibitors of calcium oxalate crystallization, and pyridoxine in responsive cases, can help to maintain renal function in compliant subjects. In end-stage renal disease patients, the best outcomes have been achieved with combined liver-kidney transplantation which corrects the enzyme defect.
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spelling doaj-art-e2f44121de4346cea321664d05a9cf402025-08-20T02:21:14ZengWileyInternational Journal of Nephrology2090-214X2090-21582011-01-01201110.4061/2011/864580864580Primary HyperoxaluriaJérôme Harambat0Sonia Fargue1Justine Bacchetta2Cécile Acquaviva3Pierre Cochat4Service de Pédiatrie, Centre de référence Maladies Rénales Rares du Sud-Ouest, Centre Hospitalier Universitaire de Bordeaux, 33076 Bordeaux, FranceService de Pédiatrie, Centre de référence des Maladies Rénales Rares, Hospices Civils de Lyon et Université de Lyon, 69677 Bron, FranceService de Pédiatrie, Centre de référence des Maladies Rénales Rares, Hospices Civils de Lyon et Université de Lyon, 69677 Bron, FranceMaladies Héréditaires du Métabolisme, Centre de Biologie et Pathologie Est, Hospices Civils de Lyon, 69677 Bron, FranceService de Pédiatrie, Centre de référence des Maladies Rénales Rares, Hospices Civils de Lyon et Université de Lyon, 69677 Bron, FrancePrimary hyperoxalurias (PH) are inborn errors in the metabolism of glyoxylate and oxalate. PH type 1, the most common form, is an autosomal recessive disorder caused by a deficiency of the liver-specific enzyme alanine, glyoxylate aminotransferase (AGT) resulting in overproduction and excessive urinary excretion of oxalate. Recurrent urolithiasis and nephrocalcinosis are the hallmarks of the disease. As glomerular filtration rate decreases due to progressive renal damage, oxalate accumulates leading to systemic oxalosis. Diagnosis is often delayed and is based on clinical and sonographic findings, urinary oxalate assessment, DNA analysis, and, if necessary, direct AGT activity measurement in liver biopsy tissue. Early initiation of conservative treatment, including high fluid intake, inhibitors of calcium oxalate crystallization, and pyridoxine in responsive cases, can help to maintain renal function in compliant subjects. In end-stage renal disease patients, the best outcomes have been achieved with combined liver-kidney transplantation which corrects the enzyme defect.http://dx.doi.org/10.4061/2011/864580
spellingShingle Jérôme Harambat
Sonia Fargue
Justine Bacchetta
Cécile Acquaviva
Pierre Cochat
Primary Hyperoxaluria
International Journal of Nephrology
title Primary Hyperoxaluria
title_full Primary Hyperoxaluria
title_fullStr Primary Hyperoxaluria
title_full_unstemmed Primary Hyperoxaluria
title_short Primary Hyperoxaluria
title_sort primary hyperoxaluria
url http://dx.doi.org/10.4061/2011/864580
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AT soniafargue primaryhyperoxaluria
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AT cecileacquaviva primaryhyperoxaluria
AT pierrecochat primaryhyperoxaluria