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Enzyme replacement therapy and Fabry nephropathy.
Clin J Am Soc Nephrol. 2010 Feb; 5(2):371-8.CJ

Abstract

Involvement of the kidneys in Fabry disease ("nephropathy") occurs in male and female individuals. The majority of patients with progressive nephropathy will have significant proteinuria and develop progressive loss of kidney function, leading to ESRD. All too often, treating physicians may ignore "normal" serum creatinine levels or "minimal" proteinuria and fail to assess properly the severity of kidney involvement and institute appropriate management. Fabry nephropathy is treatable, even in patients with fairly advanced disease. Although the cornerstone of therapy remains enzyme replacement therapy with agalsidase, this treatment alone does not reduce urine protein excretion. Treatment with angiotensin receptor blockers or angiotensin-converting enzyme inhibitors must be added to enzyme replacement therapy to reduce urine protein excretion with the hope that this will stabilize kidney function. Kidney function, with at least estimated GFR based on serum creatinine and measurements of urinary protein, should be measured at every clinic visit, and the rate of change of the estimated GFR should be followed over time. Antiproteinuric therapy can be dosed to a prespecified urine protein target rather than a specific BP goal, with the proviso that successful therapy will usually lower the BP below the goal of 130/80 mmHg that is used for other forms of kidney disease. The overall goal for treating Fabry nephropathy is to reduce the rate of loss of GFR to -1 ml/min per 1.73 m(2)/yr, which is that seen in the normal adult population. A systematic approach is presented for reaching this goal in the individual patient.

Authors+Show Affiliations

Division of Nephrology, Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama 35294-0007, USA. dwarnock@uab.eduNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

20007680

Citation

Warnock, David G., et al. "Enzyme Replacement Therapy and Fabry Nephropathy." Clinical Journal of the American Society of Nephrology : CJASN, vol. 5, no. 2, 2010, pp. 371-8.
Warnock DG, Daina E, Remuzzi G, et al. Enzyme replacement therapy and Fabry nephropathy. Clin J Am Soc Nephrol. 2010;5(2):371-8.
Warnock, D. G., Daina, E., Remuzzi, G., & West, M. (2010). Enzyme replacement therapy and Fabry nephropathy. Clinical Journal of the American Society of Nephrology : CJASN, 5(2), 371-8. https://doi.org/10.2215/CJN.06900909
Warnock DG, et al. Enzyme Replacement Therapy and Fabry Nephropathy. Clin J Am Soc Nephrol. 2010;5(2):371-8. PubMed PMID: 20007680.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Enzyme replacement therapy and Fabry nephropathy. AU - Warnock,David G, AU - Daina,Erica, AU - Remuzzi,Giuseppe, AU - West,Michael, Y1 - 2009/12/10/ PY - 2009/12/17/entrez PY - 2009/12/17/pubmed PY - 2010/5/8/medline SP - 371 EP - 8 JF - Clinical journal of the American Society of Nephrology : CJASN JO - Clin J Am Soc Nephrol VL - 5 IS - 2 N2 - Involvement of the kidneys in Fabry disease ("nephropathy") occurs in male and female individuals. The majority of patients with progressive nephropathy will have significant proteinuria and develop progressive loss of kidney function, leading to ESRD. All too often, treating physicians may ignore "normal" serum creatinine levels or "minimal" proteinuria and fail to assess properly the severity of kidney involvement and institute appropriate management. Fabry nephropathy is treatable, even in patients with fairly advanced disease. Although the cornerstone of therapy remains enzyme replacement therapy with agalsidase, this treatment alone does not reduce urine protein excretion. Treatment with angiotensin receptor blockers or angiotensin-converting enzyme inhibitors must be added to enzyme replacement therapy to reduce urine protein excretion with the hope that this will stabilize kidney function. Kidney function, with at least estimated GFR based on serum creatinine and measurements of urinary protein, should be measured at every clinic visit, and the rate of change of the estimated GFR should be followed over time. Antiproteinuric therapy can be dosed to a prespecified urine protein target rather than a specific BP goal, with the proviso that successful therapy will usually lower the BP below the goal of 130/80 mmHg that is used for other forms of kidney disease. The overall goal for treating Fabry nephropathy is to reduce the rate of loss of GFR to -1 ml/min per 1.73 m(2)/yr, which is that seen in the normal adult population. A systematic approach is presented for reaching this goal in the individual patient. SN - 1555-905X UR - https://www.unboundmedicine.com/medline/citation/20007680/Enzyme_replacement_therapy_and_Fabry_nephropathy_ L2 - https://cjasn.asnjournals.org/cgi/pmidlookup?view=long&pmid=20007680 DB - PRIME DP - Unbound Medicine ER -