Fabry Disease, Proteinuria
Conditions
Keywords
proteinuria, ARBs, agalsidase-beta, MDRD estimated GFR, Chronic kidney disease
Brief summary
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Our hypothesis is that using drugs that reduce urine protein excretion (ACE inhibitors and ARBs) will have a beneficial effect on patients with Fabry disease who already are receiving enzyme replacement therapy. A longitudinal, observational study is being undertaken to determine the utility of these agents in Fabry disease, realizing that these agents are primarily indicated for reducing systemic blood pressure, and most patients with Fabry disease have relatively low blood pressures at baseline.
Detailed description
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Our hypothesis is that using drugs that reduce urine protein excretion (ACE inhibitors and ARBs) will have a beneficial effect on patients with Fabry disease who already are receiving enzyme replacement therapy. A longitudinal, observational study is being undertaken to determine the utility of these agents in Fabry disease, realizing that these agents are primarily indicated for reducing systemic blood pressure, and most patients with Fabry disease have relatively low blood pressures at baseline.
Interventions
None listed
Sponsors
University of Alabama at Birmingham
Study design
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
14 Years to 95 Years
Healthy volunteers
No
Inclusion criteria
* genetically confirmed Fabry disease * institution of commercially available agalsidase-beta
Exclusion criteria
* s/p kidney transplant
Countries
United States
Outcome results
None listed