Antiproteinuric therapy and fabry nephropathy: sustained reduction of proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta

J Am Soc Nephrol. 2007 Sep;18(9):2609-17. doi: 10.1681/ASN.2006121400. Epub 2007 Jul 26.


This report describes an open-label, nonrandomized, prospective evaluation of the effects of angiotensin-converting enzyme inhibitor and angiotensin receptor blocker therapy on patients who have Fabry disease and also received enzyme replacement therapy with agalsidase-beta, given at 1 mg/kg body wt every 2 wk. Previous placebo-controlled phase III and phase IV trials with agalsidase-beta demonstrated clearing of globotriaosylceramide from vascular endothelia but little effect on proteinuria or progressive loss of kidney function in patients with Fabry disease and severe chronic kidney disease marked by overt proteinuria and/or estimated GFR <60 ml/min per 1.73 m2. Angiotensin-converting enzyme inhibitor and/or angiotensin receptor blocker therapy is the standard of care for patients with proteinuric kidney diseases, but their use is challenging in patients with Fabry disease and low or low-normal baseline systemic BP. A group of patients with Fabry disease were treated with antiproteinuric therapy, in conjunction with agalsidase-beta; sustained reductions in proteinuria with stabilization of kidney function were achieved in a group of six patients who had severe Fabry nephropathy; the progression rate was -0.23 +/- 1.12 ml/min per 1.73 m2 per yr with 30 mo of follow-up.

Publication types

  • Clinical Trial

MeSH terms

  • Adult
  • Angiotensin II Type 1 Receptor Blockers / administration & dosage
  • Angiotensin II Type 1 Receptor Blockers / therapeutic use*
  • Angiotensin-Converting Enzyme Inhibitors / administration & dosage
  • Angiotensin-Converting Enzyme Inhibitors / therapeutic use*
  • Blood Pressure
  • Creatinine / urine
  • Drug Administration Schedule
  • Drug Therapy, Combination
  • Fabry Disease / complications*
  • Female
  • Glomerular Filtration Rate
  • Humans
  • Isoenzymes / administration & dosage
  • Isoenzymes / therapeutic use*
  • Kidney / drug effects
  • Kidney / physiopathology
  • Kidney Diseases / drug therapy*
  • Kidney Diseases / etiology*
  • Kidney Diseases / physiopathology
  • Male
  • Middle Aged
  • Prospective Studies
  • Proteinuria / drug therapy*
  • Proteinuria / physiopathology
  • Severity of Illness Index
  • Treatment Outcome
  • alpha-Galactosidase / administration & dosage
  • alpha-Galactosidase / therapeutic use*


  • Angiotensin II Type 1 Receptor Blockers
  • Angiotensin-Converting Enzyme Inhibitors
  • Isoenzymes
  • Creatinine
  • alpha-Galactosidase
  • agalsidase beta