title: The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy.
abstract: OBJECTIVE
  Fabry disease is a rare X-linked disease with multisystemic manifestations. This study investigated the effectiveness of long-term enzyme replacement therapy with agalsidase alfa in Fabry nephropathy treatment.
  
  
  METHODS
  In this observational study, data on patients receiving agalsidase alfa (0.2 mg/kg every other week) were extracted from the Fabry Outcome Survey, an international registry of patients with Fabry disease. Serum creatinine and estimated GFR (eGFR) at baseline and after ≥5 years of treatment were assessed; 24-hour urinary protein excretion and BP measurements were also reviewed. The eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration formula. Patients with an eGFR <30 ml/min per 1.73 m(2) were excluded.
  
  
  RESULTS
  Renal function was assessed in 208 patients (mean enzyme replacement therapy, 7.4 years; range, 5.0-11.2 years). Mean yearly change in eGFR was -2.2 ml/min per 1.73 m(2) in men and -0.7 ml/min per 1.73 m(2) in women (95% confidence limits, -2.8; -1.7 and -1.4; 0.0, respectively). Patients with 24-hour protein excretion >1 g/24 h had poorer renal function at baseline and follow-up compared with patients with protein excretion of 500-1000 mg/24 h or with proteinuria <500 mg/24 h. Renal function was worse in patients with baseline arterial hypertension, and there was a more rapid yearly decline compared with normotensive patients.
  
  
  CONCLUSIONS
  This study suggests that long-term agalsidase alfa therapy is able to stabilize the rate of Fabry nephropathy progression in women and is associated with a mild to moderate decline of renal function in men.
authors.affiliation: Belcolle Hospital, Nephrology and Dialysis, Strada Sammartinese snc, IT-01100 Viterbo, Italy. sandro.feriozzi@tiscali.it
authors.forename: Sandro
authors.initials: S
authors.lastname: Feriozzi
authors.forename: Joan
authors.initials: J
authors.lastname: Torras
authors.forename: Markus
authors.initials: M
authors.lastname: Cybulla
authors.forename: Kathy
authors.initials: K
authors.lastname: Nicholls
authors.forename: Gere
authors.initials: G
authors.lastname: Sunder-Plassmann
authors.forename: Michael
authors.initials: M
authors.lastname: West
mesh_terms: D000328:Adult; D057911:Angiotensin Receptor Antagonists / Q000627:therapeutic use; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000627:therapeutic use; D056947:Enzyme Replacement Therapy; D000795:Fabry Disease / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D006973:Hypertension / Q000503:physiopathology; D007527:Isoenzymes / Q000627:therapeutic use; D007674:Kidney Diseases / Q000188:drug therapy* / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D011507:Proteinuria / Q000503:physiopathology; D011994:Recombinant Proteins; D000519:alpha-Galactosidase / Q000627:therapeutic use*
chemical_list: D057911:Angiotensin Receptor Antagonists; D000806:Angiotensin-Converting Enzyme Inhibitors; D007527:Isoenzymes; D011994:Recombinant Proteins; C000627036:agalsidase alfa; D000519:alpha-Galactosidase