The Effectiveness of Long-Term Agalsidase Alfa Therapy in the Treatment of Fabry Nephropathy

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. In this observational study, data on patients receiving agalsidase alfa (0.2 mg/kg every...

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Published in:	Clinical journal of the American Society of Nephrology 2012-01, Vol.7 (1), p.60-69
Main Authors:	Feriozzi, Sandro, Torras, Joan, Cybulla, Markus, Nicholls, Kathy, Sunder-Plassmann, Gere, West, Michael
Format:	Article
Language:	eng
Subjects:	Adult alpha-Galactosidase - therapeutic use Angiotensin Receptor Antagonists - therapeutic use Angiotensin-Converting Enzyme Inhibitors - therapeutic use Enzyme Replacement Therapy Fabry Disease - complications Fabry Disease - drug therapy Fabry Disease - physiopathology Female Glomerular Filtration Rate Humans Hypertension - physiopathology Isoenzymes - therapeutic use Kidney Diseases - drug therapy Kidney Diseases - physiopathology Male Middle Aged Original Proteinuria - physiopathology Recombinant Proteins
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Summary:	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. 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 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
ISSN:	1555-9041 1555-905X