title: [Fabry's disease: a comparison of the effectiveness of two different enzyme replacement therapies].
abstract: BACKGROUND
  Anderson-Fabry disease (AFD) is a multiorgan X-linked lysosomal storage disease that particularly affects the heart, kidneys, and cerebrovascular system. Current treatment is with enzyme replacement therapy (ERT), using either beta-galactosidase ('Fabrazyme') or alpha-galactosidase ('Replagal'). From June 2009, it was recommended that patients switch to alpha-galactosidase due to a manufacturing shortage of beta-galactosidase. This study assesses the effect of switching to alpha-galactosidase on clinical outcomes in patients with AFD previously treated with beta-galactosidase.
  
  
  METHODS
  Ten patients (seven male, three female) with genetically confirmed AFD and at least 48 months continuous data collected during treatment with beta-galactosidase 1 mg/kg every other week, were switched to alpha-galactosidase 0.2 mg/kg every other week for at least 20 months, with prospective clinical evaluations performed every 6 months. Pre-switch data was collected retrospectively from patient charts. Cardiac functional parameters were assessed using magnetic resonance imaging.
  
  
  RESULTS
  Results showed that renal function generally remained stable after the switch. Cardiac mass had already decreased significantly (p < 0.05 vs pre-ERT) after introduction of beta-galactosidase and remained unchanged after switching to alpha-galactosidase. Symptoms of pain and health status scores did not deteriorate during alpha-galactosidase therapy. Adverse events were mostly mild and infusion -related.
  
  
  CONCLUSIONS
  In conclusion, switching to alpha-galactosidse was generally well tolerated and associated with stable clinical status and preservation of both renal and cardiac function.
authors.forename: Antonio
authors.initials: A
authors.lastname: Pisani
authors.forename: Bianca
authors.initials: B
authors.lastname: Visciano
authors.forename: Ivana
authors.initials: I
authors.lastname: Capuano
authors.forename: Antonello
authors.initials: A
authors.lastname: Mancini
authors.forename: Eleonora
authors.initials: E
authors.lastname: Riccio
mesh_terms: D000328:Adult; D004305:Dose-Response Relationship, Drug; D057915:Drug Substitution; D056947:Enzyme Replacement Therapy / Q000379:methods*; D000795:Fabry Disease / Q000188:drug therapy* / Q000201:enzymology*; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D019028:Magnetic Resonance Imaging, Cine; D008297:Male; D008875:Middle Aged; D011788:Quality of Life; D012189:Retrospective Studies; D016896:Treatment Outcome; D000519:alpha-Galactosidase / Q000627:therapeutic use*; D001616:beta-Galactosidase / Q000627:therapeutic use*
chemical_list: D000519:alpha-Galactosidase; D001616:beta-Galactosidase