title: Clinical course of patients with Fabry disease who were switched from agalsidase-β to agalsidase-α.
abstract: BACKGROUND
  Between 2009 and 2012, there was a worldwide shortage of agalsidase-β for the treatment of Fabry disease. Therefore, alternative treatments were needed, including switching to a different enzyme-replacement therapy.
  
  
  OBJECTIVE
  This is an ongoing observational study assessing the effects of switching from agalsidase-β (1.0 mg/kg every other week) to agalsidase-α (0.2 mg/kg every other week) in 11 patients with Fabry disease.
  
  
  METHODS
  Clinical data were collected for 5 years-2 years before switching and 3 years after switching.
  
  
  RESULTS
  Measures of renal function such as estimated glomerular filtration rate remained stable during the 3 years after switching to agalsidase-α. Improvements in cardiac mass were recorded in both male and female patients 12 months after switching to agalsidase-α, and the benefit was maintained during 36 months of follow-up. There was no significant difference in the severity of pain experienced by patients before and after switching enzyme-replacement therapy, and no difference in quality-of-life parameters. Agalsidase-α was generally well tolerated, and no patients experienced allergy or developed antibodies to agalsidase-α.
  
  
  CONCLUSIONS
  This observational study supports the safety of switching from agalsidase-β to agalsidase-α at the approved doses, with no loss of efficacy. It also suggests that if an infusion-related allergic reaction occurs in a patient receiving agalsidase-β, switching to agalsidase-α may be a viable option.
authors.affiliation: LSD Center and Hematology, Nagoya Central Hospital, Nagoya, Japan.
authors.forename: Kazuya
authors.initials: K
authors.lastname: Tsuboi
authors.affiliation: Otorhinolaryngology, Nagoya Central Hospital, Nagoya, Japan.
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Yamamoto
mesh_terms: D000328:Adult; D000368:Aged; D003404:Creatinine / Q000652:urine; D056947:Enzyme Replacement Therapy / Q000379:methods*; D000795:Fabry Disease / Q000188:drug therapy*; D005260:Female; D005500:Follow-Up Studies; D005919:Glomerular Filtration Rate / Q000187:drug effects; D006801:Humans; D007527:Isoenzymes / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D011507:Proteinuria / Q000652:urine; D013997:Time Factors; D016896:Treatment Outcome; D000519:alpha-Galactosidase / Q000627:therapeutic use*
chemical_list: D007527:Isoenzymes; D003404:Creatinine; D000519:alpha-Galactosidase; C459420:agalsidase beta