title: Clinical observations on enzyme replacement therapy in patients with Fabry disease and the switch from agalsidase beta to agalsidase alfa.
abstract: BACKGROUND
  Fabry disease is an X-linked inherited lysosomal storage disease that can be treated with the enzymes of agalsidase beta (Fabrazyme) and agalsidase alfa (Replagal). Since June 2009, viral contamination of Genzyme's production facility has resulted in a worldwide shortage of agalsidase beta, leading to the switch to agalsidase alfa for patients with Fabry disease in Taiwan.
  
  
  METHODS
  The medical records were retrospectively reviewed for nine male patients with Fabry disease from the start of agalsidase beta treatment until the switch to agalsidase alfa for at least 1 year.
  
  
  RESULTS
  After 12-112 months of enzyme replacement therapy (ERT), decreased plasma globotriaosylsphingosine (lyso-Gb3) was found in five out of seven patients, indicating improvement in disease severity. Among the six patients with available echocardiographic data at baseline and after ERT, all six experienced reductions of left ventricular mass index. Renal function, including microalbuminuria and estimated glomerular filtration rate, showed stability after ERT. Mainz Severity Score Index scores revealed that all nine patients remained stable at 12 months after switching to agalsidase alfa. ERT improved or stabilized cardiac status and stabilized renal function, while reducing plasma lyso-Gb3. ERT was well tolerated, even among the three patients who had hypersensitivity reactions.
  
  
  CONCLUSIONS
  The switch of ERT from agalsidase beta to agalsidase alfa appears to be safe after 1 year of follow-up for Taiwanese patients with Fabry disease.
authors.affiliation: Institute of Clinical Medicine, National Yang-Ming University, Taipei, Taiwan, ROC; Department of Pediatrics, Mackay Memorial Hospital, Taipei, Taiwan, ROC; Department of Medicine, Mackay Medical College, New Taipei City, Taiwan, ROC; Mackay Junior College of Medicine, Nursing, and Management, Taipei, Taiwan, ROC; Taiwan Clinical Trial Consortium in Fabry Disease, ROC.
authors.forename: Hsiang-Yu
authors.initials: HY
authors.lastname: Lin
authors.affiliation: Institute of Clinical Medicine, National Yang-Ming University, Taipei, Taiwan, ROC; Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Yu-Hsiu
authors.initials: YH
authors.lastname: Huang
authors.affiliation: Institute of Clinical Medicine, National Yang-Ming University, Taipei, Taiwan, ROC; Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Neonatal Screening Center, Chinese Foundation of Health, Taipei, Taiwan, ROC.
authors.forename: Hsuan-Chieh
authors.initials: HC
authors.lastname: Liao
authors.affiliation: Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Hao-Chuan
authors.initials: HC
authors.lastname: Liu
authors.affiliation: Institute of Clinical Medicine, National Yang-Ming University, Taipei, Taiwan, ROC; Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Ting-Rong
authors.initials: TR
authors.lastname: Hsu
authors.affiliation: Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Chia-I
authors.initials: CI
authors.lastname: Shen
authors.affiliation: Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Shao-Tzu
authors.initials: ST
authors.lastname: Li
authors.affiliation: Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Cheng-Fang
authors.initials: CF
authors.lastname: Li
authors.affiliation: Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Nursing Department, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Li-Hong
authors.initials: LH
authors.lastname: Lee
authors.affiliation: Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Pi-Chang
authors.initials: PC
authors.lastname: Lee
authors.affiliation: Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC.
authors.forename: Chun-Kai
authors.initials: CK
authors.lastname: Huang
authors.affiliation: Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Neonatal Screening Center, Chinese Foundation of Health, Taipei, Taiwan, ROC.
authors.forename: Chuan-Chi
authors.initials: CC
authors.lastname: Chiang
authors.affiliation: Department of Pediatrics, Mackay Memorial Hospital, Taipei, Taiwan, ROC; Department of Medicine, Mackay Medical College, New Taipei City, Taiwan, ROC; Mackay Junior College of Medicine, Nursing, and Management, Taipei, Taiwan, ROC.
authors.forename: Shuan-Pei
authors.initials: SP
authors.lastname: Lin
authors.affiliation: Institute of Clinical Medicine, National Yang-Ming University, Taipei, Taiwan, ROC; Taiwan Clinical Trial Consortium in Fabry Disease, ROC; Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC. Electronic address: dmniu1111@yahoo.com.tw.
authors.forename: Dau-Ming
authors.initials: DM
authors.lastname: Niu
mesh_terms: D000293:Adolescent; D000328:Adult; D056947:Enzyme Replacement Therapy / Q000379:methods*; D000795:Fabry Disease / Q000188:drug therapy*; D006801:Humans; D007527:Isoenzymes / Q000009:adverse effects / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D011994:Recombinant Proteins; D012189:Retrospective Studies; D000519:alpha-Galactosidase / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D007527:Isoenzymes; D011994:Recombinant Proteins; C000627036:agalsidase alfa; D000519:alpha-Galactosidase; C459420:agalsidase beta
keywords: Fabrazyme; Fabry disease; Replagal; agalsidase alfa; agalsidase beta; enzyme replacement therapy