title: Effect of reduced agalsidase Beta dosage in fabry patients: the Australian experience.
abstract: BACKGROUND
  In Australia, enzyme replacement therapy (ERT) for Fabry Disease (FD), both Agalsidase alfa (Replagal, Shire HGT) and beta (Fabrazyme, Genzyme), is funded and monitored through a specific government program. Agalsidase beta supply has been rationed by Genzyme since 2009 due to manufacturing issues. Consequently, the Australian Fabry Disease Advisory Committee has treated patients on Agalsidase beta at 50% of their usual dose from mid-2009, with a further reduction to 30% for some patients from late 2009.
  
  
  OBJECTIVE
  To determine the clinical effect of Agalsidase beta dose reduction in the Australian FD patient cohort.
  
  
  METHODS
  A questionnaire assessing FD symptoms was administered to 40 patients on long-term ERT. Clinical data from The Fabry Registry for patients receiving Agalsidase alfa or beta, for at least 2 years prior to the time of enforced Agalsidase beta dose reduction, were reviewed. Disease burden and quality of life (QOL) were graded using the Disease Severity Scoring System, Mainz Severity Score Index, Brief Pain Inventory and Short Form 36 Health Survey at 2 years before dose reduction, at the time of dose reduction and at the most recent clinical review following dose reduction.
  
  
  RESULTS
  Disease severity and QOL scores did not change between the ERT groups. Males on Agalsidase beta reported lower energy levels after dose reduction, while no change was reported by females on either product or by males on a stable dose of Agalsidase alfa.
  
  
  CONCLUSIONS
  This study suggests that energy levels in male patients worsen after dose reduction of Agalsidase beta.
authors: Nephrology Department, Royal Melbourne Hospital, Grattan St, Parkville, VIC, 3050, Australia, joanna.ghali@monash.edu.
authors: Joanna
authors: J
authors: Ghali
authors: Kathy
authors: K
authors: Nicholls
authors: Charles
authors: C
authors: Denaro
authors: David
authors: D
authors: Sillence
authors: Ian
authors: I
authors: Chapman
authors: Jack
authors: J
authors: Goldblatt
authors: Mark
authors: M
authors: Thomas
authors: Janice
authors: J
authors: Fletcher