Clinical course of patients with Fabry disease who were switched from agalsidase-β to agalsidase-α.

[fabry disease]

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 .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 .Clinical data were collected for 5 years -2 years before switching and 3 years after switching .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 .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 .Genet Med 16 10 , 766 -772 .

Diseases
Validation

Diseases presenting "female patients" symptom

adrenomyeloneuropathy

aromatase deficiency

benign recurrent intrahepatic cholestasis

carcinoma of the gallbladder

classical phenylketonuria

congenital adrenal hyperplasia

cushing syndrome

erdheim-chester disease

esophageal adenocarcinoma

esophageal carcinoma

esophageal squamous cell carcinoma

fabry disease

familial hypocalciuric hypercalcemia

familial mediterranean fever

heparin-induced thrombocytopenia

lymphangioleiomyomatosis

oligodontia

papillon-lefèvre syndrome

phenylketonuria

pyruvate dehydrogenase deficiency

sneddon syndrome

trochlear dysplasia

von hippel-lindau disease

wolf-hirschhorn syndrome

x-linked adrenoleukodystrophy

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