title: Kidney transplantation and enzyme replacement therapy in patients with Fabry disease.
abstract: During Fabry disease, progressive glycosphingolipid deposition in the kidney causes gradual deterioration of renal function with proteinuria, uremia and hypertension. This results in end-stage renal disease (ESRD) which is one of the leading causes of morbidity and premature mortality in affected patients. Given the excellent graft and patient survival generally nowadays, kidney transplantation is the first choice to correct renal dysfunction and improve the overall prognosis of patients with renal failure because of Fabry disease. The benefit of enzyme-replacement therapy (ERT) in kidney transplanted Fabry patients has been controversially discussed and long-term trials focusing on the effectiveness of agalsidase in this patient population are needed.
authors.affiliation: FGM, Center of Internal Medicine, Department of Nephrology and Rheumatology, Müllheim, Germany.
authors.forename: Markus
authors.initials: M
authors.lastname: Cybulla
authors.forename: Christine
authors.initials: C
authors.lastname: Kurschat
authors.forename: Michael
authors.initials: M
authors.lastname: West
authors.forename: Kathy
authors.initials: K
authors.lastname: Nicholls
authors.forename: Joan
authors.initials: J
authors.lastname: Torras
authors.forename: Gere
authors.initials: G
authors.lastname: Sunder-Plassmann
authors.forename: Sandro
authors.initials: S
authors.lastname: Feriozzi
mesh_terms: D018450:Disease Progression; D056947:Enzyme Replacement Therapy; D000795:Fabry Disease / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007527:Isoenzymes / Q000627:therapeutic use*; D007676:Kidney Failure, Chronic / Q000209:etiology / Q000601:surgery; D016030:Kidney Transplantation; D000519:alpha-Galactosidase / Q000627:therapeutic use*
chemical_list: D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta