title: Kidney transplantation in patients with Fabry disease.
abstract: Little is known about the effects of enzyme replacement therapy (ERT) in kidney transplant recipients with Fabry disease. Clinical characteristics of transplant recipients in the Fabry Outcome Survey (FOS) were therefore examined in patients with Fabry disease with or without ERT. Of the 837 European patients in FOS (March 2006), 34 male patients and two female patients had received kidney transplants. Mean age at transplantation was 37.6 +/- 10.9 years, mean time since transplantation was 7.7 +/- 6.4 years, median estimated glomerular filtration rate (eGFR) was 44.4 ml/min/1.73 m(2), and median proteinuria was 296 mg/24 h. Of 27 patients with baseline data, 59% had hypertension, 74% had left ventricular hypertrophy, 22% had cardiac valve disease, 30% had arrhythmia, and 22% had transient ischaemic attacks and 15% stroke. Twenty patients (74%; two female patients, 18 male patients) were receiving ERT with agalsidase alfa. At enrollment or at the start of ERT, median eGFRs were 59 and 35 ml/min/1.73 m(2) (P = 0.05) and median proteinuria levels were 240 and 420 mg/24 h (not significant) in treated and untreated patients respectively. Renal function remained stable in patients receiving ERT. In conclusion, agalsidase alfa is well tolerated in patients with Fabry disease who have undergone renal transplantation.
authors.affiliation: Department of Nephrology, University Hospital of Freiburg, Germany. markus.cybulla@uniklinik-freiburg.de
authors.forename: Markus
authors.initials: M
authors.lastname: Cybulla
authors.forename: Kerstin Nanette
authors.initials: KN
authors.lastname: Walter
authors.forename: Andreas
authors.initials: A
authors.lastname: Schwarting
authors.forename: Raffaelle
authors.initials: R
authors.lastname: Divito
authors.forename: Sandro
authors.initials: S
authors.lastname: Feriozzi
authors.forename: Gere
authors.initials: G
authors.lastname: Sunder-Plassmann
mesh_terms: D000293:Adolescent; D000328:Adult; D015897:Comorbidity; D000795:Fabry Disease / Q000150:complications* / Q000188:drug therapy; D005260:Female; D006801:Humans; D007527:Isoenzymes / Q000627:therapeutic use*; D007676:Kidney Failure, Chronic / Q000453:epidemiology / Q000209:etiology* / Q000601:surgery; D007677:Kidney Function Tests; D016030:Kidney Transplantation; D008297:Male; D008875:Middle Aged; D011994:Recombinant Proteins; D055815:Young Adult; D000519:alpha-Galactosidase / Q000627:therapeutic use*
chemical_list: D007527:Isoenzymes; D011994:Recombinant Proteins; C000627036:agalsidase alfa; D000519:alpha-Galactosidase