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Kidney Int ; 65(4): 1381-5, 2004 Apr.
Article in English | MEDLINE | ID: mdl-15086478

ABSTRACT

BACKGROUND: We sought to assess the safety and efficacy of enzyme replacement therapy (ERT) with recombinant human-alpha-galactosidase A (rh-alpha-Gal A) in kidney transplant recipients with Fabry disease, a previously unstudied population. METHODS: Three male kidney transplant recipients with biochemically, genetically, and histologically confirmed Fabry disease and documented Fabry myocardiopathy received the rh-alpha-Gal A, agalsidase beta, 1 mg/kg of body weight every 2 weeks by intravenous infusion and were monitored biochemically, clinically, and electrocardiographically and echocardiographically for 18 months. RESULTS: Patients showed biochemical, clinical/functional, and morphologic response to ERT. Plasma globotriaosylceramide decreased 23% to 50%. Extremity pain resolved within 2 months in the patient with this manifestation. On echocardiography, left ventricular mass, end diastolic diameter (EDD), and cardiac contractility, shown by ejection fraction (EF), improved in 2 of the 3 patients receiving essentially all planned infusions. EDD and EF remained basically stable, but cardiac morphologic abnormalities progressed in the other patient, who had a 5-month interruption in ERT after the initial month. Mild mitral insufficiency persisted in all patients, as did atrial fibrillation in the affected individual. After a combined total of 116 infusions, no treatment-related adverse event, intolerance, or seroconversion was seen. Renal function remained stable and the immunosuppression regimen unchanged in all patients. CONCLUSION: Our pilot study provides preliminary evidence that ERT with agalsidase beta, 1 mg/kg every 2 weeks, is safe and often effective against extra-renal manifestations in kidney transplant patients with Fabry disease. Studies with longer courses of this and higher doses of ERT are merited in this population.


Subject(s)
Fabry Disease/drug therapy , Fabry Disease/surgery , Isoenzymes/therapeutic use , Kidney Transplantation , alpha-Galactosidase/therapeutic use , Adult , Atrial Fibrillation/etiology , Atrial Fibrillation/physiopathology , Echocardiography , Electrocardiography , Extremities , Fabry Disease/complications , Fabry Disease/diagnosis , Heart/drug effects , Heart/physiopathology , Humans , Male , Middle Aged , Mitral Valve Insufficiency/etiology , Mitral Valve Insufficiency/physiopathology , Pain/physiopathology , Pilot Projects , Recombinant Proteins/therapeutic use , Treatment Outcome , Trihexosylceramides/blood
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