Efficacy and safety of long-term enzyme replacement therapy with agalsidase alfa or agalsidase beta in adult patients with Fabry disease

Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis.

Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry disease in patients enrolled in the Fabry Outcome Survey (FOS) were compared with published findings for untreated patients with Fabry disease. Data were extracted from FOS, a Shire-sponsored database, for comparison with data from three published studies. Outcomes evaluated were the annualized ra...

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease

BACKGROUND Fabry disease results from deficient α-galactosidase A activity and globotriaosylceramide accumulation causing renal insufficiency, strokes, hypertrophic cardiomyopathy and early demise. We assessed the 10-year outcome of recombinant α-galactosidase A therapy. METHODS The outcomes (severe clinical events, renal function, cardiac structure) of 52/58 patients with classic Fabry disea...

Enzyme replacement therapy with agalsidase alfa in children with Fabry disease.

AIM To assess the effects of enzyme replacement therapy (ERT) in children with Fabry disease. METHODS Safety and efficacy of ERT with agalsidase alfa, 0.2 mg/kg infused over 40 minutes every 2 weeks for 23 weeks, were studied in a multicentre open-label trial in nine boys and four girls. Median age at the start of the study was 11.0 years (range 3.5-18 years). RESULTS Fifty-four adverse eve...

Agalsidase alfa: enzyme therapy for Anderson–Fabry disease

Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events

This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. Cardiac events occurred at younger ages than cerebrovascular or renal events, cerebrovascular events were more frequent in females than males, and m...