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Background: Fabry disease is a rare X-linked lysosomal storage disease caused by defective activity of the lysosomal enzyme α-galactosidase A. The objective was to study the baseline characteristics of Fabry patients in Sweden, Furthermore, to study the effect of 4 years of ERT (enzyme replacement therapy) in an open study of a subcohort of Fabry men with no or mild proteinuria (<1 gram/day). Patients and methods: Thirty-four Swedish Fabry patients were enrolled in the baseline study during a 10 year period. We present baseline data for both men and women. The effect of 4 years of ERT was studied in an open study of a subcohort of 12 Fabry men with no or mild proteinuria. In the first 12-18 months this subcohort received the licensed dose of agalsidase alfa (Replagal ® ) 0.2 mg/kg body weight every other week and the men were then switched to a reduced dose of agalsidase beta (Fabrazyme ®), 0.5 mg/kg body weight every other week. Results: Swedish Fabry patients showed a similar prevalence of classical symptoms compared to Fabry patients from other countries, except for a lower prevalence of renal complications in the Swedish women. The 4 years of ERT of men with no or mild proteinuria resulted in maintained kidney function. In addition, there was no increase in left ventricular mass. Conclusion: The study indicates that ERT stabilizes kidney function in Fabry patients with no or mild proteinuria and has a cardioprotective effect.

Adult Fabry patients in Sweden - Baseline data and Outcome of Four-Year Enzyme Replacement Treatment in a Male Cohort without Proteinuria