Enzyme replacement therapy for Fabry's disease.

摘要

A Mehta and colleagues claim that, for patients with Fabry's disease treated with agalsidase alfa, the "rate of fall of estimated glomerular filtration rate (GFR) is substantially slower" than for historical controls. We feel that this claim is not supported by the reported data.Rates of fall of GFR cannot be evaluated without considering pro-teinuria, which was not addressed in this study. This omission is surprising considering the emphasis given to this point in the natural history of Fabry's disease by Schiffmann and colleagues.