Enzyme replacement therapy (ERT) with agalsidase alfa may slow deterioration of kidney function in men with Fabry disease, provided the treatment is started early, suggests a new study.
Without treatment, Fabry disease causes progressive loss of kidney function, eventually leading to end-stage renal disease.
AdvertisementAgalsidase alfa is an enzyme produced from human cells.
"The results provide further evidence that ERT with agalsidase alfa may slow the progression of kidney disease, provided that ERT is initiated early in the disease process," said Michael L. West, MD (Dalhousie University, Canada).
For the study, researchers pooled the results of three previous clinical trials of ERT with agalsidase alfa in 108 men with Fabry disease-a rare genetic disorder.
During treatment with an inactive placebo, kidney function declined rapidly.
By comparison, during treatment with agalsidase alfa (1 to 4.5 years), the rate of decline slowed considerably.
The response to treatment was not as good for patients with lower initial kidney function.
"This underlines the importance of prompt diagnosis and intervention in patients with Fabry disease," West said.
The study has been published in the online edition of the Journal of the American Society of Nephrology (JASN).