Abstract
Aim: Fabry disease is a rare lysosomal storage disorder caused by deficient activity of α-galactosidase A, resulting in progressive cellular accumulation of glycolipids, which may ultimately result in end-stage renal disease. We examined the effects of enzyme replacement therapy (ERT) with A galsidase-α on renal function using data from a large international database, the Fabry Outcome Survey (FOS). Methods: This analysis was based on 1,040 serum creatinine measurements in 201 patients with Fabry disease, aged 20 - 60 years, with serum creatinine concentrations of less than 2 mg/dl and duration of ERT of up to 4.7 years. Both pretreatment and treatment data were used to examine independent predictors of changes in serum creatinine. In a second approach longitudinal serum creatinine measurements from 1 year before treatment, at baseline and 1 and 2 years after the start of treatment were analyzed in 20 patients with chronic kidney disease (CKD) Stage 2 and 3. Results: We found an independent negative association between serum creatinine and time on Agalsidase-α treatment (p < 0.05). Renal function declined significantly (p < 0.05) in the year before treatment. After 1 year of treatment, however, the decline in estimated glomerular filtration rate had been halted, and renal function was preserved for up to 2 years. Conclusions: In conclusion, ERT with Agalsidase-α is associated with decrease of serum creatinine and may prevent the deterioration of renal function in patients with Fabry disease.
Original language | English (US) |
---|---|
Pages (from-to) | 77-84 |
Number of pages | 8 |
Journal | Clinical Nephrology |
Volume | 66 |
Issue number | 2 |
State | Published - Aug 2006 |
Externally published | Yes |
Keywords
- Chronic kidney disease
- Creatinine
- Enzyme replacement therapy
- Fabry disease
- Fabry Outcome Survery
- Glomerular filtration rate
ASJC Scopus subject areas
- Nephrology