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Enzyme replacement therapy and renal function in 201 patients with Fabry disease.
Clin Nephrol. 2006 Aug; 66(2):77-84.CN

Abstract

AIM

Fabry disease is a rare lysosomal storage disorder caused by deficient activity of alpha-galactosidase A, resulting in progressive cellular accumulation of glycolipids, which may ultimately result in endstage renal disease. We examined the effects of enzyme replacement therapy (ERT) with Agalsidase-alpha 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-alpha 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-alpha is associated with decrease of serum creatinine and may prevent the deterioration of renal function in patients with Fabry disease.

Authors+Show Affiliations

Department of Nephrology, University of Mainz, Mainz, Germany.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

16939062

Citation

Schwarting, A, et al. "Enzyme Replacement Therapy and Renal Function in 201 Patients With Fabry Disease." Clinical Nephrology, vol. 66, no. 2, 2006, pp. 77-84.
Schwarting A, Dehout F, Feriozzi S, et al. Enzyme replacement therapy and renal function in 201 patients with Fabry disease. Clin Nephrol. 2006;66(2):77-84.
Schwarting, A., Dehout, F., Feriozzi, S., Beck, M., Mehta, A., & Sunder-Plassmann, G. (2006). Enzyme replacement therapy and renal function in 201 patients with Fabry disease. Clinical Nephrology, 66(2), 77-84.
Schwarting A, et al. Enzyme Replacement Therapy and Renal Function in 201 Patients With Fabry Disease. Clin Nephrol. 2006;66(2):77-84. PubMed PMID: 16939062.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Enzyme replacement therapy and renal function in 201 patients with Fabry disease. AU - Schwarting,A, AU - Dehout,F, AU - Feriozzi,S, AU - Beck,M, AU - Mehta,A, AU - Sunder-Plassmann,G, AU - ,, PY - 2006/8/31/pubmed PY - 2007/1/4/medline PY - 2006/8/31/entrez SP - 77 EP - 84 JF - Clinical nephrology JO - Clin Nephrol VL - 66 IS - 2 N2 - AIM: Fabry disease is a rare lysosomal storage disorder caused by deficient activity of alpha-galactosidase A, resulting in progressive cellular accumulation of glycolipids, which may ultimately result in endstage renal disease. We examined the effects of enzyme replacement therapy (ERT) with Agalsidase-alpha 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-alpha 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-alpha is associated with decrease of serum creatinine and may prevent the deterioration of renal function in patients with Fabry disease. SN - 0301-0430 UR - https://www.unboundmedicine.com/medline/citation/16939062/Enzyme_replacement_therapy_and_renal_function_in_201_patients_with_Fabry_disease_ L2 - http://babysfirsttest.org/newborn-screening/conditions/fabry-disease DB - PRIME DP - Unbound Medicine ER -