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Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation.
Nephrol Dial Transplant. 2012 Mar; 27(3):1042-9.ND

Abstract

BACKGROUND

The purpose of this study was to identify determinants of renal disease progression in adults with Fabry disease during treatment with agalsidase beta.

METHODS

Renal function was evaluated in 151 men and 62 women from the Fabry Registry who received agalsidase beta at an average dose of 1 mg/kg/2 weeks for at least 2 years. Patients were categorized into quartiles based on slopes of estimated glomerular filtration rate (eGFR) during treatment. Multivariate logistic regression analyses were used to identify factors associated with renal disease progression.

RESULTS

Men within the first quartile had a mean eGFR slope of -0.1 mL/min/1.73m(2)/year, whereas men with the most rapid renal disease progression (Quartile 4) had a mean eGFR slope of -6.7 mL/min/1.73m(2)/year. The risk factor most strongly associated with renal disease progression was averaged urinary protein:creatinine ratio (UP/Cr) ≥1 g/g (odds ratio 112, 95% confidence interval (95% CI) 4-3109, P = 0.0054). Longer time from symptom onset to treatment was also associated with renal disease progression (odds ratio 19, 95% CI 2-184, P = 0.0098). Women in Quartile 4 had the highest averaged UP/Cr (mean 1.8 g/g) and the most rapid renal disease progression: (mean slope -4.4 mL/min/1.73m(2)/year).

CONCLUSIONS

Adults with Fabry disease are at risk for progressive loss of eGFR despite enzyme replacement therapy, particularly if proteinuria is ≥1 g/g. Men with little urinary protein excretion and those who began receiving agalsidase beta sooner after the onset of symptoms had stable renal function. These findings suggest that early intervention may lead to optimal renal outcomes.

Authors+Show Affiliations

University of Alabama at Birmingham, Birmingham, AL, USA. dwarnock@uab.eduNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

21804088

Citation

Warnock, David G., et al. "Renal Outcomes of Agalsidase Beta Treatment for Fabry Disease: Role of Proteinuria and Timing of Treatment Initiation." Nephrology, Dialysis, Transplantation : Official Publication of the European Dialysis and Transplant Association - European Renal Association, vol. 27, no. 3, 2012, pp. 1042-9.
Warnock DG, Ortiz A, Mauer M, et al. Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation. Nephrol Dial Transplant. 2012;27(3):1042-9.
Warnock, D. G., Ortiz, A., Mauer, M., Linthorst, G. E., Oliveira, J. P., Serra, A. L., Maródi, L., Mignani, R., Vujkovac, B., Beitner-Johnson, D., Lemay, R., Cole, J. A., Svarstad, E., Waldek, S., Germain, D. P., & Wanner, C. (2012). Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation. Nephrology, Dialysis, Transplantation : Official Publication of the European Dialysis and Transplant Association - European Renal Association, 27(3), 1042-9. https://doi.org/10.1093/ndt/gfr420
Warnock DG, et al. Renal Outcomes of Agalsidase Beta Treatment for Fabry Disease: Role of Proteinuria and Timing of Treatment Initiation. Nephrol Dial Transplant. 2012;27(3):1042-9. PubMed PMID: 21804088.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation. AU - Warnock,David G, AU - Ortiz,Alberto, AU - Mauer,Michael, AU - Linthorst,Gabor E, AU - Oliveira,João P, AU - Serra,Andreas L, AU - Maródi,László, AU - Mignani,Renzo, AU - Vujkovac,Bojan, AU - Beitner-Johnson,Dana, AU - Lemay,Roberta, AU - Cole,J Alexander, AU - Svarstad,Einar, AU - Waldek,Stephen, AU - Germain,Dominique P, AU - Wanner,Christoph, AU - ,, Y1 - 2011/07/29/ PY - 2011/8/2/entrez PY - 2011/8/2/pubmed PY - 2012/9/15/medline SP - 1042 EP - 9 JF - Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association JO - Nephrol Dial Transplant VL - 27 IS - 3 N2 - BACKGROUND: The purpose of this study was to identify determinants of renal disease progression in adults with Fabry disease during treatment with agalsidase beta. METHODS: Renal function was evaluated in 151 men and 62 women from the Fabry Registry who received agalsidase beta at an average dose of 1 mg/kg/2 weeks for at least 2 years. Patients were categorized into quartiles based on slopes of estimated glomerular filtration rate (eGFR) during treatment. Multivariate logistic regression analyses were used to identify factors associated with renal disease progression. RESULTS: Men within the first quartile had a mean eGFR slope of -0.1 mL/min/1.73m(2)/year, whereas men with the most rapid renal disease progression (Quartile 4) had a mean eGFR slope of -6.7 mL/min/1.73m(2)/year. The risk factor most strongly associated with renal disease progression was averaged urinary protein:creatinine ratio (UP/Cr) ≥1 g/g (odds ratio 112, 95% confidence interval (95% CI) 4-3109, P = 0.0054). Longer time from symptom onset to treatment was also associated with renal disease progression (odds ratio 19, 95% CI 2-184, P = 0.0098). Women in Quartile 4 had the highest averaged UP/Cr (mean 1.8 g/g) and the most rapid renal disease progression: (mean slope -4.4 mL/min/1.73m(2)/year). CONCLUSIONS: Adults with Fabry disease are at risk for progressive loss of eGFR despite enzyme replacement therapy, particularly if proteinuria is ≥1 g/g. Men with little urinary protein excretion and those who began receiving agalsidase beta sooner after the onset of symptoms had stable renal function. These findings suggest that early intervention may lead to optimal renal outcomes. SN - 1460-2385 UR - https://www.unboundmedicine.com/medline/citation/21804088/Renal_outcomes_of_agalsidase_beta_treatment_for_Fabry_disease:_role_of_proteinuria_and_timing_of_treatment_initiation_ L2 - https://academic.oup.com/ndt/article-lookup/doi/10.1093/ndt/gfr420 DB - PRIME DP - Unbound Medicine ER -