What question did this study set out to answer?

To identify determinants of renal disease progression in adults with Fabry disease receiving long-term agalsidase beta therapy.

July 29, 2011Nephrology Dialysis TransplantationOpen Access

Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation

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To identify determinants of renal disease progression in adults with Fabry disease receiving long-term agalsidase beta therapy.
Evaluated 151 men and 62 women from the Fabry Registry treated with agalsidase beta at an average dose of 1 mg/kg every 2 weeks for at least 2 years.
Categorized patients into quartiles based on slopes of estimated glomerular filtration rate (eGFR) and identified progression factors using multivariate logistic regression.
An averaged urinary protein:creatinine ratio (UP/Cr) ≥1 g/g was strongly associated with renal disease progression (OR 112, 95% CI 4–3109, P = 0.0054), with male eGFR slopes spanning from -0.1 (Quartile 1) to -6.7 mL/min/1.73m²/year (Quartile 4).
Longer delay from symptom onset to treatment initiation significantly increased the odds of renal progression (OR 19, 95% CI 2–184, P = 0.0098).
Women experiencing the most rapid decline (Quartile 4) had an average eGFR slope of -4.4 mL/min/1.73m²/year and the highest average UP/Cr of 1.8 g/g.

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Implication

Registry study demonstrates that early treatment and controlled proteinuria preserve kidney function in Fabry disease, indicating early intervention prevents organ decline.

Cite This Study

Warnock et al. (2011) studied this question.

synapsesocial.com/papers/6a8cf3b1ea63fdd6cbf5551d https://doi.org/10.1093/ndt/gfr420

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Implication

Registry study demonstrates that early treatment and controlled proteinuria preserve kidney function in Fabry disease, indicating early intervention prevents organ decline.

Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation

Key Points

Discussion

Implication

Cite This Study

Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation

Key Points

Discussion

Implication

Cite This Study

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