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The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy.
Clin J Am Soc Nephrol. 2012 Jan; 7(1):60-9.CJ

Abstract

BACKGROUND AND OBJECTIVES

Fabry disease is a rare X-linked disease with multisystemic manifestations. This study investigated the effectiveness of long-term enzyme replacement therapy with agalsidase alfa in Fabry nephropathy treatment.

DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS

In this observational study, data on patients receiving agalsidase alfa (0.2 mg/kg every other week) were extracted from the Fabry Outcome Survey, an international registry of patients with Fabry disease. Serum creatinine and estimated GFR (eGFR) at baseline and after ≥5 years of treatment were assessed; 24-hour urinary protein excretion and BP measurements were also reviewed. The eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration formula. Patients with an eGFR <30 ml/min per 1.73 m(2) were excluded.

RESULTS

Renal function was assessed in 208 patients (mean enzyme replacement therapy, 7.4 years; range, 5.0-11.2 years). Mean yearly change in eGFR was -2.2 ml/min per 1.73 m(2) in men and -0.7 ml/min per 1.73 m(2) in women (95% confidence limits, -2.8; -1.7 and -1.4; 0.0, respectively). Patients with 24-hour protein excretion >1 g/24 h had poorer renal function at baseline and follow-up compared with patients with protein excretion of 500-1000 mg/24 h or with proteinuria <500 mg/24 h. Renal function was worse in patients with baseline arterial hypertension, and there was a more rapid yearly decline compared with normotensive patients.

CONCLUSIONS

This study suggests that long-term agalsidase alfa therapy is able to stabilize the rate of Fabry nephropathy progression in women and is associated with a mild to moderate decline of renal function in men.

Authors+Show Affiliations

Belcolle Hospital, Nephrology and Dialysis, Strada Sammartinese snc, IT-01100 Viterbo, Italy. sandro.feriozzi@tiscali.itNo 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

22246281

Citation

Feriozzi, Sandro, et al. "The Effectiveness of Long-term Agalsidase Alfa Therapy in the Treatment of Fabry Nephropathy." Clinical Journal of the American Society of Nephrology : CJASN, vol. 7, no. 1, 2012, pp. 60-9.
Feriozzi S, Torras J, Cybulla M, et al. The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy. Clin J Am Soc Nephrol. 2012;7(1):60-9.
Feriozzi, S., Torras, J., Cybulla, M., Nicholls, K., Sunder-Plassmann, G., & West, M. (2012). The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy. Clinical Journal of the American Society of Nephrology : CJASN, 7(1), 60-9. https://doi.org/10.2215/CJN.03130411
Feriozzi S, et al. The Effectiveness of Long-term Agalsidase Alfa Therapy in the Treatment of Fabry Nephropathy. Clin J Am Soc Nephrol. 2012;7(1):60-9. PubMed PMID: 22246281.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy. AU - Feriozzi,Sandro, AU - Torras,Joan, AU - Cybulla,Markus, AU - Nicholls,Kathy, AU - Sunder-Plassmann,Gere, AU - West,Michael, AU - ,, PY - 2012/1/17/entrez PY - 2012/1/17/pubmed PY - 2012/5/23/medline SP - 60 EP - 9 JF - Clinical journal of the American Society of Nephrology : CJASN JO - Clin J Am Soc Nephrol VL - 7 IS - 1 N2 - BACKGROUND AND OBJECTIVES: Fabry disease is a rare X-linked disease with multisystemic manifestations. This study investigated the effectiveness of long-term enzyme replacement therapy with agalsidase alfa in Fabry nephropathy treatment. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: In this observational study, data on patients receiving agalsidase alfa (0.2 mg/kg every other week) were extracted from the Fabry Outcome Survey, an international registry of patients with Fabry disease. Serum creatinine and estimated GFR (eGFR) at baseline and after ≥5 years of treatment were assessed; 24-hour urinary protein excretion and BP measurements were also reviewed. The eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration formula. Patients with an eGFR <30 ml/min per 1.73 m(2) were excluded. RESULTS: Renal function was assessed in 208 patients (mean enzyme replacement therapy, 7.4 years; range, 5.0-11.2 years). Mean yearly change in eGFR was -2.2 ml/min per 1.73 m(2) in men and -0.7 ml/min per 1.73 m(2) in women (95% confidence limits, -2.8; -1.7 and -1.4; 0.0, respectively). Patients with 24-hour protein excretion >1 g/24 h had poorer renal function at baseline and follow-up compared with patients with protein excretion of 500-1000 mg/24 h or with proteinuria <500 mg/24 h. Renal function was worse in patients with baseline arterial hypertension, and there was a more rapid yearly decline compared with normotensive patients. CONCLUSIONS: This study suggests that long-term agalsidase alfa therapy is able to stabilize the rate of Fabry nephropathy progression in women and is associated with a mild to moderate decline of renal function in men. SN - 1555-905X UR - https://www.unboundmedicine.com/medline/citation/22246281/The_effectiveness_of_long_term_agalsidase_alfa_therapy_in_the_treatment_of_Fabry_nephropathy_ L2 - https://cjasn.asnjournals.org/cgi/pmidlookup?view=long&amp;pmid=22246281 DB - PRIME DP - Unbound Medicine ER -