Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3

Vedder, Anouk C.; Breunig, Frank; Donker‐Koopman, Wilma E.; Mills, Kevin; Young, Elisabeth; Winchester, Bryan; Berge, Ineke J. M. ten; Groener, J.E.M.; Aerts, Johannes M. F. G.; Wanner, Christoph; Hollak, Carla E. M.

doi:10.1016/j.ymgme.2008.03.003

Cited by 117 publications

(132 citation statements)

References 30 publications

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“…Hence, our observations support previous reports that suggested similar milligram-to-milligram in vitro biocheical potency and clinical effect. [34][35][36][37][38] Our study also supports previous clinical studies that have shown dose-dependent effects on various surrogate endpoints indicating a higher efficiency of agalsidase beta, 1 mg/kg every other week, than agalsidase alfa, 0.2 mg/kg every other week, 23,27,[37][38][39][40][41] but further studies are needed to clarify the issue of equipotency of the two currently available drugs.…”

Section: Discussionsupporting

confidence: 86%

“…Previous recommendations and recent studies mainly focus on adult patients and the need of treatment of patients with mild-to-moderate disease, 25,26 at least before clinically advanced disease, such as proteinuria .1 g/d and CKD stage III, is reached. 9,14,16,25,26 In addition, despite treatment with agalsidase alfa or agalsidase beta at the licensed doses, disease progresses in different organs in a subset of patients, 27 as was also observed in a 16-year-old boy in our study who had progression of cardiac disease (need of pacemaker) despite excellent renal effect (patient 5). This indicates that cardiac and kidney disease may respond differently to early ERT, and close collaboration between nephrologists and cardiologists is mandatory in the follow-up of these patients.…”

Section: Discussionsupporting

confidence: 73%

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Agalsidase Benefits Renal Histology in Young Patients with Fabry Disease

Tøndel

Bostad

Larsen

et al. 2013

Journal of the American Society of Nephrology

223

212

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The effect of early-onset enzyme replacement therapy on renal morphologic features in Fabry disease is largely unknown. Here, we evaluated the effect of 5 years of treatment with agalsidase alfa or agalsidase beta in 12 consecutive patients age 7-33 years (median age, 16.5 years). We performed renal biopsies at baseline and after 5 years of enzyme replacement therapy; 7 patients had additional biopsies after 1 and 3 years. After a median of 65 months, biopsy findings from all patients showed total clearance of glomerular endothelial and mesangial cell inclusions, and findings from 2 patients showed complete clearance of inclusions from epithelial cells of the distal tubule. The 4 patients who received the highest dose of agalsidase exhibited substantial clearance of podocyte inclusions, and the youngest patient had nearly complete clearance of these inclusions. Linear regression analysis showed a highly significant correlation between podocyte globotriaocylceramide clearance and cumulative agalsidase dose (r50.804; P50.002). Microalbuminuria normalized in five patients. In summary, long-term enzyme replacement therapy in young patients can result in complete globotriaocylceramide clearance of mesangial and glomerular endothelial cells across all dosage regimens, and clearance of podocyte inclusions is dosedependent.

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Section: Discussionsupporting

confidence: 86%

Section: Discussionsupporting

confidence: 73%

Agalsidase Benefits Renal Histology in Young Patients with Fabry Disease

Tøndel

Bostad

Larsen

et al. 2013

Journal of the American Society of Nephrology

223

212

View full text Add to dashboard Cite

show abstract

“…Most, but not all (Koskenvuo et al 2008;KovacevicPreradovic et al 2008), prior studies that investigated the effects of ERT (agalsidase beta or agalsidase alfa) on cardiac functional parameters reported improvements in cardiac parameters after initiation of ERT (Weidemann et al 2003;Beck et al 2004;Hughes et al 2008;Vedder et al 2008;Imbriaco et al 2009). Results of the current study were in agreement with the majority of these studies, i.e., LV mass and wall thickness decreased significantly from pre-ERT values after initiation of ERT, which in this case was with agalsidase beta (retrospective data; see Table 1).…”

Section: Discussionmentioning

confidence: 99%

Effects of Switching from Agalsidase Beta to Agalsidase Alfa in 10 Patients with Anderson-Fabry Disease

et al. 2012

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“…It can be used as an indicator of the metabolic effect of infused ␣-galactosidase A for ERT (17,18). As soon as 2 wk after initiation of ERT, urinary Gb 3 levels decrease significantly (5) but may increase again when anti-␣-galactosidase A antibodies develop (17,19). Thus, mea-suring this glycosphingolipid may indicate the presence of such antibodies and help monitor patients during toleration efforts.…”

Section: What Do We Know?mentioning

confidence: 99%

Biomarkers of Fabry Disease Nephropathy

Schiffmann¹,

Waldek²,

Benigni³

et al. 2010

Clinical Journal of the American Society of Nephrology

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It is suggested that biomarkers of renal complications of Fabry disease are likely to be useful for diagnosis and to follow the natural disease progression or the effect of specific therapeutic interventions. Traditionally, globotriaosylceramide (Gb 3 ) in urine has been used to evaluate the effect of specific therapy, such as enzyme replacement therapy (ERT). Although urinary Gb 3 decreases significantly with ERT, it has not yet been shown to be a valid surrogate marker in treatment trials. We propose a detailed study of the nature and origin of Gb 3 combined with a prospective collaborative trial that combines Gb 3 changes with the effect of ERT on clinical nephrological outcome measures. Existing biomarkers such as general proteinuria/ albuminuria or specific proteins such as N-acetyl-␤-D-glucosaminidase should be evaluated along with novel proteomic or metabolomic studies for biomarker discovery using mass spectrometry or nuclear magnetic resonance. Standard scoring of all pathologic aspects of kidney biopsies may also be a promising way to assess the effect of therapy.

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Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3

Cited by 117 publications

References 30 publications

Agalsidase Benefits Renal Histology in Young Patients with Fabry Disease

Agalsidase Benefits Renal Histology in Young Patients with Fabry Disease

Effects of Switching from Agalsidase Beta to Agalsidase Alfa in 10 Patients with Anderson-Fabry Disease

Biomarkers of Fabry Disease Nephropathy

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