Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
| Autor: | Bichet, Daniel G., Torra Balcells, Roser, Wallace, Eric, Hughes, Derralynn A, Giugliani, Roberto, Skuban, Nina, Krusinska, Eva, Feldt-Rasmussen, Ulla, Schiffmann, Raphael, Nicholls, Kathy, Universitat Autònoma de Barcelona |
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| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
Medicine (General)
Fenótipo α-Gal A α-galactosidase A Chaperone urologic and male genital diseases Doença de Fabry chemistry.chemical_compound Endocrinology Gb globotriaosylceramide Migalastat Biology (General) RI renin inhibitor biology Enzyme replacement therapy Egfr estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation medicine.anatomical_structure LVMi left ventricular mass index GLP-HEK Good Laboratory Practice-validated human embryonic kidney Q1 quartile 1 Q3 quartile 3 Corrigendum Research Paper medicine.medical_specialty Classic phenotype Efficacy QH301-705.5 Globotriaosylceramide Urology Renal function R5-920 White blood cell Genetics medicine Molecular Biology Fabry disease Alpha-galactosidase business.industry eGFRCKD-EPI estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation Gb3 globotriaosylceramide medicine.disease Testes de função renal Resultado do tratamento chemistry biology.protein Eficácia business Kidney disease |
| Zdroj: | Mol Genet Metab Rep Molecular Genetics and Metabolism Reports r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau instname Molecular Genetics and Metabolism Reports, Vol 28, Iss, Pp 100786-(2021) Repositório Institucional da UFRGS Universidade Federal do Rio Grande do Sul (UFRGS) instacron:UFRGS Dipòsit Digital de Documents de la UAB Universitat Autònoma de Barcelona Bichet, D G, Torra, R, Wallace, E, Hughes, D, Giugliani, R, Skuban, N, Krusinska, E, Feldt-Rasmussen, U, Schiffmann, R & Nicholls, K 2021, ' Long-term follow-up of renal function in patients treated with migalastat for Fabry disease ', Molecular Genetics and Metabolism Reports, vol. 28, 100786 . https://doi.org/10.1016/j.ymgmr.2021.100786 |
| ISSN: | 2214-4269 |
| DOI: | 10.1016/j.ymgmr.2021.100786 |
| Popis: | The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)–naive and ERT-experienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical trials and open-label extension studies was conducted to evaluate long-term changes in renal function in patients with Fabry disease and amenable GLA variants who were treated with migalastat for ≥2 years during these studies. The analysis included ERT-naive (n = 36 [23 females]; mean age 45 years; mean baseline estimated glomerular filtration rate (eGFR), 91.4 mL/min/mL/1.73 m2) and ERT-experienced (n = 42 [24 females]; mean age, 50 years; mean baseline eGFR, 89.2 mL/min/1.73m2) patients with amenable variants who received migalastat 123 mg every other day for ≥2 years. The annualized rate of change from baseline to last observation in estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation (eGFRCKD-EPI) was calculated by both simple linear regression and a random coefficient model. In ERT-naive patients, mean annualized rates of change from baseline in eGFRCKD-EPI were − 1.6 mL/min/1.73 m2 overall and − 1.8 mL/min/1.73 m2 and − 1.4 mL/min/1.73 m2 in male and female patients, respectively, as estimated by simple linear regression. In ERT-experienced patients, mean annualized rates of change from baseline in eGFRCKD-EPI were − 1.6 mL/min/1.73 m2 overall and − 2.6 mL/min/1.73 m2 and − 0.8 mL/min/1.73 m2 in male and female patients, respectively. Mean annualized rate of change in eGFRCKD-EPI in ERT-naive patients with the classic phenotype (defined by white blood cell alpha galactosidase A [α-Gal A] activity of Highlights • Migalastat-treated patients had stable renal function for up to 8.6 years. • Migalastat stabilized eGFR in ERT-naive and ERT-experienced males and females. • Migalastat stabilized eGFR in patients with advanced disease at baseline. • Mean annualized rate of eGFR change was −1.6 mL/min/1.73 m2 overall (simple regression). |
| Databáze: | OpenAIRE |
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