التفاصيل البيبلوغرافية
| العنوان: |
Long-term follow-up of renal function in patients treated with migalastat for Fabry disease |
| المؤلفون: |
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 |
| سنة النشر: |
2021 |
| المجموعة: |
Universitat Autònoma de Barcelona: Dipòsit Digital de Documents de la UAB |
| مصطلحات موضوعية: |
Classic phenotype, Efficacy, Fabry disease, Chaperone, Migalastat, Renal function, α-Gal A, α-galactosidase A, Gb, globotriaosylceramide, Egfr, estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation, GLP-HEK, Good Laboratory Practice-validated human embryonic kidney, LVMi, left ventricular mass index, Q1, quartile 1, Q3, quartile 3, RI, renin inhibitor |
| الوصف: |
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 m 2) and ERT-experienced (n = 42 [24 females]; mean age, 50 years; mean baseline eGFR, 89.2 mL/min/1.73m 2) 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 (eGFR) was calculated by both simple linear regression and a random coefficient model. In ERT-naive patients, mean annualized rates of change from baseline in eGFR were − 1.6 mL/min/1.73 m 2 overall and − 1.8 mL/min/1.73 m 2 and − 1.4 mL/min/1.73 m 2 in male and female patients, respectively, as estimated by simple linear regression. In ERT-experienced patients, mean annualized rates of change from baseline in eGFR were − 1.6 mL/min/1.73 m 2 overall and − 2.6 mL/min/1.73 m 2 and − 0.8 mL/min/1.73 m 2 in male and female patients, respectively. Mean annualized rate of change in eGFR in ERT-naive patients with the classic phenotype (defined by white blood cell alpha galactosidase A [α-Gal A] activity of <3% of normal and multiorgan system involvement) was −1.7 mL/min/1.73 m 2. When calculated using the random coefficient model, which adjusted for sex, age, and baseline renal function, the annualized eGFR change was minimal (mean: −0.1 and 0.1 mL/min/1.73 m 2 in ERT-naive and ERT-experienced patients, respectively). In conclusion, patients with Fabry disease ... |
| نوع الوثيقة: |
article in journal/newspaper |
| وصف الملف: |
application/pdf |
| اللغة: |
English |
| Relation: |
Molecular Genetics and Metabolism Reports; Vol. 28 (august 2021); https://ddd.uab.cat/record/248892; urn:10.1016/j.ymgmr.2021.100786; urn:oai:ddd.uab.cat:248892; urn:pmcid:PMC8353473; urn:pmc-uid:8353473; urn:pmid:34401344; urn:oai:pubmedcentral.nih.gov:8353473; urn:oai:egreta.uab.cat:publications/5684c99f-0a5c-4d29-9323-9f9c80a720f9 |
| الاتاحة: |
https://ddd.uab.cat/record/248892 |
| Rights: |
open access ; Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades. ; https://creativecommons.org/licenses/by-nc-nd/4.0/ |
| رقم الانضمام: |
edsbas.C15FDC0B |
| قاعدة البيانات: |
BASE |