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Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients: a pre-treatment vs. post-treatment analysis
C. Wanner, U. Feldt-Rasmussen, A. Jovanovic, A. Linhart, M. Yang, E. Ponce, E. Brand, DP. Germain, DA. Hughes, JL. Jefferies, AM. Martins, A. Nowak, B. Vujkovac, F. Weidemann, ML. West, A. Ortiz
Jazyk angličtina Země Velká Británie
Typ dokumentu časopisecké články, práce podpořená grantem
Grantová podpora
-
Sanofi Genzyme - International
NLK
Directory of Open Access Journals
od 2014
Free Medical Journals
od 2014
PubMed Central
od 2015
Europe PubMed Central
od 2015
ProQuest Central
od 2014-09-01
Open Access Digital Library
od 2014-09-01
Open Access Digital Library
od 2014-01-01
Health & Medicine (ProQuest)
od 2014-09-01
Wiley-Blackwell Open Access Titles
od 2014
ROAD: Directory of Open Access Scholarly Resources
od 2014
PubMed
32100468
DOI
10.1002/ehf2.12647
Knihovny.cz E-zdroje
- MeSH
- alfa-galaktosidasa MeSH
- enzymová substituční terapie MeSH
- Fabryho nemoc * komplikace diagnóza farmakoterapie MeSH
- izoenzymy MeSH
- kardiomyopatie * MeSH
- ledviny MeSH
- lidé MeSH
- Check Tag
- lidé MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
AIMS: Long-term treatment effect studies in large female Fabry patient groups are challenging to design because of phenotype heterogeneity and lack of appropriate comparison groups, and have not been reported. We compared long-term cardiomyopathy and kidney function outcomes after agalsidase beta treatment with preceding treatment-naive outcomes. METHODS AND RESULTS: Self-controlled pretreatment and post-treatment comparison (piecewise mixed linear modelling) included Fabry female patients ≥18 years at treatment initiation who received agalsidase beta (0.9-1.1 mg/kg every other week) for ≥2 years, with ≥2 pretreatment and ≥2 post-treatment outcome measurements during 10-year follow-up. Left ventricular posterior wall thickness (LVPWT)/interventricular septal thickness (IVST) and estimated glomerular filtration rate (eGFR, Chronic Kidney Disease Epidemiology Collaboration creatinine equation) analyses included 42 and 86 patients, respectively, aged 50.0 and 46.3 years at treatment initiation, respectively. LVPWT and IVST increased pretreatment (follow-up 3.5 years) but stabilized during 3.6 years of treatment (LVPWT: n = 38, slope difference [95% confidence interval (CI)] = -0.41 [-0.68, -0.15] mm/year, Ppre-post difference <0.01; IVST: n = 38, slope difference = -0.32 [-0.67, 0.02] mm/year, Ppre-post difference = 0.07). These findings were not modified by renal involvement or antiproteinuric agent use. Compared with the treatment-naive period (follow-up 3.6 years), eGFR decline remained modest and stabilized within normal ranges during 4.1 years of treatment (slope difference, 95% CI: -0.13 [-1.15, 0.89] mL/min/1.73m2 /year, Ppre-post difference = 0.80). CONCLUSIONS: Cardiac hypertrophy, progressing during pretreatment follow-up, appeared to stabilize during sustained agalsidase beta treatment. eGFR decline remained within normal ranges. This suggests that treatment may prevent further Fabry-related progression of cardiomyopathy in female patients and maintain normal kidney function.
Department of Internal Medicine General Hospital Slovenj Gradec Slovenj Gradec Slovenia
Department of Medical Endocrinology Rigshospitalet Copenhagen University Hospital Copenhagen Denmark
Department of Medicine Division of Nephrology Dalhousie University Halifax Nova Scotia Canada
Department of Medicine Division of Nephrology University Hospital Würzburg Würzburg Germany
Department of Paediatrics Federal University of São Paulo São Paulo Brazil
Epidemiology and Biostatistics Sanofi Genzyme Cambridge MA USA
Global Medical Affairs Rare Diseases Sanofi Genzyme Cambridge MA USA
Medical Clinic 1 Klinikum Vest Knappschaftskrankenhaus Recklinghausen Germany
Unidad de Dialisis IIS Fundación Jiménez Díaz UAM IRSIN REDINREN Madrid Spain
Citace poskytuje Crossref.org
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- $a AIMS: Long-term treatment effect studies in large female Fabry patient groups are challenging to design because of phenotype heterogeneity and lack of appropriate comparison groups, and have not been reported. We compared long-term cardiomyopathy and kidney function outcomes after agalsidase beta treatment with preceding treatment-naive outcomes. METHODS AND RESULTS: Self-controlled pretreatment and post-treatment comparison (piecewise mixed linear modelling) included Fabry female patients ≥18 years at treatment initiation who received agalsidase beta (0.9-1.1 mg/kg every other week) for ≥2 years, with ≥2 pretreatment and ≥2 post-treatment outcome measurements during 10-year follow-up. Left ventricular posterior wall thickness (LVPWT)/interventricular septal thickness (IVST) and estimated glomerular filtration rate (eGFR, Chronic Kidney Disease Epidemiology Collaboration creatinine equation) analyses included 42 and 86 patients, respectively, aged 50.0 and 46.3 years at treatment initiation, respectively. LVPWT and IVST increased pretreatment (follow-up 3.5 years) but stabilized during 3.6 years of treatment (LVPWT: n = 38, slope difference [95% confidence interval (CI)] = -0.41 [-0.68, -0.15] mm/year, Ppre-post difference <0.01; IVST: n = 38, slope difference = -0.32 [-0.67, 0.02] mm/year, Ppre-post difference = 0.07). These findings were not modified by renal involvement or antiproteinuric agent use. Compared with the treatment-naive period (follow-up 3.6 years), eGFR decline remained modest and stabilized within normal ranges during 4.1 years of treatment (slope difference, 95% CI: -0.13 [-1.15, 0.89] mL/min/1.73m2 /year, Ppre-post difference = 0.80). CONCLUSIONS: Cardiac hypertrophy, progressing during pretreatment follow-up, appeared to stabilize during sustained agalsidase beta treatment. eGFR decline remained within normal ranges. This suggests that treatment may prevent further Fabry-related progression of cardiomyopathy in female patients and maintain normal kidney function.
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