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Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients: a pre-treatment vs. post-treatment analysis

Wanner, C; Feldt-Rasmussen, U; Jovanovic, A; Linhart, A; Yang, M; Ponce, E; Brand, E; ... Ortiz, A; + view all (2020) Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients: a pre-treatment vs. post-treatment analysis. ESC Heart Failure , 7 (3) pp. 825-834. 10.1002/ehf2.12647. Green open access

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Abstract

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.

Type: Article
Title: Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients: a pre-treatment vs. post-treatment analysis
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1002/ehf2.12647
Publisher version: https://doi.org/10.1002/ehf2.12647
Language: English
Additional information: This is an open access article under the terms of the Creative Commons Attribution‐NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. http://creativecommons.org/licenses/by-nc/4.0/
Keywords: Agalsidase beta; Enzyme replacement therapy; Fabry disease; Cardiomyopathy; Kidney function; Female patient
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: https://discovery.ucl.ac.uk/id/eprint/10093287
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