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Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients: a pre-treatment vs. post-treatment analysis
- Source :
- ESC Heart Failure, ESC Heart Failure, Wiley, 2020, 7 (3), pp.825-834. ⟨10.1002/ehf2.12647⟩, ESC Heart Failure, Vol 7, Iss 3, Pp 825-834 (2020), Wanner, C, Feldt-Rasmussen, U, Jovanovic, A, Linhart, A, Yang, M, Ponce, E, Brand, E, Germain, D P, Hughes, D A, Jefferies, J L, Martins, A M, Nowak, A, Vujkovac, B, Weidemann, F, West, M L & Ortiz, A 2020, ' Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients : a pre-treatment vs. post-treatment analysis ', ESC heart failure, vol. 7, no. 3, pp. 825-834 . https://doi.org/10.1002/ehf2.12647
- Publication Year :
- 2020
- Publisher :
- HAL CCSD, 2020.
-
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 pre–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.
- Subjects :
- Male
lcsh:Diseases of the circulatory (Cardiovascular) system
Adolescent
Cardiomyopathy
10265 Clinic for Endocrinology and Diabetology
Agalsidase beta
610 Medicine & health
Kidney
2705 Cardiology and Cardiovascular Medicine
Kidney function
Original Research Articles
Humans
ddc:610
Original Research Article
Language
Fabry disease
Myocardium
Female patients
Isoenzymes
lcsh:RC666-701
alpha-Galactosidase
Enzyme replacement therapy
Female
[SDV.IB]Life Sciences [q-bio]/Bioengineering
Glycolipids
Cardiomyopathies
Subjects
Details
- Language :
- English
- ISSN :
- 20555822
- Database :
- OpenAIRE
- Journal :
- ESC Heart Failure, ESC Heart Failure, Wiley, 2020, 7 (3), pp.825-834. ⟨10.1002/ehf2.12647⟩, ESC Heart Failure, Vol 7, Iss 3, Pp 825-834 (2020), Wanner, C, Feldt-Rasmussen, U, Jovanovic, A, Linhart, A, Yang, M, Ponce, E, Brand, E, Germain, D P, Hughes, D A, Jefferies, J L, Martins, A M, Nowak, A, Vujkovac, B, Weidemann, F, West, M L & Ortiz, A 2020, ' Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients : a pre-treatment vs. post-treatment analysis ', ESC heart failure, vol. 7, no. 3, pp. 825-834 . https://doi.org/10.1002/ehf2.12647
- Accession number :
- edsair.pmid.dedup....316920ab44a4e1a97d804f8d38c614c0