Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis
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...
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| Format: | Article |
| Language: | English |
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Wiley
2020-06-01
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| Series: | ESC Heart Failure |
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| Online Access: | https://doi.org/10.1002/ehf2.12647 |
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| author | Christoph Wanner Ulla Feldt‐Rasmussen Ana Jovanovic Aleš Linhart Meng Yang Elvira Ponce Eva Brand Dominique P. Germain Derralynn A. Hughes John L. Jefferies Ana Maria Martins Albina Nowak Bojan Vujkovac Frank Weidemann Michael L. West Alberto Ortiz |
| author_facet | Christoph Wanner Ulla Feldt‐Rasmussen Ana Jovanovic Aleš Linhart Meng Yang Elvira Ponce Eva Brand Dominique P. Germain Derralynn A. Hughes John L. Jefferies Ana Maria Martins Albina Nowak Bojan Vujkovac Frank Weidemann Michael L. West Alberto Ortiz |
| author_sort | Christoph Wanner |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-d245bf80912e439eb9ef9a86af5c8b32 |
| institution | Kabale University |
| issn | 2055-5822 |
| language | English |
| publishDate | 2020-06-01 |
| publisher | Wiley |
| record_format | Article |
| series | ESC Heart Failure |
| spelling | doaj-art-d245bf80912e439eb9ef9a86af5c8b322025-02-03T10:25:46ZengWileyESC Heart Failure2055-58222020-06-017382583410.1002/ehf2.12647Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysisChristoph Wanner0Ulla Feldt‐Rasmussen1Ana Jovanovic2Aleš Linhart3Meng Yang4Elvira Ponce5Eva Brand6Dominique P. Germain7Derralynn A. Hughes8John L. Jefferies9Ana Maria Martins10Albina Nowak11Bojan Vujkovac12Frank Weidemann13Michael L. West14Alberto Ortiz15Department of Medicine, Division of Nephrology University Hospital Würzburg Würzburg GermanyDepartment of Medical Endocrinology Rigshospitalet, Copenhagen University Hospital Copenhagen DenmarkThe Mark Holland Unit, Department of Endocrinology and Metabolic Medicine Salford Royal NHS Foundation Trust Salford UK2nd Department of Medicine, Department of Cardiovascular Medicine, First Faculty of Medicine Charles University, General University Hospital Prague Czech RepublicEpidemiology & Biostatistics Sanofi Genzyme Cambridge MA USAGlobal Medical Affairs Rare Diseases Sanofi Genzyme Cambridge MA USAInternal Medicine D, Department of Nephrology, Hypertension and Rheumatology University Hospital Münster Münster GermanyFrench Referral Center for Fabry Disease, Division of Medical Genetics and INSERM U1179 University of Versailles, Paris‐Saclay University Montigny FranceLysosomal Storage Disorder Unit Royal Free London NHS Foundation Trust and, University College London London UKDepartment of Medicine, Division of Cardiovascular Diseases University of Tennessee Health Science Center Memphis TN USADepartment of Paediatrics Federal University of São Paulo São Paulo BrazilDepartment of Internal Medicine University Hospital of Zürich, University of Zürich Zürich SwitzerlandDepartment of Internal Medicine General Hospital Slovenj Gradec Slovenj Gradec SloveniaMedical Clinic I, Klinikum Vest Knappschaftskrankenhaus Recklinghausen GermanyDepartment of Medicine, Division of Nephrology Dalhousie University Halifax Nova Scotia CanadaUnidad de Dialisis IIS‐Fundación Jiménez Díaz, UAM, IRSIN, REDINREN Madrid SpainAbstract 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.https://doi.org/10.1002/ehf2.12647Agalsidase betaEnzyme replacement therapyFabry diseaseCardiomyopathyKidney functionFemale patients |
| spellingShingle | Christoph Wanner Ulla Feldt‐Rasmussen Ana Jovanovic Aleš Linhart Meng Yang Elvira Ponce Eva Brand Dominique P. Germain Derralynn A. Hughes John L. Jefferies Ana Maria Martins Albina Nowak Bojan Vujkovac Frank Weidemann Michael L. West Alberto Ortiz Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis ESC Heart Failure Agalsidase beta Enzyme replacement therapy Fabry disease Cardiomyopathy Kidney function Female patients |
| title | Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis |
| title_full | Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis |
| title_fullStr | Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis |
| title_full_unstemmed | Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis |
| title_short | Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis |
| title_sort | cardiomyopathy and kidney function in agalsidase beta treated female fabry patients a pre treatment vs post treatment analysis |
| topic | Agalsidase beta Enzyme replacement therapy Fabry disease Cardiomyopathy Kidney function Female patients |
| url | https://doi.org/10.1002/ehf2.12647 |
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