A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta
BackgroundAgalsidase beta is used to treat Fabry disease (FD); however, data on cardiac and cerebrovascular outcomes with agalsidase beta treatment come from studies with limited numbers of patients.MethodsA systematic literature review of studies reporting on the efficacy and effectiveness of agals...
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Frontiers Media S.A.
2025-01-01
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| Series: | Frontiers in Cardiovascular Medicine |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fcvm.2024.1415547/full |
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| author | Gavin Y. Oudit Gavin Y. Oudit Pronabesh DasMahapatra Nicole Lyn Florence R. Wilson Adekemi Adeyemi Chae Sung Lee Ana Crespo Mehdi Namdar |
| author_facet | Gavin Y. Oudit Gavin Y. Oudit Pronabesh DasMahapatra Nicole Lyn Florence R. Wilson Adekemi Adeyemi Chae Sung Lee Ana Crespo Mehdi Namdar |
| author_sort | Gavin Y. Oudit |
| collection | DOAJ |
| description | BackgroundAgalsidase beta is used to treat Fabry disease (FD); however, data on cardiac and cerebrovascular outcomes with agalsidase beta treatment come from studies with limited numbers of patients.MethodsA systematic literature review of studies reporting on the efficacy and effectiveness of agalsidase beta in FD was conducted. Studies were identified in searches of MEDLINE, Embase, and the Cochrane Central Register of Controlled Trials from January 2000–June 2022. Outcomes of interest included cardiac structure and mass, cardiac events, and cerebrovascular events.ResultsFifty-two citations (41 studies) were included. Reductions in interventricular septal thickness (IVST) and/or left ventricular posterior wall thickness (LVPWT) were demonstrated in six studies (follow-up 1–6 years, n = 4 using echocardiography, n = 2 cardiac MRI). IVST ranged from 12.1–14.9 mm at baseline and 10.8–14.1 mm at follow-up (all p < 0.05). LVPWT ranged from 11.7–16.0 mm at baseline and 10.7–13.0 mm at follow-up (all p < 0.05). Significant reductions in cardiac mass were demonstrated after 1 year of treatment in a single-arm study using cardiac MRI [left ventricular mass (LVM) 193–178 g; LVM index 102–94 g/m2; both p < 0.05]. Rates of composite cardiac events (3.8%–24.0%; four studies, follow-up 2–10 years) and cerebrovascular events (0.0%–18.9%; 12 studies, follow-up 1–10 years) were numerically lower than rates for placebo (follow-up 3 years).ConclusionLiterature over the last 20 years indicates that agalsidase beta treatment may lead to stabilization or regression of cardiac structural thickness and mass, and reduction in cardiac and cerebrovascular events relative to placebo. |
| format | Article |
| id | doaj-art-6f9ffe238d224e5ba82fbf08d0a95cf0 |
| institution | Kabale University |
| issn | 2297-055X |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Frontiers Media S.A. |
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| series | Frontiers in Cardiovascular Medicine |
| spelling | doaj-art-6f9ffe238d224e5ba82fbf08d0a95cf02025-01-21T08:36:57ZengFrontiers Media S.A.Frontiers in Cardiovascular Medicine2297-055X2025-01-011110.3389/fcvm.2024.14155471415547A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase BetaGavin Y. Oudit0Gavin Y. Oudit1Pronabesh DasMahapatra2Nicole Lyn3Florence R. Wilson4Adekemi Adeyemi5Chae Sung Lee6Ana Crespo7Mehdi Namdar8Division of Cardiology, Department of Medicine, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, CanadaHeart Function Clinic, Mazankowski Alberta Heart Institute, University of Alberta, Edmonton, AB, CanadaSpecialty Care, Sanofi, Cambridge, MA, United StatesSpecialty Care, Sanofi, Cambridge, MA, United StatesPRECISIONheor, Vancouver, BC, CanadaPRECISIONheor, Vancouver, BC, CanadaSpecialty Care, Sanofi, Cambridge, MA, United StatesSpecialty Care, Sanofi, Milan, ItalyCardiology Division, Geneva University Hospitals, Geneva, SwitzerlandBackgroundAgalsidase beta is used to treat Fabry disease (FD); however, data on cardiac and cerebrovascular outcomes with agalsidase beta treatment come from studies with limited numbers of patients.MethodsA systematic literature review of studies reporting on the efficacy and effectiveness of agalsidase beta in FD was conducted. Studies were identified in searches of MEDLINE, Embase, and the Cochrane Central Register of Controlled Trials from January 2000–June 2022. Outcomes of interest included cardiac structure and mass, cardiac events, and cerebrovascular events.ResultsFifty-two citations (41 studies) were included. Reductions in interventricular septal thickness (IVST) and/or left ventricular posterior wall thickness (LVPWT) were demonstrated in six studies (follow-up 1–6 years, n = 4 using echocardiography, n = 2 cardiac MRI). IVST ranged from 12.1–14.9 mm at baseline and 10.8–14.1 mm at follow-up (all p < 0.05). LVPWT ranged from 11.7–16.0 mm at baseline and 10.7–13.0 mm at follow-up (all p < 0.05). Significant reductions in cardiac mass were demonstrated after 1 year of treatment in a single-arm study using cardiac MRI [left ventricular mass (LVM) 193–178 g; LVM index 102–94 g/m2; both p < 0.05]. Rates of composite cardiac events (3.8%–24.0%; four studies, follow-up 2–10 years) and cerebrovascular events (0.0%–18.9%; 12 studies, follow-up 1–10 years) were numerically lower than rates for placebo (follow-up 3 years).ConclusionLiterature over the last 20 years indicates that agalsidase beta treatment may lead to stabilization or regression of cardiac structural thickness and mass, and reduction in cardiac and cerebrovascular events relative to placebo.https://www.frontiersin.org/articles/10.3389/fcvm.2024.1415547/fullFabry diseaseagalsidase betagenetic disorderscardiaccardiovascularenzyme replacement therapy |
| spellingShingle | Gavin Y. Oudit Gavin Y. Oudit Pronabesh DasMahapatra Nicole Lyn Florence R. Wilson Adekemi Adeyemi Chae Sung Lee Ana Crespo Mehdi Namdar A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta Frontiers in Cardiovascular Medicine Fabry disease agalsidase beta genetic disorders cardiac cardiovascular enzyme replacement therapy |
| title | A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta |
| title_full | A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta |
| title_fullStr | A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta |
| title_full_unstemmed | A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta |
| title_short | A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta |
| title_sort | systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with fabry disease treated with agalsidase beta |
| topic | Fabry disease agalsidase beta genetic disorders cardiac cardiovascular enzyme replacement therapy |
| url | https://www.frontiersin.org/articles/10.3389/fcvm.2024.1415547/full |
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