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SYSTEMATIC REVIEW article

Front. Cardiovasc. Med.
Sec. Cardiovascular Metabolism
Volume 11 - 2024 | doi: 10.3389/fcvm.2024.1415547

A Systematic Literature Review to Evaluate the Cardiac and Cerebrovascular Outcomes of Patients with Fabry Disease Treated with Agalsidase Beta

Provisionally accepted
Gavin Y Oudit Gavin Y Oudit 1*Pronabesh DasMahapatra Pronabesh DasMahapatra 2Nicole Lyn Nicole Lyn 2Florence R Wilson Florence R Wilson 3Adekemi Adeyemi Adekemi Adeyemi 3Chae Sung Lee Chae Sung Lee 2Ana Crespo Ana Crespo 4Mehdi Namdar Mehdi Namdar 5
  • 1 Division of Cardiology, Department of Medicine, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, Canada
  • 2 Sanofi, Cambridge, MA, United States
  • 3 Precision Medicine Group, Vancouver, Canada
  • 4 Sanofi S.p.A., Milan, Lombardy, Italy
  • 5 University Hospitals of Geneva, Geneva, Geneva, Switzerland

The final, formatted version of the article will be published soon.

    Background: Agalsidase 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.Methods: A 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.Results: Fifty-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/m 2 ; 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).Literature 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.

    Keywords: Fabry Disease, Agalsidase beta, genetic disorders, Cardiac, cardiovascular, Enzyme Replacement Therapy

    Received: 10 Apr 2024; Accepted: 28 Nov 2024.

    Copyright: © 2024 Oudit, DasMahapatra, Lyn, Wilson, Adeyemi, Lee, Crespo and Namdar. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Gavin Y Oudit, Division of Cardiology, Department of Medicine, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, Canada

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.