Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study.

ABSTRACT: Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7

SUBMITTER: Yang A 

PROVIDER: S-EPMC10817061 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature