Elevated Endothelial Microparticles in Fabry Children Decreased After Enzyme Replacement Therapy

Abstract

To the Editor: Fabry disease (FD), an X-linked metabolic disorder, is caused by insufficient activity of the lysosomal enzyme alpha galactosidase A (α-gal A). This results in impaired catabolism of globotriaosylceramide ([Gb3] also called ceramidetrihexoside) and its subsequent accumulation leading to endothelial dysfunction and other anomalies.1 At present, enzyme replacement therapy (ERT) is the only available therapeutic approach for patients with Fabry disease. However, in adult patients with advanced FD, ERT has its limitations.2 Therefore, studies have been initiated to analyze the efficacy of ERT started at an earlier stage of FD.3,4 In general, the identification of markers that could serve as indicators of disease severity, prognosis, and possible effects of specific therapies is highly desirable in genetic disorders. Because it has been documented that elevated counts of endothelial cell derived membrane microparticles can be present in the circulation of patients with a variety of diseases with a vascular injury component, we explored whether microparticles (MPs) could serve as a potential marker.5 We investigated the …