Assessment of plasma lyso-Gb₃ for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease.

ABSTRACT:

Purpose

To assess the utility of globotriaosylsphingosine (lyso-Gb₃) for clinical monitoring of treatment response in patients with Fabry disease receiving migalastat.

Methods

A post hoc analysis evaluated data from 97 treatment-naive and enzyme replacement therapy (ERT)-experienced patients with migalastat-amenable GLA variants from FACETS (NCT00925301) and ATTRACT (NCT01218659) and subsequent open-label extension studies. The relationship between plasma lyso-Gb₃ and measures of Fabry disease progression (left ventricular mass index [LVMi], estimated glomerular filtration rate [eGFR], and pain) and the relationship between lyso-Gb₃ and incidence of Fabry-associated clinical events (FACEs) were assessed in both groups. The relationship betwee

SUBMITTER: Bichet DG 

PROVIDER: S-EPMC7790748 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature