New insights from the application of the FAbry STabilization indEX in a large population of Fabry cases.

ABSTRACT:

Background

The FAbry STabilization indEX (FASTEX) is an innovative index allowing the assessment of clinical stability over time in Fabry disease patients. This index was developed in a population of 28 male patients with the classical form of Fabry disease.

Objectives

The aim of the study was to test the accuracy of the FASTEX in evaluating Fabry disease stability in 132 male and female patients with classical and non-classical Fabry disease from nine Italian centres and it also aimed to define the sensitivity and specificity of this new tool. In particular, we aimed to investigate the correlation between the FASTEX and clinical judgement in a large-scale cohort of the study population.

Methods

Statistical methods applied to this investigation included the calculatio

SUBMITTER: Mignani R 

PROVIDER: S-EPMC6425459 | biostudies-literature | 2019 Feb

REPOSITORIES: biostudies-literature