Do clinical guidelines facilitate or impede drivers of treatment in Fabry disease?

ABSTRACT:

Background

Variable disease progression confounds accurate prognosis in Fabry disease. Evidence supports the long-term benefit of early intervention with disease-specific therapy, but current guidelines recommend treatment initiation based on signs that may present too late to avoid irreversible organ damage. Findings from the 'PRoposing Early Disease Indicators for Clinical Tracking in Fabry Disease' (PREDICT-FD) initiative included expert consensus on 27 early indicators of disease progression in Fabry disease and on drivers of and barriers to treatment initiation in Fabry disease. Here, we compared the PREDICT-FD indicators with guidance from the European Fabry Working Group and various national guidelines to identify differences in signs supporting treatment initiation and how

SUBMITTER: Hughes DA 

PROVIDER: S-EPMC8822651 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature