Management goals of type 1 Gaucher disease in South Africa: An expert Delphi consensus document on good clinical practice.

ABSTRACT:

Background

Gaucher disease is a rare autosomal recessive glycosphingolipid storage disease that ultimately leads to reduced life expectancy. Management of Gaucher disease is challenging due to its wide genotypic and phenotypic variability and changing clinical manifestations due to effective treatment. Deliberation between experts is essential to discuss daily clinical practice and identify controversies regarding the management of Gaucher disease. The usefulness of methods like Delphi surveys is suitable for setting up consensus recommendations for different clinical scenarios.

Objectives

The goal of this study was to develop an expert consensus document for the management of type 1 Gaucher disease by local experts.

Methods

A modified e-Delphi was carried out to deve

SUBMITTER: Louw VJ 

PROVIDER: S-EPMC10443848 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature