Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project.

ABSTRACT:

Background

The availability of multiple treatments for type 1 Gaucher disease increases the need for real-life studies to evaluate treatment efficacy and safety and provide clinicians with more information to choose the best personalized therapy for their patients.

Aims

To determine whether treatment with eliglustat produces, in adult GD1 patients, ans optimal response in daily clinical practice.

Methods

We designed a real-life study with 2 years of follow-up (TRAZELGA [GEE-ELI-2017-01]) to uniformly evaluate the response and adverse events to eliglustat treatment. This study, conducted in 30 patients across Spain and previously treated with other therapies, included the evaluation of safety and efficacy by assessing visceral enlargement, bone disease (DEXA and T and

SUBMITTER: Serrano-Gonzalo I 

PROVIDER: S-EPMC10722815 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature