Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.

ABSTRACT:

Background

Eliglustat is a first-line oral treatment for adults with Gaucher disease type 1 who have an extensive, intermediate or poor CYP2D6 metabolizer phenotype (> 90% of patients). Whereas enzyme replacement therapy for Gaucher disease has been widely used for more than two decades, eliglustat has only been in commercial use since 2014. Clinicians and patients want to better understand which adverse events are most commonly associated with eliglustat, as well as their severity, frequency, and duration.

Methods

This pooled analysis of treatment-emergent adverse events combines data from four completed eliglustat clinical trials involving 393 Gaucher disease type 1 patients. It represents 1400 patient-years of eliglustat exposure and a mean treatment duration of 3.6 years

SUBMITTER: Peterschmitt MJ 

PROVIDER: S-EPMC6555985 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature