title: Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.
abstract: Eliglustat is a recently approved oral therapy in the United States and Europe for adults with Gaucher disease type 1 who are CYP2D6 extensive, intermediate, or poor metabolizers (> 90% of patients) that has been shown to decrease spleen and liver volume and increase hemoglobin concentrations and platelet counts in untreated adults with Gaucher disease type 1 and maintain these parameters in patients previously stabilized on enzyme replacement therapy. In a post-hoc analysis, we compared the results of eliglustat treatment in treatment-naïve patients in two clinical studies with the results of imiglucerase treatment among a cohort of treatment-naïve patients with comparable baseline hematologic and visceral parameters in the International Collaborative Gaucher Group Gaucher Registry. Organ volumes and hematologic parameters improved from baseline in both treatment groups, with a time course and degree of improvement in eliglustat-treated patients similar to imiglucerase-treated patients.
authors.affiliation: Sanofi Genzyme, 500 Kendall Street, Cambridge, MA 02142, USA.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Ibrahim
authors.affiliation: Sanofi Genzyme, 500 Kendall Street, Cambridge, MA 02142, USA.
authors.forename: Lisa H
authors.initials: LH
authors.lastname: Underhill
authors.affiliation: Sanofi Genzyme, 500 Kendall Street, Cambridge, MA 02142, USA.
authors.forename: John S
authors.initials: JS
authors.lastname: Taylor
authors.affiliation: Sanofi Genzyme, 500 Kendall Street, Cambridge, MA 02142, USA.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Angell
authors.affiliation: Sanofi Genzyme, 500 Kendall Street, Cambridge, MA 02142, USA.
authors.forename: M Judith
authors.initials: MJ
authors.lastname: Peterschmitt
keywords: Acid-β-glucosidase deficiency; Eliglustat; Enzyme replacement therapy; Gaucher disease type 1; Imiglucerase; Substrate reduction therapy