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Ibrahim J, Underhill LH, Taylor JS, et al. 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. Mol Genet Metab Rep. 2016;8:17-9doi: 10.1016/j.ymgmr.2016.06.003.
Ibrahim, J., Underhill, L. H., Taylor, J. S., Angell, J., & Peterschmitt, M. J. (2016). 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. Molecular genetics and metabolism reports, 817-9. https://doi.org/10.1016/j.ymgmr.2016.06.003
Ibrahim, Jennifer, et al. "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." Molecular genetics and metabolism reports vol. 8 (2016): 17-9. doi: https://doi.org/10.1016/j.ymgmr.2016.06.003
Ibrahim J, Underhill LH, Taylor JS, Angell J, Peterschmitt MJ. 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. Mol Genet Metab Rep. 2016 Jun 27;8:17-9. doi: 10.1016/j.ymgmr.2016.06.003. eCollection 2016 Sep. PMID: 27408819; PMCID: PMC4927653.
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Mol Genet Metab Rep. 2016 Jun 27;8:17-9. doi: 10.1016/j.ymgmr.2016.06.003. eCollection 2016 Sep.

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.

Molecular genetics and metabolism reports

Jennifer Ibrahim, Lisa H Underhill, John S Taylor, Jennifer Angell, M Judith Peterschmitt

Affiliations

  1. Sanofi Genzyme, 500 Kendall Street, Cambridge, MA 02142, USA.

PMID: 27408819 PMCID: PMC4927653 DOI: 10.1016/j.ymgmr.2016.06.003

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.

Keywords: Acid-β-glucosidase deficiency; Eliglustat; Enzyme replacement therapy; Gaucher disease type 1; Imiglucerase; Substrate reduction therapy

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