Should eliglustat be first line therapy for patients with type 1 Gaucher disease? Definitions of safety and efficacy

Affiliations

¹ Gaucher Clinic, Shaare Zedek Medical Center, 12 Bayit Street, Jerusalem 91031, Israel.
² Genetics Services of Western Australia, 374 Bagot Rd, Subiaco, WA 6008, Australia.
³ Department of Clinical Haematology, Royal Melbourne Hospital and Victorian Comprehensive Cancer Centre, 300 Grattan St, Parkville, VIC 3050, Australia.. Electronic address: jeff.szer@mh.org.au.

Review

Ari Zimran et al. Blood Cells Mol Dis. 2018 Feb.

. 2018 Feb:68:14-16.

doi: 10.1016/j.bcmd.2017.09.003. Epub 2017 Sep 14.

¹ Gaucher Clinic, Shaare Zedek Medical Center, 12 Bayit Street, Jerusalem 91031, Israel.
² Genetics Services of Western Australia, 374 Bagot Rd, Subiaco, WA 6008, Australia.
³ Department of Clinical Haematology, Royal Melbourne Hospital and Victorian Comprehensive Cancer Centre, 300 Grattan St, Parkville, VIC 3050, Australia.. Electronic address: jeff.szer@mh.org.au.

No abstract available

Keywords: Clinical trial design; Eliglustat; Enzyme replacement; Gaucher; Substrate reduction.

Comment in

Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.
Mistry PK, Balwani M, Baris HN, Turkia HB, Burrow TA, Charrow J, Cox GF, Danda S, Dragosky M, Drelichman G, El-Beshlawy A, Fraga C, Freisens S, Gaemers S, Hadjiev E, Kishnani PS, Lukina E, Maison-Blanche P, Martins AM, Pastores G, Petakov M, Peterschmitt MJ, Rosenbaum H, Rosenbloom B, Underhill LH, Cox TM. Mistry PK, et al. Blood Cells Mol Dis. 2018 Jul;71:71-74. doi: 10.1016/j.bcmd.2018.04.001. Epub 2018 Apr 9. Blood Cells Mol Dis. 2018. PMID: 29680197 No abstract available.