Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
Authors Serratrice C, Carballo S, Serratrice J, Stirnemann J
Received 27 June 2016
Accepted for publication 10 September 2016
Published 14 October 2016 Volume 2016:11 Pages 37—47
Checked for plagiarism Yes
Review by Single-blind
Peer reviewers approved by Dr Amy Norman
Peer reviewer comments 3
Editor who approved publication: Professor Garry Walsh
Christine Serratrice,1 Sebastian Carballo,2 Jacques Serratrice,2 Jérome Stirnemann2
1Department of Internal Medicine and Rehabilitation, Geneva University Hospital, Thonex, Switzerland; 2Department of General Internal Medicine, Geneva University Hospital, Geneva, Switzerland
Introduction: Gaucher disease is the first lysosomal disease to benefit from enzyme replacement therapy, thus serving as model for numerous other lysosomal diseases. Alglucerase was the first glucocerebrosidase purified from placental extracts, and this was then replaced by imiglucerase – a Chinese hamster ovary cell-derived glucocerebrosidase.
Aim: The aim was to review the evidence underlying the use of imiglucerase in Gaucher disease type 1
Evidence review: Data from clinical trials and Gaucher Registries were analyzed.
Conclusion: Imiglucerase has been prescribed and found to have an excellent efficacy and safety profile. We report herein the evidence-based data published for 26 years justifying the use of imiglucerase.
Keywords: Gaucher disease, lysosomal disease, imiglucerase, treatment, therapeutic goals, safety
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