Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy

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...

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Autores principales: Serratrice, Christine, Carballo, Sebastian, Serratrice, Jacques, Stirnemann, Jérome
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2016
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5072572/
https://www.ncbi.nlm.nih.gov/pubmed/27790078
http://dx.doi.org/10.2147/CE.S93717
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author Serratrice, Christine
Carballo, Sebastian
Serratrice, Jacques
Stirnemann, Jérome
author_facet Serratrice, Christine
Carballo, Sebastian
Serratrice, Jacques
Stirnemann, Jérome
author_sort Serratrice, Christine
collection PubMed
description 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.
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spelling pubmed-50725722016-10-27 Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy Serratrice, Christine Carballo, Sebastian Serratrice, Jacques Stirnemann, Jérome Core Evid Review 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. Dove Medical Press 2016-10-14 /pmc/articles/PMC5072572/ /pubmed/27790078 http://dx.doi.org/10.2147/CE.S93717 Text en © 2016 Serratrice et al. This work is published and licensed by Dove Medical Press Limited The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License (http://creativecommons.org/licenses/by-nc/3.0/). By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed.
spellingShingle Review
Serratrice, Christine
Carballo, Sebastian
Serratrice, Jacques
Stirnemann, Jérome
Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
title Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
title_full Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
title_fullStr Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
title_full_unstemmed Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
title_short Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
title_sort imiglucerase in the management of gaucher disease type 1: an evidence-based review of its place in therapy
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5072572/
https://www.ncbi.nlm.nih.gov/pubmed/27790078
http://dx.doi.org/10.2147/CE.S93717
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