Taliglucerase alfa leads to favorable bone marrow responses in patients with type I Gaucher disease

Taliglucerase alfa (Protalix Biotherapeutics, Israel) is a carrot-cell-expressed recombinant human beta-glucocerebrosidase recently approved in the United States for the treatment of type 1 Gaucher disease (GD). As bone disease is one of the most debilitating features of GD, quantification of bone m...

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Bibliographic Details
Published in:Blood cells, molecules, & diseases molecules, & diseases, 2013-03, Vol.50 (3), p.206-211
Main Authors: van Dussen, L., Zimran, A., Akkerman, E.M., Aerts, J.M.F.G., Petakov, M., Elstein, D., Rosenbaum, H., Aviezer, D., Brill-Almon, E., Chertkoff, R., Maas, M., Hollak, C.E.M.
Format: Article
Language:English
Subjects:
Adipose Tissue - metabolism
Adult
Aged
Antibodies - immunology
Antibodies, Neutralizing - immunology
Bone Marrow - drug effects
Bone Marrow - metabolism
Bone marrow fat fraction
Enzyme Replacement Therapy - adverse effects
Female
Gaucher disease
Gaucher Disease - drug therapy
Glucosylceramidase - administration & dosage
Glucosylceramidase - immunology
Glucosylceramidase - therapeutic use
Humans
Male
Middle Aged
Quantitative chemical shift imaging
Taliglucerase alfa
Treatment Outcome
Young Adult
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Summary:Taliglucerase alfa (Protalix Biotherapeutics, Israel) is a carrot-cell-expressed recombinant human beta-glucocerebrosidase recently approved in the United States for the treatment of type 1 Gaucher disease (GD). As bone disease is one of the most debilitating features of GD, quantification of bone marrow involvement is important for monitoring the response to treatment. Therefore, bone marrow fat fraction (Ff) measured by quantitative chemical shift imaging (QCSI) was included as exploratory parameter to evaluate bone marrow response in treatment naïve GD patients participating in a double-blind, randomized phase III study. Eight GD patients with intact spleens were treated with 30 or 60U/kg biweekly. Ff results were compared to outcomes in 15 untreated Dutch GD patients with a follow-up interval of 1year. Five taliglucerase alfa treated patients had a Ff below the threshold that relates to complication risk (
ISSN:1079-9796
1096-0961
DOI:10.1016/j.bcmd.2012.11.001