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Taliglucerase alfa leads to favorable bone marrow responses in patients with type I Gaucher disease
- Source :
- Blood cells, molecules & diseases, 50(3), 206-211. Academic Press Inc.
- Publication Year :
- 2013
- Publisher :
- Elsevier BV, 2013.
-
Abstract
- 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 (
- Subjects :
- Adult
Male
medicine.medical_specialty
Bone disease
Adipose tissue
Gastroenterology
Antibodies
Young Adult
03 medical and health sciences
0302 clinical medicine
Bone Marrow
Internal medicine
medicine
Humans
Enzyme Replacement Therapy
Young adult
Molecular Biology
Aged
030304 developmental biology
0303 health sciences
Gaucher Disease
business.industry
Cell Biology
Hematology
Enzyme replacement therapy
Middle Aged
medicine.disease
Antibodies, Neutralizing
Taliglucerase alfa
3. Good health
Surgery
Glucosylceramidase
Treatment Outcome
medicine.anatomical_structure
Adipose Tissue
030220 oncology & carcinogenesis
Molecular Medicine
Female
Bone marrow
Complication
business
Subjects
Details
- ISSN :
- 10799796
- Volume :
- 50
- Database :
- OpenAIRE
- Journal :
- Blood Cells, Molecules, and Diseases
- Accession number :
- edsair.doi.dedup.....86167d66aec94ebdaddd9f5d0d730e5e