Taliglucerase alfa leads to favorable bone marrow responses in patients with type I Gaucher disease
| Autor: | Mario Maas, Raul Chertkoff, Hanna Rosenbaum, Johannes M. F. G. Aerts, Ari Zimran, Milan Petakov, Carla E. M. Hollak, Deborah Elstein, Einat Brill-Almon, David Aviezer, Erik M. Akkerman, L. van Dussen |
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| Přispěvatelé: | Other departments, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, ANS - Amsterdam Neuroscience, Radiology and Nuclear Medicine, ACS - Amsterdam Cardiovascular Sciences, Medical Biochemistry, AMS - Amsterdam Movement Sciences, Endocrinology |
| Rok vydání: | 2013 |
| Předmět: |
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 |
| Zdroj: | Blood cells, molecules & diseases, 50(3), 206-211. Academic Press Inc. |
| ISSN: | 1079-9796 |
| Popis: | 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 ( |
| Databáze: | OpenAIRE |
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