Taliglucerase Alfa Shows Positive Effect on Bone Marrow Infiltration of Gaucher Cells
In type 1 Gaucher disease, infiltration of bone marrow by Gaucher cells reduces the bone marrow fat fraction (FF) and increases the risk of skeletal manifestations. Preliminary data from a single-institution study found administration of taliglucerase alfa (Elelyso) enzyme replacement therapy (ERT) improved bone marrow FF in ERT-naïve patients (n = 15) and patients who switched from imiglucerase ERT (n = 11). Bone marrow FF was assessed using quantitative chemical shift imaging. In the treatment-naïve cohort, median lumbar spine FF increased from 0.27 at baseline to 0.37 at year 1 and 0.42 at year 2. In the cohort of switched patients, median lumbar spine FF increased from 0.24 at baseline to 0.3 at year 1 and 0.34 at year 2. Larger studies are needed to confirm the findings. Protalix Biotherapeutics contributed to the study’s funding.
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Zimran A, Dinur T, Revel-Vilk S, et al. Improvement in bone marrow infiltration in patients with type I Gaucher disease treated with taliglucerase alfa. J Inherit Metab Dis. 2018 Jul 31. doi: 10.1007/s10545-018-0195-y.
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