Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase
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- 作者：Laurie Smitha ; ldsmith@cmh.edu" class="auth_mail" title="E-mail the corresponding author ; William Rheadb ; Joel Charrowc ; Suma P. Shankard ; e ; Ashish Bavdekarf ; Nicola Longog ; Rebecca Mardachh ; Paul Harmatzi ; Thomas Hangartnerj ; Hak-Myung Leek ; Eric Crombezl ; Gregory M. Pastoresm
- 关键词：AE ; adverse event ; BMB ; bone marrow burden ; BW ; body weight ; CCL18 ; chemokine (C&ndash ; C motif) ligand 18 ; EOW ; every other week ; ERT ; enzyme replacement therapy ; GD ; Gaucher disease ; ICH ; International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use ; IgG ; immunoglobulin G ; IRAE ; infusion-related adverse event ; LS ; lumbar spine ; MN ; multiples of normal ; MRI ; magnetic resonance imaging ; SAE ; serious adverse event ; TEAE ; treatment-emergent ad
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：February 2016
- 年：2016
- 卷：117
- 期：2
- 页码：164-171
- 全文大小：781 K
文摘
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Pediatric patients received up to 5.8 years of velaglucerase alfa therapy.
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The safety profile in pediatric patients was consistent with that seen in adults.
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A positive effect on key efficacy parameters was seen in treatment-naïve patients.
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Key efficacy parameters were stable in patients who switched from imiglucerase.