戈谢病患者改用塔利格卢酶的现实世界经验：一个病例系列

IF 1.9 4区医学 Q3 GENETICS & HEREDITY

Molecular Genetics and Metabolism Reports Pub Date : 2025-09-24 DOI:10.1016/j.ymgmr.2025.101257

Angelo Rizzolo , Marie-Claude Miron , Jean-François Delisle , Nathalie Alos , Philippe M. Campeau , François Mercier

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引用次数: 0

摘要

戈谢病（GD）的酶替代疗法（ERT）有效地预防了这种遗传性溶酶体贮积疾病的骨骼、内脏和血液学并发症。Taliglucerase是三种市售ERT产品之一，并于2016年成为加拿大qu忧郁省推荐的一线治疗药物。因此，19名患者从伊米格鲁酶转为塔利格鲁酶，但超过四分之一的患者出现了明显的副作用。在这里，我们总结了这些患者的临床过程，并描述了6个疑似产品相关的不良反应。

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Real-world experience of switching to taliglucerase among patients with Gaucher disease in Québec: A case series

Enzyme replacement therapy (ERT) for Gaucher disease (GD) effectively prevents skeletal, visceral, and hematologic complications of this inherited, lysosomal storage disorder. Taliglucerase is one of the three commercially available ERT products and became the recommended first-line therapy in Québec, Canada in 2016. Thus, 19 patients were switched from imiglucerase to taliglucerase, but more than a quarter experienced significant side effects. Here, we summarize these patients' clinical course and describe 6 suspected product-related adverse-effects.

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来源期刊

Molecular Genetics and Metabolism Reports Biochemistry, Genetics and Molecular Biology-Endocrinology

CiteScore

4.00

自引率

5.30%

发文量

105

审稿时长

33 days

期刊介绍： Molecular Genetics and Metabolism Reports is an open access journal that publishes molecular and metabolic reports describing investigations that use the tools of biochemistry and molecular biology for studies of normal and diseased states. In addition to original research articles, sequence reports, brief communication reports and letters to the editor are considered.