Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher disease

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• 作者：Deborah Elstein^a ; ¹ ; ^{delstein-c@shire.com} ; T. Andrew Burrow^b ; Joel Charrow^c ; Pilar Giraldo^d ; ^e ; Atul Mehta^f ; Gregory M. Pastores^g ; ^h ; Hak-Myung Leeⁱ ; Bjö ; rn Mellgard^j ; Ari Zimran^a
• 关键词：AE ; adverse event ; EAP ; early access program ; ERT ; enzyme replacement therapy ; GD1 ; type 1 Gaucher disease ; IRAE ; infusion-related adverse event
• 刊名：Molecular Genetics and Metabolism
• 出版年：2017
• 出版时间：January-February 2017
• 年：2017
• 卷：120
• 期：1-2
• 页码：111-115
• 全文大小：308 K
• 卷排序：120

文摘

A home therapy option was implemented in velaglucerase alfa clinical studies. 104 patients in 4 open-label studies received ≥ 1 velaglucerase alfa home infusion. Most home infusion patients received > 80% of their infusions at home. Fewer than 10% of patients experienced infusion-related adverse events at home.