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

Deborah Elstein*, T. Andrew Burrow, Joel Charrow, Pilar Giraldo, Atul Mehta, Gregory M. Pastores, Hak Myung Lee, Björn Mellgard, Ari Zimran

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


The introduction of a home therapy option during clinical trials of velaglucerase alfa in patients with type 1 Gaucher disease marked the first time that home infusions have been permitted during a clinical trial for an investigational drug for Gaucher disease. Home infusions were an available option in 4 open-label velaglucerase alfa clinical studies to eligible patients who received their initial infusions at a clinic. Patients who participated in the home therapy option and received at least 10% of their infusions at home (n = 100) received a range of 11.6%–100% of their scheduled infusions at home (median 87.5%), excluding infusions received at the clinic during protocol-mandated visits. The length of time over which individual patients received home therapy ranged from 13 days to 4.56 years (median 0.57 years). During the time that home therapy was available, 2904 of 3572 (81.3%) infusions were administered at home. Ten patients experienced 62 infusion-related adverse events (IRAEs) during 38 home infusions, with malaise, pain, hypertension, fatigue, and headache being reported most frequently. No notable differences were found between the type and severity of IRAEs experienced at home and those experienced at the clinic. Home infusions administered by trained and qualified medical personnel were successfully introduced into the velaglucerase alfa clinical development program, and fewer than 10% of patients experienced IRAEs in the home setting. Local labeling and practice guidelines should be consulted for administration of velaglucerase alfa infusions at home.

Original languageEnglish (US)
Pages (from-to)111-115
Number of pages5
JournalMolecular Genetics and Metabolism
Issue number1-2
StatePublished - Jan 1 2017


  • Enzyme replacement therapy
  • Gaucher disease
  • Home therapy
  • Velaglucerase alfa

ASJC Scopus subject areas

  • Genetics
  • Endocrinology
  • Molecular Biology
  • Biochemistry
  • Endocrinology, Diabetes and Metabolism


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