title: 13,845 home therapy infusions with velaglucerase alfa exemplify safety of velaglucerase alfa and increased compliance to every-other-week intravenous enzyme replacement therapy for Gaucher disease.
abstract: BACKGROUND
  Lifelong intravenous (IV) enzyme replacement therapy (ERT) every other week for Gaucher disease is appreciated as decreasing quality of life in a palpable way.
  
  
  OBJECTIVE
  To review the Israeli experience with the home therapy option for IV velaglucerase alfa (Shire, Lexington MA USA) infusions every-other-week in the clinical trial context, in the early access program (EAP) during a shortage with the standard commercial ERT, and currently with the commercially available drug (VPRIV, Shire).
  
  
  RESULTS
  Among 24 patients participating in trials, 1654 infusions were at home; in the EAP and commercial setting, 12,191 infusions were performed at home for a total of 154 patients with 98.4% compliance. There were no incidents of serious adverse events.
  
  
  CONCLUSIONS
  This is the first review of experience of 174 patients and 13,845 intravenous infusions of velaglucerase alfa for Gaucher in the home setting, underscoring its safety.
authors.affiliation: Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel. Electronic address: elstein@szmc.org.il.
authors.forename: Deborah
authors.initials: D
authors.lastname: Elstein
authors.affiliation: Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel.
authors.forename: Aya
authors.initials: A
authors.lastname: Abrahamov
authors.affiliation: Medison Pharma Ltd, Petach Tikva, Israel.
authors.forename: Anat
authors.initials: A
authors.lastname: Oz
authors.affiliation: Medison Pharma Ltd, Petach Tikva, Israel.
authors.forename: Naama
authors.initials: N
authors.lastname: Arbel
authors.affiliation: Genetics Institute, Rambam Medical Center, Haifa, Israel.
authors.forename: Hagit
authors.initials: H
authors.lastname: Baris
authors.affiliation: Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel.
authors.forename: Ari
authors.initials: A
authors.lastname: Zimran
mesh_terms: D004334:Drug Administration Schedule; D056947:Enzyme Replacement Therapy / Q000009:adverse effects / Q000379:methods; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000008:administration & dosage*; D018718:Home Infusion Therapy / Q000009:adverse effects; D006801:Humans; D055118:Medication Adherence
chemical_list: D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human
keywords: Gaucher disease; Home therapy; Intravenous administration; Quality of life; Safety; Velaglucerase alfa