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Genet Med. 2014 May;16(5):359-66. doi: 10.1038/gim.2013.154. Epub 2013 Nov 21.

A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability.

Author information

  • 1Departments of Neurology and Pediatrics, New York University School of Medicine, New York, New York, USA.
  • 2Cedars-Sinai/Tower Hematology Oncology, Beverly Hills, California, USA.
  • 3University Research Foundation for Lysosomal Storage Diseases, Coral Springs, Florida, USA.
  • 4Lysosomal Research and Treatment Unit, Fairfax, Virginia, USA.
  • 5Children's Hospital Medical Center, Cincinnati, Ohio, USA.
  • 6Shire, Lexington, Massachusetts, USA.



To evaluate the safety of velaglucerase alfa in patients with type 1 Gaucher disease who received velaglucerase alfa in the US treatment protocol HGT-GCB-058 ( identifier NCT00954460) during a global supply shortage of imiglucerase.


This multicenter open-label treatment protocol enrolled patients who were either treatment naïve or had been receiving imiglucerase. Patients received intravenous velaglucerase alfa every other week at a dose of 60 U/kg (treatment naïve) or 15-60 U/kg (previously treated).


A total of 211 (including six treatment-naïve) patients were enrolled. Among the 205 previously treated patients, 35 (17.1%) experienced an adverse event considered related to study drug. Among the six treatment-naïve patients, one had an adverse event considered related to study drug. Infusion-related adverse events occurred in 28 (13.3%) of the 211 patients and usually occurred during the first three infusions. De novo, nonneutralizing, anti-velaglucerase alfa antibodies developed during treatment in one (<1.0%) previously treated patient and none of the treatment-naïve patients.


The currently observed safety profile was consistent with those previously reported for imiglucerase and velaglucerase alfa phase III clinical trials. These results support the safety of initiating treatment with velaglucerase alfa or transitioning patients from imiglucerase therapy to velaglucerase alfa therapy.

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