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Velaglucerase Alfa (VPRIV) Enzyme Replacement Therapy in Patients with Gaucher Disease: Long-Term Data from Phase III Clinical Trials
American Journal of Hematology
  • Derralynn A. Hughes
  • Derlis Gonzalez
  • Elena A. Lukina
  • Atul Mehta
  • Madhulika Kabra
  • Deborah Elstein
  • Isaac Kisinovsky
  • Pilar Giraldo
  • Ashish Bavdekar
  • Thomas N. Hangartner, Wright State University - Main Campus
  • Nan Wang
  • Eric Crombez
  • Ari Zimran
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Type 1 Gaucher disease is an inherited lysosomal enzyme deficiency with variable age of symptom onset. Common presenting signs include thrombocytopenia, anemia, hepatosplenomegaly, bone abnormalities, and, additionally in children, growth failure. Fifty-seven patients aged 3–62 years at the baseline of two phase III trials for velaglucerase alfa treatment were enrolled in the single extension study. In the extension, they received every-other-week velaglucerase alfa intravenous infusions for 1.2–4.8 years at 60 U/kg, although 10 patients experienced dose reduction. No patient experienced a drug-related serious adverse event or withdrew due to an adverse event. One patient died following a convulsion that was reported as unrelated to the study drug. Only one patient tested positive for anti-velaglucerase alfa antibodies. Combining the experience of the initial phase III trials and the extension study, significant improvements were observed in the first 24 months from baseline in hematology variables, organ volumes, plasma biomarkers, and, in adults, the lumbar spine bone mineral density Z-score. Improvements were maintained over longer-term treatment. Velaglucerase alfa had a good long-term safety and tolerability profile, and patients continued to respond clinically, which is consistent with the results of the extension study to the phase I/II trial of velaglucerase alfa.

© 2015 The Authors. American Journal of Hematology published by Wiley Periodicals, Inc.

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Citation Information
Derralynn A. Hughes, Derlis Gonzalez, Elena A. Lukina, Atul Mehta, et al.. "Velaglucerase Alfa (VPRIV) Enzyme Replacement Therapy in Patients with Gaucher Disease: Long-Term Data from Phase III Clinical Trials" American Journal of Hematology Vol. 90 Iss. 7 (2015) p. 584 - 591 ISSN: 03618609
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