Derralynn A Hughes, Derlis E Gonzalez, Elena A Lukina, Atul Mehta, Madhulika Kabra, Deborah Elstein, Isaac Kisinovsky, Pilar Giraldo, Ashish Bavdekar, Thomas N Hangartner, Nan Wang, Eric Crombez, Ari Zimran
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...
July 2015: American Journal of Hematology