{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T06:08:39Z","timestamp":1790662119047,"version":"4.1.0"},"reference-count":23,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2013,2,9]],"date-time":"2013-02-09T00:00:00Z","timestamp":1360368000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2013,3]]},"abstract":"<jats:p>Enzyme replacement therapy for Gaucher disease (GD) has been available since 1991. This study compared the efficacy and safety of velaglucerase alfa with imiglucerase, the previous standard of care. A 9\u2010month, global, randomized, double\u2010blind, non\u2010inferiority study compared velaglucerase alfa with imiglucerase (60 U\/kg every other week) in treatment\u2010na\u00efve patients aged 3\u201373 years with anemia and either thrombocytopenia or organomegaly. The primary endpoint was the difference between groups in mean change from baseline to 9 months in hemoglobin concentration. 35 patients were randomized: 34 received study drug (intent\u2010to\u2010treat: 17 per arm), 20 were splenectomized. Baseline characteristics were similar in the two groups. The per\u2010protocol population included 15 patients per arm. The mean treatment difference for hemoglobin concentration from baseline to 9 months (velaglucerase alfa minus imiglucerase) was 0.14 and 0.16 g\/dL in the intent\u2010to\u2010treat and per\u2010protocol populations, respectively. The lower bound of the 97.5% one\u2010sided confidence interval in both populations lay within the pre\u2010defined non\u2010inferiority margin of \u22121.0 g\/dL, confirming that velaglucerase alfa is non\u2010inferior to imiglucerase. There were no statistically significant differences in the secondary endpoints. Most adverse events were mild to moderate. No patient receiving velaglucerase alfa developed antibodies to either drug, whereas four patients (23.5%) receiving imiglucerase developed IgG antibodies to imiglucerase, which were cross\u2010reactive with velaglucerase alfa in one patient. This study demonstrates the efficacy and safety of velaglucerase alfa compared with imiglucerase in adult and pediatric patients with GD clinically characterized as Type 1. Differences in immunogenicity were also observed. Am. J. 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