{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T01:16:15Z","timestamp":1793322975525,"version":"4.5.0"},"reference-count":23,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2013,1,22]],"date-time":"2013-01-22T00:00:00Z","timestamp":1358812800000},"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>Velaglucerase alfa is a glucocerebrosidase produced by gene activation technology in a human fibroblast cell line (<jats:styled-content style=\"fixed-case\">HT<\/jats:styled-content>\u20101080), and it is indicated as an enzyme replacement therapy (<jats:styled-content style=\"fixed-case\">ERT<\/jats:styled-content>) for the treatment of Gaucher disease type 1 (<jats:styled-content style=\"fixed-case\">GD1<\/jats:styled-content>). This multicenter, open\u2010label, 12\u2010month study examined the safety and efficacy of velaglucerase alfa in patients with <jats:styled-content style=\"fixed-case\">GD1<\/jats:styled-content> previously receiving imiglucerase. Eligible patients, \u22652 years old and clinically stable on imiglucerase therapy, were switched to velaglucerase alfa at a dose equal to their prior imiglucerase dose. Infusion durations were 1 hr every other week. Forty patients received velaglucerase alfa (18 male, 22 female; four previously splenectomized; age range 9\u201371 years). Velaglucerase alfa was generally well tolerated with most adverse events (<jats:styled-content style=\"fixed-case\">AEs<\/jats:styled-content>) of mild or moderate severity. The three most frequently reported AEs were headache (12 of 40 patients), arthralgia (9 of 40 patients), and nasopharyngitis (8 of 40 patients). No patients developed antibodies to velaglucerase alfa. There was one serious <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content> considered treatment\u2010related: a Grade 2 anaphylactoid reaction within 30 min of the first infusion. The patient withdrew; this was the only <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>\u2010related withdrawal. Hemoglobin concentrations, platelet counts, and spleen and liver volumes remained stable through 12 months. In conclusion, adult and pediatric patients with <jats:styled-content style=\"fixed-case\">GD1<\/jats:styled-content>, previously treated with imiglucerase, successfully transitioned to velaglucerase alfa, which was generally well tolerated and demonstrated efficacy over 12 months' treatment consistent with that observed in the velaglucerase alfa Phase 3 clinical trial program. Am. J. 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