482 Safety and Efficacy of Velaglucerase Alfa in Gaucher Disease Type 1 Patients Previously Treated with Imiglucerase

Objective: To examine safety and efficacy of velaglucerase alfa every other week in patients with Gaucher disease previously receiving imiglucerase. Methods: This open-label, multicenter, 12-month study, enrolled 41 patients ≥2 years to receive velaglucerase alfa at a dose equalling their prior imig...

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Veröffentlicht in:Pediatric research 2010-11, Vol.68 (Suppl 1), p.246-247
Hauptverfasser: Grabowski, G, Pastores, G, Mardach, R, Eng, C, Smith, L, Fernhoff, P, Charrow, J, Heisel-Kurth, M, Longo, N, Rhead, W, Harmatz, P, Mehta, A, Giraldo, P, Tylki-Szymanska, A, Zimran, A, Crombez, E
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Sprache:eng
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Zusammenfassung:Objective: To examine safety and efficacy of velaglucerase alfa every other week in patients with Gaucher disease previously receiving imiglucerase. Methods: This open-label, multicenter, 12-month study, enrolled 41 patients ≥2 years to receive velaglucerase alfa at a dose equalling their prior imiglucerase regimen, with infusions administered over 1-hour. Results: Forty patients received study drug (18 male, 22 female; 4 previously splenectomized; age range, 9-71 years, 25% < 18 years). Median prior imiglucerase use was 67 months (range 22-192 months). Velaglucerase alfa doses were: ≤22.5U/ kg (n=14), 22.5-37.5U/kg (n=12), 37.5-52.5U/kg (n=7), and >52.5U/kg (n=7). Velaglucerase alfa was generally well tolerated with most AEs of mild or moderate severity. No patient experienced a lifethreatening AE; 7 severe AEs (in 5 patients), and 5 treatment-emergent serious AEs (in 4 patients) were reported. One patient discontinued due to a hypersensitivity reaction; this patient did not have IgG or IgE antibodies. No patients developed IgG antibodies to velaglucerase alfa. Clinical parameters were sustained at therapeutic levels through 1 year ( table ). Table Conclusions: Adult and pediatric Gaucher disease patients, previously treated with imiglucerase for ≥6 months, were successfully transitioned to velaglucerase alfa, with stability in clinical disease measures over 12 months. Velaglucerase alfa is approved in the U.S.
ISSN:0031-3998
1530-0447
DOI:10.1203/00006450-201011001-00482