title: Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase.
abstract: Velaglucerase alfa is a glucocerebrosidase produced by gene activation technology in a human fibroblast cell line (HT-1080), and it is indicated as an enzyme replacement therapy (ERT) for the treatment of Gaucher disease type 1 (GD1). This multicenter, open-label, 12-month study examined the safety and efficacy of velaglucerase alfa in patients with GD1 previously receiving imiglucerase. Eligible patients, ≥2 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 9-71 years). Velaglucerase alfa was generally well tolerated with most adverse events (AEs) 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 AE considered treatment-related: a grade 2 anaphylactoid reaction within 30 min of the first infusion. The patient withdrew; this was the only AE-related withdrawal. Hemoglobin concentrations, platelet counts, and spleen and liver volumes remained stable through 12 months. In conclusion, adult and pediatric patients with GD1, 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.
authors: Shaare Zedek Medical Center and Hebrew University-Hadassah Medical School, Jerusalem, Israel.
authors: Ari
authors: A
authors: Zimran
authors: Gregory M
authors: GM
authors: Pastores
authors: Anna
authors: A
authors: Tylki-Szymanska
authors: Derralynn A
authors: DA
authors: Hughes
authors: Deborah
authors: D
authors: Elstein
authors: Rebecca
authors: R
authors: Mardach
authors: Christine
authors: C
authors: Eng
authors: Laurie
authors: L
authors: Smith
authors: Margaret
authors: M
authors: Heisel-Kurth
authors: Joel
authors: J
authors: Charrow
authors: Paul
authors: P
authors: Harmatz
authors: Paul
authors: P
authors: Fernhoff
authors: William
authors: W
authors: Rhead
authors: Nicola
authors: N
authors: Longo
authors: Pilar
authors: P
authors: Giraldo
authors: Juan A
authors: JA
authors: Ruiz
authors: David
authors: D
authors: Zahrieh
authors: Eric
authors: E
authors: Crombez
authors: Gregory A
authors: GA
authors: Grabowski
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D004334:Drug Administration Schedule; D057915:Drug Substitution; D056947:Enzyme Replacement Therapy; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy* / Q000201:enzymology / Q000235:genetics; D005962:Glucosylceramidase / Q000172:deficiency* / Q000235:genetics / Q000494:pharmacology / Q000627:therapeutic use; D006454:Hemoglobins / Q000032:analysis; D006801:Humans; D007275:Injections, Intravenous; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D011994:Recombinant Proteins / Q000494:pharmacology / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D006454:Hemoglobins; D011994:Recombinant Proteins; D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human; C090568:imiglucerase