title: A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24months.
abstract: Enzyme replacement therapy (ERT) with exogenous glucocerebrosidase is indicated to treat symptomatic Gaucher disease (GD), a rare, inherited metabolic disorder. ERT with velaglucerase alfa, which is produced in a human cell line using gene activation technology, was studied in a 12-month phase III trial in Japanese patients with type 1 or 3 GD who were switched from imiglucerase ERT (n=6); the current, open-label, 12-month extension study was designed to assess longer-term safety and efficacy. Two adult and three pediatric patients (aged <18years) were enrolled into the extension study. Every-other-week intravenous infusions were administered for 63-78weeks at average doses between 51.5 and 60.7units/kg. Three non-serious adverse events were considered related to velaglucerase alfa treatment, but no patient discontinued from the study. Six serious but non-drug-related adverse events were reported. No patient tested positive for anti-velaglucerase alfa antibodies. Hemoglobin concentrations, platelet counts, and liver and spleen volumes (normalized to body weight) in these patients were generally stable over a cumulative 24-month period from the baseline of the parent trial. The data suggest that velaglucerase alfa was well tolerated and maintained clinical stability in Japanese GD patients over 2years after switching from imiglucerase. ClinicalTrials.gov identifier NCT01842841.
authors.affiliation: Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan. Electronic address: hiroy@jikei.ac.jp.
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Ida
authors.affiliation: Department of Pediatrics, Osaka City University Graduate School of Medicine, Osaka, Japan.
authors.forename: Akemi
authors.initials: A
authors.lastname: Tanaka
authors.affiliation: Department of Pediatrics, Hamamatsu University School of Medicine, Hamamatsu, Japan. Electronic address: matsuba@hama-med.ac.jp.
authors.forename: Tomoko
authors.initials: T
authors.lastname: Matsubayashi
authors.affiliation: Department of Metabolism, Chiba Children's Hospital, Chiba, Japan. Electronic address: kmuraya@mri.biglobe.ne.jp.
authors.forename: Kei
authors.initials: K
authors.lastname: Murayama
authors.affiliation: Department of Pediatrics, Iwata City Hospital, Iwata, Japan. Electronic address: hongot47@gmail.com.
authors.forename: Teruaki
authors.initials: T
authors.lastname: Hongo
authors.affiliation: Shire, Lexington, MA, USA. Electronic address: halee@shire.com.
authors.forename: Hak-Myung
authors.initials: HM
authors.lastname: Lee
authors.affiliation: Shire, Zug, Switzerland. Electronic address: bmellgard@shire.com.
authors.forename: Björn
authors.initials: B
authors.lastname: Mellgard
mesh_terms: D000906:Antibodies / Q000032:analysis; D044466:Asian People; D057915:Drug Substitution; D056947:Enzyme Replacement Therapy / Q000379:methods; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000008:administration & dosage / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D006801:Humans; D016896:Treatment Outcome
chemical_list: D000906:Antibodies; D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human; C090568:imiglucerase
keywords: Enzyme replacement therapy; Gaucher disease; Japan; Neuronopathic; Velaglucerase alfa