A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24 ce:hsp>months

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• 作者：Hiroyuki Ida^a ; ^{hiroy@jikei.ac.jp" class="auth_mail" title="E-mail the corresponding author} ; Akemi Tanaka^b ; Tomoko Matsubayashi^c ; ^{matsuba@hama-med.ac.jp" class="auth_mail" title="E-mail the corresponding author} ; Kei Murayama^d ; ^{kmuraya@mri.biglobe.ne.jp" class="auth_mail" title="E-mail the corresponding author} ; Teruaki Hongo^e ; ^{hongot47@gmail.com" class="auth_mail" title="E-mail the corresponding author} ; Hak-Myung Lee^f ; ^{halee@shire.com" class="auth_mail" title="E-mail the corresponding author} ; Bjö ; rn Mellgard^g ; ^{bmellgard@shire.com" class="auth_mail" title="E-mail the corresponding author}
• 关键词：AE ; adverse event ; BMB ; bone marrow burden ; BMD ; bone mineral density ; BSAP ; bone-specific alkaline phosphatase ; CCL18 ; chemokine (C-C motif) ligand 18 ; CTx ; type I collagen telopeptides C-terminal cross-links ; DXA ; dual energy X-ray absorptiometry ; EOW ; every other week ; ERT ; enzyme replacement therapy ; GD ; Gaucher disease ; MRI ; magnetic resonance imaging ; NTx ; type I collagen telopeptides N-terminal cross-links ; SAE ; serious adverse event
• 刊名：Blood Cells, Molecules and Diseases
• 出版年：2016
• 出版时间：July 2016
• 年：2016
• 卷：59
• 期：Complete
• 页码：140-147
• 全文大小：562 K

文摘

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 < 18 years) were enrolled into the extension study. Every-other-week intravenous infusions were administered for 63–78 weeks at average doses between 51.5 and 60.7 units/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 2 years after switching from imiglucerase. class="interref" data-locatorType="url" data-locatorKey="http://ClinicalTrials.gov">ClinicalTrials.gov identifier class="interref" data-locatorType="ctgov" data-locatorKey="NCT01842841">NCT01842841.