A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability
| Autor: | Gregory A. Grabowski, Gabriel M. Cohn, Neal J. Weinreb, Ozlem Goker-Alpan, Gregory M. Pastores, Barry E. Rosenbloom, David Zahrieh |
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| Jazyk: | angličtina |
| Rok vydání: | 2013 |
| Předmět: |
Adult
Male medicine.medical_specialty Treatment protocol Imiglucerase Adolescent Disease velaglucerase alfa type 1 Gaucher disease Gastroenterology Antibodies Young Adult imiglucerase Internal medicine Medicine Humans In patient antidrug antibodies Enzyme Replacement Therapy Original Research Article Child Genetics (clinical) Aged Aged 80 and over Gaucher Disease business.industry Velaglucerase alfa OPEN LABEL TREATMENT Enzyme replacement therapy Middle Aged Recombinant Proteins Surgery Tolerability Glucosylceramidase Female business medicine.drug |
| Zdroj: | Genetics in Medicine |
| ISSN: | 1530-0366 1098-3600 |
| Popis: | Purpose: To evaluate the safety of velaglucerase alfa in patients with type 1 Gaucher disease who received velaglucerase alfa in the US treatment protocol HGT-GCB-058 (ClinicalTrials.gov identifier NCT00954460) during a global supply shortage of imiglucerase. Methods: This multicenter open-label treatment protocol enrolled patients who were either treatment naïve or had been receiving imiglucerase. Patients received intravenous velaglucerase alfa every other week at a dose of 60 U/kg (treatment naïve) or 15–60 U/kg (previously treated). Results: A total of 211 (including six treatment-naïve) patients were enrolled. Among the 205 previously treated patients, 35 (17.1%) experienced an adverse event considered related to study drug. Among the six treatment-naïve patients, one had an adverse event considered related to study drug. Infusion-related adverse events occurred in 28 (13.3%) of the 211 patients and usually occurred during the first three infusions. De novo, nonneutralizing, anti–velaglucerase alfa antibodies developed during treatment in one ( |
| Databáze: | OpenAIRE |
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