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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
medicine
Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease
American Journal of Hematology, Volume 88, No. 3, Year 2013
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Description
Enzyme replacement therapy for Gaucher disease (GD) has been available since 1991. This study compared the efficacy and safety of velaglucerase alfa with imiglucerase, the previous standard of care. A 9-month, global, randomized, double-blind, non-inferiority study compared velaglucerase alfa with imiglucerase (60 U/kg every other week) in treatment-naïve patients aged 3-73 years with anemia and either thrombocytopenia or organomegaly. The primary endpoint was the difference between groups in mean change from baseline to 9 months in hemoglobin concentration. 35 patients were randomized: 34 received study drug (intent-to-treat: 17 per arm), 20 were splenectomized. Baseline characteristics were similar in the two groups. The per-protocol population included 15 patients per arm. The mean treatment difference for hemoglobin concentration from baseline to 9 months (velaglucerase alfa minus imiglucerase) was 0.14 and 0.16 g/dL in the intent-to-treat and per-protocol populations, respectively. The lower bound of the 97.5% one-sided confidence interval in both populations lay within the pre-defined non-inferiority margin of -1.0 g/dL, confirming that velaglucerase alfa is non-inferior to imiglucerase. There were no statistically significant differences in the secondary endpoints. Most adverse events were mild to moderate. No patient receiving velaglucerase alfa developed antibodies to either drug, whereas four patients (23.5%) receiving imiglucerase developed IgG antibodies to imiglucerase, which were cross-reactive with velaglucerase alfa in one patient. This study demonstrates the efficacy and safety of velaglucerase alfa compared with imiglucerase in adult and pediatric patients with GD clinically characterized as Type 1. Differences in immunogenicity were also observed. © 2012 Wiley Periodicals, Inc.
Authors & Co-Authors
Ben Turkia, Hadhami
Tunisia, Tunis
Hôpital la Rabta
Gonzalez, Derlis E.
Paraguay, Asuncion
Sanatorio Español
Barton, Norman W.
United States, Lexington
Shire Human Genetic Therapies, Inc.
Zimran, Ari
Israel, Jerusalem
Hebrew University of Jerusalem
Kabra, Madhulika
India, New Delhi
All India Institute of Medical Sciences, new Delhi
Lukina, Elena A.
Russian Federation, Moscow
National Medical Research Center for Hematology
Giraldo, Pilar
Spain, Zaragoza
Hospital Miguel Servet
Kisinovsky, Isaac
Argentina, Buenos Aires
Your Health S.a.
Bavdekar, Ashish R.
India, Pune
Kem Hospital
Dridi, Marie Françoise Ben
Tunisia, Tunis
Hôpital la Rabta
Gupta, Neerja
India, New Delhi
All India Institute of Medical Sciences, new Delhi
Kishnani, Priya S.
United States, Durham
Duke University Medical Center
Sureshkumar, E. K.
India, Kozhikode
Malabar Institute of Medical Sciences Ltd.
Wang, Nan
United States, Lexington
Shire Human Genetic Therapies, Inc.
Crombez, Eric
United States, Lexington
Shire Human Genetic Therapies, Inc.
Bhirangi, Kiran
United States, Lexington
Shire Human Genetic Therapies, Inc.
Mehta, Atul B.
United Kingdom, London
Ucl Medical School
Statistics
Citations: 57
Authors: 17
Affiliations: 12
Identifiers
Doi:
10.1002/ajh.23382
ISSN:
03618609
e-ISSN:
10968652
Research Areas
Disability
Health System And Policy
Study Design
Cross Sectional Study