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Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease.
Am J Hematol. 2013 Mar; 88(3):179-84.AJ

Abstract

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.

Authors+Show Affiliations

La Rabta Hospital, Tunis, Tunisia.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Comparative Study
Journal Article
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

23400823

Citation

Ben Turkia, Hadhami, et al. "Velaglucerase Alfa Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Gaucher Disease." American Journal of Hematology, vol. 88, no. 3, 2013, pp. 179-84.
Ben Turkia H, Gonzalez DE, Barton NW, et al. Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease. Am J Hematol. 2013;88(3):179-84.
Ben Turkia, H., Gonzalez, D. E., Barton, N. W., Zimran, A., Kabra, M., Lukina, E. A., Giraldo, P., Kisinovsky, I., Bavdekar, A., Ben Dridi, M. F., Gupta, N., Kishnani, P. S., Sureshkumar, E. K., Wang, N., Crombez, E., Bhirangi, K., & Mehta, A. (2013). Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease. American Journal of Hematology, 88(3), 179-84. https://doi.org/10.1002/ajh.23382
Ben Turkia H, et al. Velaglucerase Alfa Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Gaucher Disease. Am J Hematol. 2013;88(3):179-84. PubMed PMID: 23400823.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease. AU - Ben Turkia,Hadhami, AU - Gonzalez,Derlis E, AU - Barton,Norman W, AU - Zimran,Ari, AU - Kabra,Madhulika, AU - Lukina,Elena A, AU - Giraldo,Pilar, AU - Kisinovsky,Isaac, AU - Bavdekar,Ashish, AU - Ben Dridi,Marie-Françoise, AU - Gupta,Neerja, AU - Kishnani,Priya S, AU - Sureshkumar,E K, AU - Wang,Nan, AU - Crombez,Eric, AU - Bhirangi,Kiran, AU - Mehta,Atul, Y1 - 2013/02/09/ PY - 2012/10/30/received PY - 2012/11/23/revised PY - 2012/12/12/accepted PY - 2013/2/13/entrez PY - 2013/2/13/pubmed PY - 2013/4/13/medline SP - 179 EP - 84 JF - American journal of hematology JO - Am. J. Hematol. VL - 88 IS - 3 N2 - 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. SN - 1096-8652 UR - https://www.unboundmedicine.com/medline/citation/23400823/Velaglucerase_alfa_enzyme_replacement_therapy_compared_with_imiglucerase_in_patients_with_Gaucher_disease_ L2 - https://doi.org/10.1002/ajh.23382 DB - PRIME DP - Unbound Medicine ER -