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Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imiglucerase.
Blood Cells Mol Dis. 2018 02; 68:163-172.BC

Abstract

Taliglucerase alfa is an enzyme replacement therapy approved for treatment of Gaucher disease (GD) in children and adults in several countries. This multicenter extension study assessed the efficacy and safety of taliglucerase alfa in pediatric patients with GD who were treatment-naïve (n=10) or switched from imiglucerase (n=5). Patients received taliglucerase alfa 30 or 60U/kg (treatment-naïve) or the same dose as previously treated with imiglucerase every other week. In treatment-naïve patients, taliglucerase alfa 30 and 60U/kg, respectively, reduced mean spleen volume (-18.6 multiples of normal [MN] and -26.0MN), liver volume (-0.8MN and -0.9MN), and chitotriosidase activity (-72.7% and -84.4%), and increased mean Hb concentration (+2.0g/dL and +2.3g/dL) and mean platelet count (+38,200/mm3 and +138,250/mm3) from baseline through 36 total months of treatment. In patients previously treated with imiglucerase, these disease parameters remained stable through 33 total months of treatment with taliglucerase alfa. Most adverse events were mild/moderate; treatment was well tolerated. These findings extend the taliglucerase alfa safety and efficacy profile and demonstrate long-term clinical improvement in treatment-naïve children receiving taliglucerase alfa and maintenance of disease stability in children switched to taliglucerase alfa. Treatment was well-tolerated, with no new safety signals. This study is registered at www.clinicaltrials.gov as NCT01411228.

Authors+Show Affiliations

Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School, Jerusalem, Israel. Electronic address: azimran@gmail.com.Instituto Privado de Hematologia e Investigacion Clinica, Asuncion, Paraguay.Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School, Jerusalem, Israel.Faculty of Health Sciences, University of the Witwatersrand, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.Faculty of Health Sciences, University of the Witwatersrand, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.CIBERER, Hospital Universitario Miguel Servet, IIS Aragon, Zaragoza, Spain.Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Disease, Belgrade University Medical School, Belgrade, Serbia.Genetics Service, Department of Paediatric Medicine, KK Women's and Children's Hospital, Singapore.Protalix BioTherapeutics, Carmiel, Israel.

Pub Type(s)

Clinical Trial
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

27839981

Citation

Zimran, Ari, et al. "Long-term Safety and Efficacy of Taliglucerase Alfa in Pediatric Gaucher Disease Patients Who Were Treatment-naïve or Previously Treated With Imiglucerase." Blood Cells, Molecules & Diseases, vol. 68, 2018, pp. 163-172.
Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, et al. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imiglucerase. Blood Cells Mol Dis. 2018;68:163-172.
Zimran, A., Gonzalez-Rodriguez, D. E., Abrahamov, A., Cooper, P. A., Varughese, S., Giraldo, P., Petakov, M., Tan, E. S., & Chertkoff, R. (2018). Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imiglucerase. Blood Cells, Molecules & Diseases, 68, 163-172. https://doi.org/10.1016/j.bcmd.2016.10.005
Zimran A, et al. Long-term Safety and Efficacy of Taliglucerase Alfa in Pediatric Gaucher Disease Patients Who Were Treatment-naïve or Previously Treated With Imiglucerase. Blood Cells Mol Dis. 2018;68:163-172. PubMed PMID: 27839981.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imiglucerase. AU - Zimran,Ari, AU - Gonzalez-Rodriguez,Derlis Emilio, AU - Abrahamov,Aya, AU - Cooper,Peter A, AU - Varughese,Sheeba, AU - Giraldo,Pilar, AU - Petakov,Milan, AU - Tan,Ee Shien, AU - Chertkoff,Raul, Y1 - 2016/10/20/ PY - 2016/10/12/received PY - 2016/10/19/accepted PY - 2016/11/15/pubmed PY - 2018/7/17/medline PY - 2016/11/15/entrez KW - Enzyme replacement therapy KW - Gaucher disease KW - Imiglucerase KW - Pediatrics KW - Taliglucerase alfa SP - 163 EP - 172 JF - Blood cells, molecules & diseases JO - Blood Cells Mol. Dis. VL - 68 N2 - Taliglucerase alfa is an enzyme replacement therapy approved for treatment of Gaucher disease (GD) in children and adults in several countries. This multicenter extension study assessed the efficacy and safety of taliglucerase alfa in pediatric patients with GD who were treatment-naïve (n=10) or switched from imiglucerase (n=5). Patients received taliglucerase alfa 30 or 60U/kg (treatment-naïve) or the same dose as previously treated with imiglucerase every other week. In treatment-naïve patients, taliglucerase alfa 30 and 60U/kg, respectively, reduced mean spleen volume (-18.6 multiples of normal [MN] and -26.0MN), liver volume (-0.8MN and -0.9MN), and chitotriosidase activity (-72.7% and -84.4%), and increased mean Hb concentration (+2.0g/dL and +2.3g/dL) and mean platelet count (+38,200/mm3 and +138,250/mm3) from baseline through 36 total months of treatment. In patients previously treated with imiglucerase, these disease parameters remained stable through 33 total months of treatment with taliglucerase alfa. Most adverse events were mild/moderate; treatment was well tolerated. These findings extend the taliglucerase alfa safety and efficacy profile and demonstrate long-term clinical improvement in treatment-naïve children receiving taliglucerase alfa and maintenance of disease stability in children switched to taliglucerase alfa. Treatment was well-tolerated, with no new safety signals. This study is registered at www.clinicaltrials.gov as NCT01411228. SN - 1096-0961 UR - https://www.unboundmedicine.com/medline/citation/27839981/Long_term_safety_and_efficacy_of_taliglucerase_alfa_in_pediatric_Gaucher_disease_patients_who_were_treatment_naïve_or_previously_treated_with_imiglucerase_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1079-9796(16)30221-2 DB - PRIME DP - Unbound Medicine ER -