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Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.
Am J Hematol. 2016 07; 91(7):661-5.AJ

Abstract

Taliglucerase alfa is the first available plant cell-expressed human recombinant therapeutic protein. It is indicated for treatment of patients with type 1 Gaucher disease (GD) in adult and pediatric patients in several countries. Study PB-06-002 examined the safety and efficacy of taliglucerase alfa for 9 months in patients who previously received imiglucerase. The results of adult patients from Study PB-06-002 who continued receiving taliglucerase alfa in extension Study PB-06-003 for up to 36 months are reported here. Eighteen patients received at least one dose of taliglucerase alfa in Study PB-06-003; 10 patients completed 36 total months of therapy, and four patients who transitioned to commercial drug completed 30-33 months of treatment. In patients who completed 36 total months of treatment, mean percent (±standard error) changes from baseline/time of switch to taliglucerase alfa to 36 months were as follows: hemoglobin concentration, -1.0% (±1.9%; n = 10); platelet count, +9.3% (±9.8%; n = 10); spleen volume measured in multiples of normal (MN), -19.8% (±9.9%; n = 7); liver volume measured in MN, +0.9% (±5.4%; n = 8); chitotriosidase activity, -51.5% (±8.1%; n = 10); and CCL18 concentration, -36.5 (±8.0%; n = 10). Four patients developed antidrug antibodies, including one with evidence of neutralizing activity in vitro. All treatment-related adverse events were mild or moderate and transient. The 36-month results of switching from imiglucerase to taliglucerase alfa treatment in adults with GD provide further data on the clinical safety and efficacy of taliglucerase alfa beyond the initial 9 months of the original study. www.clinicaltrials.gov identifier NCT00705939. Am. J. Hematol. 91:661-665, 2016. © 2016 Wiley Periodicals, Inc.

Authors+Show Affiliations

National Centre for Inherited Metabolic Disorders, Mater Misericordiae University Hospital, Dublin, Ireland.Department of Genetics, Emory University School of Medicine, Atlanta, Georgia. Department of Ophthalmology, Emory University School of Medicine, Atlanta, Georgia.Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Diseases, Belgrade University Medical School, Belgrade, Serbia.CIBERER, Hospital Universitario Miguel Servet, Zaragoza, Spain.Rambam Medical Center, Haifa, Israel.Mount Sinai Hospital, Toronto, Ontario, Canada.Royal Melbourne Hospital, Victoria, Australia.Protalix BioTherapeutics, Carmiel, Israel.Protalix BioTherapeutics, Carmiel, Israel.Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel.

Pub Type(s)

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

Language

eng

PubMed ID

27102949

Citation

Pastores, Gregory M., et al. "Enzyme Replacement Therapy With Taliglucerase Alfa: 36-month Safety and Efficacy Results in Adult Patients With Gaucher Disease Previously Treated With Imiglucerase." American Journal of Hematology, vol. 91, no. 7, 2016, pp. 661-5.
Pastores GM, Shankar SP, Petakov M, et al. Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. Am J Hematol. 2016;91(7):661-5.
Pastores, G. M., Shankar, S. P., Petakov, M., Giraldo, P., Rosenbaum, H., Amato, D. J., Szer, J., Chertkoff, R., Brill-Almon, E., & Zimran, A. (2016). Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. American Journal of Hematology, 91(7), 661-5. https://doi.org/10.1002/ajh.24399
Pastores GM, et al. Enzyme Replacement Therapy With Taliglucerase Alfa: 36-month Safety and Efficacy Results in Adult Patients With Gaucher Disease Previously Treated With Imiglucerase. Am J Hematol. 2016;91(7):661-5. PubMed PMID: 27102949.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. AU - Pastores,Gregory M, AU - Shankar,Suma P, AU - Petakov,Milan, AU - Giraldo,Pilar, AU - Rosenbaum,Hanna, AU - Amato,Dominick J, AU - Szer,Jeffrey, AU - Chertkoff,Raul, AU - Brill-Almon,Einat, AU - Zimran,Ari, Y1 - 2016/05/18/ PY - 2015/09/25/received PY - 2016/04/18/revised PY - 2016/04/19/accepted PY - 2016/4/23/entrez PY - 2016/4/23/pubmed PY - 2017/7/18/medline SP - 661 EP - 5 JF - American journal of hematology JO - Am. J. Hematol. VL - 91 IS - 7 N2 - Taliglucerase alfa is the first available plant cell-expressed human recombinant therapeutic protein. It is indicated for treatment of patients with type 1 Gaucher disease (GD) in adult and pediatric patients in several countries. Study PB-06-002 examined the safety and efficacy of taliglucerase alfa for 9 months in patients who previously received imiglucerase. The results of adult patients from Study PB-06-002 who continued receiving taliglucerase alfa in extension Study PB-06-003 for up to 36 months are reported here. Eighteen patients received at least one dose of taliglucerase alfa in Study PB-06-003; 10 patients completed 36 total months of therapy, and four patients who transitioned to commercial drug completed 30-33 months of treatment. In patients who completed 36 total months of treatment, mean percent (±standard error) changes from baseline/time of switch to taliglucerase alfa to 36 months were as follows: hemoglobin concentration, -1.0% (±1.9%; n = 10); platelet count, +9.3% (±9.8%; n = 10); spleen volume measured in multiples of normal (MN), -19.8% (±9.9%; n = 7); liver volume measured in MN, +0.9% (±5.4%; n = 8); chitotriosidase activity, -51.5% (±8.1%; n = 10); and CCL18 concentration, -36.5 (±8.0%; n = 10). Four patients developed antidrug antibodies, including one with evidence of neutralizing activity in vitro. All treatment-related adverse events were mild or moderate and transient. The 36-month results of switching from imiglucerase to taliglucerase alfa treatment in adults with GD provide further data on the clinical safety and efficacy of taliglucerase alfa beyond the initial 9 months of the original study. www.clinicaltrials.gov identifier NCT00705939. Am. J. Hematol. 91:661-665, 2016. © 2016 Wiley Periodicals, Inc. SN - 1096-8652 UR - https://www.unboundmedicine.com/medline/citation/27102949/Enzyme_replacement_therapy_with_taliglucerase_alfa:_36_month_safety_and_efficacy_results_in_adult_patients_with_Gaucher_disease_previously_treated_with_imiglucerase_ L2 - https://doi.org/10.1002/ajh.24399 DB - PRIME DP - Unbound Medicine ER -