Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naïve patients with Gaucher disease

Abstract

Taliglucerase alfa is an intravenous enzyme replacement therapy approved for treatment of type 1 Gaucher disease (GD), and is the first available plant cell-expressed recombinant therapeutic protein. Herein, we report long-term safety and efficacy results of taliglucerase alfa in treatment-naïve adult patients with GD. Patients were randomized to receive taliglucerase alfa 30 or 60 U/kg every other week, and 23 patients completed 36 months of treatment. Taliglucerase alfa (30 U/kg; 60 U/kg, respectively) resulted in mean decreases in spleen volume (50.1%; 64.6%) and liver volume (25.6%; 24.4%) with mean increases in hemoglobin concentration (16.0%; 35.8%) and platelet count (45.7%; 114.0%), and mean decreases in chitotriosidase activity (71.5%; 82.2%). All treatment-related adverse events were mild to moderate in intensity and transient. The most common adverse events were nasopharyngitis, arthralgia, upper respiratory tract infection, headache, pain in extremity, and hypertension. These 36-month results of taliglucerase alfa in treatment-naïve adult patients with GD demonstrate continued improvement in disease parameters with no new safety concerns. These findings extend the taliglucerase alfa clinical safety and efficacy dataset. www.clinicaltrials.gov identifier NCT00705939. Am. J. Hematol. 91:656-660, 2016. © 2016 Wiley Periodicals, Inc.

© 2016 The Authors. American Journal of Hematology Published by Wiley Periodicals, Inc.

Figures

Figure 1

Spleen volume, expressed as multiples of normal (MN), where normal spleen volume is 2 mL/kg times body weight (kg), and liver volume, expressed as MN, where normal liver volume is 25 mL/kg times body weight (kg), through 36 total months of taliglucerase alfa treatment. At each time point, values represent the mean based on all patients with available data. Error bars represent standard error. [Color figure can be viewed in the online issue, which is available at wileyonlinelibrary.com.]

Figure 2

Hemoglobin concentration and platelet count through 36 total months of taliglucerase alfa treatment. One patient did not have hemoglobin concentration values at 36 months and was excluded from the analysis. At each time point, values represent the mean based on all patients with available data. Error bars represent standard error. [Color figure can be viewed in the online issue, which is available at wileyonlinelibrary.com.]

Figure 3

Mean percent change in chitotriosidase activity and CCL18 concentration mean percent change from baseline through 36 total months of taliglucerase alfa treatment. One patient in the 60 U/kg dose group (30–111) was found to have no chitotriosidase activity at baseline; therefore, no chitotriosidase activity data were available during the study, and the patient was excluded from chitotriosidase analysis but was followed via CCL18. At each time point, values represent the mean based on all patients with available data. Error bars represent standard error. [Color figure can be viewed in the online issue, which is available at wileyonlinelibrary.com.]