A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase

作者：Pastores Gregory M^*; Petakov Milan; Giraldo Pilar; Rosenbaum Hanna; Szer Jeffrey; Deegan Patrick B; Amato Dominick J; Mengel Eugen; Tan Ee Shien; Chertkoff Raul; Brill Almon Einat; Zimran Ari

来源：Blood Cells, Molecules, and Diseases, 2014, 53(4): 253-260.

DOI：10.1016/j.bcmd.2014.05.004

• Gaucher disease
• Enzyme replacement therapy
• Taliglucerase alfa
• Imiglucerase

Taliglucerase alfa is beta-glucosidase enzyme replacement therapy (ERT) approved in the US and other countries for the treatment of Gaucher disease (GD) in adults and is approved in pediatric and adult patients in Australia and Canada. It is the first approved plant cell-expressed recombinant human protein. A Phase 3, multicenter, open-label, 9-month study assessed safety and efficacy of switching to taliglucerase alfa in adult and pediatric patients with GD treated with imiglucerase for at least the previous 2 years. Patients with stable disease were offered taliglucerase alfa treatment using the same dose (9-60 U/kg body weight) and regimen of administration (every 2 weeks) as imiglucerase. This report summarizes results from 26 adult and 5 pediatric patients who participated in the trial. Disease parameters (spleen and liver volumes, hemoglobin concentration, platelet count, and biomarker levels) remained stable through 9 months of treatment in adults and children following the switch from imiglucerase. All treatment-related adverse events were mild or moderate in severity and transient in nature. Exploratory parameters of linear growth and development showed positive outcomes in pediatric patients. These findings provide evidence of the efficacy and safety profile of taliglucerase alfa as an ERT for GD in patients previously treated with imiglucerase. This trial was registered at www.clinicaltrials.gov as # NCT00712348.

• 出版日期2014-12