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Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 18.

Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.

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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.
Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel.


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. identifier NCT00705939. Am. J. Hematol. 91:661-665, 2016.

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