Long‐term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment‐naïve patients with Gaucher disease |
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| Authors: | Ari Zimran Gloria Durán Atul Mehta Pilar Giraldo Hanna Rosenbaum Fiorina Giona Dominick J. Amato Milan Petakov Eduardo Terreros Muñoz Sergio Eduardo Solorio‐Meza Peter A. Cooper Sheeba Varughese Raul Chertkoff Einat Brill‐Almon |
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| Affiliation: | 1. Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School, Jerusalem, Israel;2. Pontificia Universidad Catolica de Chile, Santiago, Chile;3. Royal Free and University College School of Medicine, Royal Free Hospital, London, United Kingdom;4. CIBERER, Hospital Universitario Miguel Servet, Zaragoza, Spain;5. Rambam Medical Center, Haifa, Israel;6. Sapienza University of Rome, Rome, Italy;7. Mount Sinai Hospital, Toronto, Ontario, Canada;8. Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Disease, Belgrade University Medical School, Belgrade, Serbia;9. Servicio de Hematologia, Centro Médico Nacional Siglo XXI, México City, México;10. Hospital de Especialidades No1, Leon, México;11. Faculty of Health Sciences, University of the Witwatersrand & Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa;12. Protalix BioTherapeutics, Carmiel, Israel |
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| 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. |
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