Zimran, Ari (7006390817)Ari (7006390817)ZimranDurán, Gloria (54779673100)Gloria (54779673100)DuránGiraldo, Pilar (7007060971)Pilar (7007060971)GiraldoRosenbaum, Hanna (7005497035)Hanna (7005497035)RosenbaumGiona, Fiorina (6701332902)Fiorina (6701332902)GionaPetakov, Milan (7003976693)Milan (7003976693)PetakovTerreros Muñoz, Eduardo (8246124100)Eduardo (8246124100)Terreros MuñozSolorio-Meza, Sergio Eduardo (6603867633)Sergio Eduardo (6603867633)Solorio-MezaCooper, Peter A. (7402087887)Peter A. (7402087887)CooperVarughese, Sheeba (55756661100)Sheeba (55756661100)VarugheseAlon, Sari (56678491100)Sari (56678491100)AlonChertkoff, Raul (6507776646)Raul (6507776646)Chertkoff2025-06-122025-06-122019https://doi.org/10.1016/j.bcmd.2016.07.002https://www.scopus.com/inward/record.uri?eid=2-s2.0-84994351540&doi=10.1016%2fj.bcmd.2016.07.002&partnerID=40&md5=e3ea3df7ac07afdf67ea0607df4cc243https://remedy.med.bg.ac.rs/handle/123456789/5520Taliglucerase alfa, the first available plant cell–expressed recombinant therapeutic protein, is an enzyme replacement therapy approved for Gaucher disease (GD). PB-06-001, a pivotal phase 3, multicenter, randomized, double-blind, parallel-dose study investigated taliglucerase alfa 30 or 60 U/kg every other week through 9 months in treatment-naïve adults with GD; 30-month extension study PB-06-003 followed. Patients completing PB-06-001 and PB-06-003 could continue treatment in PB-06-007. Nineteen patients enrolled in PB-06-007 (30 U/kg, n = 8; 60 U/kg, n = 9; dose adjusted, n = 2); 17 completed 5 total years of treatment. In these 3 groups, respectively, taliglucerase alfa resulted in mean decreases in spleen volume (− 8.7, − 6.9, − 12.4 multiples of normal), liver volume (− 0.6, − 0.4, − 0.5 multiples of normal), chitotriosidase activity (− 83.1%, − 93.4%, − 87.9%), and chemokine (C[sbnd]C motif) ligand 18 concentration (− 66.7%, − 83.3%, − 78.9%), as well as mean increases in hemoglobin concentration (+ 2.1, + 2.1, + 1.8 mg/dL) and platelet count (+ 31,871, + 106,800, + 34,000/mm3). The most common adverse events were nasopharyngitis and arthralgia. Most adverse events were mild/moderate; no serious adverse events were considered treatment-related. These results demonstrate continued improvement of disease parameters during 5 years of taliglucerase alfa therapy in 17 treatment-naive patients with no new safety concerns, extending the taliglucerase alfa clinical efficacy and safety dataset. This study was registered at www.clinicaltrials.gov as NCT01422187. © 2016 Elsevier Inc.AnemiaChemokine (C[sbnd]C motif) ligand 18ChitotriosidaseEnzyme replacement therapyGaucher diseaseHepatomegalySplenomegalyTaliglucerase alfaThrombocytopenia