title: 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.
authors.affiliation: Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School, Jerusalem, Israel.
authors.forename: Ari
authors.initials: A
authors.lastname: Zimran
authors.affiliation: Pontificia Universidad Catolica de Chile, Santiago, Chile.
authors.forename: Gloria
authors.initials: G
authors.lastname: Durán
authors.affiliation: Royal Free and University College School of Medicine, Royal Free Hospital, London, United Kingdom.
authors.forename: Atul
authors.initials: A
authors.lastname: Mehta
authors.affiliation: CIBERER, Hospital Universitario Miguel Servet, Zaragoza, Spain.
authors.forename: Pilar
authors.initials: P
authors.lastname: Giraldo
authors.affiliation: Rambam Medical Center, Haifa, Israel.
authors.forename: Hanna
authors.initials: H
authors.lastname: Rosenbaum
authors.affiliation: Sapienza University of Rome, Rome, Italy.
authors.forename: Fiorina
authors.initials: F
authors.lastname: Giona
authors.affiliation: Mount Sinai Hospital, Toronto, Ontario, Canada.
authors.forename: Dominick J
authors.initials: DJ
authors.lastname: Amato
authors.affiliation: Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Disease, Belgrade University Medical School, Belgrade, Serbia.
authors.forename: Milan
authors.initials: M
authors.lastname: Petakov
authors.affiliation: Servicio de Hematologia, Centro Médico Nacional Siglo XXI, México City, México.
authors.forename: Eduardo Terreros
authors.initials: ET
authors.lastname: Muñoz
authors.affiliation: Hospital de Especialidades No1, Leon, México.
authors.forename: Sergio Eduardo
authors.initials: SE
authors.lastname: Solorio-Meza
authors.affiliation: Faculty of Health Sciences, University of the Witwatersrand & Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
authors.forename: Peter A
authors.initials: PA
authors.lastname: Cooper
authors.affiliation: Faculty of Health Sciences, University of the Witwatersrand & Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
authors.forename: Sheeba
authors.initials: S
authors.lastname: Varughese
authors.affiliation: Protalix BioTherapeutics, Carmiel, Israel.
authors.forename: Raul
authors.initials: R
authors.lastname: Chertkoff
authors.affiliation: Protalix BioTherapeutics, Carmiel, Israel.
authors.forename: Einat
authors.initials: E
authors.lastname: Brill-Almon
mesh_terms: D000328:Adult; D000368:Aged; D056947:Enzyme Replacement Therapy / Q000009:adverse effects / Q000379:methods; D005260:Female; D005776:Gaucher Disease / Q000150:complications / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D006801:Humans; D008099:Liver / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009929:Organ Size / Q000187:drug effects; D013154:Spleen / Q000473:pathology; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D005962:Glucosylceramidase; C582473:taliglucerase alfa