title: Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease.
abstract: Taliglucerase alfa is a plant cell-expressed beta-glucocerebrosidase approved in the United States, Israel, Australia, Canada, and other countries for enzyme replacement therapy in adults with Type 1 Gaucher disease (GD), for treatment of pediatric patients in the United States, Australia, and Canada, and for the hematologic manifestations of Type 3 GD in pediatric patients in Canada. This multicenter, randomized, double-blind, parallel-dose, 12-month study assessed efficacy and safety of taliglucerase alfa in pediatric patients with GD. Eleven children were randomized to taliglucerase alfa 30U/kg (n=6) or 60U/kg (n=5) per infusion every other week. From baseline to month 12, the following changes were noted in the taliglucerase alfa 30-U/kg and 60-U/kg dose groups, respectively: median hemoglobin concentrations increased by 12.2% and 14.2%; the interquartile ranges of median percent change in hemoglobin levels from baseline were 20.6 and 10.4, respectively; mean spleen volume decreased from 22.2 to 14.0 multiples of normal (MN) and from 29.4 to 12.9 MN; mean liver volume decreased from 1.8 to 1.5 MN and from 2.2 to 1.7 MN; platelet counts increased by 30.9% and 73.7%; and chitotriosidase activity was reduced by 58.5% and 66.1%. Nearly all adverse events were mild/moderate, unrelated to treatment, and transient. One patient presented with treatment-related gastroenteritis reported as a serious adverse event due to the need for hospitalization for rehydration. No patient discontinued. These data suggest that taliglucerase alfa has the potential to be a therapeutic treatment option for children with GD. This study was registered at www.clinicaltrials.gov as NCT01132690.
authors.affiliation: Gaucher Clinic, Shaare Zedek Medical Center, 12 Bayit Street, Jerusalem 01931, Israel; Hadassah Medical School - Hebrew University, Jerusalem, Israel. Electronic address: azimran@gmail.com.
authors.forename: Ari
authors.initials: A
authors.lastname: Zimran
authors.affiliation: Instituto Privado de Hematologia e Investigacion Clinica (I.P.H.I.C.), Asuncion, Paraguay. Electronic address: gderlis@conexion.com.py.
authors.forename: Derlis Emilio
authors.initials: DE
authors.lastname: Gonzalez-Rodriguez
authors.affiliation: Gaucher Clinic, Shaare Zedek Medical Center, 12 Bayit Street, Jerusalem 01931, Israel; Hadassah Medical School - Hebrew University, Jerusalem, Israel. Electronic address: abrahamova@szmc.org.il.
authors.forename: Aya
authors.initials: A
authors.lastname: Abrahamov
authors.affiliation: Gaucher Clinic, Shaare Zedek Medical Center, 12 Bayit Street, Jerusalem 01931, Israel; Hadassah Medical School - Hebrew University, Jerusalem, Israel. Electronic address: elstein@szmc.org.il.
authors.forename: Deborah
authors.initials: D
authors.lastname: Elstein
authors.affiliation: Protalix BioTherapeutics, 2 Snunit Street, Science Park, POB 455, Carmiel 20100, Israel. Electronic address: Alona.Paz@protalix.com.
authors.forename: Alona
authors.initials: A
authors.lastname: Paz
authors.affiliation: Protalix BioTherapeutics, 2 Snunit Street, Science Park, POB 455, Carmiel 20100, Israel. Electronic address: einata@protalix.com.
authors.forename: Einat
authors.initials: E
authors.lastname: Brill-Almon
authors.affiliation: Protalix BioTherapeutics, 2 Snunit Street, Science Park, POB 455, Carmiel 20100, Israel. Electronic address: raul@protalix.com.
authors.forename: Raul
authors.initials: R
authors.lastname: Chertkoff
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D056947:Enzyme Replacement Therapy; D005260:Female; D005776:Gaucher Disease / Q000097:blood* / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000009:adverse effects / Q000627:therapeutic use*; D006454:Hemoglobins / Q000378:metabolism*; D006801:Humans; D008297:Male
chemical_list: D006454:Hemoglobins; D005962:Glucosylceramidase; C582473:taliglucerase alfa
keywords: Enzyme replacement therapy; Gaucher disease; Pediatrics; Taliglucerase alfa