{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,20]],"date-time":"2026-02-20T16:05:59Z","timestamp":1771603559511,"version":"3.50.1"},"reference-count":8,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2016,4,24]],"date-time":"2016-04-24T00:00:00Z","timestamp":1461456000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2016,7]]},"abstract":"<jats:p>Taliglucerase alfa is an intravenous enzyme replacement therapy approved for treatment of type 1 Gaucher disease (GD), and is the first available plant cell\u2013expressed recombinant therapeutic protein. Herein, we report long\u2010term safety and efficacy results of taliglucerase alfa in treatment\u2010na\u00efve 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\u2010related 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\u2010month results of taliglucerase alfa in treatment\u2010na\u00efve 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. <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/www.clinicaltrials.gov\">www.clinicaltrials.gov<\/jats:ext-link> identifier NCT00705939. Am. J. Hematol. 91:656\u2013660, 2016. \u00a9 2016 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/ajh.24369","type":"journal-article","created":{"date-parts":[[2016,3,19]],"date-time":"2016-03-19T16:16:27Z","timestamp":1458404187000},"page":"656-660","source":"Crossref","is-referenced-by-count":20,"title":["Long\u2010term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment\u2010na\u00efve patients with Gaucher disease"],"prefix":"10.1002","volume":"91","author":[{"given":"Ari","family":"Zimran","sequence":"first","affiliation":[{"name":"Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School Jerusalem Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gloria","family":"Dur\u00e1n","sequence":"additional","affiliation":[{"name":"Pontificia Universidad Catolica de Chile Santiago Chile"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Atul","family":"Mehta","sequence":"additional","affiliation":[{"name":"Royal Free and University College School of Medicine, Royal Free Hospital London United Kingdom"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pilar","family":"Giraldo","sequence":"additional","affiliation":[{"name":"CIBERER, Hospital Universitario Miguel Servet Zaragoza Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hanna","family":"Rosenbaum","sequence":"additional","affiliation":[{"name":"Rambam Medical Center Haifa Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fiorina","family":"Giona","sequence":"additional","affiliation":[{"name":"Sapienza University of Rome Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dominick J.","family":"Amato","sequence":"additional","affiliation":[{"name":"Mount Sinai Hospital Toronto Ontario Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Milan","family":"Petakov","sequence":"additional","affiliation":[{"name":"Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Disease, Belgrade University Medical School Belgrade Serbia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eduardo Terreros","family":"Mu\u00f1oz","sequence":"additional","affiliation":[{"name":"Servicio de Hematologia Centro M\u00e9dico Nacional Siglo XXI M\u00e9xico City M\u00e9xico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sergio Eduardo","family":"Solorio\u2010Meza","sequence":"additional","affiliation":[{"name":"Hospital de Especialidades No1 Leon M\u00e9xico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter A.","family":"Cooper","sequence":"additional","affiliation":[{"name":"Faculty of Health Sciences University of the Witwatersrand &amp; Charlotte Maxeke Johannesburg Academic Hospital Johannesburg South Africa"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sheeba","family":"Varughese","sequence":"additional","affiliation":[{"name":"Faculty of Health Sciences University of the Witwatersrand &amp; Charlotte Maxeke Johannesburg Academic Hospital Johannesburg South Africa"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Raul","family":"Chertkoff","sequence":"additional","affiliation":[{"name":"Protalix BioTherapeutics Carmiel Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Einat","family":"Brill\u2010Almon","sequence":"additional","affiliation":[{"name":"Protalix BioTherapeutics Carmiel Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2016,4,24]]},"reference":[{"key":"e_1_2_6_2_1","volume-title":"Elelyso [package insert]","year":"2015"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/nbt0612-472"},{"key":"e_1_2_6_4_1","unstructured":"National Institutes of Health. A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients with Gaucher Disease NCT00376168; 2012. Available at:http:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT00376168 accessed on June 25 2015."},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-07-366955"},{"key":"e_1_2_6_6_1","unstructured":"Clinicaltrials.gov. 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