{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T01:37:49Z","timestamp":1790386669669,"version":"4.1.0"},"reference-count":14,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2016,5,18]],"date-time":"2016-05-18T00:00:00Z","timestamp":1463529600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/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 the first available plant cell\u2010expressed human recombinant therapeutic protein. It is indicated for treatment of patients with type 1 Gaucher disease (GD) in adult and pediatric patients in several countries. Study PB\u201006\u2010002 examined the safety and efficacy of taliglucerase alfa for 9 months in patients who previously received imiglucerase. The results of adult patients from Study PB\u201006\u2010002 who continued receiving taliglucerase alfa in extension Study PB\u201006\u2010003 for up to 36 months are reported here. Eighteen patients received at least one dose of taliglucerase alfa in Study PB\u201006\u2010003; 10 patients completed 36 total months of therapy, and four patients who transitioned to commercial drug completed 30\u201333 months of treatment. In patients who completed 36 total months of treatment, mean percent (\u00b1standard error) changes from baseline\/time of switch to taliglucerase alfa to 36 months were as follows: hemoglobin concentration, \u22121.0% (\u00b11.9%; <jats:italic>n<\/jats:italic>\u2009=\u200910); platelet count, +9.3% (\u00b19.8%; <jats:italic>n<\/jats:italic>\u2009=\u200910); spleen volume measured in multiples of normal (MN), \u221219.8% (\u00b19.9%; <jats:italic>n<\/jats:italic>\u2009=\u20097); liver volume measured in MN, +0.9% (\u00b15.4%; <jats:italic>n<\/jats:italic>\u2009=\u20098); chitotriosidase activity, \u221251.5% (\u00b18.1%; <jats:italic>n<\/jats:italic>\u2009=\u200910); and CCL18 concentration, \u221236.5 (\u00b18.0%; <jats:italic>n<\/jats:italic>\u2009=\u200910). Four patients developed antidrug antibodies, including one with evidence of neutralizing activity in vitro. All treatment\u2010related adverse events were mild or moderate and transient. The 36\u2010month results of switching from imiglucerase to taliglucerase alfa treatment in adults with GD provide further data on the clinical safety and efficacy of taliglucerase alfa beyond the initial 9 months of the original study. <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. 91:661\u2013665, 2016. \u00a9 2016 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/ajh.24399","type":"journal-article","created":{"date-parts":[[2016,4,22]],"date-time":"2016-04-22T21:50:00Z","timestamp":1461361800000},"page":"661-665","source":"Crossref","is-referenced-by-count":27,"title":["Enzyme replacement therapy with taliglucerase alfa: 36\u2010month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase"],"prefix":"10.1002","volume":"91","author":[{"given":"Gregory M.","family":"Pastores","sequence":"first","affiliation":[{"name":"National Centre for Inherited Metabolic Disorders Mater Misericordiae University Hospital Dublin Ireland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suma P.","family":"Shankar","sequence":"additional","affiliation":[{"name":"Department of Genetics Emory University School of Medicine Atlanta Georgia"},{"name":"Department of Ophthalmology Emory University School of Medicine Atlanta Georgia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Milan","family":"Petakov","sequence":"additional","affiliation":[{"name":"Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Diseases, Belgrade University Medical School Belgrade Serbia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pilar","family":"Giraldo","sequence":"additional","affiliation":[{"name":"CIBERER Hospital Universitario Miguel Servet Zaragoza Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanna","family":"Rosenbaum","sequence":"additional","affiliation":[{"name":"Rambam Medical Center Haifa Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dominick J.","family":"Amato","sequence":"additional","affiliation":[{"name":"Mount Sinai Hospital Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Szer","sequence":"additional","affiliation":[{"name":"Royal Melbourne Hospital Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raul","family":"Chertkoff","sequence":"additional","affiliation":[{"name":"Protalix BioTherapeutics Carmiel Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Einat","family":"Brill\u2010Almon","sequence":"additional","affiliation":[{"name":"Protalix BioTherapeutics Carmiel Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ari","family":"Zimran","sequence":"additional","affiliation":[{"name":"Gaucher Clinic Shaare Zedek Medical Center Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2016,5,18]]},"reference":[{"key":"e_1_2_6_2_1","volume-title":"The Online Metabolic and Molecular Basis of Inherited Disease","author":"Grabowski GA","year":"2010"},{"key":"e_1_2_6_3_1","volume-title":"The Online Metabolic and Molecular Bases of Inherited Disease","author":"Grabowski GA","year":"2010"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199105233242104"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bcmd.2009.09.006"},{"key":"e_1_2_6_6_1","first-page":"81","article-title":"Imiglucerase in the treatment of Gaucher disease: A history and perspective","volume":"6","author":"Deegan PB","year":"2012","journal-title":"Drug Des Dev Ther"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/nbt0612-472"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2014.02.011"},{"key":"e_1_2_6_9_1","unstructured":"National Institutes of Health. Switchover trial from imiglucerase to plant cell expressed recombinant human glucocerebrosidase [NCT00712348]. June 10 2013; Available at:http:\/\/clinicaltrials.gov\/ct2\/show\/NCT00712348?term=nct00712348&rank=1. Accessed: January 27 2015."},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bcmd.2014.05.004"},{"key":"e_1_2_6_11_1","unstructured":"National Institutes of Health. Plant cell expressed recombinant human glucocerebrosidase extension trial [NCT00705939]. August 13 2013; Available at:http:\/\/clinicaltrials.gov\/ct2\/show\/NCT00705939?term=nct00705939&rank=1. Accessed: October 8 2015."},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-07-366955"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bcmd.2010.10.009"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1053\/j.seminhematol.2004.07.009"},{"key":"e_1_2_6_15_1","unstructured":"Elelyso [package insert]. New York NY: Pfizer Labs;2015."}],"container-title":["American Journal of Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fajh.24399","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajh.24399","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/ajh.24399","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajh.24399","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,23]],"date-time":"2023-09-23T20:58:36Z","timestamp":1695502716000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ajh.24399"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,5,18]]},"references-count":14,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2016,7]]}},"alternative-id":["10.1002\/ajh.24399"],"URL":"https:\/\/doi.org\/10.1002\/ajh.24399","archive":["Portico"],"relation":{},"ISSN":["0361-8609","1096-8652"],"issn-type":[{"value":"0361-8609","type":"print"},{"value":"1096-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,5,18]]}}}