{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T02:22:19Z","timestamp":1790043739308,"version":"4.0.1"},"reference-count":22,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2013,2,6]],"date-time":"2013-02-06T00:00:00Z","timestamp":1360108800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2013,3]]},"abstract":"<jats:p>Type 1 Gaucher disease (GD1), resulting from glucocerebrosidase deficiency, leads to splenomegaly, hepatomegaly, anemia, thrombocytopenia, and bone involvement. Current standard treatment is enzyme replacement therapy. Velaglucerase alfa is an enzyme replacement product for GD1, with the same amino acid sequence as naturally occurring human glucocerebrosidase. This multinational, Phase 3 trial evaluated the efficacy and safety of two doses of velaglucerase alfa in 25 treatment\u2010na\u00efve, anemic patients with GD1 (4\u201362 years of age), randomized to intravenous velaglucerase alfa 60 U\/kg (<jats:italic>n<\/jats:italic>=12) or 45 U\/kg body weight (<jats:italic>n<\/jats:italic>=13) every other week for 12 months. The primary endpoint was change from baseline in hemoglobin concentration in the 60 U\/kg arm. At 12 months, mean hemoglobin concentrations increased from baseline [60 U\/kg: +23.3%; +2.43 g\/dL (<jats:italic>P<\/jats:italic>&lt;0.001); 45 U\/kg: +23.8%; +2.44 g\/dL (<jats:italic>P<\/jats:italic>&lt;0.001)], as did mean platelet counts [60 U\/kg: +65.9%; +50.9 \u00d7 10<jats:sup>9<\/jats:sup>\/L (<jats:italic>P<\/jats:italic>=0.002); 45 U\/kg: +66.4%; +40.9 \u00d7 10<jats:sup>9<\/jats:sup>\/L(<jats:italic>P<\/jats:italic>=0.01)]. Mean splenic volume decreased from baseline [60 U\/kg: \u221250.4%, from 14.0 to 5.8 multiples of normal (MN) (<jats:italic>P<\/jats:italic>=0.003); 45 U\/kg: \u221239.9%, from 14.5 to 9.5 MN (<jats:italic>P<\/jats:italic>=0.009)]. No drug\u2010related serious adverse events or withdrawals were observed. One patient developed antibodies. Velaglucerase alfa was generally well tolerated and effective for adults and children with GD1 in this study. All disease\u2010specific parameters measured demonstrated clinically meaningful improvements after 12 months. Am. J. Hematol. 88:166\u2013171, 2013. \u00a9 2012 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/ajh.23381","type":"journal-article","created":{"date-parts":[[2012,12,19]],"date-time":"2012-12-19T10:04:19Z","timestamp":1355911459000},"page":"166-171","source":"Crossref","is-referenced-by-count":77,"title":["Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double\u2010blind, multinational, Phase 3 study"],"prefix":"10.1002","volume":"88","author":[{"given":"Derlis E.","family":"Gonzalez","sequence":"first","affiliation":[{"name":"Sanatorio Espa\u00f1ol Asunci\u00f3n Paraguay"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hadhami Ben","family":"Turkia","sequence":"additional","affiliation":[{"name":"La Rabta Hospital Tunis Tunisia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena A.","family":"Lukina","sequence":"additional","affiliation":[{"name":"Hematology Research Center Moscow Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isaac","family":"Kisinovsky","sequence":"additional","affiliation":[{"name":"Your Health S. A. BuenosAires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie\u2010Fran\u00e7oise Ben","family":"Dridi","sequence":"additional","affiliation":[{"name":"La Rabta Hospital Tunis Tunisia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deborah","family":"Elstein","sequence":"additional","affiliation":[{"name":"Shaare Zedek Medical Center and Hebrew University\u2010Hadassah Medical School Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Zahrieh","sequence":"additional","affiliation":[{"name":"Shire Human Genetic Therapies, Inc. Lexington Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Crombez","sequence":"additional","affiliation":[{"name":"Shire Human Genetic Therapies, Inc. Lexington Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kiran","family":"Bhirangi","sequence":"additional","affiliation":[{"name":"Shire Human Genetic Therapies, Inc. Lexington Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Norman W.","family":"Barton","sequence":"additional","affiliation":[{"name":"Shire Human Genetic Therapies, Inc. Lexington Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ari","family":"Zimran","sequence":"additional","affiliation":[{"name":"Shaare Zedek Medical Center and Hebrew University\u2010Hadassah Medical School Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2013,2,6]]},"reference":[{"key":"e_1_2_6_2_1","first-page":"3635","volume-title":"The Metabolic and Molecular Bases of Inherited Disease","author":"Beutler E","year":"2001"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0950-3536(97)80033-9"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.7326\/0003-4819-122-1-199501010-00005"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(95)91038-7"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1093\/glycob\/cwp138"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-02-268649"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M301804200"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cca.2007.02.042"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-05-1612"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jim.2011.07.020"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.2307\/2529712"},{"key":"e_1_2_6_13_1","first-page":"65","article-title":"A simple sequentially rejective multiple test procedure","volume":"6","author":"Holm S","year":"1979","journal-title":"Scand J Statist"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0074-7696(06)52001-7"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2005-12-5072"},{"key":"e_1_2_6_16_1","first-page":"724","volume-title":"Current methods of autopsy practice","author":"Ludwig J","year":"1979"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bcmd.2007.02.008"},{"key":"e_1_2_6_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2006.09.003"},{"key":"e_1_2_6_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0009-8981(01)00798-7"},{"key":"e_1_2_6_20_1","doi-asserted-by":"publisher","DOI":"10.1373\/clinchem.2005.061838"},{"key":"e_1_2_6_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2009.12.014"},{"key":"e_1_2_6_22_1","doi-asserted-by":"publisher","DOI":"10.1093\/ndt\/gfn370"},{"key":"e_1_2_6_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bcmd.2010.07.008"}],"container-title":["American Journal of Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fajh.23381","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajh.23381","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,10,16]],"date-time":"2023-10-16T09:26:00Z","timestamp":1697448360000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ajh.23381"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,2,6]]},"references-count":22,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2013,3]]}},"alternative-id":["10.1002\/ajh.23381"],"URL":"https:\/\/doi.org\/10.1002\/ajh.23381","archive":["Portico"],"relation":{},"ISSN":["0361-8609","1096-8652"],"issn-type":[{"value":"0361-8609","type":"print"},{"value":"1096-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2013,2,6]]}}}