{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T15:34:11Z","timestamp":1791646451260,"version":"4.3.4"},"reference-count":21,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2020,6,24]],"date-time":"2020-06-24T00:00:00Z","timestamp":1592956800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2020,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Eliglustat is a first\u2010line oral therapy for adults with Gaucher disease type 1 (GD1) with extensive, intermediate, or poor CYP2D6\u2010metabolizer phenotypes (90% of patients). We report real\u2010world outcomes after 2\u2009years of eliglustat therapy in the International Collaborative Gaucher Group Gaucher Registry (NCT00358943). As of January 2019, baseline and 2\u2010year data (\u00b11 year) were available for 231 eliglustat\u2010treated GD1 patients: 19 treatment\u2010na\u00efve (zero splenectomized) and 212 ERT patients who switched to eliglustat (36 splenectomized). Most patients (89%) were from the United States, where eliglustat was first approved. In treatment\u2010na\u00efve patients, mean hemoglobin increased from 12.4 to 13.4 g\/dL (<jats:italic>P<\/jats:italic>\u2009=\u2009.004, n\u2009=\u200918), mean platelet count increased from 113 to 156\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L (<jats:italic>P<\/jats:italic>\u2009&lt;\u2009.001, n\u2009=\u200917); mean spleen volume decreased from 7.4 to 3.5 multiples of normal (MN) (<jats:italic>P<\/jats:italic>\u2009=\u2009.02, n\u2009=\u20097); mean liver volume remained normal (n\u2009=\u20097), and median spine Z\u2010score was unchanged (\u22121.3 to \u22121.2, n\u2009=\u20096). In non\u2010splenectomized switch patients, mean hemoglobin remained stable\/non\u2010anemic (n\u2009=\u2009167); mean platelet count remained stable\/normal (n\u2009=\u2009165); mean spleen volume decreased from 3.3 to 2.8 MN (<jats:italic>P<\/jats:italic>\u2009&lt;\u2009.001, n\u2009=\u200964); mean liver volume remained normal (n\u2009=\u200963), and median lumbar spine Z\u2010score improved from \u22120.7 to \u22120.4 (<jats:italic>P<\/jats:italic>\u2009=\u2009.014, n\u2009=\u200968). In splenectomized switch patients, mean hemoglobin remained stable\/non\u2010anemic (n\u2009=\u200931); mean platelet count increased from 297 to 324\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L (non\u2010significant, n\u2009=\u200929); mean liver volume remained normal (n\u2009=\u200913); median spine Z\u2010score improved from \u22120.8 to \u22120.6 (non\u2010significant, n\u2009=\u200911). Median chitotriosidase decreased in all groups (<jats:italic>P<\/jats:italic>\u2009&lt;\u2009.01 for all). These real\u2010world results are consistent with eliglustat clinical trial results demonstrating long\u2010term benefit in treatment\u2010na\u00efve patients and stability in ERT switch patients.<\/jats:p>","DOI":"10.1002\/ajh.25875","type":"journal-article","created":{"date-parts":[[2020,5,21]],"date-time":"2020-05-21T22:38:56Z","timestamp":1590100736000},"page":"1038-1046","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":32,"title":["Real\u2010world effectiveness of eliglustat in treatment\u2010na\u00efve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry"],"prefix":"10.1002","volume":"95","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3447-6421","authenticated-orcid":false,"given":"Pramod K.","family":"Mistry","sequence":"first","affiliation":[{"name":"Department of Internal Medicine Yale University  New Haven Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manisha","family":"Balwani","sequence":"additional","affiliation":[{"name":"Department of Genetics and Genomic Sciences Icahn School of Medicine at Mount Sinai  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Charrow","sequence":"additional","affiliation":[{"name":"Division of Genetics, Ann &amp; Robert H. Lurie Children's Hospital of Chicago, and Department of Pediatrics, Northwestern University Feinberg School of Medicine  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Priya","family":"Kishnani","sequence":"additional","affiliation":[{"name":"Division of Medical Genetics, Department of Pediatrics Duke University  Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claus","family":"Niederau","sequence":"additional","affiliation":[{"name":"Department of Medicine Katholisches Klinikum Oberhausen  Oberhausen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa H.","family":"Underhill","sequence":"additional","affiliation":[{"name":"Global Medical Affairs, Rare Hematology Sanofi Genzyme  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monica R.","family":"McClain","sequence":"additional","affiliation":[{"name":"Global Medical Affairs, Rare Hematology Sanofi Genzyme  Cambridge Massachusetts USA"},{"name":"American College of Medical Genetics and Genomics  Bethesda Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,6,24]]},"reference":[{"key":"e_1_2_8_2_1","unstructured":"GrabowskiGA PetskoGA KolodnyEH. Gaucher disease. In: Valle D Beaudet AL Vogelstein B et al. eds.OMMBID: The Online Metabolic and Molecular Bases of Inherited Disease. New York NY: McGraw\u2010Hill; 2013.http:\/\/ommbid.mhmedical.com\/content.aspx?bookid=2709&sectionid=225546056. Accessed August 22 2019."},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00431-003-1362-0"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1001\/archinte.160.18.2835"},{"issue":"6","key":"e_1_2_8_5_1","first-page":"1","article-title":"Disease state awareness in Gaucher disease: a Q&A expert roundtable discussion","volume":"10","author":"Mistry PK","year":"2012","journal-title":"Clin Adv Hematol Oncol"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)61522-6"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature21368"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-010-9055-0"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1600-0404.2006.00774.x"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awq198"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1038\/clpt.2013.2"},{"key":"e_1_2_8_12_1","unstructured":"CERDELGA (eliglustat) [package insert]. Waterford Ireland: Genzyme Corporation A Sanofi Company; 2018.http:\/\/www.cerdelga.com\/pdf\/cerdelga_prescribing_information.pdf. 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