{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T08:45:07Z","timestamp":1792658707383,"version":"4.5.0"},"reference-count":33,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2012,9,14]],"date-time":"2012-09-14T00:00:00Z","timestamp":1347580800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2013,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>We studied the effect of long\u2010term alglucerase\/imiglucerase (Ceredase\u00ae\/Cerezyme\u00ae, Genzyme, a Sanofi company, Cambridge, MA, USA) treatment on hematological, visceral, and bone manifestations of Gaucher disease type 1 (GD1).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The International Collaborative Gaucher Group (ICGG) Gaucher Registry identified GD1 patients treated with alglucerase\/imiglucerase who had dose and clinical data at first infusion and after 10 years of follow\u2010up. Data for hemoglobin, platelet count, organ volumes, bone pain, and bone crisis were analyzed. Tests of the null hypothesis (no change from first infusion to 10 years) were performed using<jats:italic> t<\/jats:italic> tests for within\u2010patient absolute change in continuous measurements and McNemar\/chi\u2010square tests for change in distributions using categorical values. An alpha level of 0.05 designated statistical significance.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>As of October 2011, 557 nonsplenectomized and 200 splenectomized patients met the inclusion criteria. The majority of GD1 patients had at least one N370S allele. Compared with nonsplenectomized patients at first infusion, splenectomized patients had lower percentages of anemia (26.0 % vs. 42.8 %) and thrombocytopenia (14.2 % vs. 76.3 %), similar percentages of moderate or severe hepatomegaly (81.2 % vs. 80.0 %), and higher percentages of bone pain (88.9 % vs. 52.4 %) and bone crises (38.3 % vs. 16.0 %). After 10 years, both groups showed significant (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.05) improvements in mean hemoglobin levels, platelet count, liver, and spleen (nonsplenectomized) volumes, and bone crises. Initial dosing in both groups ranged from &lt;15 U\/kg to \u226490 U\/kg every 2 weeks. After 10 years, the majority was receiving 15 to \u226445 U\/kg every 2 weeks.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Ten years of imiglucerase treatment results in sustainable improvements in all GD1 parameters.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-012-9528-4","type":"journal-article","created":{"date-parts":[[2012,9,13]],"date-time":"2012-09-13T14:21:00Z","timestamp":1347546060000},"page":"543-553","source":"Crossref","is-referenced-by-count":122,"title":["Long\u2010term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment"],"prefix":"10.1002","volume":"36","author":[{"given":"Neal J.","family":"Weinreb","sequence":"first","affiliation":[{"name":"University Research Foundation for Lysosomal Storage Diseases, Inc, Northwest Oncology Hematology Associates PA 8170 Royal Palm Boulevard Coral Springs FL 33065 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jack","family":"Goldblatt","sequence":"additional","affiliation":[{"name":"Genetic Services &amp; Familial Cancer Program of Western Australia, School of Pediatrics and Child Health University of Western Australia Perth WA Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacobo","family":"Villalobos","sequence":"additional","affiliation":[{"name":"Universidad Central de Venezuela Caracas Venezuela"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Charrow","sequence":"additional","affiliation":[{"name":"Division of Genetics, Birth Defects and Metabolism, Ann and Robert H. Lurie Children's Hospital of Chicago Chicago IL USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. Alexander","family":"Cole","sequence":"additional","affiliation":[{"name":"Genzyme, a Sanofi company Cambridge MA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcelo","family":"Kerstenetzky","sequence":"additional","affiliation":[{"name":"Instituto de Medicina Integral Professor Fernando Figueira Recife Pernambuco Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephan","family":"vom Dahl","sequence":"additional","affiliation":[{"name":"Klinik f\u00fcr Innere Medizin, St. Franziskus\u2010Hospital Cologne Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla","family":"Hollak","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Division of Endocrinology and Metabolism, Academic Medical Center University of Amsterdam Amsterdam The 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