{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T02:10:56Z","timestamp":1791943856768,"version":"4.3.4"},"reference-count":42,"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:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"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>Background<\/jats:title><jats:p>Dyslipidemia in Gaucher disease includes reduced total, low\u2010density lipoprotein (LDL)\u2010, and high\u2010density lipoprotein (HDL)\u2010cholesterol (C). No prospective analysis of lipid profile changes in treatment\u2010na\u00efve patients under enzyme replacement therapy (ERT) is available.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We analyzed lipid profile changes during ERT in a prospective controlled manner. Twelve treatment\u2010na\u00efve patients, Gaucher disease type 1 (GD1), 29.5 \u00b1 12.9 years, 4M\/8F. Diagnosis was made by enzymatic measurement and mutational analysis. Total\u2010, LDL\u2010, and HDL\u2010C, triglycerides (TG), and LDL subfractions were assessed before the start of ERT with imiglucerase and biannually for 3 years. Patients were matched with healthy controls before and after 3 years of ERT.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>At baseline, we found severely reduced HDL\u2010C concentrations (23.6 \u00b1 5.4 mg\/dl) and enhanced LDL\/HDL ratios (3.1 \u00b1 0.7). HDL\u2010C increased after 6 months (29.2 \u00b1 5.7,<jats:italic>p<\/jats:italic>\u2009=\u20090.023), LDL\/HDL ratio decreased after 30 months (2.5 \u00b1 0.5,<jats:italic>p<\/jats:italic>\u2009=\u20090.039). TG, even not consistently enhanced at baseline (128 \u00b1 31.3 mg\/dl), yet higher than in controls (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001), decreased after 18 months, being comparable with controls after 3 years of ERT. Small, dense LDL (mg\/dl) increased continuously without significant difference to controls. After 3 years of ERT, only reduced HDL\u2010C concentrations persisted as a potentially atherogenic alteration; however, mean concentrations markedly improved (42.9 \u00b1 8.3 mg\/dl,<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001). Lipid parameters correlated with six markers of disease severity.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>This is the first prospective controlled study regarding lipid profile dynamics during ERT (glucocerebrosidase) in initially treatment\u2010na\u00efve GD1 patients. The most important changes were reduced HDL\u2010C and enhanced LDL\/HDL ratio. Their dynamics during ERT and correlations with markers of disease activity suggest that they can be considered markers of disease severity and follow\u2010up in Gaucher patients under treatment.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-012-9529-3","type":"journal-article","created":{"date-parts":[[2012,9,13]],"date-time":"2012-09-13T14:01:06Z","timestamp":1347544866000},"page":"555-563","source":"Crossref","is-referenced-by-count":13,"title":["Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study"],"prefix":"10.1002","volume":"36","author":[{"given":"Anca","family":"Zimmermann","sequence":"first","affiliation":[{"name":"Department of Endocrinology and Metabolic Diseases, 1st Clinic of Internal Medicine Johannes Gutenberg University Langenbeckstr. 1 Mainz 55131 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