Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study
Background Dyslipidemia in Gaucher disease includes reduced total, low-density lipoprotein (LDL)-, and high-density lipoprotein (HDL)-cholesterol (C). No prospective analysis of lipid profile changes in treatment-naïve patients under enzyme replacement therapy (ERT) is available. Methods We analyzed...
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| Veröffentlicht in: | Journal of inherited metabolic disease 2013-05, Vol.36 (3), p.555-563 |
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| creator | Zimmermann, Anca Grigorescu-Sido, Paula Rossmann, Heidi Lackner, Karl J. Drugan, Cristina Al Khzouz, Camelia Bucerzan, Simona Naşcu, Ioana Zimmermann, Tim Leucuţa, Daniel Weber, Matthias M. |
| description | Background
Dyslipidemia in Gaucher disease includes reduced total, low-density lipoprotein (LDL)-, and high-density lipoprotein (HDL)-cholesterol (C). No prospective analysis of lipid profile changes in treatment-naïve patients under enzyme replacement therapy (ERT) is available.
Methods
We analyzed lipid profile changes during ERT in a prospective controlled manner. Twelve treatment-naïve patients, Gaucher disease type 1 (GD1), 29.5 ± 12.9 years, 4M/8F. Diagnosis was made by enzymatic measurement and mutational analysis. Total-, LDL-, and HDL-C, 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.
Results
At baseline, we found severely reduced HDL-C concentrations (23.6 ± 5.4 mg/dl) and enhanced LDL/HDL ratios (3.1 ± 0.7). HDL-C increased after 6 months (29.2 ± 5.7,
p
= 0.023), LDL/HDL ratio decreased after 30 months (2.5 ± 0.5,
p
= 0.039). TG, even not consistently enhanced at baseline (128 ± 31.3 mg/dl), yet higher than in controls (
p
|
| doi_str_mv | 10.1007/s10545-012-9529-3 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_1350152165</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2983536901</sourcerecordid><originalsourceid>FETCH-LOGICAL-c4505-2d577e772dd341f48e2a0bb38eb67b181930367163a564387f087af92bdb4a623</originalsourceid><addsrcrecordid>eNqFkV2L1TAQhoMo7nH1B3gjARG8qWaSJmm9k11dV1YE0eswbaeeLG1ObVqX-gf826b0-IEgXgXC887My8PYQxDPQAj7PILQuc4EyKzUsszULbYDbVUmjdG32U5ADllRan3C7sV4LYQoC63vshMpS2usMTv2_XwJ2Pua13sMnynyQ8s7P_iGD-Oh9R1xH_iHQ4_BY-ADTp7CFPmNn_b8Aud6TyNvfCSMxKdlIA58Dk36pPBt6YmPNHRYU59SfEowDssLjuvwOFA9-a_E4zQ3y312p8Uu0oPje8o-vX718exNdvX-4vLs5VVW51roTDbaWrJWNo3Koc0LkiiqShVUGVtBAaUSylgwCrXJVWFbUVhsS1k1VY5GqlP2dJubLvgyU5xc72NNXYeBDnN0oLQALcHohD7-C70-zGNI162UMYUCCYmCjapTpThS64bR9zguDoRbLbnNkkuW3GrJqZR5dJw8Vz01vxI_tSTgyRHAWGPXjhhqH39z1ghdwLrcbtxNMrX8f7N7e_nuXGi9lpNbMqZQEj_-Ue6fd_8Aa5W6Xg</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>1356683121</pqid></control><display><type>article</type><title>Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study</title><source>MEDLINE</source><source>Wiley Journals</source><source>SpringerLink Journals - AutoHoldings</source><creator>Zimmermann, Anca ; Grigorescu-Sido, Paula ; Rossmann, Heidi ; Lackner, Karl J. ; Drugan, Cristina ; Al Khzouz, Camelia ; Bucerzan, Simona ; Naşcu, Ioana ; Zimmermann, Tim ; Leucuţa, Daniel ; Weber, Matthias M.</creator><creatorcontrib>Zimmermann, Anca ; Grigorescu-Sido, Paula ; Rossmann, Heidi ; Lackner, Karl J. ; Drugan, Cristina ; Al Khzouz, Camelia ; Bucerzan, Simona ; Naşcu, Ioana ; Zimmermann, Tim ; Leucuţa, Daniel ; Weber, Matthias M.</creatorcontrib><description>Background
Dyslipidemia in Gaucher disease includes reduced total, low-density lipoprotein (LDL)-, and high-density lipoprotein (HDL)-cholesterol (C). No prospective analysis of lipid profile changes in treatment-naïve patients under enzyme replacement therapy (ERT) is available.
Methods
We analyzed lipid profile changes during ERT in a prospective controlled manner. Twelve treatment-naïve patients, Gaucher disease type 1 (GD1), 29.5 ± 12.9 years, 4M/8F. Diagnosis was made by enzymatic measurement and mutational analysis. Total-, LDL-, and HDL-C, 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.
Results
At baseline, we found severely reduced HDL-C concentrations (23.6 ± 5.4 mg/dl) and enhanced LDL/HDL ratios (3.1 ± 0.7). HDL-C increased after 6 months (29.2 ± 5.7,
p
= 0.023), LDL/HDL ratio decreased after 30 months (2.5 ± 0.5,
p
= 0.039). TG, even not consistently enhanced at baseline (128 ± 31.3 mg/dl), yet higher than in controls (
p
< 0.001), 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-C concentrations persisted as a potentially atherogenic alteration; however, mean concentrations markedly improved (42.9 ± 8.3 mg/dl,
p
< 0.001). Lipid parameters correlated with six markers of disease severity.
Conclusions
This is the first prospective controlled study regarding lipid profile dynamics during ERT (glucocerebrosidase) in initially treatment-naïve GD1 patients. The most important changes were reduced HDL-C 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-up in Gaucher patients under treatment.</description><identifier>ISSN: 0141-8955</identifier><identifier>EISSN: 1573-2665</identifier><identifier>DOI: 10.1007/s10545-012-9529-3</identifier><identifier>PMID: 22976766</identifier><identifier>CODEN: JIMDDP</identifier><language>eng</language><publisher>Dordrecht: Springer Netherlands</publisher><subject>Adolescent ; Adult ; Biochemistry ; Biological and medical sciences ; Enzyme Replacement Therapy ; Errors of metabolism ; Female ; Follow-Up Studies ; Gaucher Disease - blood ; Gaucher Disease - drug therapy ; Gaucher Disease - epidemiology ; Glucosylceramidase - administration & dosage ; Glucosylceramidase - therapeutic use ; Human Genetics ; Humans ; Internal Medicine ; Lipid Metabolism - drug effects ; Lipids (lysosomal enzyme disorders, storage diseases) ; Lipids - blood ; Male ; Medical genetics ; Medical sciences ; Medicine ; Medicine & Public Health ; Metabolic Diseases ; Middle Aged ; Original Article ; Pediatrics ; Prospective Studies ; Romania - epidemiology ; Young Adult</subject><ispartof>Journal of inherited metabolic disease, 2013-05, Vol.36 (3), p.555-563</ispartof><rights>SSIEM and Springer 2012</rights><rights>2013 SSIEM</rights><rights>2014 INIST-CNRS</rights><rights>SSIEM and Springer Science+Business Media Dordrecht 2013</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4505-2d577e772dd341f48e2a0bb38eb67b181930367163a564387f087af92bdb4a623</citedby><cites>FETCH-LOGICAL-c4505-2d577e772dd341f48e2a0bb38eb67b181930367163a564387f087af92bdb4a623</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s10545-012-9529-3$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s10545-012-9529-3$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,780,784,1417,27924,27925,41488,42557,45574,45575,51319</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=27605811$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/22976766$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Zimmermann, Anca</creatorcontrib><creatorcontrib>Grigorescu-Sido, Paula</creatorcontrib><creatorcontrib>Rossmann, Heidi</creatorcontrib><creatorcontrib>Lackner, Karl J.</creatorcontrib><creatorcontrib>Drugan, Cristina</creatorcontrib><creatorcontrib>Al Khzouz, Camelia</creatorcontrib><creatorcontrib>Bucerzan, Simona</creatorcontrib><creatorcontrib>Naşcu, Ioana</creatorcontrib><creatorcontrib>Zimmermann, Tim</creatorcontrib><creatorcontrib>Leucuţa, Daniel</creatorcontrib><creatorcontrib>Weber, Matthias M.</creatorcontrib><title>Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study</title><title>Journal of inherited metabolic disease</title><addtitle>J Inherit Metab Dis</addtitle><addtitle>J Inherit Metab Dis</addtitle><description>Background
Dyslipidemia in Gaucher disease includes reduced total, low-density lipoprotein (LDL)-, and high-density lipoprotein (HDL)-cholesterol (C). No prospective analysis of lipid profile changes in treatment-naïve patients under enzyme replacement therapy (ERT) is available.
Methods
We analyzed lipid profile changes during ERT in a prospective controlled manner. Twelve treatment-naïve patients, Gaucher disease type 1 (GD1), 29.5 ± 12.9 years, 4M/8F. Diagnosis was made by enzymatic measurement and mutational analysis. Total-, LDL-, and HDL-C, 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.
Results
At baseline, we found severely reduced HDL-C concentrations (23.6 ± 5.4 mg/dl) and enhanced LDL/HDL ratios (3.1 ± 0.7). HDL-C increased after 6 months (29.2 ± 5.7,
p
= 0.023), LDL/HDL ratio decreased after 30 months (2.5 ± 0.5,
p
= 0.039). TG, even not consistently enhanced at baseline (128 ± 31.3 mg/dl), yet higher than in controls (
p
< 0.001), 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-C concentrations persisted as a potentially atherogenic alteration; however, mean concentrations markedly improved (42.9 ± 8.3 mg/dl,
p
< 0.001). Lipid parameters correlated with six markers of disease severity.
Conclusions
This is the first prospective controlled study regarding lipid profile dynamics during ERT (glucocerebrosidase) in initially treatment-naïve GD1 patients. The most important changes were reduced HDL-C 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-up in Gaucher patients under treatment.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Biochemistry</subject><subject>Biological and medical sciences</subject><subject>Enzyme Replacement Therapy</subject><subject>Errors of metabolism</subject><subject>Female</subject><subject>Follow-Up Studies</subject><subject>Gaucher Disease - blood</subject><subject>Gaucher Disease - drug therapy</subject><subject>Gaucher Disease - epidemiology</subject><subject>Glucosylceramidase - administration & dosage</subject><subject>Glucosylceramidase - therapeutic use</subject><subject>Human Genetics</subject><subject>Humans</subject><subject>Internal Medicine</subject><subject>Lipid Metabolism - drug effects</subject><subject>Lipids (lysosomal enzyme disorders, storage diseases)</subject><subject>Lipids - blood</subject><subject>Male</subject><subject>Medical genetics</subject><subject>Medical sciences</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Metabolic Diseases</subject><subject>Middle Aged</subject><subject>Original Article</subject><subject>Pediatrics</subject><subject>Prospective Studies</subject><subject>Romania - epidemiology</subject><subject>Young Adult</subject><issn>0141-8955</issn><issn>1573-2665</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2013</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><recordid>eNqFkV2L1TAQhoMo7nH1B3gjARG8qWaSJmm9k11dV1YE0eswbaeeLG1ObVqX-gf826b0-IEgXgXC887My8PYQxDPQAj7PILQuc4EyKzUsszULbYDbVUmjdG32U5ADllRan3C7sV4LYQoC63vshMpS2usMTv2_XwJ2Pua13sMnynyQ8s7P_iGD-Oh9R1xH_iHQ4_BY-ADTp7CFPmNn_b8Aud6TyNvfCSMxKdlIA58Dk36pPBt6YmPNHRYU59SfEowDssLjuvwOFA9-a_E4zQ3y312p8Uu0oPje8o-vX718exNdvX-4vLs5VVW51roTDbaWrJWNo3Koc0LkiiqShVUGVtBAaUSylgwCrXJVWFbUVhsS1k1VY5GqlP2dJubLvgyU5xc72NNXYeBDnN0oLQALcHohD7-C70-zGNI162UMYUCCYmCjapTpThS64bR9zguDoRbLbnNkkuW3GrJqZR5dJw8Vz01vxI_tSTgyRHAWGPXjhhqH39z1ghdwLrcbtxNMrX8f7N7e_nuXGi9lpNbMqZQEj_-Ue6fd_8Aa5W6Xg</recordid><startdate>201305</startdate><enddate>201305</enddate><creator>Zimmermann, Anca</creator><creator>Grigorescu-Sido, Paula</creator><creator>Rossmann, Heidi</creator><creator>Lackner, Karl J.</creator><creator>Drugan, Cristina</creator><creator>Al Khzouz, Camelia</creator><creator>Bucerzan, Simona</creator><creator>Naşcu, Ioana</creator><creator>Zimmermann, Tim</creator><creator>Leucuţa, Daniel</creator><creator>Weber, Matthias M.</creator><general>Springer Netherlands</general><general>Springer</general><general>Blackwell Publishing Ltd</general><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7QP</scope><scope>7TK</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>BENPR</scope><scope>CCPQU</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>K9.</scope><scope>M0S</scope><scope>M1P</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>7X8</scope></search><sort><creationdate>201305</creationdate><title>Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study</title><author>Zimmermann, Anca ; Grigorescu-Sido, Paula ; Rossmann, Heidi ; Lackner, Karl J. ; Drugan, Cristina ; Al Khzouz, Camelia ; Bucerzan, Simona ; Naşcu, Ioana ; Zimmermann, Tim ; Leucuţa, Daniel ; Weber, Matthias M.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4505-2d577e772dd341f48e2a0bb38eb67b181930367163a564387f087af92bdb4a623</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2013</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Biochemistry</topic><topic>Biological and medical sciences</topic><topic>Enzyme Replacement Therapy</topic><topic>Errors of metabolism</topic><topic>Female</topic><topic>Follow-Up Studies</topic><topic>Gaucher Disease - blood</topic><topic>Gaucher Disease - drug therapy</topic><topic>Gaucher Disease - epidemiology</topic><topic>Glucosylceramidase - administration & dosage</topic><topic>Glucosylceramidase - therapeutic use</topic><topic>Human Genetics</topic><topic>Humans</topic><topic>Internal Medicine</topic><topic>Lipid Metabolism - drug effects</topic><topic>Lipids (lysosomal enzyme disorders, storage diseases)</topic><topic>Lipids - blood</topic><topic>Male</topic><topic>Medical genetics</topic><topic>Medical sciences</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Metabolic Diseases</topic><topic>Middle Aged</topic><topic>Original Article</topic><topic>Pediatrics</topic><topic>Prospective Studies</topic><topic>Romania - epidemiology</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Zimmermann, Anca</creatorcontrib><creatorcontrib>Grigorescu-Sido, Paula</creatorcontrib><creatorcontrib>Rossmann, Heidi</creatorcontrib><creatorcontrib>Lackner, Karl J.</creatorcontrib><creatorcontrib>Drugan, Cristina</creatorcontrib><creatorcontrib>Al Khzouz, Camelia</creatorcontrib><creatorcontrib>Bucerzan, Simona</creatorcontrib><creatorcontrib>Naşcu, Ioana</creatorcontrib><creatorcontrib>Zimmermann, Tim</creatorcontrib><creatorcontrib>Leucuţa, Daniel</creatorcontrib><creatorcontrib>Weber, Matthias M.</creatorcontrib><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Calcium & Calcified Tissue Abstracts</collection><collection>Neurosciences Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Medical Database (Alumni Edition)</collection><collection>ProQuest Pharma Collection</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>ProQuest Central</collection><collection>ProQuest One Community College</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central China</collection><collection>MEDLINE - Academic</collection><jtitle>Journal of inherited metabolic disease</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Zimmermann, Anca</au><au>Grigorescu-Sido, Paula</au><au>Rossmann, Heidi</au><au>Lackner, Karl J.</au><au>Drugan, Cristina</au><au>Al Khzouz, Camelia</au><au>Bucerzan, Simona</au><au>Naşcu, Ioana</au><au>Zimmermann, Tim</au><au>Leucuţa, Daniel</au><au>Weber, Matthias M.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study</atitle><jtitle>Journal of inherited metabolic disease</jtitle><stitle>J Inherit Metab Dis</stitle><addtitle>J Inherit Metab Dis</addtitle><date>2013-05</date><risdate>2013</risdate><volume>36</volume><issue>3</issue><spage>555</spage><epage>563</epage><pages>555-563</pages><issn>0141-8955</issn><eissn>1573-2665</eissn><coden>JIMDDP</coden><abstract>Background
Dyslipidemia in Gaucher disease includes reduced total, low-density lipoprotein (LDL)-, and high-density lipoprotein (HDL)-cholesterol (C). No prospective analysis of lipid profile changes in treatment-naïve patients under enzyme replacement therapy (ERT) is available.
Methods
We analyzed lipid profile changes during ERT in a prospective controlled manner. Twelve treatment-naïve patients, Gaucher disease type 1 (GD1), 29.5 ± 12.9 years, 4M/8F. Diagnosis was made by enzymatic measurement and mutational analysis. Total-, LDL-, and HDL-C, 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.
Results
At baseline, we found severely reduced HDL-C concentrations (23.6 ± 5.4 mg/dl) and enhanced LDL/HDL ratios (3.1 ± 0.7). HDL-C increased after 6 months (29.2 ± 5.7,
p
= 0.023), LDL/HDL ratio decreased after 30 months (2.5 ± 0.5,
p
= 0.039). TG, even not consistently enhanced at baseline (128 ± 31.3 mg/dl), yet higher than in controls (
p
< 0.001), 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-C concentrations persisted as a potentially atherogenic alteration; however, mean concentrations markedly improved (42.9 ± 8.3 mg/dl,
p
< 0.001). Lipid parameters correlated with six markers of disease severity.
Conclusions
This is the first prospective controlled study regarding lipid profile dynamics during ERT (glucocerebrosidase) in initially treatment-naïve GD1 patients. The most important changes were reduced HDL-C 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-up in Gaucher patients under treatment.</abstract><cop>Dordrecht</cop><pub>Springer Netherlands</pub><pmid>22976766</pmid><doi>10.1007/s10545-012-9529-3</doi><tpages>9</tpages></addata></record> |
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| ispartof | Journal of inherited metabolic disease, 2013-05, Vol.36 (3), p.555-563 |
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| source | MEDLINE; Wiley Journals; SpringerLink Journals - AutoHoldings |
| subjects | Adolescent Adult Biochemistry Biological and medical sciences Enzyme Replacement Therapy Errors of metabolism Female Follow-Up Studies Gaucher Disease - blood Gaucher Disease - drug therapy Gaucher Disease - epidemiology Glucosylceramidase - administration & dosage Glucosylceramidase - therapeutic use Human Genetics Humans Internal Medicine Lipid Metabolism - drug effects Lipids (lysosomal enzyme disorders, storage diseases) Lipids - blood Male Medical genetics Medical sciences Medicine Medicine & Public Health Metabolic Diseases Middle Aged Original Article Pediatrics Prospective Studies Romania - epidemiology Young Adult |
| title | Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study |
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