Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease
The level of plasma globotriaosylsphingosine (lysoGb3) is an indication of disease severity in Fabry disease (FD) and its decrease during enzyme replacement therapy could be a reflection of treatment efficacy. Early treatment of FD may improve clinical outcome, but data to support this hypothesis ar...
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| Veröffentlicht in: | Molecular genetics and metabolism 2017-06, Vol.121 (2), p.157-161 |
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| creator | Arends, Maarten Wijburg, Frits A. Wanner, Christoph Vaz, Frédéric M. van Kuilenburg, André B.P. Hughes, Derralynn A. Biegstraaten, Marieke Mehta, Atul Hollak, Carla E.M. Langeveld, Mirjam |
| description | The level of plasma globotriaosylsphingosine (lysoGb3) is an indication of disease severity in Fabry disease (FD) and its decrease during enzyme replacement therapy could be a reflection of treatment efficacy. Early treatment of FD may improve clinical outcome, but data to support this hypothesis are scarce. In this study we compared lysoGb3 decrease after ERT initiation in men with classical FD who started ERT before the age of 25 (early-treatment) with those who started later in life (late-treatment).
Treatment naïve men with classical FD from three centers of excellence in Europe were included. Measurements of lysoGb3 levels by tandem mass spectroscopy and antibodies by an inhibitory assay were performed in a single laboratory. Results were adjusted for lysoGb3 at baseline, first ERT (i.e. agalsidase alfa or beta) and the average ERT dose.
85 patients were included, 21 in the early-treatment and 64 in the late-treatment group. LysoGb3 level at baseline was not different between the two groups (112 vs 114nmol/L, p=0.92). The adjusted odds ratio for reaching a lysoGb3 level |
| doi_str_mv | 10.1016/j.ymgme.2017.05.001 |
| format | Article |
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Treatment naïve men with classical FD from three centers of excellence in Europe were included. Measurements of lysoGb3 levels by tandem mass spectroscopy and antibodies by an inhibitory assay were performed in a single laboratory. Results were adjusted for lysoGb3 at baseline, first ERT (i.e. agalsidase alfa or beta) and the average ERT dose.
85 patients were included, 21 in the early-treatment and 64 in the late-treatment group. LysoGb3 level at baseline was not different between the two groups (112 vs 114nmol/L, p=0.92). The adjusted odds ratio for reaching a lysoGb3 level<20nmol/L was 7.38 for the early-treatment versus late-treatment group (95% CI: 1.91–34.04, p=0.006). The adjusted lysoGb3 levels one year after ERT initiation was 12.9nmol/L lower in the early-treatment (95% CI: −20.1–−5.8, p<0.001) compared to the late-treatment group.
The current retrospective cohort study shows that initiation of ERT at younger age in men with classical Fabry disease results in a better biochemical response.
•Baseline lysoGb3 values of early-treated (<25years of age) and late-treated (≥25years of age) classical FD men are similar.•LysoGb3 reduction following enzyme replacement therapy is more pronounced in the early-treatment group.•Even at young age a significant proportion of the patients already showed signs of organ involvement.</description><identifier>ISSN: 1096-7192</identifier><identifier>EISSN: 1096-7206</identifier><identifier>DOI: 10.1016/j.ymgme.2017.05.001</identifier><identifier>PMID: 28495078</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Adolescent ; Adult ; Age Factors ; Aged ; Antibodies - blood ; Child ; Cohort Studies ; Early-treatment ; Enzyme replacement therapy ; Enzyme Replacement Therapy - methods ; Europe ; Fabry disease ; Fabry Disease - blood ; Fabry Disease - drug therapy ; Globotriaosylsphingosine ; Glycolipids - blood ; Glycolipids - immunology ; Humans ; LysoGb3 ; Male ; Middle Aged ; Retrospective Studies ; Sphingolipids - blood ; Sphingolipids - immunology ; Tandem Mass Spectrometry ; Treatment Outcome ; Young Adult</subject><ispartof>Molecular genetics and metabolism, 2017-06, Vol.121 (2), p.157-161</ispartof><rights>2017 Elsevier Inc.</rights><rights>Copyright © 2017 Elsevier Inc. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c359t-17c8edb166fab60b7cbbb10af002b248329d18620928b8685f9d30805e89a5003</citedby><cites>FETCH-LOGICAL-c359t-17c8edb166fab60b7cbbb10af002b248329d18620928b8685f9d30805e89a5003</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://www.sciencedirect.com/science/article/pii/S1096719217301567$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,776,780,3537,27901,27902,65306</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/28495078$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Arends, Maarten</creatorcontrib><creatorcontrib>Wijburg, Frits A.</creatorcontrib><creatorcontrib>Wanner, Christoph</creatorcontrib><creatorcontrib>Vaz, Frédéric M.</creatorcontrib><creatorcontrib>van Kuilenburg, André B.P.</creatorcontrib><creatorcontrib>Hughes, Derralynn A.</creatorcontrib><creatorcontrib>Biegstraaten, Marieke</creatorcontrib><creatorcontrib>Mehta, Atul</creatorcontrib><creatorcontrib>Hollak, Carla E.M.</creatorcontrib><creatorcontrib>Langeveld, Mirjam</creatorcontrib><title>Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease</title><title>Molecular genetics and metabolism</title><addtitle>Mol Genet Metab</addtitle><description>The level of plasma globotriaosylsphingosine (lysoGb3) is an indication of disease severity in Fabry disease (FD) and its decrease during enzyme replacement therapy could be a reflection of treatment efficacy. Early treatment of FD may improve clinical outcome, but data to support this hypothesis are scarce. In this study we compared lysoGb3 decrease after ERT initiation in men with classical FD who started ERT before the age of 25 (early-treatment) with those who started later in life (late-treatment).
Treatment naïve men with classical FD from three centers of excellence in Europe were included. Measurements of lysoGb3 levels by tandem mass spectroscopy and antibodies by an inhibitory assay were performed in a single laboratory. Results were adjusted for lysoGb3 at baseline, first ERT (i.e. agalsidase alfa or beta) and the average ERT dose.
85 patients were included, 21 in the early-treatment and 64 in the late-treatment group. LysoGb3 level at baseline was not different between the two groups (112 vs 114nmol/L, p=0.92). The adjusted odds ratio for reaching a lysoGb3 level<20nmol/L was 7.38 for the early-treatment versus late-treatment group (95% CI: 1.91–34.04, p=0.006). The adjusted lysoGb3 levels one year after ERT initiation was 12.9nmol/L lower in the early-treatment (95% CI: −20.1–−5.8, p<0.001) compared to the late-treatment group.
The current retrospective cohort study shows that initiation of ERT at younger age in men with classical Fabry disease results in a better biochemical response.
•Baseline lysoGb3 values of early-treated (<25years of age) and late-treated (≥25years of age) classical FD men are similar.•LysoGb3 reduction following enzyme replacement therapy is more pronounced in the early-treatment group.•Even at young age a significant proportion of the patients already showed signs of organ involvement.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Age Factors</subject><subject>Aged</subject><subject>Antibodies - blood</subject><subject>Child</subject><subject>Cohort Studies</subject><subject>Early-treatment</subject><subject>Enzyme replacement therapy</subject><subject>Enzyme Replacement Therapy - methods</subject><subject>Europe</subject><subject>Fabry disease</subject><subject>Fabry Disease - blood</subject><subject>Fabry Disease - drug therapy</subject><subject>Globotriaosylsphingosine</subject><subject>Glycolipids - blood</subject><subject>Glycolipids - immunology</subject><subject>Humans</subject><subject>LysoGb3</subject><subject>Male</subject><subject>Middle Aged</subject><subject>Retrospective Studies</subject><subject>Sphingolipids - blood</subject><subject>Sphingolipids - immunology</subject><subject>Tandem Mass Spectrometry</subject><subject>Treatment Outcome</subject><subject>Young Adult</subject><issn>1096-7192</issn><issn>1096-7206</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2017</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kcFu1DAQhiMEoqXlCZAqH7lsGDubxD5wQBULSJW40LNlO5Ndr5w49SSLwpPwuLhsy5GTLc_3e6T_K4p3HEoOvPlwLNdhP2ApgLcl1CUAf1FcclDNphXQvHy-cyUuijdExwzwWm1fFxdCblUNrbwsfu_MKS7J2IAM-x7dzGLP0KSwshMmWogFMyOj2aTzaPy1DsgSTsE4HHCc2XzAZKaVxZHlRxoM24do45y8ibQGmg5-3EfyI7KAJwzE_Mhykv3084G5HCHvTGA7Y9PKOk9oCK-LV70JhG-fzqvifvf5x-3Xzd33L99uP91tXFWrecNbJ7GzvGl6YxuwrbPWcjA9gLBiKyuhOi4bAUpIKxtZ96qrQEKNUpkaoLoq3p__nVJ8WJBmPXhyGIIZMS6kuVQq98iFzGh1Rl2KRAl7PSU_mLRqDvpRiT7qv0r0oxINtc6N59TN04LFDtj9yzw7yMDHM5CbwZPHpMl5HB12PmUfuov-vwv-AMDGoZ4</recordid><startdate>201706</startdate><enddate>201706</enddate><creator>Arends, Maarten</creator><creator>Wijburg, Frits A.</creator><creator>Wanner, Christoph</creator><creator>Vaz, Frédéric M.</creator><creator>van Kuilenburg, André B.P.</creator><creator>Hughes, Derralynn A.</creator><creator>Biegstraaten, Marieke</creator><creator>Mehta, Atul</creator><creator>Hollak, Carla E.M.</creator><creator>Langeveld, Mirjam</creator><general>Elsevier Inc</general><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>7X8</scope></search><sort><creationdate>201706</creationdate><title>Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease</title><author>Arends, Maarten ; Wijburg, Frits A. ; Wanner, Christoph ; Vaz, Frédéric M. ; van Kuilenburg, André B.P. ; Hughes, Derralynn A. ; Biegstraaten, Marieke ; Mehta, Atul ; Hollak, Carla E.M. ; Langeveld, Mirjam</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c359t-17c8edb166fab60b7cbbb10af002b248329d18620928b8685f9d30805e89a5003</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2017</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Age Factors</topic><topic>Aged</topic><topic>Antibodies - blood</topic><topic>Child</topic><topic>Cohort Studies</topic><topic>Early-treatment</topic><topic>Enzyme replacement therapy</topic><topic>Enzyme Replacement Therapy - methods</topic><topic>Europe</topic><topic>Fabry disease</topic><topic>Fabry Disease - blood</topic><topic>Fabry Disease - drug therapy</topic><topic>Globotriaosylsphingosine</topic><topic>Glycolipids - blood</topic><topic>Glycolipids - immunology</topic><topic>Humans</topic><topic>LysoGb3</topic><topic>Male</topic><topic>Middle Aged</topic><topic>Retrospective Studies</topic><topic>Sphingolipids - blood</topic><topic>Sphingolipids - immunology</topic><topic>Tandem Mass Spectrometry</topic><topic>Treatment Outcome</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Arends, Maarten</creatorcontrib><creatorcontrib>Wijburg, Frits A.</creatorcontrib><creatorcontrib>Wanner, Christoph</creatorcontrib><creatorcontrib>Vaz, Frédéric M.</creatorcontrib><creatorcontrib>van Kuilenburg, André B.P.</creatorcontrib><creatorcontrib>Hughes, Derralynn A.</creatorcontrib><creatorcontrib>Biegstraaten, Marieke</creatorcontrib><creatorcontrib>Mehta, Atul</creatorcontrib><creatorcontrib>Hollak, Carla E.M.</creatorcontrib><creatorcontrib>Langeveld, Mirjam</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Molecular genetics and metabolism</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Arends, Maarten</au><au>Wijburg, Frits A.</au><au>Wanner, Christoph</au><au>Vaz, Frédéric M.</au><au>van Kuilenburg, André B.P.</au><au>Hughes, Derralynn A.</au><au>Biegstraaten, Marieke</au><au>Mehta, Atul</au><au>Hollak, Carla E.M.</au><au>Langeveld, Mirjam</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease</atitle><jtitle>Molecular genetics and metabolism</jtitle><addtitle>Mol Genet Metab</addtitle><date>2017-06</date><risdate>2017</risdate><volume>121</volume><issue>2</issue><spage>157</spage><epage>161</epage><pages>157-161</pages><issn>1096-7192</issn><eissn>1096-7206</eissn><abstract>The level of plasma globotriaosylsphingosine (lysoGb3) is an indication of disease severity in Fabry disease (FD) and its decrease during enzyme replacement therapy could be a reflection of treatment efficacy. Early treatment of FD may improve clinical outcome, but data to support this hypothesis are scarce. In this study we compared lysoGb3 decrease after ERT initiation in men with classical FD who started ERT before the age of 25 (early-treatment) with those who started later in life (late-treatment).
Treatment naïve men with classical FD from three centers of excellence in Europe were included. Measurements of lysoGb3 levels by tandem mass spectroscopy and antibodies by an inhibitory assay were performed in a single laboratory. Results were adjusted for lysoGb3 at baseline, first ERT (i.e. agalsidase alfa or beta) and the average ERT dose.
85 patients were included, 21 in the early-treatment and 64 in the late-treatment group. LysoGb3 level at baseline was not different between the two groups (112 vs 114nmol/L, p=0.92). The adjusted odds ratio for reaching a lysoGb3 level<20nmol/L was 7.38 for the early-treatment versus late-treatment group (95% CI: 1.91–34.04, p=0.006). The adjusted lysoGb3 levels one year after ERT initiation was 12.9nmol/L lower in the early-treatment (95% CI: −20.1–−5.8, p<0.001) compared to the late-treatment group.
The current retrospective cohort study shows that initiation of ERT at younger age in men with classical Fabry disease results in a better biochemical response.
•Baseline lysoGb3 values of early-treated (<25years of age) and late-treated (≥25years of age) classical FD men are similar.•LysoGb3 reduction following enzyme replacement therapy is more pronounced in the early-treatment group.•Even at young age a significant proportion of the patients already showed signs of organ involvement.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>28495078</pmid><doi>10.1016/j.ymgme.2017.05.001</doi><tpages>5</tpages></addata></record> |
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| subjects | Adolescent Adult Age Factors Aged Antibodies - blood Child Cohort Studies Early-treatment Enzyme replacement therapy Enzyme Replacement Therapy - methods Europe Fabry disease Fabry Disease - blood Fabry Disease - drug therapy Globotriaosylsphingosine Glycolipids - blood Glycolipids - immunology Humans LysoGb3 Male Middle Aged Retrospective Studies Sphingolipids - blood Sphingolipids - immunology Tandem Mass Spectrometry Treatment Outcome Young Adult |
| title | Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease |
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