A revised home treatment algorithm for Fabry disease: Influence of antibody formation
Enzyme replacement therapy for Fabry disease, consisting of biweekly infusions, interferes daily life. Home treatment proved beneficial. We evaluated a previously reported home treatment algorithm aiming to shorten the period of in-hospital infusions, while ascertaining patient safety. Retrospective...
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| Veröffentlicht in: | Molecular genetics and metabolism 2013-02, Vol.108 (2), p.132-137 |
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| creator | Smid, B.E. Hoogendijk, S.L. Wijburg, F.A. Hollak, C.E.M. Linthorst, G.E. |
| description | Enzyme replacement therapy for Fabry disease, consisting of biweekly infusions, interferes daily life. Home treatment proved beneficial. We evaluated a previously reported home treatment algorithm aiming to shorten the period of in-hospital infusions, while ascertaining patient safety.
Retrospective analysis on clinical records of treated Fabry patients. Potentially predictive factors for infusion associated reactions (IARs) were studied: agalsidase antibodies, agalsidase product and dose, FOS-SSI scores, and GLA activity and mutation. A questionnaire evaluated patient satisfaction and compliance.
Seventy-nine patients were included (41 males, 46% agalsidase antibody positive (AB+)). 85% received home treatment. Home treatment complications were erroneous fast infusion rates (n=4) causing IARs and, rarely, venous access problems. The single SAE was unrelated to home treatment. IgG antibody status was significantly associated with IARs (89% vs. 26% p-value |
| doi_str_mv | 10.1016/j.ymgme.2012.12.005 |
| format | Article |
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Retrospective analysis on clinical records of treated Fabry patients. Potentially predictive factors for infusion associated reactions (IARs) were studied: agalsidase antibodies, agalsidase product and dose, FOS-SSI scores, and GLA activity and mutation. A questionnaire evaluated patient satisfaction and compliance.
Seventy-nine patients were included (41 males, 46% agalsidase antibody positive (AB+)). 85% received home treatment. Home treatment complications were erroneous fast infusion rates (n=4) causing IARs and, rarely, venous access problems. The single SAE was unrelated to home treatment. IgG antibody status was significantly associated with IARs (89% vs. 26% p-value<0.01). Negative antibody status did not preclude IARs. Except for three AB+ patients, all first IARs occurred within 13 infusions. IARs occurred more frequently in patients using agalsidase beta 1.0mg/kg/eow than agalsidase alpha or beta 0.2mg/kg/eow, but the time to first IAR did not differ between groups. Four AB+ males experienced IARs after a dose increase. Compliance between home and in-hospital treatment was similar. Most patients preferred home treatment.
In this study home therapy for Fabry disease was safe and improved patient satisfaction. We propose a revised algorithm which allows safe home-treatment in all male patients after 13 instead of 26 infusions, irrespective of ERT preparation or dose. Furthermore, AB+ patients with dosage increase may experience new or increased IARs, necessitating in-hospital observations.
► Home therapy with enzyme replacement therapy for Fabry disease is safe. ► Home therapy can safely be initiated in all male patients after 13 infusions. ► Agalsidase dosage increase in AB+ patients necessitates in-hospital observations.</description><identifier>ISSN: 1096-7192</identifier><identifier>EISSN: 1096-7206</identifier><identifier>DOI: 10.1016/j.ymgme.2012.12.005</identifier><identifier>PMID: 23332169</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Adolescent ; Adult ; Agalsidase ; Aged ; Algorithms ; alpha-Galactosidase - immunology ; Antibodies ; Antibodies - blood ; Antibodies - immunology ; Antibody Formation ; Child ; Enzyme replacement therapy ; Enzyme Replacement Therapy - adverse effects ; Fabry disease ; Fabry Disease - immunology ; Fabry Disease - therapy ; Fabry's disease ; Female ; Home Infusion Therapy - adverse effects ; Home therapy ; Humans ; Immunoglobulin G - blood ; Immunoglobulin G - immunology ; Isoenzymes - immunology ; Lysosomal storage disorders ; Male ; Middle Aged ; Young Adult</subject><ispartof>Molecular genetics and metabolism, 2013-02, Vol.108 (2), p.132-137</ispartof><rights>2012 Elsevier Inc.</rights><rights>Copyright © 2012 Elsevier Inc. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c392t-6eb9f52b372c69de14d3713374dabf5bcdb9ba770e05d9001ab4cdbf8b1c7be83</citedby><cites>FETCH-LOGICAL-c392t-6eb9f52b372c69de14d3713374dabf5bcdb9ba770e05d9001ab4cdbf8b1c7be83</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://www.sciencedirect.com/science/article/pii/S1096719212007184$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,776,780,3537,27901,27902,65306</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/23332169$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Smid, B.E.</creatorcontrib><creatorcontrib>Hoogendijk, S.L.</creatorcontrib><creatorcontrib>Wijburg, F.A.</creatorcontrib><creatorcontrib>Hollak, C.E.M.</creatorcontrib><creatorcontrib>Linthorst, G.E.</creatorcontrib><title>A revised home treatment algorithm for Fabry disease: Influence of antibody formation</title><title>Molecular genetics and metabolism</title><addtitle>Mol Genet Metab</addtitle><description>Enzyme replacement therapy for Fabry disease, consisting of biweekly infusions, interferes daily life. Home treatment proved beneficial. We evaluated a previously reported home treatment algorithm aiming to shorten the period of in-hospital infusions, while ascertaining patient safety.
Retrospective analysis on clinical records of treated Fabry patients. Potentially predictive factors for infusion associated reactions (IARs) were studied: agalsidase antibodies, agalsidase product and dose, FOS-SSI scores, and GLA activity and mutation. A questionnaire evaluated patient satisfaction and compliance.
Seventy-nine patients were included (41 males, 46% agalsidase antibody positive (AB+)). 85% received home treatment. Home treatment complications were erroneous fast infusion rates (n=4) causing IARs and, rarely, venous access problems. The single SAE was unrelated to home treatment. IgG antibody status was significantly associated with IARs (89% vs. 26% p-value<0.01). Negative antibody status did not preclude IARs. Except for three AB+ patients, all first IARs occurred within 13 infusions. IARs occurred more frequently in patients using agalsidase beta 1.0mg/kg/eow than agalsidase alpha or beta 0.2mg/kg/eow, but the time to first IAR did not differ between groups. Four AB+ males experienced IARs after a dose increase. Compliance between home and in-hospital treatment was similar. Most patients preferred home treatment.
In this study home therapy for Fabry disease was safe and improved patient satisfaction. We propose a revised algorithm which allows safe home-treatment in all male patients after 13 instead of 26 infusions, irrespective of ERT preparation or dose. Furthermore, AB+ patients with dosage increase may experience new or increased IARs, necessitating in-hospital observations.
► Home therapy with enzyme replacement therapy for Fabry disease is safe. ► Home therapy can safely be initiated in all male patients after 13 infusions. ► Agalsidase dosage increase in AB+ patients necessitates in-hospital observations.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Agalsidase</subject><subject>Aged</subject><subject>Algorithms</subject><subject>alpha-Galactosidase - immunology</subject><subject>Antibodies</subject><subject>Antibodies - blood</subject><subject>Antibodies - immunology</subject><subject>Antibody Formation</subject><subject>Child</subject><subject>Enzyme replacement therapy</subject><subject>Enzyme Replacement Therapy - adverse effects</subject><subject>Fabry disease</subject><subject>Fabry Disease - immunology</subject><subject>Fabry Disease - therapy</subject><subject>Fabry's disease</subject><subject>Female</subject><subject>Home Infusion Therapy - adverse effects</subject><subject>Home therapy</subject><subject>Humans</subject><subject>Immunoglobulin G - blood</subject><subject>Immunoglobulin G - immunology</subject><subject>Isoenzymes - immunology</subject><subject>Lysosomal storage disorders</subject><subject>Male</subject><subject>Middle Aged</subject><subject>Young Adult</subject><issn>1096-7192</issn><issn>1096-7206</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2013</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqNkMlKQzEUhoMoDtUnECRLN60Z7hTBhRQnKLjRdchwrqbc3GiSCn17U6suRTiQQ_j-_OFD6JSSGSW0uVjO1v7Fw4wRymZlCKl30CElopm2jDS7PzsV7AAdpbQkhNJaVPvogHHOGW3EIXq-xhE-XAKLX4MHnCOo7GHMWA0vIbr86nEfIr5VOq6xLaBKcIkfxn5YwWgAhx6rMTsd7HoDepVdGI_RXq-GBCff5wQ93948ze-ni8e7h_n1Ymq4YHnagBZ9zTRvmWmEBVpZ3lLO28oq3dfaWC20alsCpLaifF_pqtz1naam1dDxCTrfvvsWw_sKUpbeJQPDoEYIqyQp62pKeFf9C2Udr0nVFJRvURNDShF6-RadV3EtKZEb9XIpv9TLjfqSlEV9SZ19F6y0B_ub-XFdgKstAMXIh4Mok3Ebh9ZFMFna4P4s-ASN55aQ</recordid><startdate>201302</startdate><enddate>201302</enddate><creator>Smid, B.E.</creator><creator>Hoogendijk, S.L.</creator><creator>Wijburg, F.A.</creator><creator>Hollak, C.E.M.</creator><creator>Linthorst, G.E.</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><scope>8FD</scope><scope>FR3</scope><scope>P64</scope><scope>RC3</scope></search><sort><creationdate>201302</creationdate><title>A revised home treatment algorithm for Fabry disease: Influence of antibody formation</title><author>Smid, B.E. ; Hoogendijk, S.L. ; Wijburg, F.A. ; Hollak, C.E.M. ; Linthorst, G.E.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c392t-6eb9f52b372c69de14d3713374dabf5bcdb9ba770e05d9001ab4cdbf8b1c7be83</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2013</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Agalsidase</topic><topic>Aged</topic><topic>Algorithms</topic><topic>alpha-Galactosidase - immunology</topic><topic>Antibodies</topic><topic>Antibodies - blood</topic><topic>Antibodies - immunology</topic><topic>Antibody Formation</topic><topic>Child</topic><topic>Enzyme replacement therapy</topic><topic>Enzyme Replacement Therapy - adverse effects</topic><topic>Fabry disease</topic><topic>Fabry Disease - immunology</topic><topic>Fabry Disease - therapy</topic><topic>Fabry's disease</topic><topic>Female</topic><topic>Home Infusion Therapy - adverse effects</topic><topic>Home therapy</topic><topic>Humans</topic><topic>Immunoglobulin G - blood</topic><topic>Immunoglobulin G - immunology</topic><topic>Isoenzymes - immunology</topic><topic>Lysosomal storage disorders</topic><topic>Male</topic><topic>Middle Aged</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Smid, B.E.</creatorcontrib><creatorcontrib>Hoogendijk, S.L.</creatorcontrib><creatorcontrib>Wijburg, F.A.</creatorcontrib><creatorcontrib>Hollak, C.E.M.</creatorcontrib><creatorcontrib>Linthorst, G.E.</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><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><jtitle>Molecular genetics and metabolism</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Smid, B.E.</au><au>Hoogendijk, S.L.</au><au>Wijburg, F.A.</au><au>Hollak, C.E.M.</au><au>Linthorst, G.E.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>A revised home treatment algorithm for Fabry disease: Influence of antibody formation</atitle><jtitle>Molecular genetics and metabolism</jtitle><addtitle>Mol Genet Metab</addtitle><date>2013-02</date><risdate>2013</risdate><volume>108</volume><issue>2</issue><spage>132</spage><epage>137</epage><pages>132-137</pages><issn>1096-7192</issn><eissn>1096-7206</eissn><abstract>Enzyme replacement therapy for Fabry disease, consisting of biweekly infusions, interferes daily life. Home treatment proved beneficial. We evaluated a previously reported home treatment algorithm aiming to shorten the period of in-hospital infusions, while ascertaining patient safety.
Retrospective analysis on clinical records of treated Fabry patients. Potentially predictive factors for infusion associated reactions (IARs) were studied: agalsidase antibodies, agalsidase product and dose, FOS-SSI scores, and GLA activity and mutation. A questionnaire evaluated patient satisfaction and compliance.
Seventy-nine patients were included (41 males, 46% agalsidase antibody positive (AB+)). 85% received home treatment. Home treatment complications were erroneous fast infusion rates (n=4) causing IARs and, rarely, venous access problems. The single SAE was unrelated to home treatment. IgG antibody status was significantly associated with IARs (89% vs. 26% p-value<0.01). Negative antibody status did not preclude IARs. Except for three AB+ patients, all first IARs occurred within 13 infusions. IARs occurred more frequently in patients using agalsidase beta 1.0mg/kg/eow than agalsidase alpha or beta 0.2mg/kg/eow, but the time to first IAR did not differ between groups. Four AB+ males experienced IARs after a dose increase. Compliance between home and in-hospital treatment was similar. Most patients preferred home treatment.
In this study home therapy for Fabry disease was safe and improved patient satisfaction. We propose a revised algorithm which allows safe home-treatment in all male patients after 13 instead of 26 infusions, irrespective of ERT preparation or dose. Furthermore, AB+ patients with dosage increase may experience new or increased IARs, necessitating in-hospital observations.
► Home therapy with enzyme replacement therapy for Fabry disease is safe. ► Home therapy can safely be initiated in all male patients after 13 infusions. ► Agalsidase dosage increase in AB+ patients necessitates in-hospital observations.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>23332169</pmid><doi>10.1016/j.ymgme.2012.12.005</doi><tpages>6</tpages></addata></record> |
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| ispartof | Molecular genetics and metabolism, 2013-02, Vol.108 (2), p.132-137 |
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| language | eng |
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| subjects | Adolescent Adult Agalsidase Aged Algorithms alpha-Galactosidase - immunology Antibodies Antibodies - blood Antibodies - immunology Antibody Formation Child Enzyme replacement therapy Enzyme Replacement Therapy - adverse effects Fabry disease Fabry Disease - immunology Fabry Disease - therapy Fabry's disease Female Home Infusion Therapy - adverse effects Home therapy Humans Immunoglobulin G - blood Immunoglobulin G - immunology Isoenzymes - immunology Lysosomal storage disorders Male Middle Aged Young Adult |
| title | A revised home treatment algorithm for Fabry disease: Influence of antibody formation |
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