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A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B
Introduction Extended half‐life factor IX concentrates (EHL‐FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL‐FIX for persons with severe haemophilia B. Aim The lo...
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| Published in: | Haemophilia : the official journal of the World Federation of Hemophilia 2020-03, Vol.26 (2), p.278-281 |
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| Main Authors: | , , , , , , , |
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| cites | cdi_FETCH-LOGICAL-c3536-fcaaf81ce5661998703462e9807c0af317212107c2dc53308651f76c99ef7e453 |
| container_end_page | 281 |
| container_issue | 2 |
| container_start_page | 278 |
| container_title | Haemophilia : the official journal of the World Federation of Hemophilia |
| container_volume | 26 |
| creator | Rampotas, Alexandros Desborough, Michael J. R. Raza‐Burton, Sayma Taylor, Stephanie Wilkinson, Alice Hall, Georgina W Shapiro, Susan Curry, Nicola |
| description | Introduction
Extended half‐life factor IX concentrates (EHL‐FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL‐FIX for persons with severe haemophilia B.
Aim
The low doses used in real world are approximately half of the doses used in clinical trials. We aim to assess the efficacy and safety of low dose EHL‐FIX.
Methods
Data from a cohort of 13 patients who were switched from standard half‐life factor IX (SHL‐FIX) to Alprolix® (mean dose 31.5 IU/kg) and seven patients who switched from standard half‐life factor IX to Idelvion® (mean dose 20.2 IU/kg) were included.
Results
The median annualized bleeding rate was similar for SHL‐FIX (median 3, interquartile range [IQR] 1‐5) and EHL‐FIX (median 3, IQR 1‐5.25). Quality of life scores, measured using the European Quality of Life 5 Dimensions assessment were similar for SHL‐FIX (median 0.76, IQR: 0.63‐0.84) and EHL‐FIX (median 0.79, IQR: 0.58‐0.88).
Conclusion
This study shows that EHL‐FIX given at low doses can be effective for prevention of bleeding for persons with severe haemophilia B. |
| doi_str_mv | 10.1111/hae.13936 |
| format | article |
| fullrecord | <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_2362066295</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2362066295</sourcerecordid><originalsourceid>FETCH-LOGICAL-c3536-fcaaf81ce5661998703462e9807c0af317212107c2dc53308651f76c99ef7e453</originalsourceid><addsrcrecordid>eNp1kctKxDAYhYMoXkYXvoAE3OiiTi42bZajqDMguFFwV2L6x0bSdkzauex8BJ_RJzHjqAvBbE4CXw7nPz9Ch5Sc0XiGlYIzyiUXG2iXcpEmLKVic3VPaZIzKnbQXggvhFDOiNhGO1Fyngm5ixYjHGzz7ABraDoP2EPn2zAF3dkZ4ND15RK3Brt2jss2AJ76dlotnVrYgOe2qzAsOmhKKHGlnPl4e3fWADZKd63Hk0dsogSYQbSOMev42Tqr8MU-2jLKBTj41gF6uL66vxwnt3c3k8vRbaJ5ykVitFImpxpSIaiUeUb4uWAgc5JpogynGaOMxgcrdco5yUVKTSa0lGAyOE_5AJ2sfWPw1x5CV9Q2aHBONdD2oWBcxE4Ekyv0-A_60va-iekilUmRU56xSJ2uKR17Ch5MMfW2Vn5ZUFKs1lHEOYuvdUT26Nuxf6qh_CV_-o_AcA3MrYPl_07FeHS1tvwE-ZCUVQ</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2379681372</pqid></control><display><type>article</type><title>A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B</title><source>Wiley</source><creator>Rampotas, Alexandros ; Desborough, Michael J. R. ; Raza‐Burton, Sayma ; Taylor, Stephanie ; Wilkinson, Alice ; Hall, Georgina W ; Shapiro, Susan ; Curry, Nicola</creator><creatorcontrib>Rampotas, Alexandros ; Desborough, Michael J. R. ; Raza‐Burton, Sayma ; Taylor, Stephanie ; Wilkinson, Alice ; Hall, Georgina W ; Shapiro, Susan ; Curry, Nicola</creatorcontrib><description>Introduction
Extended half‐life factor IX concentrates (EHL‐FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL‐FIX for persons with severe haemophilia B.
Aim
The low doses used in real world are approximately half of the doses used in clinical trials. We aim to assess the efficacy and safety of low dose EHL‐FIX.
Methods
Data from a cohort of 13 patients who were switched from standard half‐life factor IX (SHL‐FIX) to Alprolix® (mean dose 31.5 IU/kg) and seven patients who switched from standard half‐life factor IX to Idelvion® (mean dose 20.2 IU/kg) were included.
Results
The median annualized bleeding rate was similar for SHL‐FIX (median 3, interquartile range [IQR] 1‐5) and EHL‐FIX (median 3, IQR 1‐5.25). Quality of life scores, measured using the European Quality of Life 5 Dimensions assessment were similar for SHL‐FIX (median 0.76, IQR: 0.63‐0.84) and EHL‐FIX (median 0.79, IQR: 0.58‐0.88).
Conclusion
This study shows that EHL‐FIX given at low doses can be effective for prevention of bleeding for persons with severe haemophilia B.</description><identifier>ISSN: 1351-8216</identifier><identifier>EISSN: 1365-2516</identifier><identifier>DOI: 10.1111/hae.13936</identifier><identifier>PMID: 32083769</identifier><language>eng</language><publisher>England: Wiley Subscription Services, Inc</publisher><subject>annual bleeding rate ; Bleeding ; bleeding disorders ; Clinical trials ; Coagulation factors ; extended half‐life factors ; factor IX ; Factor IX deficiency ; haemophilia ; Hemophilia ; Prophylaxis ; Quality of life</subject><ispartof>Haemophilia : the official journal of the World Federation of Hemophilia, 2020-03, Vol.26 (2), p.278-281</ispartof><rights>2020 John Wiley & Sons Ltd</rights><rights>2020 John Wiley & Sons Ltd.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c3536-fcaaf81ce5661998703462e9807c0af317212107c2dc53308651f76c99ef7e453</citedby><cites>FETCH-LOGICAL-c3536-fcaaf81ce5661998703462e9807c0af317212107c2dc53308651f76c99ef7e453</cites><orcidid>0000-0002-3849-0688 ; 0000-0002-1951-5616 ; 0000-0003-2689-1780 ; 0000-0002-2681-5860</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1111%2Fhae.13936$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1111%2Fhae.13936$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>315,786,790,27957,27958,50923,51032</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/32083769$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Rampotas, Alexandros</creatorcontrib><creatorcontrib>Desborough, Michael J. R.</creatorcontrib><creatorcontrib>Raza‐Burton, Sayma</creatorcontrib><creatorcontrib>Taylor, Stephanie</creatorcontrib><creatorcontrib>Wilkinson, Alice</creatorcontrib><creatorcontrib>Hall, Georgina W</creatorcontrib><creatorcontrib>Shapiro, Susan</creatorcontrib><creatorcontrib>Curry, Nicola</creatorcontrib><title>A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B</title><title>Haemophilia : the official journal of the World Federation of Hemophilia</title><addtitle>Haemophilia</addtitle><description>Introduction
Extended half‐life factor IX concentrates (EHL‐FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL‐FIX for persons with severe haemophilia B.
Aim
The low doses used in real world are approximately half of the doses used in clinical trials. We aim to assess the efficacy and safety of low dose EHL‐FIX.
Methods
Data from a cohort of 13 patients who were switched from standard half‐life factor IX (SHL‐FIX) to Alprolix® (mean dose 31.5 IU/kg) and seven patients who switched from standard half‐life factor IX to Idelvion® (mean dose 20.2 IU/kg) were included.
Results
The median annualized bleeding rate was similar for SHL‐FIX (median 3, interquartile range [IQR] 1‐5) and EHL‐FIX (median 3, IQR 1‐5.25). Quality of life scores, measured using the European Quality of Life 5 Dimensions assessment were similar for SHL‐FIX (median 0.76, IQR: 0.63‐0.84) and EHL‐FIX (median 0.79, IQR: 0.58‐0.88).
Conclusion
This study shows that EHL‐FIX given at low doses can be effective for prevention of bleeding for persons with severe haemophilia B.</description><subject>annual bleeding rate</subject><subject>Bleeding</subject><subject>bleeding disorders</subject><subject>Clinical trials</subject><subject>Coagulation factors</subject><subject>extended half‐life factors</subject><subject>factor IX</subject><subject>Factor IX deficiency</subject><subject>haemophilia</subject><subject>Hemophilia</subject><subject>Prophylaxis</subject><subject>Quality of life</subject><issn>1351-8216</issn><issn>1365-2516</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><recordid>eNp1kctKxDAYhYMoXkYXvoAE3OiiTi42bZajqDMguFFwV2L6x0bSdkzauex8BJ_RJzHjqAvBbE4CXw7nPz9Ch5Sc0XiGlYIzyiUXG2iXcpEmLKVic3VPaZIzKnbQXggvhFDOiNhGO1Fyngm5ixYjHGzz7ABraDoP2EPn2zAF3dkZ4ND15RK3Brt2jss2AJ76dlotnVrYgOe2qzAsOmhKKHGlnPl4e3fWADZKd63Hk0dsogSYQbSOMev42Tqr8MU-2jLKBTj41gF6uL66vxwnt3c3k8vRbaJ5ykVitFImpxpSIaiUeUb4uWAgc5JpogynGaOMxgcrdco5yUVKTSa0lGAyOE_5AJ2sfWPw1x5CV9Q2aHBONdD2oWBcxE4Ekyv0-A_60va-iekilUmRU56xSJ2uKR17Ch5MMfW2Vn5ZUFKs1lHEOYuvdUT26Nuxf6qh_CV_-o_AcA3MrYPl_07FeHS1tvwE-ZCUVQ</recordid><startdate>202003</startdate><enddate>202003</enddate><creator>Rampotas, Alexandros</creator><creator>Desborough, Michael J. R.</creator><creator>Raza‐Burton, Sayma</creator><creator>Taylor, Stephanie</creator><creator>Wilkinson, Alice</creator><creator>Hall, Georgina W</creator><creator>Shapiro, Susan</creator><creator>Curry, Nicola</creator><general>Wiley Subscription Services, Inc</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7T5</scope><scope>8FD</scope><scope>FR3</scope><scope>H94</scope><scope>P64</scope><scope>RC3</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0002-3849-0688</orcidid><orcidid>https://orcid.org/0000-0002-1951-5616</orcidid><orcidid>https://orcid.org/0000-0003-2689-1780</orcidid><orcidid>https://orcid.org/0000-0002-2681-5860</orcidid></search><sort><creationdate>202003</creationdate><title>A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B</title><author>Rampotas, Alexandros ; Desborough, Michael J. R. ; Raza‐Burton, Sayma ; Taylor, Stephanie ; Wilkinson, Alice ; Hall, Georgina W ; Shapiro, Susan ; Curry, Nicola</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c3536-fcaaf81ce5661998703462e9807c0af317212107c2dc53308651f76c99ef7e453</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2020</creationdate><topic>annual bleeding rate</topic><topic>Bleeding</topic><topic>bleeding disorders</topic><topic>Clinical trials</topic><topic>Coagulation factors</topic><topic>extended half‐life factors</topic><topic>factor IX</topic><topic>Factor IX deficiency</topic><topic>haemophilia</topic><topic>Hemophilia</topic><topic>Prophylaxis</topic><topic>Quality of life</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Rampotas, Alexandros</creatorcontrib><creatorcontrib>Desborough, Michael J. R.</creatorcontrib><creatorcontrib>Raza‐Burton, Sayma</creatorcontrib><creatorcontrib>Taylor, Stephanie</creatorcontrib><creatorcontrib>Wilkinson, Alice</creatorcontrib><creatorcontrib>Hall, Georgina W</creatorcontrib><creatorcontrib>Shapiro, Susan</creatorcontrib><creatorcontrib>Curry, Nicola</creatorcontrib><collection>PubMed</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><collection>MEDLINE - Academic</collection><jtitle>Haemophilia : the official journal of the World Federation of Hemophilia</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Rampotas, Alexandros</au><au>Desborough, Michael J. R.</au><au>Raza‐Burton, Sayma</au><au>Taylor, Stephanie</au><au>Wilkinson, Alice</au><au>Hall, Georgina W</au><au>Shapiro, Susan</au><au>Curry, Nicola</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B</atitle><jtitle>Haemophilia : the official journal of the World Federation of Hemophilia</jtitle><addtitle>Haemophilia</addtitle><date>2020-03</date><risdate>2020</risdate><volume>26</volume><issue>2</issue><spage>278</spage><epage>281</epage><pages>278-281</pages><issn>1351-8216</issn><eissn>1365-2516</eissn><notes>ObjectType-Article-1</notes><notes>SourceType-Scholarly Journals-1</notes><notes>ObjectType-Feature-2</notes><notes>content type line 23</notes><abstract>Introduction
Extended half‐life factor IX concentrates (EHL‐FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL‐FIX for persons with severe haemophilia B.
Aim
The low doses used in real world are approximately half of the doses used in clinical trials. We aim to assess the efficacy and safety of low dose EHL‐FIX.
Methods
Data from a cohort of 13 patients who were switched from standard half‐life factor IX (SHL‐FIX) to Alprolix® (mean dose 31.5 IU/kg) and seven patients who switched from standard half‐life factor IX to Idelvion® (mean dose 20.2 IU/kg) were included.
Results
The median annualized bleeding rate was similar for SHL‐FIX (median 3, interquartile range [IQR] 1‐5) and EHL‐FIX (median 3, IQR 1‐5.25). Quality of life scores, measured using the European Quality of Life 5 Dimensions assessment were similar for SHL‐FIX (median 0.76, IQR: 0.63‐0.84) and EHL‐FIX (median 0.79, IQR: 0.58‐0.88).
Conclusion
This study shows that EHL‐FIX given at low doses can be effective for prevention of bleeding for persons with severe haemophilia B.</abstract><cop>England</cop><pub>Wiley Subscription Services, Inc</pub><pmid>32083769</pmid><doi>10.1111/hae.13936</doi><tpages>4</tpages><orcidid>https://orcid.org/0000-0002-3849-0688</orcidid><orcidid>https://orcid.org/0000-0002-1951-5616</orcidid><orcidid>https://orcid.org/0000-0003-2689-1780</orcidid><orcidid>https://orcid.org/0000-0002-2681-5860</orcidid></addata></record> |
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| ispartof | Haemophilia : the official journal of the World Federation of Hemophilia, 2020-03, Vol.26 (2), p.278-281 |
| issn | 1351-8216 1365-2516 |
| language | eng |
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| source | Wiley |
| subjects | annual bleeding rate Bleeding bleeding disorders Clinical trials Coagulation factors extended half‐life factors factor IX Factor IX deficiency haemophilia Hemophilia Prophylaxis Quality of life |
| title | A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B |
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