Bleeding control improves after switching to emicizumab: Real-world experience of 177 children in the PedNet registry
Despite the rapid uptake of emicizumab in the paediatric haemophilia A (HA) population, real-world data on the safety and efficacy is limited. To report on bleeding and safety in paediatric patients receiving emicizumab prophylaxis. Data were extracted from the multicentre prospective observational...
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| Veröffentlicht in: | Haemophilia : the official journal of the World Federation of Hemophilia 2024-05, Vol.30 (3), p.685-692 |
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| creator | van der Zwet, Konrad de Kovel, Marloes Motwani, Jayashree van Geet, Chris Nolan, Beatrice Glosli, Heidi Escuriola Ettingshausen, Carmen Königs, Christoph Kenet, Gili Fischer, Kathelijn |
| description | Despite the rapid uptake of emicizumab in the paediatric haemophilia A (HA) population, real-world data on the safety and efficacy is limited.
To report on bleeding and safety in paediatric patients receiving emicizumab prophylaxis.
Data were extracted from the multicentre prospective observational PedNet Registry (NCT02979119). Children with haemophilia A, and ≥50 FVIII exposures or inhibitors present receiving emicizumab maintenance therapy were analysed. Data were summarized as medians with interquartile range (IQR, P25-P75). Mean (95% confidence interval (CI)), annualized (joint) bleeding rate (A(J)BR) during emicizumab and ≤2 years before emicizumab prophylaxis were modelled and compared using negative binomial regression.
Total of 177 patients started emicizumab at median 8.6 years (IQR 4.8-13.1), most had no FVIII inhibitors (64%). Follow up before emicizumab was median: 1.68 years (IQR: 1.24-1.90) and during emicizumab: 1.32 years (IQR: .94-2.11). In patients without inhibitors, mean ABR reduced after starting emicizumab from 2.41 (CI 1.98-2.95) to 1.11 (CI .90-1.36, p |
| doi_str_mv | 10.1111/hae.15015 |
| format | Article |
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To report on bleeding and safety in paediatric patients receiving emicizumab prophylaxis.
Data were extracted from the multicentre prospective observational PedNet Registry (NCT02979119). Children with haemophilia A, and ≥50 FVIII exposures or inhibitors present receiving emicizumab maintenance therapy were analysed. Data were summarized as medians with interquartile range (IQR, P25-P75). Mean (95% confidence interval (CI)), annualized (joint) bleeding rate (A(J)BR) during emicizumab and ≤2 years before emicizumab prophylaxis were modelled and compared using negative binomial regression.
Total of 177 patients started emicizumab at median 8.6 years (IQR 4.8-13.1), most had no FVIII inhibitors (64%). Follow up before emicizumab was median: 1.68 years (IQR: 1.24-1.90) and during emicizumab: 1.32 years (IQR: .94-2.11). In patients without inhibitors, mean ABR reduced after starting emicizumab from 2.41 (CI 1.98-2.95) to 1.11 (CI .90-1.36, p < .001), while AJBR reduced from.74 (CI .56-.98) to.31 (CI .21-.46, p < .001). Concordantly, in patients with inhibitors, mean ABR reduced from 5.08 (CI 4.08-6.38) to .75 (CI .56-1.01, p < .001), while AJBR reduced from 1.90 (CI 1.42-2.58) to .34 (CI .21-.56, p < .001). Five emicizumab-related adverse events were reported (3% of the cohort), including one patient with antidrug antibodies.
This study showed improved bleeding control compared to previous treatment and a favourable safety profile during emicizumab therapy in paediatric haemophilia A patients.</description><identifier>ISSN: 1351-8216</identifier><identifier>EISSN: 1365-2516</identifier><identifier>DOI: 10.1111/hae.15015</identifier><identifier>PMID: 38578720</identifier><language>eng</language><publisher>England: Wiley Subscription Services, Inc</publisher><subject>Adolescent ; Antibodies, Bispecific - pharmacology ; Antibodies, Bispecific - therapeutic use ; Antibodies, Monoclonal, Humanized - therapeutic use ; Bleeding ; Child ; Child, Preschool ; Disease prevention ; Factor VIII - therapeutic use ; Female ; Hemophilia ; Hemophilia A - drug therapy ; Hemorrhage ; Humans ; Male ; Patients ; Pediatrics ; Prophylaxis ; Prospective Studies ; Registries ; Safety</subject><ispartof>Haemophilia : the official journal of the World Federation of Hemophilia, 2024-05, Vol.30 (3), p.685-692</ispartof><rights>2024 The Authors. Haemophilia published by John Wiley & Sons Ltd.</rights><rights>2024. This article is published under http://creativecommons.org/licenses/by-nc-nd/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><cites>FETCH-LOGICAL-c308t-c77d5c2099727fdba30d5b0955e84e9fb975ecf01c4f49781c05202e59cf02c63</cites><orcidid>0000-0002-1279-2557 ; 0000-0001-9082-1416 ; 0000-0001-7126-6613 ; 0000-0003-3521-1863</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,780,784,27924,27925</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/38578720$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>van der Zwet, Konrad</creatorcontrib><creatorcontrib>de Kovel, Marloes</creatorcontrib><creatorcontrib>Motwani, Jayashree</creatorcontrib><creatorcontrib>van Geet, Chris</creatorcontrib><creatorcontrib>Nolan, Beatrice</creatorcontrib><creatorcontrib>Glosli, Heidi</creatorcontrib><creatorcontrib>Escuriola Ettingshausen, Carmen</creatorcontrib><creatorcontrib>Königs, Christoph</creatorcontrib><creatorcontrib>Kenet, Gili</creatorcontrib><creatorcontrib>Fischer, Kathelijn</creatorcontrib><creatorcontrib>PedNet Investigators</creatorcontrib><creatorcontrib>the PedNet Investigators</creatorcontrib><title>Bleeding control improves after switching to emicizumab: Real-world experience of 177 children in the PedNet registry</title><title>Haemophilia : the official journal of the World Federation of Hemophilia</title><addtitle>Haemophilia</addtitle><description>Despite the rapid uptake of emicizumab in the paediatric haemophilia A (HA) population, real-world data on the safety and efficacy is limited.
To report on bleeding and safety in paediatric patients receiving emicizumab prophylaxis.
Data were extracted from the multicentre prospective observational PedNet Registry (NCT02979119). Children with haemophilia A, and ≥50 FVIII exposures or inhibitors present receiving emicizumab maintenance therapy were analysed. Data were summarized as medians with interquartile range (IQR, P25-P75). Mean (95% confidence interval (CI)), annualized (joint) bleeding rate (A(J)BR) during emicizumab and ≤2 years before emicizumab prophylaxis were modelled and compared using negative binomial regression.
Total of 177 patients started emicizumab at median 8.6 years (IQR 4.8-13.1), most had no FVIII inhibitors (64%). Follow up before emicizumab was median: 1.68 years (IQR: 1.24-1.90) and during emicizumab: 1.32 years (IQR: .94-2.11). In patients without inhibitors, mean ABR reduced after starting emicizumab from 2.41 (CI 1.98-2.95) to 1.11 (CI .90-1.36, p < .001), while AJBR reduced from.74 (CI .56-.98) to.31 (CI .21-.46, p < .001). Concordantly, in patients with inhibitors, mean ABR reduced from 5.08 (CI 4.08-6.38) to .75 (CI .56-1.01, p < .001), while AJBR reduced from 1.90 (CI 1.42-2.58) to .34 (CI .21-.56, p < .001). Five emicizumab-related adverse events were reported (3% of the cohort), including one patient with antidrug antibodies.
This study showed improved bleeding control compared to previous treatment and a favourable safety profile during emicizumab therapy in paediatric haemophilia A patients.</description><subject>Adolescent</subject><subject>Antibodies, Bispecific - pharmacology</subject><subject>Antibodies, Bispecific - therapeutic use</subject><subject>Antibodies, Monoclonal, Humanized - therapeutic use</subject><subject>Bleeding</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Disease prevention</subject><subject>Factor VIII - therapeutic use</subject><subject>Female</subject><subject>Hemophilia</subject><subject>Hemophilia A - drug therapy</subject><subject>Hemorrhage</subject><subject>Humans</subject><subject>Male</subject><subject>Patients</subject><subject>Pediatrics</subject><subject>Prophylaxis</subject><subject>Prospective Studies</subject><subject>Registries</subject><subject>Safety</subject><issn>1351-8216</issn><issn>1365-2516</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNpdkU1PwzAMhiMEgjE48AdQJC5w6MhH0zTcAPElTYAQnKs2dVmmthlJCoxfT8bXAV9sWc9r2X4R2qNkQmMcz0qYUEGoWEMjyjORMEGz9VUtaJIzmm2hbe_nhFDOSLaJtnguZC4ZGaHhrAWoTf-Mte2Dsy023cLZV_C4bAI47N9M0LMVECyGzmjzMXRldYIfoGyTN-vaGsP7ApyBXgO2DaZS4qhoawc9Nj0OM8D3UN9CwA6ejQ9uuYM2mrL1sPuTx-jp8uLx_DqZ3l3dnJ9OE81JHhItZS00I0pJJpu6KjmpRUWUEJCnoJpKSQG6IVSnTapkTjURjDAQKjaZzvgYHX7PjSe9DOBD0RmvoW3LHuzgC054yqKSkoge_EPndnB93C5SIuU0E0pF6uib0s5676ApFs50pVsWlBQrL4roRfHlRWT3fyYOVQf1H_n7fP4Jg1aEEg</recordid><startdate>202405</startdate><enddate>202405</enddate><creator>van der Zwet, Konrad</creator><creator>de Kovel, Marloes</creator><creator>Motwani, Jayashree</creator><creator>van Geet, Chris</creator><creator>Nolan, Beatrice</creator><creator>Glosli, Heidi</creator><creator>Escuriola Ettingshausen, Carmen</creator><creator>Königs, Christoph</creator><creator>Kenet, Gili</creator><creator>Fischer, Kathelijn</creator><general>Wiley Subscription Services, 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>7T5</scope><scope>8FD</scope><scope>FR3</scope><scope>H94</scope><scope>P64</scope><scope>RC3</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0002-1279-2557</orcidid><orcidid>https://orcid.org/0000-0001-9082-1416</orcidid><orcidid>https://orcid.org/0000-0001-7126-6613</orcidid><orcidid>https://orcid.org/0000-0003-3521-1863</orcidid></search><sort><creationdate>202405</creationdate><title>Bleeding control improves after switching to emicizumab: Real-world experience of 177 children in the PedNet registry</title><author>van der Zwet, Konrad ; de Kovel, Marloes ; Motwani, Jayashree ; van Geet, Chris ; Nolan, Beatrice ; Glosli, Heidi ; Escuriola Ettingshausen, Carmen ; Königs, Christoph ; Kenet, Gili ; Fischer, Kathelijn</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c308t-c77d5c2099727fdba30d5b0955e84e9fb975ecf01c4f49781c05202e59cf02c63</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><topic>Adolescent</topic><topic>Antibodies, Bispecific - pharmacology</topic><topic>Antibodies, Bispecific - therapeutic use</topic><topic>Antibodies, Monoclonal, Humanized - therapeutic use</topic><topic>Bleeding</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Disease prevention</topic><topic>Factor VIII - therapeutic use</topic><topic>Female</topic><topic>Hemophilia</topic><topic>Hemophilia A - drug therapy</topic><topic>Hemorrhage</topic><topic>Humans</topic><topic>Male</topic><topic>Patients</topic><topic>Pediatrics</topic><topic>Prophylaxis</topic><topic>Prospective Studies</topic><topic>Registries</topic><topic>Safety</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>van der Zwet, Konrad</creatorcontrib><creatorcontrib>de Kovel, Marloes</creatorcontrib><creatorcontrib>Motwani, Jayashree</creatorcontrib><creatorcontrib>van Geet, Chris</creatorcontrib><creatorcontrib>Nolan, Beatrice</creatorcontrib><creatorcontrib>Glosli, Heidi</creatorcontrib><creatorcontrib>Escuriola Ettingshausen, Carmen</creatorcontrib><creatorcontrib>Königs, Christoph</creatorcontrib><creatorcontrib>Kenet, Gili</creatorcontrib><creatorcontrib>Fischer, Kathelijn</creatorcontrib><creatorcontrib>PedNet Investigators</creatorcontrib><creatorcontrib>the PedNet Investigators</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><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>van der Zwet, Konrad</au><au>de Kovel, Marloes</au><au>Motwani, Jayashree</au><au>van Geet, Chris</au><au>Nolan, Beatrice</au><au>Glosli, Heidi</au><au>Escuriola Ettingshausen, Carmen</au><au>Königs, Christoph</au><au>Kenet, Gili</au><au>Fischer, Kathelijn</au><aucorp>PedNet Investigators</aucorp><aucorp>the PedNet Investigators</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Bleeding control improves after switching to emicizumab: Real-world experience of 177 children in the PedNet registry</atitle><jtitle>Haemophilia : the official journal of the World Federation of Hemophilia</jtitle><addtitle>Haemophilia</addtitle><date>2024-05</date><risdate>2024</risdate><volume>30</volume><issue>3</issue><spage>685</spage><epage>692</epage><pages>685-692</pages><issn>1351-8216</issn><eissn>1365-2516</eissn><abstract>Despite the rapid uptake of emicizumab in the paediatric haemophilia A (HA) population, real-world data on the safety and efficacy is limited.
To report on bleeding and safety in paediatric patients receiving emicizumab prophylaxis.
Data were extracted from the multicentre prospective observational PedNet Registry (NCT02979119). Children with haemophilia A, and ≥50 FVIII exposures or inhibitors present receiving emicizumab maintenance therapy were analysed. Data were summarized as medians with interquartile range (IQR, P25-P75). Mean (95% confidence interval (CI)), annualized (joint) bleeding rate (A(J)BR) during emicizumab and ≤2 years before emicizumab prophylaxis were modelled and compared using negative binomial regression.
Total of 177 patients started emicizumab at median 8.6 years (IQR 4.8-13.1), most had no FVIII inhibitors (64%). Follow up before emicizumab was median: 1.68 years (IQR: 1.24-1.90) and during emicizumab: 1.32 years (IQR: .94-2.11). In patients without inhibitors, mean ABR reduced after starting emicizumab from 2.41 (CI 1.98-2.95) to 1.11 (CI .90-1.36, p < .001), while AJBR reduced from.74 (CI .56-.98) to.31 (CI .21-.46, p < .001). Concordantly, in patients with inhibitors, mean ABR reduced from 5.08 (CI 4.08-6.38) to .75 (CI .56-1.01, p < .001), while AJBR reduced from 1.90 (CI 1.42-2.58) to .34 (CI .21-.56, p < .001). Five emicizumab-related adverse events were reported (3% of the cohort), including one patient with antidrug antibodies.
This study showed improved bleeding control compared to previous treatment and a favourable safety profile during emicizumab therapy in paediatric haemophilia A patients.</abstract><cop>England</cop><pub>Wiley Subscription Services, Inc</pub><pmid>38578720</pmid><doi>10.1111/hae.15015</doi><tpages>8</tpages><orcidid>https://orcid.org/0000-0002-1279-2557</orcidid><orcidid>https://orcid.org/0000-0001-9082-1416</orcidid><orcidid>https://orcid.org/0000-0001-7126-6613</orcidid><orcidid>https://orcid.org/0000-0003-3521-1863</orcidid><oa>free_for_read</oa></addata></record> |
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| subjects | Adolescent Antibodies, Bispecific - pharmacology Antibodies, Bispecific - therapeutic use Antibodies, Monoclonal, Humanized - therapeutic use Bleeding Child Child, Preschool Disease prevention Factor VIII - therapeutic use Female Hemophilia Hemophilia A - drug therapy Hemorrhage Humans Male Patients Pediatrics Prophylaxis Prospective Studies Registries Safety |
| title | Bleeding control improves after switching to emicizumab: Real-world experience of 177 children in the PedNet registry |
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