Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A
Identifieur interne : 000982 ( Pmc/Checkpoint ); précédent : 000981; suivant : 000983Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A
Auteurs : Johnny Mahlangu [Afrique du Sud] ; Kazimierz Kuliczkowski [Pologne] ; Faraizah Abdul Karim [Malaisie] ; Oleksandra Stasyshyn [Ukraine] ; Marina V. Kosinova [Russie] ; Lynda Mae Lepatan [Philippines] ; Aleksander Skotnicki [Pologne] ; Lisa N. Boggio [États-Unis] ; Robert Klamroth [Allemagne] ; Johannes Oldenburg [Allemagne] ; Andrzej Hellmann [Pologne] ; Elena Santagostino [Italie] ; Ross I. Baker [Australie] ; Kathelijn Fischer [Pays-Bas] ; Joan C. Gill [États-Unis] ; Stephanie P G [Australie] ; Pratima Chowdary [Royaume-Uni] ; Miguel A. Escobar [États-Unis] ; Claudia Djambas Khayat [Liban] ; Luminita Rusen [Roumanie] ; Debra Bensen-Kennedy [États-Unis] ; Nicole Blackman [États-Unis] ; Tharin Limsakun [États-Unis] ; Alex Veldman [Allemagne] ; Katie St. Ledger [États-Unis] ; Ingrid Pabinger [Autriche]Source :
- Blood [ 0006-4971 ] ; 2016.
Abstract
rVIII-SingleChain is a novel rFVIII, designed to have high stability and high binding affinity for VWF. In severe hemophilia A patients, rVIII-SingleChain was well tolerated and resulted in low bleeding rates, when dosed twice per week.
Url:
DOI: 10.1182/blood-2016-01-687434
PubMed: 27330001
PubMed Central: 4974198
Affiliations:
- Afrique du Sud, Allemagne, Australie, Autriche, Italie, Liban, Malaisie, Pays-Bas, Philippines, Pologne, Roumanie, Royaume-Uni, Russie, Ukraine, États-Unis
- Angleterre, Berlin, District de Cologne, District de Giessen, Gauteng, Grand Londres, Hesse (Land), Illinois, Lombardie, Pennsylvanie, Rhénanie-du-Nord-Westphalie, Texas, Utrecht (province), Vienne (Autriche), Wisconsin
- Berlin, Bonn, Johannesbourg, Londres, Marbourg, Milan, Utrecht, Vienne (Autriche)
- Université du Witwatersrand
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PMC:4974198Le document en format XML
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<author><name sortKey="Klamroth, Robert" sort="Klamroth, Robert" uniqKey="Klamroth R" first="Robert" last="Klamroth">Robert Klamroth</name>
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<author><name sortKey="Oldenburg, Johannes" sort="Oldenburg, Johannes" uniqKey="Oldenburg J" first="Johannes" last="Oldenburg">Johannes Oldenburg</name>
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<author><name sortKey="Santagostino, Elena" sort="Santagostino, Elena" uniqKey="Santagostino E" first="Elena" last="Santagostino">Elena Santagostino</name>
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<author><name sortKey="P G, Stephanie" sort="P G, Stephanie" uniqKey="P G S" first="Stephanie" last="P G">Stephanie P G</name>
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<author><name sortKey="Chowdary, Pratima" sort="Chowdary, Pratima" uniqKey="Chowdary P" first="Pratima" last="Chowdary">Pratima Chowdary</name>
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<author><name sortKey="Khayat, Claudia Djambas" sort="Khayat, Claudia Djambas" uniqKey="Khayat C" first="Claudia Djambas" last="Khayat">Claudia Djambas Khayat</name>
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<author><name sortKey="St Ledger, Katie" sort="St Ledger, Katie" uniqKey="St Ledger K" first="Katie" last="St. Ledger">Katie St. Ledger</name>
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<author><name sortKey="Pabinger, Ingrid" sort="Pabinger, Ingrid" uniqKey="Pabinger I" first="Ingrid" last="Pabinger">Ingrid Pabinger</name>
<affiliation wicri:level="3"><nlm:aff id="aff23">Clinical Division of Haematology and Haemostaseology, Medical Clinic I, Medical University Vienna, Vienna, Austria</nlm:aff>
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<placeName><settlement type="city">Vienne (Autriche)</settlement>
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<author><name sortKey="Karim, Faraizah Abdul" sort="Karim, Faraizah Abdul" uniqKey="Karim F" first="Faraizah Abdul" last="Karim">Faraizah Abdul Karim</name>
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<author><name sortKey="Stasyshyn, Oleksandra" sort="Stasyshyn, Oleksandra" uniqKey="Stasyshyn O" first="Oleksandra" last="Stasyshyn">Oleksandra Stasyshyn</name>
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<author><name sortKey="Kosinova, Marina V" sort="Kosinova, Marina V" uniqKey="Kosinova M" first="Marina V." last="Kosinova">Marina V. Kosinova</name>
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<author><name sortKey="Oldenburg, Johannes" sort="Oldenburg, Johannes" uniqKey="Oldenburg J" first="Johannes" last="Oldenburg">Johannes Oldenburg</name>
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<settlement type="city">Bonn</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Hellmann, Andrzej" sort="Hellmann, Andrzej" uniqKey="Hellmann A" first="Andrzej" last="Hellmann">Andrzej Hellmann</name>
<affiliation wicri:level="1"><nlm:aff id="aff11">Department of Hematology and Transplantation, Medical University of Gdansk, Gdansk, Poland;</nlm:aff>
<country xml:lang="fr">Pologne</country>
<wicri:regionArea>Department of Hematology and Transplantation, Medical University of Gdansk, Gdansk</wicri:regionArea>
<wicri:noRegion>Gdansk</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Santagostino, Elena" sort="Santagostino, Elena" uniqKey="Santagostino E" first="Elena" last="Santagostino">Elena Santagostino</name>
<affiliation wicri:level="3"><nlm:aff id="aff12">A. Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy;</nlm:aff>
<country xml:lang="fr">Italie</country>
<wicri:regionArea>A. Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan</wicri:regionArea>
<placeName><settlement type="city">Milan</settlement>
<region nuts="2">Lombardie</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Baker, Ross I" sort="Baker, Ross I" uniqKey="Baker R" first="Ross I." last="Baker">Ross I. Baker</name>
<affiliation wicri:level="1"><nlm:aff id="aff13">Western Australian Centre for Thrombosis and Haemostasis, Murdoch University, Perth, Australia;</nlm:aff>
<country xml:lang="fr">Australie</country>
<wicri:regionArea>Western Australian Centre for Thrombosis and Haemostasis, Murdoch University, Perth</wicri:regionArea>
<wicri:noRegion>Perth</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Fischer, Kathelijn" sort="Fischer, Kathelijn" uniqKey="Fischer K" first="Kathelijn" last="Fischer">Kathelijn Fischer</name>
<affiliation wicri:level="3"><nlm:aff id="aff14">Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands;</nlm:aff>
<country xml:lang="fr">Pays-Bas</country>
<wicri:regionArea>Van Creveldkliniek, University Medical Center Utrecht, Utrecht</wicri:regionArea>
<placeName><settlement type="city">Utrecht</settlement>
<region nuts="2" type="province">Utrecht (province)</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Gill, Joan C" sort="Gill, Joan C" uniqKey="Gill J" first="Joan C." last="Gill">Joan C. Gill</name>
<affiliation wicri:level="2"><nlm:aff id="aff15">Blood Center of Wisconsin and Medical College of Wisconsin, Milwaukee, WI;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Wisconsin</region>
</placeName>
<wicri:cityArea>Blood Center of Wisconsin and Medical College of Wisconsin, Milwaukee</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="P G, Stephanie" sort="P G, Stephanie" uniqKey="P G S" first="Stephanie" last="P G">Stephanie P G</name>
<affiliation wicri:level="1"><nlm:aff id="aff16">Royal Perth Hospital, Perth, Australia;</nlm:aff>
<country xml:lang="fr">Australie</country>
<wicri:regionArea>Royal Perth Hospital, Perth</wicri:regionArea>
<wicri:noRegion>Perth</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Chowdary, Pratima" sort="Chowdary, Pratima" uniqKey="Chowdary P" first="Pratima" last="Chowdary">Pratima Chowdary</name>
<affiliation wicri:level="3"><nlm:aff id="aff17">Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, United Kingdom;</nlm:aff>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London</wicri:regionArea>
<placeName><settlement type="city">Londres</settlement>
<region type="country">Angleterre</region>
<region type="région" nuts="1">Grand Londres</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Escobar, Miguel A" sort="Escobar, Miguel A" uniqKey="Escobar M" first="Miguel A." last="Escobar">Miguel A. Escobar</name>
<affiliation wicri:level="2"><nlm:aff id="aff18">University of Texas Health Science Center and The Gulf States Hemophilia and Thrombophilia Center, Houston, TX;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Texas</region>
</placeName>
<wicri:cityArea>University of Texas Health Science Center and The Gulf States Hemophilia and Thrombophilia Center, Houston</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="Khayat, Claudia Djambas" sort="Khayat, Claudia Djambas" uniqKey="Khayat C" first="Claudia Djambas" last="Khayat">Claudia Djambas Khayat</name>
<affiliation wicri:level="1"><nlm:aff id="aff19">Lebanon Department of Pediatrics, Hotel Dieu de France Hospital, Beirut, Lebanon;</nlm:aff>
<country xml:lang="fr">Liban</country>
<wicri:regionArea>Lebanon Department of Pediatrics, Hotel Dieu de France Hospital, Beirut</wicri:regionArea>
<wicri:noRegion>Beirut</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Rusen, Luminita" sort="Rusen, Luminita" uniqKey="Rusen L" first="Luminita" last="Rusen">Luminita Rusen</name>
<affiliation wicri:level="1"><nlm:aff id="aff20">Sanador SRL, Bucharest, Romania;</nlm:aff>
<country xml:lang="fr">Roumanie</country>
<wicri:regionArea>Sanador SRL, Bucharest</wicri:regionArea>
<wicri:noRegion>Bucharest</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Bensen Kennedy, Debra" sort="Bensen Kennedy, Debra" uniqKey="Bensen Kennedy D" first="Debra" last="Bensen-Kennedy">Debra Bensen-Kennedy</name>
<affiliation wicri:level="2"><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
<wicri:cityArea>Clinical Research and Development, CSL Behring, King of Prussia</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="Blackman, Nicole" sort="Blackman, Nicole" uniqKey="Blackman N" first="Nicole" last="Blackman">Nicole Blackman</name>
<affiliation wicri:level="2"><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
<wicri:cityArea>Clinical Research and Development, CSL Behring, King of Prussia</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="Limsakun, Tharin" sort="Limsakun, Tharin" uniqKey="Limsakun T" first="Tharin" last="Limsakun">Tharin Limsakun</name>
<affiliation wicri:level="2"><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
<wicri:cityArea>Clinical Research and Development, CSL Behring, King of Prussia</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="Veldman, Alex" sort="Veldman, Alex" uniqKey="Veldman A" first="Alex" last="Veldman">Alex Veldman</name>
<affiliation wicri:level="3"><nlm:aff wicri:cut="; and" id="aff22">Clinical Research and Development, CSL Behring, Marburg, Germany</nlm:aff>
<country xml:lang="fr">Allemagne</country>
<wicri:regionArea>Clinical Research and Development, CSL Behring, Marburg</wicri:regionArea>
<placeName><region type="land" nuts="1">Hesse (Land)</region>
<region type="district" nuts="2">District de Giessen</region>
<settlement type="city">Marbourg</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="St Ledger, Katie" sort="St Ledger, Katie" uniqKey="St Ledger K" first="Katie" last="St. Ledger">Katie St. Ledger</name>
<affiliation wicri:level="2"><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
<wicri:cityArea>Clinical Research and Development, CSL Behring, King of Prussia</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="Pabinger, Ingrid" sort="Pabinger, Ingrid" uniqKey="Pabinger I" first="Ingrid" last="Pabinger">Ingrid Pabinger</name>
<affiliation wicri:level="3"><nlm:aff id="aff23">Clinical Division of Haematology and Haemostaseology, Medical Clinic I, Medical University Vienna, Vienna, Austria</nlm:aff>
<country xml:lang="fr">Autriche</country>
<wicri:regionArea>Clinical Division of Haematology and Haemostaseology, Medical Clinic I, Medical University Vienna, Vienna</wicri:regionArea>
<placeName><settlement type="city">Vienne (Autriche)</settlement>
<region nuts="2" type="province">Vienne (Autriche)</region>
</placeName>
</affiliation>
</author>
</analytic>
<series><title level="j">Blood</title>
<idno type="ISSN">0006-4971</idno>
<idno type="eISSN">1528-0020</idno>
<imprint><date when="2016">2016</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
</fileDesc>
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</teiHeader>
<front><div type="abstract" xml:lang="en"><title>Key Points</title>
<p><list list-type="bullet"><list-item><p>rVIII-SingleChain is a novel rFVIII, designed to have high stability and high binding affinity for VWF.</p>
</list-item>
<list-item><p>In severe hemophilia A patients, rVIII-SingleChain was well tolerated and resulted in low bleeding rates, when dosed twice per week.</p>
</list-item>
</list>
</p>
</div>
</front>
</TEI>
<pmc article-type="research-article"><pmc-comment>The publisher of this article does not allow downloading of the full text in XML form.</pmc-comment>
<front><journal-meta><journal-id journal-id-type="nlm-ta">Blood</journal-id>
<journal-id journal-id-type="iso-abbrev">Blood</journal-id>
<journal-id journal-id-type="hwp">bloodjournal</journal-id>
<journal-id journal-id-type="pmc">blood</journal-id>
<journal-id journal-id-type="publisher-id">Blood</journal-id>
<journal-title-group><journal-title>Blood</journal-title>
</journal-title-group>
<issn pub-type="ppub">0006-4971</issn>
<issn pub-type="epub">1528-0020</issn>
<publisher><publisher-name>American Society of Hematology</publisher-name>
<publisher-loc>Washington, DC</publisher-loc>
</publisher>
</journal-meta>
<article-meta><article-id pub-id-type="pmid">27330001</article-id>
<article-id pub-id-type="pmc">4974198</article-id>
<article-id pub-id-type="publisher-id">2016/687434</article-id>
<article-id pub-id-type="doi">10.1182/blood-2016-01-687434</article-id>
<article-categories><subj-group subj-group-type="hwp-journal-coll"><subject>29</subject>
<subject>8</subject>
<subject>37</subject>
</subj-group>
<subj-group subj-group-type="heading"><subject>Clinical Trials and Observations</subject>
</subj-group>
</article-categories>
<title-group><article-title>Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A</article-title>
</title-group>
<contrib-group><contrib contrib-type="author"><name><surname>Mahlangu</surname>
<given-names>Johnny</given-names>
</name>
<xref ref-type="aff" rid="aff1">1</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Kuliczkowski</surname>
<given-names>Kazimierz</given-names>
</name>
<xref ref-type="aff" rid="aff2">2</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Karim</surname>
<given-names>Faraizah Abdul</given-names>
</name>
<xref ref-type="aff" rid="aff3">3</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Stasyshyn</surname>
<given-names>Oleksandra</given-names>
</name>
<xref ref-type="aff" rid="aff4">4</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Kosinova</surname>
<given-names>Marina V.</given-names>
</name>
<xref ref-type="aff" rid="aff5">5</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Lepatan</surname>
<given-names>Lynda Mae</given-names>
</name>
<xref ref-type="aff" rid="aff6">6</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Skotnicki</surname>
<given-names>Aleksander</given-names>
</name>
<xref ref-type="aff" rid="aff7">7</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Boggio</surname>
<given-names>Lisa N.</given-names>
</name>
<xref ref-type="aff" rid="aff8">8</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Klamroth</surname>
<given-names>Robert</given-names>
</name>
<xref ref-type="aff" rid="aff9">9</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Oldenburg</surname>
<given-names>Johannes</given-names>
</name>
<xref ref-type="aff" rid="aff10">10</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Hellmann</surname>
<given-names>Andrzej</given-names>
</name>
<xref ref-type="aff" rid="aff11">11</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Santagostino</surname>
<given-names>Elena</given-names>
</name>
<xref ref-type="aff" rid="aff12">12</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Baker</surname>
<given-names>Ross I.</given-names>
</name>
<xref ref-type="aff" rid="aff13">13</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Fischer</surname>
<given-names>Kathelijn</given-names>
</name>
<xref ref-type="aff" rid="aff14">14</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Gill</surname>
<given-names>Joan C.</given-names>
</name>
<xref ref-type="aff" rid="aff15">15</xref>
</contrib>
<contrib contrib-type="author"><name><surname>P’Ng</surname>
<given-names>Stephanie</given-names>
</name>
<xref ref-type="aff" rid="aff16">16</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Chowdary</surname>
<given-names>Pratima</given-names>
</name>
<xref ref-type="aff" rid="aff17">17</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Escobar</surname>
<given-names>Miguel A.</given-names>
</name>
<xref ref-type="aff" rid="aff18">18</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Khayat</surname>
<given-names>Claudia Djambas</given-names>
</name>
<xref ref-type="aff" rid="aff19">19</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Rusen</surname>
<given-names>Luminita</given-names>
</name>
<xref ref-type="aff" rid="aff20">20</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Bensen-Kennedy</surname>
<given-names>Debra</given-names>
</name>
<xref ref-type="aff" rid="aff21">21</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Blackman</surname>
<given-names>Nicole</given-names>
</name>
<xref ref-type="aff" rid="aff21">21</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Limsakun</surname>
<given-names>Tharin</given-names>
</name>
<xref ref-type="aff" rid="aff21">21</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Veldman</surname>
<given-names>Alex</given-names>
</name>
<xref ref-type="aff" rid="aff22">22</xref>
</contrib>
<contrib contrib-type="author"><name><surname>St. Ledger</surname>
<given-names>Katie</given-names>
</name>
<xref ref-type="aff" rid="aff21">21</xref>
</contrib>
<contrib contrib-type="author" corresp="yes"><name><surname>Pabinger</surname>
<given-names>Ingrid</given-names>
</name>
<xref ref-type="aff" rid="aff23">23</xref>
</contrib>
<contrib contrib-type="author"><collab>for the AFFINITY Investigators</collab>
</contrib>
<aff id="aff1"><label>1</label>
Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, National Health Laboratory Service, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa;</aff>
<aff id="aff2"><label>2</label>
Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland;</aff>
<aff id="aff3"><label>3</label>
Hemophilia Clinic, National Blood Centre, Kuala Lumpur, Malaysia;</aff>
<aff id="aff4"><label>4</label>
Institute of Blood Pathology and Transfusion Medicine, National Academy of Medical Sciences of Ukraine, Lviv, Ukraine;</aff>
<aff id="aff5"><label>5</label>
Kemerovo Regional Clinical Hospital, Kemerovo, Russia;</aff>
<aff id="aff6"><label>6</label>
Perpetual Succour Hospital, Cebu, Philippines;</aff>
<aff id="aff7"><label>7</label>
Krakow Medical Centre, Krakow, Poland;</aff>
<aff id="aff8"><label>8</label>
Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL;</aff>
<aff id="aff9"><label>9</label>
Vivantes Clinic Friedrichshain, Berlin, Germany;</aff>
<aff id="aff10"><label>10</label>
Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;</aff>
<aff id="aff11"><label>11</label>
Department of Hematology and Transplantation, Medical University of Gdansk, Gdansk, Poland;</aff>
<aff id="aff12"><label>12</label>
A. Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy;</aff>
<aff id="aff13"><label>13</label>
Western Australian Centre for Thrombosis and Haemostasis, Murdoch University, Perth, Australia;</aff>
<aff id="aff14"><label>14</label>
Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands;</aff>
<aff id="aff15"><label>15</label>
Blood Center of Wisconsin and Medical College of Wisconsin, Milwaukee, WI;</aff>
<aff id="aff16"><label>16</label>
Royal Perth Hospital, Perth, Australia;</aff>
<aff id="aff17"><label>17</label>
Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, United Kingdom;</aff>
<aff id="aff18"><label>18</label>
University of Texas Health Science Center and The Gulf States Hemophilia and Thrombophilia Center, Houston, TX;</aff>
<aff id="aff19"><label>19</label>
Lebanon Department of Pediatrics, Hotel Dieu de France Hospital, Beirut, Lebanon;</aff>
<aff id="aff20"><label>20</label>
Sanador SRL, Bucharest, Romania;</aff>
<aff id="aff21"><label>21</label>
Clinical Research and Development, CSL Behring, King of Prussia, PA;</aff>
<aff id="aff22"><label>22</label>
Clinical Research and Development, CSL Behring, Marburg, Germany; and</aff>
<aff id="aff23"><label>23</label>
Clinical Division of Haematology and Haemostaseology, Medical Clinic I, Medical University Vienna, Vienna, Austria</aff>
</contrib-group>
<pub-date pub-type="ppub"><day>4</day>
<month>8</month>
<year>2016</year>
</pub-date>
<pub-date pub-type="epreprint"><day>21</day>
<month>6</month>
<year>2016</year>
</pub-date>
<pub-date pub-type="pmc-release"><day>4</day>
<month>8</month>
<year>2016</year>
</pub-date>
<pmc-comment> PMC Release delay is 0 months and 0 days and was based on the . </pmc-comment>
<volume>128</volume>
<issue>5</issue>
<fpage>630</fpage>
<lpage>637</lpage>
<history><date date-type="received"><day>05</day>
<month>1</month>
<year>2016</year>
</date>
<date date-type="accepted"><day>02</day>
<month>6</month>
<year>2016</year>
</date>
</history>
<permissions><copyright-statement>© 2016 by The American Society of Hematology</copyright-statement>
<copyright-year>2016</copyright-year>
</permissions>
<self-uri xlink:title="pdf" xlink:type="simple" xlink:href="630.pdf"></self-uri>
<abstract abstract-type="executive-summary"><title>Key Points</title>
<p><list list-type="bullet"><list-item><p>rVIII-SingleChain is a novel rFVIII, designed to have high stability and high binding affinity for VWF.</p>
</list-item>
<list-item><p>In severe hemophilia A patients, rVIII-SingleChain was well tolerated and resulted in low bleeding rates, when dosed twice per week.</p>
</list-item>
</list>
</p>
</abstract>
<abstract><p>Recombinant VIII (rVIII)-SingleChain is a novel B-domain–truncated recombinant factor VIII (rFVIII), comprised of covalently bonded factor VIII (FVIII) heavy and light chains. It was designed to have a higher binding affinity for von Willebrand factor (VWF). This phase 1/3 study investigated the efficacy and safety of rVIII-SingleChain in the treatment of bleeding episodes, routine prophylaxis, and surgical prophylaxis. Participants were ≥12 years of age, with severe hemophilia A (endogenous FVIII <1%). The participants were allocated by the investigator to receive rVIII-SingleChain in either an on-demand or prophylaxis regimen. Of the 175 patients meeting study eligibility criteria, 173 were treated with rVIII-SingleChain, prophylactically (N = 146) or on-demand (N = 27). The total cumulative exposure was 14 306 exposure days (EDs), with 120 participants reaching ≥50 EDs and 52 participants having ≥100 EDs. Hemostatic efficacy was rated by the investigator as excellent or good in 93.8% of the 835 bleeds treated and assessed. Across all prophylaxis regimens, the median annualized spontaneous bleeding rate was 0.00 (Q1, Q3: 0.0, 2.4) and the median overall annualized bleeding rate (ABR) was 1.14 (Q1, Q3: 0.0, 4.2). Surgical hemostasis was rated as excellent/good in 100% of major surgeries by the investigator. No participant developed FVIII inhibitors. In conclusion, rVIII-SingleChain is a novel rFVIII molecule showing excellent hemostatic efficacy in surgery and in the control of bleeding events, low ABR in patients on prophylaxis, and a favorable safety profile in this large clinical study. This trial was registered at <ext-link ext-link-type="uri" xlink:href="http://www.clinicaltrials.gov">www.clinicaltrials.gov</ext-link>
as #NCT01486927.</p>
</abstract>
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<meta-value>free</meta-value>
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</front>
</pmc>
<affiliations><list><country><li>Afrique du Sud</li>
<li>Allemagne</li>
<li>Australie</li>
<li>Autriche</li>
<li>Italie</li>
<li>Liban</li>
<li>Malaisie</li>
<li>Pays-Bas</li>
<li>Philippines</li>
<li>Pologne</li>
<li>Roumanie</li>
<li>Royaume-Uni</li>
<li>Russie</li>
<li>Ukraine</li>
<li>États-Unis</li>
</country>
<region><li>Angleterre</li>
<li>Berlin</li>
<li>District de Cologne</li>
<li>District de Giessen</li>
<li>Gauteng</li>
<li>Grand Londres</li>
<li>Hesse (Land)</li>
<li>Illinois</li>
<li>Lombardie</li>
<li>Pennsylvanie</li>
<li>Rhénanie-du-Nord-Westphalie</li>
<li>Texas</li>
<li>Utrecht (province)</li>
<li>Vienne (Autriche)</li>
<li>Wisconsin</li>
</region>
<settlement><li>Berlin</li>
<li>Bonn</li>
<li>Johannesbourg</li>
<li>Londres</li>
<li>Marbourg</li>
<li>Milan</li>
<li>Utrecht</li>
<li>Vienne (Autriche)</li>
</settlement>
<orgName><li>Université du Witwatersrand</li>
</orgName>
</list>
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