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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A</title><author><name sortKey="Mahlangu, Johnny" sort="Mahlangu, Johnny" uniqKey="Mahlangu J" first="Johnny" last="Mahlangu">Johnny Mahlangu</name><affiliation><nlm:aff id="aff1">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;</nlm:aff></affiliation></author><author><name sortKey="Kuliczkowski, Kazimierz" sort="Kuliczkowski, Kazimierz" uniqKey="Kuliczkowski K" first="Kazimierz" last="Kuliczkowski">Kazimierz Kuliczkowski</name><affiliation><nlm:aff id="aff2">Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland;</nlm:aff></affiliation></author><author><name sortKey="Karim, Faraizah Abdul" sort="Karim, Faraizah Abdul" uniqKey="Karim F" first="Faraizah Abdul" last="Karim">Faraizah Abdul Karim</name><affiliation><nlm:aff id="aff3">Hemophilia Clinic, National Blood Centre, Kuala Lumpur, Malaysia;</nlm:aff></affiliation></author><author><name sortKey="Stasyshyn, Oleksandra" sort="Stasyshyn, Oleksandra" uniqKey="Stasyshyn O" first="Oleksandra" last="Stasyshyn">Oleksandra Stasyshyn</name><affiliation><nlm:aff id="aff4">Institute of Blood Pathology and Transfusion Medicine, National Academy of Medical Sciences of Ukraine, Lviv, Ukraine;</nlm:aff></affiliation></author><author><name sortKey="Kosinova, Marina V" sort="Kosinova, Marina V" uniqKey="Kosinova M" first="Marina V." last="Kosinova">Marina V. Kosinova</name><affiliation><nlm:aff id="aff5">Kemerovo Regional Clinical Hospital, Kemerovo, Russia;</nlm:aff></affiliation></author><author><name sortKey="Lepatan, Lynda Mae" sort="Lepatan, Lynda Mae" uniqKey="Lepatan L" first="Lynda Mae" last="Lepatan">Lynda Mae Lepatan</name><affiliation><nlm:aff id="aff6">Perpetual Succour Hospital, Cebu, Philippines;</nlm:aff></affiliation></author><author><name sortKey="Skotnicki, Aleksander" sort="Skotnicki, Aleksander" uniqKey="Skotnicki A" first="Aleksander" last="Skotnicki">Aleksander Skotnicki</name><affiliation><nlm:aff id="aff7">Krakow Medical Centre, Krakow, Poland;</nlm:aff></affiliation></author><author><name sortKey="Boggio, Lisa N" sort="Boggio, Lisa N" uniqKey="Boggio L" first="Lisa N." last="Boggio">Lisa N. Boggio</name><affiliation><nlm:aff id="aff8">Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL;</nlm:aff></affiliation></author><author><name sortKey="Klamroth, Robert" sort="Klamroth, Robert" uniqKey="Klamroth R" first="Robert" last="Klamroth">Robert Klamroth</name><affiliation><nlm:aff id="aff9">Vivantes Clinic Friedrichshain, Berlin, Germany;</nlm:aff></affiliation></author><author><name sortKey="Oldenburg, Johannes" sort="Oldenburg, Johannes" uniqKey="Oldenburg J" first="Johannes" last="Oldenburg">Johannes Oldenburg</name><affiliation><nlm:aff id="aff10">Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;</nlm:aff></affiliation></author><author><name sortKey="Hellmann, Andrzej" sort="Hellmann, Andrzej" uniqKey="Hellmann A" first="Andrzej" last="Hellmann">Andrzej Hellmann</name><affiliation><nlm:aff id="aff11">Department of Hematology and Transplantation, Medical University of Gdansk, Gdansk, Poland;</nlm:aff></affiliation></author><author><name sortKey="Santagostino, Elena" sort="Santagostino, Elena" uniqKey="Santagostino E" first="Elena" last="Santagostino">Elena Santagostino</name><affiliation><nlm:aff id="aff12">A. Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy;</nlm:aff></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><nlm:aff id="aff13">Western Australian Centre for Thrombosis and Haemostasis, Murdoch University, Perth, Australia;</nlm:aff></affiliation></author><author><name sortKey="Fischer, Kathelijn" sort="Fischer, Kathelijn" uniqKey="Fischer K" first="Kathelijn" last="Fischer">Kathelijn Fischer</name><affiliation><nlm:aff id="aff14">Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands;</nlm:aff></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><nlm:aff id="aff15">Blood Center of Wisconsin and Medical College of Wisconsin, Milwaukee, WI;</nlm:aff></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><nlm:aff id="aff16">Royal Perth Hospital, Perth, Australia;</nlm:aff></affiliation></author><author><name sortKey="Chowdary, Pratima" sort="Chowdary, Pratima" uniqKey="Chowdary P" first="Pratima" last="Chowdary">Pratima Chowdary</name><affiliation><nlm:aff id="aff17">Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, United Kingdom;</nlm:aff></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><nlm:aff id="aff18">University of Texas Health Science Center and The Gulf States Hemophilia and Thrombophilia Center, Houston, TX;</nlm:aff></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><nlm:aff id="aff19">Lebanon Department of Pediatrics, Hotel Dieu de France Hospital, Beirut, Lebanon;</nlm:aff></affiliation></author><author><name sortKey="Rusen, Luminita" sort="Rusen, Luminita" uniqKey="Rusen L" first="Luminita" last="Rusen">Luminita Rusen</name><affiliation><nlm:aff id="aff20">Sanador SRL, Bucharest, Romania;</nlm:aff></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><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff></affiliation></author><author><name sortKey="Blackman, Nicole" sort="Blackman, Nicole" uniqKey="Blackman N" first="Nicole" last="Blackman">Nicole Blackman</name><affiliation><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff></affiliation></author><author><name sortKey="Limsakun, Tharin" sort="Limsakun, Tharin" uniqKey="Limsakun T" first="Tharin" last="Limsakun">Tharin Limsakun</name><affiliation><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff></affiliation></author><author><name sortKey="Veldman, Alex" sort="Veldman, Alex" uniqKey="Veldman A" first="Alex" last="Veldman">Alex Veldman</name><affiliation><nlm:aff wicri:cut="; and" id="aff22">Clinical Research and Development, CSL Behring, Marburg, Germany</nlm:aff></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><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff></affiliation></author><author><name sortKey="Pabinger, Ingrid" sort="Pabinger, Ingrid" uniqKey="Pabinger I" first="Ingrid" last="Pabinger">Ingrid Pabinger</name><affiliation><nlm:aff id="aff23">Clinical Division of Haematology and Haemostaseology, Medical Clinic I, Medical University Vienna, Vienna, Austria</nlm:aff></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">27330001</idno><idno type="pmc">4974198</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4974198</idno><idno type="RBID">PMC:4974198</idno><idno type="doi">10.1182/blood-2016-01-687434</idno><date when="2016">2016</date><idno type="wicri:Area/Pmc/Corpus">001809</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">001809</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A</title><author><name sortKey="Mahlangu, Johnny" sort="Mahlangu, Johnny" uniqKey="Mahlangu J" first="Johnny" last="Mahlangu">Johnny Mahlangu</name><affiliation><nlm:aff id="aff1">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;</nlm:aff></affiliation></author><author><name sortKey="Kuliczkowski, Kazimierz" sort="Kuliczkowski, Kazimierz" uniqKey="Kuliczkowski K" first="Kazimierz" last="Kuliczkowski">Kazimierz Kuliczkowski</name><affiliation><nlm:aff id="aff2">Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland;</nlm:aff></affiliation></author><author><name sortKey="Karim, Faraizah Abdul" sort="Karim, Faraizah Abdul" uniqKey="Karim F" first="Faraizah Abdul" last="Karim">Faraizah Abdul Karim</name><affiliation><nlm:aff id="aff3">Hemophilia Clinic, National Blood Centre, Kuala Lumpur, Malaysia;</nlm:aff></affiliation></author><author><name sortKey="Stasyshyn, Oleksandra" sort="Stasyshyn, Oleksandra" uniqKey="Stasyshyn O" first="Oleksandra" last="Stasyshyn">Oleksandra Stasyshyn</name><affiliation><nlm:aff id="aff4">Institute of Blood Pathology and Transfusion Medicine, National Academy of Medical Sciences of Ukraine, Lviv, Ukraine;</nlm:aff></affiliation></author><author><name sortKey="Kosinova, Marina V" sort="Kosinova, Marina V" uniqKey="Kosinova M" first="Marina V." last="Kosinova">Marina V. Kosinova</name><affiliation><nlm:aff id="aff5">Kemerovo Regional Clinical Hospital, Kemerovo, Russia;</nlm:aff></affiliation></author><author><name sortKey="Lepatan, Lynda Mae" sort="Lepatan, Lynda Mae" uniqKey="Lepatan L" first="Lynda Mae" last="Lepatan">Lynda Mae Lepatan</name><affiliation><nlm:aff id="aff6">Perpetual Succour Hospital, Cebu, Philippines;</nlm:aff></affiliation></author><author><name sortKey="Skotnicki, Aleksander" sort="Skotnicki, Aleksander" uniqKey="Skotnicki A" first="Aleksander" last="Skotnicki">Aleksander Skotnicki</name><affiliation><nlm:aff id="aff7">Krakow Medical Centre, Krakow, Poland;</nlm:aff></affiliation></author><author><name sortKey="Boggio, Lisa N" sort="Boggio, Lisa N" uniqKey="Boggio L" first="Lisa N." last="Boggio">Lisa N. Boggio</name><affiliation><nlm:aff id="aff8">Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL;</nlm:aff></affiliation></author><author><name sortKey="Klamroth, Robert" sort="Klamroth, Robert" uniqKey="Klamroth R" first="Robert" last="Klamroth">Robert Klamroth</name><affiliation><nlm:aff id="aff9">Vivantes Clinic Friedrichshain, Berlin, Germany;</nlm:aff></affiliation></author><author><name sortKey="Oldenburg, Johannes" sort="Oldenburg, Johannes" uniqKey="Oldenburg J" first="Johannes" last="Oldenburg">Johannes Oldenburg</name><affiliation><nlm:aff id="aff10">Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;</nlm:aff></affiliation></author><author><name sortKey="Hellmann, Andrzej" sort="Hellmann, Andrzej" uniqKey="Hellmann A" first="Andrzej" last="Hellmann">Andrzej Hellmann</name><affiliation><nlm:aff id="aff11">Department of Hematology and Transplantation, Medical University of Gdansk, Gdansk, Poland;</nlm:aff></affiliation></author><author><name sortKey="Santagostino, Elena" sort="Santagostino, Elena" uniqKey="Santagostino E" first="Elena" last="Santagostino">Elena Santagostino</name><affiliation><nlm:aff id="aff12">A. Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy;</nlm:aff></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><nlm:aff id="aff13">Western Australian Centre for Thrombosis and Haemostasis, Murdoch University, Perth, Australia;</nlm:aff></affiliation></author><author><name sortKey="Fischer, Kathelijn" sort="Fischer, Kathelijn" uniqKey="Fischer K" first="Kathelijn" last="Fischer">Kathelijn Fischer</name><affiliation><nlm:aff id="aff14">Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands;</nlm:aff></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><nlm:aff id="aff15">Blood Center of Wisconsin and Medical College of Wisconsin, Milwaukee, WI;</nlm:aff></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><nlm:aff id="aff16">Royal Perth Hospital, Perth, Australia;</nlm:aff></affiliation></author><author><name sortKey="Chowdary, Pratima" sort="Chowdary, Pratima" uniqKey="Chowdary P" first="Pratima" last="Chowdary">Pratima Chowdary</name><affiliation><nlm:aff id="aff17">Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, United Kingdom;</nlm:aff></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><nlm:aff id="aff18">University of Texas Health Science Center and The Gulf States Hemophilia and Thrombophilia Center, Houston, TX;</nlm:aff></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><nlm:aff id="aff19">Lebanon Department of Pediatrics, Hotel Dieu de France Hospital, Beirut, Lebanon;</nlm:aff></affiliation></author><author><name sortKey="Rusen, Luminita" sort="Rusen, Luminita" uniqKey="Rusen L" first="Luminita" last="Rusen">Luminita Rusen</name><affiliation><nlm:aff id="aff20">Sanador SRL, Bucharest, Romania;</nlm:aff></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><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff></affiliation></author><author><name sortKey="Blackman, Nicole" sort="Blackman, Nicole" uniqKey="Blackman N" first="Nicole" last="Blackman">Nicole Blackman</name><affiliation><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff></affiliation></author><author><name sortKey="Limsakun, Tharin" sort="Limsakun, Tharin" uniqKey="Limsakun T" first="Tharin" last="Limsakun">Tharin Limsakun</name><affiliation><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff></affiliation></author><author><name sortKey="Veldman, Alex" sort="Veldman, Alex" uniqKey="Veldman A" first="Alex" last="Veldman">Alex Veldman</name><affiliation><nlm:aff wicri:cut="; and" id="aff22">Clinical Research and Development, CSL Behring, Marburg, Germany</nlm:aff></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><nlm:aff id="aff21">Clinical Research and Development, CSL Behring, King of Prussia, PA;</nlm:aff></affiliation></author><author><name sortKey="Pabinger, Ingrid" sort="Pabinger, Ingrid" uniqKey="Pabinger I" first="Ingrid" last="Pabinger">Ingrid Pabinger</name><affiliation><nlm:aff id="aff23">Clinical Division of Haematology and Haemostaseology, Medical Clinic I, Medical University Vienna, Vienna, Austria</nlm:aff></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><profileDesc><textClass></textClass></profileDesc></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
<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><counts><page-count count="8"></page-count></counts><custom-meta-group><custom-meta><meta-name>access</meta-name><meta-value>free</meta-value></custom-meta></custom-meta-group></article-meta></front></pmc></record>Pour manipuler ce document sous Unix (Dilib)
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