Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A
Identifieur interne : 002011 ( Main/Merge ); précédent : 002010; suivant : 002012Efficacy 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.
Descripteurs français
- KwdFr :
- Administration par voie intraveineuse, Adolescent, Adulte, Adulte d'âge moyen, Démographie, Enfant, Facteur VIII (administration et posologie), Facteur VIII (effets indésirables), Facteur VIII (pharmacocinétique), Facteur VIII (usage thérapeutique), Humains, Hémophilie A (), Hémophilie A (traitement médicamenteux), Hémorragie (traitement médicamenteux), Hémostase (), Jeune adulte, Mâle, Protéines recombinantes (administration et posologie), Protéines recombinantes (effets indésirables), Protéines recombinantes (pharmacocinétique), Protéines recombinantes (usage thérapeutique), Relation dose-effet des médicaments, Résultat thérapeutique.
- MESH :
- administration et posologie : Facteur VIII, Protéines recombinantes.
- effets indésirables : Facteur VIII, Protéines recombinantes.
- pharmacocinétique : Facteur VIII, Protéines recombinantes.
- traitement médicamenteux : Hémophilie A, Hémorragie.
- usage thérapeutique : Facteur VIII, Protéines recombinantes.
- Administration par voie intraveineuse, Adolescent, Adulte, Adulte d'âge moyen, Démographie, Enfant, Humains, Hémophilie A, Hémostase, Jeune adulte, Mâle, Relation dose-effet des médicaments, Résultat thérapeutique.
English descriptors
- KwdEn :
- Administration, Intravenous, Adolescent, Adult, Child, Demography, Dose-Response Relationship, Drug, Factor VIII (administration & dosage), Factor VIII (adverse effects), Factor VIII (pharmacokinetics), Factor VIII (therapeutic use), Hemophilia A (drug therapy), Hemophilia A (prevention & control), Hemorrhage (drug therapy), Hemostasis (drug effects), Humans, Male, Middle Aged, Recombinant Proteins (administration & dosage), Recombinant Proteins (adverse effects), Recombinant Proteins (pharmacokinetics), Recombinant Proteins (therapeutic use), Treatment Outcome, Young Adult.
- MESH :
- chemical , administration & dosage : Factor VIII, Recombinant Proteins.
- chemical , adverse effects : Factor VIII, Recombinant Proteins.
- chemical , pharmacokinetics : Factor VIII, Recombinant Proteins.
- chemical , therapeutic use : Factor VIII, Recombinant Proteins.
- drug effects : Hemostasis.
- drug therapy : Hemophilia A, Hemorrhage.
- prevention & control : Hemophilia A.
- Administration, Intravenous, Adolescent, Adult, Child, Demography, Dose-Response Relationship, Drug, Humans, Male, Middle Aged, Treatment Outcome, Young Adult.
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
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PMC:4974198Le document en format XML
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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. 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Johannesburg Academic Hospital, Johannesburg</wicri:regionArea><orgName type="university">Université du Witwatersrand</orgName><placeName><settlement type="city">Johannesbourg</settlement><region type="region" nuts="2">Gauteng</region></placeName></affiliation></author><author><name sortKey="Kuliczkowski, Kazimierz" sort="Kuliczkowski, Kazimierz" uniqKey="Kuliczkowski K" first="Kazimierz" last="Kuliczkowski">Kazimierz Kuliczkowski</name><affiliation wicri:level="1"><nlm:aff id="aff2">Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland;</nlm:aff><country xml:lang="fr">Pologne</country><wicri:regionArea>Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw</wicri:regionArea><wicri:noRegion>Wroclaw</wicri:noRegion></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 wicri:level="1"><nlm:aff id="aff3">Hemophilia Clinic, National Blood Centre, Kuala Lumpur, Malaysia;</nlm:aff><country xml:lang="fr">Malaisie</country><wicri:regionArea>Hemophilia Clinic, National Blood Centre, Kuala Lumpur</wicri:regionArea><wicri:noRegion>Kuala Lumpur</wicri:noRegion></affiliation></author><author><name sortKey="Stasyshyn, Oleksandra" sort="Stasyshyn, Oleksandra" uniqKey="Stasyshyn O" first="Oleksandra" last="Stasyshyn">Oleksandra Stasyshyn</name><affiliation wicri:level="1"><nlm:aff id="aff4">Institute of Blood Pathology and Transfusion Medicine, National Academy of Medical Sciences of Ukraine, Lviv, Ukraine;</nlm:aff><country xml:lang="fr">Ukraine</country><wicri:regionArea>Institute of Blood Pathology and Transfusion Medicine, National Academy of Medical Sciences of Ukraine, Lviv</wicri:regionArea><wicri:noRegion>Lviv</wicri:noRegion></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 wicri:level="1"><nlm:aff id="aff5">Kemerovo Regional Clinical Hospital, Kemerovo, Russia;</nlm:aff><country xml:lang="fr">Russie</country><wicri:regionArea>Kemerovo Regional Clinical Hospital, Kemerovo</wicri:regionArea><wicri:noRegion>Kemerovo</wicri:noRegion></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 wicri:level="1"><nlm:aff id="aff6">Perpetual Succour Hospital, Cebu, Philippines;</nlm:aff><country xml:lang="fr">Philippines</country><wicri:regionArea>Perpetual Succour Hospital, Cebu</wicri:regionArea><wicri:noRegion>Cebu</wicri:noRegion></affiliation></author><author><name sortKey="Skotnicki, Aleksander" sort="Skotnicki, Aleksander" uniqKey="Skotnicki A" first="Aleksander" last="Skotnicki">Aleksander Skotnicki</name><affiliation wicri:level="1"><nlm:aff id="aff7">Krakow Medical Centre, Krakow, Poland;</nlm:aff><country xml:lang="fr">Pologne</country><wicri:regionArea>Krakow Medical Centre, Krakow</wicri:regionArea><wicri:noRegion>Krakow</wicri:noRegion></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 wicri:level="2"><nlm:aff id="aff8">Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL;</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Illinois</region></placeName><wicri:cityArea>Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago</wicri:cityArea></affiliation></author><author><name sortKey="Klamroth, Robert" sort="Klamroth, Robert" uniqKey="Klamroth R" first="Robert" last="Klamroth">Robert Klamroth</name><affiliation wicri:level="3"><nlm:aff id="aff9">Vivantes Clinic Friedrichshain, Berlin, Germany;</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Vivantes Clinic Friedrichshain, Berlin</wicri:regionArea><placeName><region type="land" nuts="3">Berlin</region><settlement type="city">Berlin</settlement></placeName></affiliation></author><author><name sortKey="Oldenburg, Johannes" sort="Oldenburg, Johannes" uniqKey="Oldenburg J" first="Johannes" last="Oldenburg">Johannes Oldenburg</name><affiliation wicri:level="3"><nlm:aff id="aff10">Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn</wicri:regionArea><placeName><region type="land" nuts="1">Rhénanie-du-Nord-Westphalie</region><region type="district" nuts="2">District de Cologne</region><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><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Administration, Intravenous</term><term>Adolescent</term><term>Adult</term><term>Child</term><term>Demography</term><term>Dose-Response Relationship, Drug</term><term>Factor VIII (administration & dosage)</term><term>Factor VIII (adverse effects)</term><term>Factor VIII (pharmacokinetics)</term><term>Factor VIII (therapeutic use)</term><term>Hemophilia A (drug therapy)</term><term>Hemophilia A (prevention & control)</term><term>Hemorrhage (drug therapy)</term><term>Hemostasis (drug effects)</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Recombinant Proteins (administration & dosage)</term><term>Recombinant Proteins (adverse effects)</term><term>Recombinant Proteins (pharmacokinetics)</term><term>Recombinant Proteins (therapeutic use)</term><term>Treatment Outcome</term><term>Young Adult</term></keywords><keywords scheme="KwdFr" xml:lang="fr"><term>Administration par voie intraveineuse</term><term>Adolescent</term><term>Adulte</term><term>Adulte d'âge moyen</term><term>Démographie</term><term>Enfant</term><term>Facteur VIII (administration et posologie)</term><term>Facteur VIII (effets indésirables)</term><term>Facteur VIII (pharmacocinétique)</term><term>Facteur VIII (usage thérapeutique)</term><term>Humains</term><term>Hémophilie A ()</term><term>Hémophilie A (traitement médicamenteux)</term><term>Hémorragie (traitement médicamenteux)</term><term>Hémostase ()</term><term>Jeune adulte</term><term>Mâle</term><term>Protéines recombinantes (administration et posologie)</term><term>Protéines recombinantes (effets indésirables)</term><term>Protéines recombinantes (pharmacocinétique)</term><term>Protéines recombinantes (usage thérapeutique)</term><term>Relation dose-effet des médicaments</term><term>Résultat thérapeutique</term></keywords><keywords scheme="MESH" type="chemical" qualifier="administration & dosage" xml:lang="en"><term>Factor VIII</term><term>Recombinant Proteins</term></keywords><keywords scheme="MESH" type="chemical" qualifier="adverse effects" xml:lang="en"><term>Factor VIII</term><term>Recombinant Proteins</term></keywords><keywords scheme="MESH" type="chemical" qualifier="pharmacokinetics" xml:lang="en"><term>Factor VIII</term><term>Recombinant Proteins</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Factor VIII</term><term>Recombinant Proteins</term></keywords><keywords scheme="MESH" qualifier="administration et posologie" xml:lang="fr"><term>Facteur VIII</term><term>Protéines recombinantes</term></keywords><keywords scheme="MESH" qualifier="drug effects" xml:lang="en"><term>Hemostasis</term></keywords><keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Hemophilia A</term><term>Hemorrhage</term></keywords><keywords scheme="MESH" qualifier="effets indésirables" xml:lang="fr"><term>Facteur VIII</term><term>Protéines recombinantes</term></keywords><keywords scheme="MESH" qualifier="pharmacocinétique" xml:lang="fr"><term>Facteur VIII</term><term>Protéines recombinantes</term></keywords><keywords scheme="MESH" qualifier="prevention & control" xml:lang="en"><term>Hemophilia A</term></keywords><keywords scheme="MESH" qualifier="traitement médicamenteux" xml:lang="fr"><term>Hémophilie A</term><term>Hémorragie</term></keywords><keywords scheme="MESH" qualifier="usage thérapeutique" xml:lang="fr"><term>Facteur VIII</term><term>Protéines recombinantes</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Administration, Intravenous</term><term>Adolescent</term><term>Adult</term><term>Child</term><term>Demography</term><term>Dose-Response Relationship, Drug</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Treatment Outcome</term><term>Young Adult</term></keywords><keywords scheme="MESH" xml:lang="fr"><term>Administration par voie intraveineuse</term><term>Adolescent</term><term>Adulte</term><term>Adulte d'âge moyen</term><term>Démographie</term><term>Enfant</term><term>Humains</term><term>Hémophilie A</term><term>Hémostase</term><term>Jeune adulte</term><term>Mâle</term><term>Relation dose-effet des médicaments</term><term>Résultat thérapeutique</term></keywords></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></record>Pour manipuler ce document sous Unix (Dilib)
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