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<h1 class="heading-title">
Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial
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data-ga-label="Monica Carpenedo">Monica Carpenedo</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Hematology and Transplant Unit, UOC Hematology and Transplant Unit Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy." href="#full-view-affiliation-4" ref="linksrc=author_aff">
4
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Kuter+DJ&amp;cauthor_id=37778358"
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data-ga-label="David J Kuter">David J Kuter</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Hematology Division, Massachusetts General Hospital, Boston, MA, USA." href="#full-view-affiliation-5" ref="linksrc=author_aff">
5
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Al-Samkari+H&amp;cauthor_id=37778358"
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data-ga-label="Hanny Al-Samkari">Hanny Al-Samkari</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Hematology Division, Massachusetts General Hospital, Boston, MA, USA." href="#full-view-affiliation-5" ref="linksrc=author_aff">
5
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Bussel+JB&amp;cauthor_id=37778358"
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data-ga-label="James B Bussel">James B Bussel</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Division of Hematology and Medical Oncology and Division of Pediatric Hematology and Oncology, Weill Cornell Medical College, New York, NY, USA." href="#full-view-affiliation-6" ref="linksrc=author_aff">
6
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Godar+M&amp;cauthor_id=37778358"
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data-ga-label="Marie Godar">Marie Godar</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="argenx, Ghent, Belgium." href="#full-view-affiliation-7" ref="linksrc=author_aff">
7
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Ayguasanosa+J&amp;cauthor_id=37778358"
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data-ga-label="Jaume Ayguasanosa">Jaume Ayguasanosa</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="argenx, Ghent, Belgium." href="#full-view-affiliation-7" ref="linksrc=author_aff">
7
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=De+Beuf+K&amp;cauthor_id=37778358"
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data-ga-label="Kristof De Beuf">Kristof De Beuf</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="argenx, Ghent, Belgium." href="#full-view-affiliation-7" ref="linksrc=author_aff">
7
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Rodeghiero+F&amp;cauthor_id=37778358"
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data-ga-label="Francesco Rodeghiero">Francesco Rodeghiero</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Haematology Project Foundation, Department of Haematology, S Bortolo Hospital, Vicenza, Italy." href="#full-view-affiliation-8" ref="linksrc=author_aff">
8
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Michel+M&amp;cauthor_id=37778358"
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data-ga-label="Marc Michel">Marc Michel</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Department of Internal Medicine, National Reference Center for Immune Cytopenias, Henri Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris-Est Créteil, Créteil, France." href="#full-view-affiliation-9" ref="linksrc=author_aff">
9
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Newland+A&amp;cauthor_id=37778358"
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data-ga-label="Adrian Newland">Adrian Newland</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Centre for Haematology, Barts and the London School of Medicine &amp; Dentistry, Queen Mary University of London, London, UK." href="#full-view-affiliation-10" ref="linksrc=author_aff">
10
</a></sup><span class="semicolon">;&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="ADVANCE Investigator Study Group">ADVANCE Investigator Study Group</a></span>
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Collaborators
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<strong class="title">ADVANCE Investigator Study Group</strong>:
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<span class="authors-list-item "><a class="full-name"
href="/?term=Fillitz+M"
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data-ga-label="Michael Fillitz">Michael Fillitz</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Knoebl+P"
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data-ga-label="Paul Knoebl">Paul Knoebl</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Meers+S"
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data-ga-label="Stef Meers">Stef Meers</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Amine+IM"
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data-ga-label="Ismail Mohamad Amine">Ismail Mohamad Amine</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Gumulec+J"
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data-ga-label="Jaromir Gumulec">Jaromir Gumulec</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Hlusi+A"
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data-ga-label="Antonin Hlusi">Antonin Hlusi</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Mayer+J"
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data-ga-label="Jiri Mayer">Jiri Mayer</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Quittet+P"
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data-ga-label="Philippe Quittet">Philippe Quittet</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Viallard+JF"
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data-ga-label="Jean-Francois Viallard">Jean-Francois Viallard</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Betaneli+M"
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data-ga-label="Magdana Betaneli">Magdana Betaneli</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Datikashvili-David+I"
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data-ga-label="Irine Datikashvili-David">Irine Datikashvili-David</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Iosava+G"
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data-ga-label="Genadi Iosava">Genadi Iosava</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Makhaldiani+L"
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data-ga-label="Levan Makhaldiani">Levan Makhaldiani</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Rejto+L"
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data-ga-label="Laszlo Rejto">Laszlo Rejto</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Sharashenidze+N"
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data-ga-label="Nino Sharashenidze">Nino Sharashenidze</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Groepper+S"
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data-ga-label="Stefanie Groepper">Stefanie Groepper</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=R%C3%B6th+A"
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data-ga-label="Alexander Röth">Alexander Röth</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Hamed+A"
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data-ga-label="Aryan Hamed">Aryan Hamed</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Illes+A"
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data-ga-label="Arpad Illes">Arpad Illes</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Carpenedo+M"
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data-ga-label="Monica Carpenedo">Monica Carpenedo</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Artoni+A"
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data-ga-label="Andrea Artoni">Andrea Artoni</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Bocchia+M"
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data-ga-label="Monica Bocchia">Monica Bocchia</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Gamberi+B"
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data-ga-label="Barbara Gamberi">Barbara Gamberi</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Borchiellini+A"
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data-ga-label="Alessandra Borchiellini">Alessandra Borchiellini</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Oliva+EN"
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data-ga-label="Esther Natalie Oliva">Esther Natalie Oliva</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Patriarca+A"
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data-ga-label="Andrea Patriarca">Andrea Patriarca</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Tomassetti+S"
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data-ga-label="Simona Tomassetti">Simona Tomassetti</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Miyakawa+Y"
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data-ga-label="Yoshitaka Miyakawa">Yoshitaka Miyakawa</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Ando+K"
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data-ga-label="Kiyoshi Ando">Kiyoshi Ando</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Handa+H"
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data-ga-label="Hiroshi Handa">Hiroshi Handa</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Katsutani+S"
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data-ga-label="Shinya Katsutani">Shinya Katsutani</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Nishiwaki+K"
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data-ga-label="Kaichi Nishiwaki">Kaichi Nishiwaki</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Ito+S"
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data-ga-label="Shoko Ito">Shoko Ito</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Ito+T"
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data-ga-label="Tomoki Ito">Tomoki Ito</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=de+Boer+F"
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data-ga-label="Fransien de Boer">Fransien de Boer</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Jansen+AJG"
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data-ga-label="A J Gerard Jansen">A J Gerard Jansen</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Ciepluch+H"
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data-ga-label="Hanna Ciepluch">Hanna Ciepluch</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Fornagiel+S"
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data-ga-label="Szymon Fornagiel">Szymon Fornagiel</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Grosicki+S"
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data-ga-label="Sebastian Grosicki">Sebastian Grosicki</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Soroka-Wojtaszko+M"
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data-ga-label="Maria Soroka-Wojtaszko">Maria Soroka-Wojtaszko</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Trelinski+J"
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data-ga-label="Jacek Trelinski">Jacek Trelinski</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Bakirov+B"
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data-ga-label="Bulat Bakirov">Bulat Bakirov</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Borisenkova+E"
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data-ga-label="Elena Borisenkova">Elena Borisenkova</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Volodicheva+E"
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data-ga-label="Elena Volodicheva">Elena Volodicheva</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Shatokhin+Y"
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data-ga-label="Yuri Shatokhin">Yuri Shatokhin</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Proydakov+A"
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data-ga-label="Andrey Proydakov">Andrey Proydakov</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Alonso+MA"
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data-ga-label="Maria Aranzazu Alonso">Maria Aranzazu Alonso</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Mingot-Castellano+ME"
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data-ga-label="María Eva Mingot-Castellano">María Eva Mingot-Castellano</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Sanchez-Gonzalez+B"
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data-ga-label="Blanca Sanchez-Gonzalez">Blanca Sanchez-Gonzalez</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Valcarcel+Ferreiras+D"
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data-ga-label="David Valcarcel Ferreiras">David Valcarcel Ferreiras</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Ayli+M"
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data-ga-label="Meltem Ayli">Meltem Ayli</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Hacibekiroglu+T"
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data-ga-label="Tuba Hacibekiroglu">Tuba Hacibekiroglu</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Demir+AM"
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data-ga-label="Ahmet Muzaffer Demir">Ahmet Muzaffer Demir</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Nal%C3%A7aci+M"
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data-ga-label="Meliha Nalçaci">Meliha Nalçaci</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Sonmez+M"
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data-ga-label="Mehmet Sonmez">Mehmet Sonmez</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Tombak+A"
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data-ga-label="Anil Tombak">Anil Tombak</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Toprak+SK"
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data-ga-label="Selami Kocak Toprak">Selami Kocak Toprak</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Turgut+B"
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<li data-affiliation-id="full-view-affiliation-1"
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><sup class="key">1</sup> Lombardi Cancer Center, Division of Hematology, Georgetown University, Washington, DC, USA. Electronic address: catherine.m.broome@gunet.georgetown.edu.</li>
<li data-affiliation-id="full-view-affiliation-2"
id="full-view-affiliation-2"
><sup class="key">2</sup> Department of Clinical Haematology, Barts Health NHS Trust, London, UK.</li>
<li data-affiliation-id="full-view-affiliation-3"
id="full-view-affiliation-3"
><sup class="key">3</sup> Department of Hematology, Saitama Medical University Hospital, Saitama, Japan.</li>
<li data-affiliation-id="full-view-affiliation-4"
id="full-view-affiliation-4"
><sup class="key">4</sup> Hematology and Transplant Unit, UOC Hematology and Transplant Unit Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy.</li>
<li data-affiliation-id="full-view-affiliation-5"
id="full-view-affiliation-5"
><sup class="key">5</sup> Hematology Division, Massachusetts General Hospital, Boston, MA, USA.</li>
<li data-affiliation-id="full-view-affiliation-6"
id="full-view-affiliation-6"
><sup class="key">6</sup> Division of Hematology and Medical Oncology and Division of Pediatric Hematology and Oncology, Weill Cornell Medical College, New York, NY, USA.</li>
<li data-affiliation-id="full-view-affiliation-7"
id="full-view-affiliation-7"
><sup class="key">7</sup> argenx, Ghent, Belgium.</li>
<li data-affiliation-id="full-view-affiliation-8"
id="full-view-affiliation-8"
><sup class="key">8</sup> Haematology Project Foundation, Department of Haematology, S Bortolo Hospital, Vicenza, Italy.</li>
<li data-affiliation-id="full-view-affiliation-9"
id="full-view-affiliation-9"
><sup class="key">9</sup> Department of Internal Medicine, National Reference Center for Immune Cytopenias, Henri Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris-Est Créteil, Créteil, France.</li>
<li data-affiliation-id="full-view-affiliation-10"
id="full-view-affiliation-10"
><sup class="key">10</sup> Centre for Haematology, Barts and the London School of Medicine &amp; Dentistry, Queen Mary University of London, London, UK.</li>
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<div class="short-view" id="short-view-heading">
<div class="publication-type" >Clinical Trial</div>
<h1 class="heading-title">
Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial
</h1>
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<span class="authors-list-item "><span class="full-name">Catherine M Broome</span><span class="citation-separator">&nbsp;et al.</span></span>
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Authors
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<span class="authors-list-item "><a class="full-name"
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data-ga-label="Catherine M Broome">Catherine M Broome</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Lombardi Cancer Center, Division of Hematology, Georgetown University, Washington, DC, USA. Electronic address: catherine.m.broome@gunet.georgetown.edu." href="#short-view-affiliation-1" ref="linksrc=author_aff">
1
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=McDonald+V&amp;cauthor_id=37778358"
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data-ga-label="Vickie McDonald">Vickie McDonald</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Department of Clinical Haematology, Barts Health NHS Trust, London, UK." href="#short-view-affiliation-2" ref="linksrc=author_aff">
2
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Yoshitaka Miyakawa">Yoshitaka Miyakawa</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Department of Hematology, Saitama Medical University Hospital, Saitama, Japan." href="#short-view-affiliation-3" ref="linksrc=author_aff">
3
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Monica Carpenedo">Monica Carpenedo</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Hematology and Transplant Unit, UOC Hematology and Transplant Unit Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy." href="#short-view-affiliation-4" ref="linksrc=author_aff">
4
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Kuter+DJ&amp;cauthor_id=37778358"
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data-ga-label="David J Kuter">David J Kuter</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Hematology Division, Massachusetts General Hospital, Boston, MA, USA." href="#short-view-affiliation-5" ref="linksrc=author_aff">
5
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Al-Samkari+H&amp;cauthor_id=37778358"
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data-ga-label="Hanny Al-Samkari">Hanny Al-Samkari</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Hematology Division, Massachusetts General Hospital, Boston, MA, USA." href="#short-view-affiliation-5" ref="linksrc=author_aff">
5
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Bussel+JB&amp;cauthor_id=37778358"
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data-ga-label="James B Bussel">James B Bussel</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Division of Hematology and Medical Oncology and Division of Pediatric Hematology and Oncology, Weill Cornell Medical College, New York, NY, USA." href="#short-view-affiliation-6" ref="linksrc=author_aff">
6
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Godar+M&amp;cauthor_id=37778358"
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data-ga-label="Marie Godar">Marie Godar</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="argenx, Ghent, Belgium." href="#short-view-affiliation-7" ref="linksrc=author_aff">
7
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Ayguasanosa+J&amp;cauthor_id=37778358"
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data-ga-label="Jaume Ayguasanosa">Jaume Ayguasanosa</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="argenx, Ghent, Belgium." href="#short-view-affiliation-7" ref="linksrc=author_aff">
7
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Kristof De Beuf">Kristof De Beuf</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="argenx, Ghent, Belgium." href="#short-view-affiliation-7" ref="linksrc=author_aff">
7
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Rodeghiero+F&amp;cauthor_id=37778358"
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data-ga-label="Francesco Rodeghiero">Francesco Rodeghiero</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Haematology Project Foundation, Department of Haematology, S Bortolo Hospital, Vicenza, Italy." href="#short-view-affiliation-8" ref="linksrc=author_aff">
8
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Michel+M&amp;cauthor_id=37778358"
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data-ga-label="Marc Michel">Marc Michel</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Department of Internal Medicine, National Reference Center for Immune Cytopenias, Henri Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris-Est Créteil, Créteil, France." href="#short-view-affiliation-9" ref="linksrc=author_aff">
9
</a></sup><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Newland+A&amp;cauthor_id=37778358"
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data-ga-label="Adrian Newland">Adrian Newland</a><sup class="affiliation-links"><span class="author-sup-separator">&nbsp;</span><a class="affiliation-link" title="Centre for Haematology, Barts and the London School of Medicine &amp; Dentistry, Queen Mary University of London, London, UK." href="#short-view-affiliation-10" ref="linksrc=author_aff">
10
</a></sup><span class="semicolon">;&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=ADVANCE+Investigator+Study+Group%5BCorporate+Author%5D"
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<h3 class="title">
Collaborators
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<li>
<strong class="title">ADVANCE Investigator Study Group</strong>:
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<span class="authors-list-item "><a class="full-name"
href="/?term=Fillitz+M"
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data-ga-label="Michael Fillitz">Michael Fillitz</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Knoebl+P"
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data-ga-label="Stef Meers">Stef Meers</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Ismail Mohamad Amine">Ismail Mohamad Amine</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Jaromir Gumulec">Jaromir Gumulec</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Antonin Hlusi">Antonin Hlusi</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Jiri Mayer">Jiri Mayer</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Philippe Quittet">Philippe Quittet</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Jean-Francois Viallard">Jean-Francois Viallard</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
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data-ga-label="Magdana Betaneli">Magdana Betaneli</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Datikashvili-David+I"
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data-ga-label="Genadi Iosava">Genadi Iosava</a><span class="comma">,&nbsp;</span></span><span class="authors-list-item "><a class="full-name"
href="/?term=Makhaldiani+L"
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<div class="affiliations">
<h3 class="title">
Affiliations
</h3>
<ul class="item-list">
<li data-affiliation-id="short-view-affiliation-1"
id="short-view-affiliation-1"
><sup class="key">1</sup> Lombardi Cancer Center, Division of Hematology, Georgetown University, Washington, DC, USA. Electronic address: catherine.m.broome@gunet.georgetown.edu.</li>
<li data-affiliation-id="short-view-affiliation-2"
id="short-view-affiliation-2"
><sup class="key">2</sup> Department of Clinical Haematology, Barts Health NHS Trust, London, UK.</li>
<li data-affiliation-id="short-view-affiliation-3"
id="short-view-affiliation-3"
><sup class="key">3</sup> Department of Hematology, Saitama Medical University Hospital, Saitama, Japan.</li>
<li data-affiliation-id="short-view-affiliation-4"
id="short-view-affiliation-4"
><sup class="key">4</sup> Hematology and Transplant Unit, UOC Hematology and Transplant Unit Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy.</li>
<li data-affiliation-id="short-view-affiliation-5"
id="short-view-affiliation-5"
><sup class="key">5</sup> Hematology Division, Massachusetts General Hospital, Boston, MA, USA.</li>
<li data-affiliation-id="short-view-affiliation-6"
id="short-view-affiliation-6"
><sup class="key">6</sup> Division of Hematology and Medical Oncology and Division of Pediatric Hematology and Oncology, Weill Cornell Medical College, New York, NY, USA.</li>
<li data-affiliation-id="short-view-affiliation-7"
id="short-view-affiliation-7"
><sup class="key">7</sup> argenx, Ghent, Belgium.</li>
<li data-affiliation-id="short-view-affiliation-8"
id="short-view-affiliation-8"
><sup class="key">8</sup> Haematology Project Foundation, Department of Haematology, S Bortolo Hospital, Vicenza, Italy.</li>
<li data-affiliation-id="short-view-affiliation-9"
id="short-view-affiliation-9"
><sup class="key">9</sup> Department of Internal Medicine, National Reference Center for Immune Cytopenias, Henri Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris-Est Créteil, Créteil, France.</li>
<li data-affiliation-id="short-view-affiliation-10"
id="short-view-affiliation-10"
><sup class="key">10</sup> Centre for Haematology, Barts and the London School of Medicine &amp; Dentistry, Queen Mary University of London, London, UK.</li>
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PMID:
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<strong class="current-id" title="PubMed ID">37778358</strong>
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10.1016/S0140-6736(23)01460-5
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Abstract
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<p>
<strong class="sub-title">
Background:
</strong>
Primary immune thrombocytopenia is an autoimmune disorder mediated partly by platelet autoantibodies, resulting in thrombocytopenia, bleeding, and constitutional symptoms. Efgartigimod, a first-in-class novel human IgG1 Fc fragment, binds the neonatal Fc receptor with high affinity and thus reduces serum IgG concentrations, including autoantibodies. The objective of this study was to evaluate the efficacy and safety of efgartigimod in adults with persistent and chronic primary immune thrombocytopenia.
</p>
<p>
<strong class="sub-title">
Methods:
</strong>
This phase 3, multicentre, randomised, double-blinded, placebo-controlled, 24-week study evaluated the efficacy and safety of intravenous efgartigimod in adults aged 18 years or older with chronic or persistent primary immune thrombocytopenia who had an average platelet count of less than 30 000, had responded to at least one previous immune thrombocytopenia therapy, and were on a concurrent therapy at baseline or had received at least a second previous immune thrombocytopenia therapy. The study took place in 71 participating sites from Asia, Europe, and North America. Patients were randomly assigned 2:1 to receive either efgartigimod (10 mg/kg) or placebo intravenously for the first 4 weeks, after which the dosing schedule could be altered to once per week or every other week depending on the patients' platelet count. The primary endpoint, evaluated in the chronic population, was sustained platelet count response (≥50 × 10<sup>9</sup> for at least 4 of the last 6 weeks). This study is registered with ClinicalTrials.gov (<a href="http://clinicaltrials.gov/show/NCT04188379" title="See in ClinicalTrials.gov">NCT04188379</a>) and is completed.
</p>
<p>
<strong class="sub-title">
Findings:
</strong>
A total of 205 patients were screened from Dec 9, 2019, to Feb 3, 2022, and 131 (86 in the efgartigimod group; 45 in the placebo group) were randomly assigned. These patients represented a population with long-term disease who had a mean time since diagnosis of 10·6 years and 67% (88/131) of whom had received at least three previous immune thrombocytopenia treatments. 22% (17/78) of patients with chronic immune thrombocytopenia receiving efgartigimod reached the primary endpoint compared with 5% (2/40) of those receiving placebo (p=0·032; adjusted difference in response, 16% [95% CI 2·6-26·4]). The median number of weeks of disease control in patients with chronic immune thrombocytopenia was 2·0 (IQR 0·0-11·0) for efgartigimod versus 0·0 (0·0-1·0) for placebo (p=0·0009). Efgartigimod was well tolerated; most adverse events were mild to moderate in severity. The most common adverse events of interest in both groups were headache (16% in efgartigimod and 13% in placebo), haematuria (16% in efgartigimod and 16% in placebo), and petechiae (15% in efgartigimod and 27% in placebo).
</p>
<p>
<strong class="sub-title">
Interpretation:
</strong>
Efgartigimod significantly increased sustained platelet count responses compared with placebo in patients with chronic immune thrombocytopenia, including those who had received multiple previous immune thrombocytopenia therapies. Upon completion of the ADVANCE IV study, patients could enroll in the ongoing open-label extension. Subcutaneous efgartigimod is currently being evaluated in patients with immune thrombocytopenia in the ADVANCE SC+ trial.
</p>
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Funding:
</strong>
argenx.
</p>
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<p xmlns:xlink="http://www.w3.org/1999/xlink" xmlns:mml="http://www.w3.org/1998/Math/MathML" xmlns:p1="http://pubmed.gov/pub-one">Declaration of interests CMB reports honoraria from Alexion, Apellis, argenx, and Sanofi; and advisory fees from Novartis and Incyte. VM reports consulting or advisory fees from Amgen, Novartis, and Sobi; and research grants from Grifols and Rigel. YM reports consulting or advisory fees from argenx, Kyowa Kirin, UCB, and Zenyaku Kogyo; and honoraria from Alexion, Chugai, Pfizer, and Sanofi. MC reports consulting or advisory fees from Amgen, argenx, and Novartis; and honoraria from Sobi. DJK reports grant support from argenx, Biocryst, Immunovant, Principia, Rigel, Takeda (Bioverativ), and UCB; consulting or advisory fees from Alexion (Syntimmune), Amgen, argenx, BioCryst, Bristol-Myers Squibb, Caremark, Cellphire, Cellularity, CRICO, Daiichi Sankyo, Hengrui, Immunovant, Incyte, Kyowa Kirin, Merck Sharp Dohme, Momenta, Novartis, Pfizer, Platelet Biogenesis, Platelet Disorder Support Association, Rigel, Sanofi (Bioveratif), Sanofi (Genzyme), Sanofi (Principia), Sobi (Dova), Takeda, UCB, and Up-To-Date; and stock ownership benefits from Rubius. HA-S reports grant support from Agios, Amgen, Novartis, Sobi, and Vaderis; and consulting or advisory fees from Agios, argenx, Forma, Moderna, Novartis, Rigel, and Sobi. JBB reports advisory fees from Amgen, argenx, AstraZeneca, Janssen, Novartis, Rigel, Sanofi, Sobi, and UCB; and data and safety monitoring fees from UCB. MG reports employment by argenx. JA reports employment by argenx. KDB reports employment by argenx. FR reports consulting or advisory fees from Amgen, argenx, Novartis, and UCB. MM reports consulting or advisory and speaker fees from Alexion, argenx, Novartis, Sanofi, and Sobi. AN reports honoraria from Amgen, Angle, argenx, Dova, Novartis, Ono, Rigel, and Shionogi; grant funding from Amgen, Novartis, and Rigel; expert testimony fees from argenx and Rigel; and consulting or advisory fees from Amgen, Angle, argenx, Dova, Novartis, Ono, Rigel, and Shionogi.</p>
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MeSH terms
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Substances
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<div id="supplemental-data" class="supplemental-data">
<h2 class="title">
Associated data
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