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

Autor: Broome, Catherine M, McDonald, Vickie, Miyakawa, Yoshitaka, Carpenedo, Monica, Kuter, David J, Al-Samkari, Hanny, Bussel, James B, Godar, Marie, Ayguasanosa, Jaume, De Beuf, Kristof, Rodeghiero, Francesco, Michel, Marc, Newland, Adrian, Fillitz, Michael, Knoebl, Paul, Meers, Stef, Amine, Ismail Mohamad, Gumulec, Jaromir, Hlusi, Antonin, Mayer, Jiri, Quittet, Philippe, Viallard, Jean-Francois, Betaneli, Magdana, Datikashvili-David, Irine, Iosava, Genadi, Makhaldiani, Levan, Rejto, Laszlo, Sharashenidze, Nino, Groepper, Stefanie, Röth, Alexander, Hamed, Aryan, Illes, Arpad, Carpenedo, Monica, Artoni, Andrea, Bocchia, Monica, Gamberi, Barbara, Borchiellini, Alessandra, Oliva, Esther Natalie, Patriarca, Andrea, Tomassetti, Simona, Miyakawa, Yoshitaka, Ando, Kiyoshi, Handa, Hiroshi, Katsutani, Shinya, Nishiwaki, Kaichi, Ito, Shoko, Ito, Tomoki, de Boer, Fransien, Jansen, A.J. Gerard, Ciepluch, Hanna, Fornagiel, Szymon, Grosicki, Sebastian, Soroka-Wojtaszko, Maria, Trelinski, Jacek, Bakirov, Bulat, Borisenkova, Elena, Volodicheva, Elena, Shatokhin, Yuri, Proydakov, Andrey, Alonso, Maria Aranzazu, Mingot-Castellano, María Eva, Sanchez-Gonzalez, Blanca, Valcarcel Ferreiras, David, Ayli, Meltem, Hacibekiroglu, Tuba, Demir, Ahmet Muzaffer, Nalçaci, Meliha, Sonmez, Mehmet, Tombak, Anil, Toprak, Selami Kocak, Turgut, Burhan, Vural, Filiz, Yagci, Munci, Altuntas, Fevzi, Turgut, Mehmet, Kaya, Emin, Romanyuk, Nataliya, McDonald, Vickie, Kazmi, Syed Rashid Saeed, Broome, Catherine, Cataland, Spero, Doshi, Ketan, Lentz, Steven R., Boxer, Michael, Rosenberg, Richard
Zdroj: The Lancet; 20230101, Issue: Preprints
Abstrakt: 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.
Databáze: Supplemental Index